Treatments for RP in general

Worldwide there are various treatments suggested by the medical world but none scientifically proves any solid results. The general idea amongst ophthalmologists is that there is no cure for Retinitis Pigmentosa. Proposed treatments go from a vitamin based medication to avoiding strong day light, to ozone or electro-stimulation. The truth is that some treatments do actually have certain effects. They tend to slow down the progress of the disease. But there is no real over all treatment for patients. All these treatments are a strain on the patients already hard every day life and in the end bring no solution.

Orfilio PelaezSince the early 50ies the Cuban Doctor Orfilio Pelaez Molina dedicated a life time of medical research to the disease Retinitis Pigmentosa. As a young doctor he was angered when a good friend of his was diagnosed with RP and the local doctor simply said there was nothing he could do: his friend would become gradually blind.

Together with a team of colleague doctors he started to assist RP patients in Cuba during the progress of their disease. All the existing treatments of the time were tried out. A lot of attention went to the psychological aspects of the disease for the patients and their environment. Parallel to the care for the patient, extensive scientific research was performed. All data about the patients were meticulously registered. New treatment methods were tried out on animals in laboratories. The team had a national mission with support on the highest level of the Cuban Ministry of Public Health. This made it possible to gather a high number of patients records, and to do research at the best centres in the country.

Thanks to these decades of care for the Cuban RP patients and the research performed around them Dr Pelaez team started in the 80ies their unique treatment for RP.

First of all we need to explain that the treatment of Dr Pelaez is a combination of a number of treatments. Some parts of the treatments are also recommended by many ophthalmologists world wide. The uniqueness of the treatment is that it is an integral approach of the patient. Unique is also the positive results of the treatment. Many analyses have been done during the years and all statistics point out to a halt in the progress of the visual loss during and after the treatment in a significative majority of the patients. Better results are obtained when treatment is applied in the early stages of the disease when patients are still young of age. There is also a statistically small number of patients that indicated an improvement of the visual field after the treatment.

What is the basis of the treatment? Dr Pelaez considered that the circulation of the ocular tissues through the coriocapillary layer should improve the metabolism of these tissues. On this correct concept the whole treatment is based. The idea is to revitalize the photoreceptors of the eye as far as they are still active. This way the progress of the vision loss is slowed down or stopped.
The method of Revitalization

There are several ways to obtain this revitalization and the combination of all these methods forms the succes of the treatment:

Revitalizing Surgery
The first part of the treatment consists in an operation on both eyes. The surgeons implant fat blood tissues in the upper choroid (a layer around the retina). This will improve the functioning of the photo-receptors of the retina that are still active.
This is a routine operation in the Camilo Cienfuegos Clinic. Patients feel side effects of the operation for a period of maximum 3 days.

Ozone Therapy
The intensity and quantity of ozone administrations is strictly regulated and based on the analysis of each patients physical condition in general and on his or her visual capacities in particular. It is administered either by rectal medication (suppositories) or by injecting it in the veins. An ozone treatment takes around 15 days and needs to be repeated annually once or twice.

Electro-stimulation
To stimulate the electro-ionic balance of the blood circulation in the eye. At the end of the patients stay the doctor explains how to continue with this treatment at home.

Medication
To stimulate the other treatments and to counter certain negative effects of them. Medication is sold in the pharmacy of the C. Cienfuegos Centre.

Other
The best an RP patient can do is live healthy. Don’t smoke as this has negative effects on the blood veins. Perform some phyiscal activity like sports or recreational activities. Eat fruits, vegetables and avoid fat. And also try to protect your eyes against heavy sunlight buy wearing sun glasses.

The Cuban Ministry of Health, Dorsemo SA de CV and Erkamed.

Treatments for RP in general

Worldwide there are various treatments suggested by the medical world but none scientifically proves any solid results. The general idea amongst ophthalmologists is that there is no cure for Retinitis Pigmentosa. Proposed treatments go from a vitamin based medication to avoiding strong day light, to ozone or electro-stimulation. The truth is that some treatments do actually have certain effects. They tend to slow down the progress of the disease. But there is no real over all treatment for patients. All these treatments are a strain on the patients already hard every day life and in the end bring no solution.

Orfilio PelaezSince the early 50ies the Cuban Doctor Orfilio Pelaez Molina dedicated a life time of medical research to the disease Retinitis Pigmentosa. As a young doctor he was angered when a good friend of his was diagnosed with RP and the local doctor simply said there was nothing he could do: his friend would become gradually blind.

Together with a team of colleague doctors he started to assist RP patients in Cuba during the progress of their disease. All the existing treatments of the time were tried out. A lot of attention went to the psychological aspects of the disease for the patients and their environment. Parallel to the care for the patient, extensive scientific research was performed. All data about the patients were meticulously registered. New treatment methods were tried out on animals in laboratories. The team had a national mission with support on the highest level of the Cuban Ministry of Public Health. This made it possible to gather a high number of patients records, and to do research at the best centres in the country.

Thanks to these decades of care for the Cuban RP patients and the research performed around them Dr Pelaez team started in the 80ies their unique treatment for RP.

First of all we need to explain that the treatment of Dr Pelaez is a combination of a number of treatments. Some parts of the treatments are also recommended by many ophthalmologists world wide. The uniqueness of the treatment is that it is an integral approach of the patient. Unique is also the positive results of the treatment. Many analyses have been done during the years and all statistics point out to a halt in the progress of the visual loss during and after the treatment in a significative majority of the patients. Better results are obtained when treatment is applied in the early stages of the disease when patients are still young of age. There is also a statistically small number of patients that indicated an improvement of the visual field after the treatment.

What is the basis of the treatment? Dr Pelaez considered that the circulation of the ocular tissues through the coriocapillary layer should improve the metabolism of these tissues. On this correct concept the whole treatment is based. The idea is to revitalize the photoreceptors of the eye as far as they are still active. This way the progress of the vision loss is slowed down or stopped.
The method of Revitalization

There are several ways to obtain this revitalization and the combination of all these methods forms the succes of the treatment:

Revitalizing Surgery
The first part of the treatment consists in an operation on both eyes. The surgeons implant fat blood tissues in the upper choroid (a layer around the retina). This will improve the functioning of the photo-receptors of the retina that are still active.
This is a routine operation in the Camilo Cienfuegos Clinic. Patients feel side effects of the operation for a period of maximum 3 days.

Ozone Therapy
The intensity and quantity of ozone administrations is strictly regulated and based on the analysis of each patients physical condition in general and on his or her visual capacities in particular. It is administered either by rectal medication (suppositories) or by injecting it in the veins. An ozone treatment takes around 15 days and needs to be repeated annually once or twice.

Electro-stimulation
To stimulate the electro-ionic balance of the blood circulation in the eye. At the end of the patients stay the doctor explains how to continue with this treatment at home.

Medication
To stimulate the other treatments and to counter certain negative effects of them. Medication is sold in the pharmacy of the C. Cienfuegos Centre.

Other
The best an RP patient can do is live healthy. Don’t smoke as this has negative effects on the blood veins. Perform some phyiscal activity like sports or recreational activities. Eat fruits, vegetables and avoid fat. And also try to protect your eyes against heavy sunlight buy wearing sun glasses.

The Cuban Ministry of Health, Dorsemo SA de CV and Erkamed.

Written by Wiggy

HARVARD MEDICAL SCHOOL MASSACHUSETTS EYE AND EAR INFIRMARY

We have now completed a 4-year clinical trial of lutein supplementation for
patients with retinitis pigmentosa on vitamin A. This study was supported by the
National Eye Institute and in part by the Foundation Fighting Blindness. The
results were reviewed for effectiveness and safety during the trial by an
independent Data and Safety Monitoring Committee.

Non-smoking adults with typical retinitis pigmentosa were randomly assigned
to lutein 12 mg or to a control tablet. All received vitamin A palmitate 15,000
IU/day. Between Years 1 and 2 of follow-up, all were encouraged to eat l-2 three-
ounce servings of oily fish per week. A paper describing this trial is published in
the Archives of Ophthatmology 128:403-411, 2010 to which you are referred.

No significant difference in rate of decline of central visual field sensitivity or
visual acuity was found when comparing those in the lutein plus vitamin A group
and those in the control plus vitamin A group. However, the patients receiving
lutein had, on average, a slower rate of loss of midperipheral visual field sensitivity
than the patients in the control group. We also observed that those with the highest
serum lutein levels or with the highest increase in macular pigment optical density
(a measure of intraretinal lutein) at follow-up had the least decline in midperipheral
field sensitivity. Those with the highest increase in macular pigment optical
density had the slowest loss of central and midperipheral field sensitivity
combined. No significant toxic effects attributable to lutein or vitamin A
supplementation were observed.

These results support the use of 12 mg/day of lutein to slow visual field loss
among non-smoking adults with typical retinitis pigmentosa on vitamin A. We
advise that most adults (for exclusions see next page) continue to take 15,000
Iu/day of vitamin A palmitate and eat I-2 three-ounce servings of oily fish per
week such as salmon or tuna which contain, among other constituents,
considerable docosahexaenoic acid (DHA). Patients with retinitis pigmentosa with
partial, early onset hearing loss (Usher syndrome type II) were included, and,
therefore, this advice applies to these patients. For those who wish to take a daily
multivitamin, the multivitamin should contain no more than 5000 IIJ of vitamin A
and no more than 30 IU of vitamin E.

Patients should have a fasting serum vitamin A and serum liver function
profile annually while on vitamin A 15,000 IU/day plus lutein 12 mg/day and take
these supplements only if these tests are normal. Patients who are pregnant or
planning to become pregnant should not take this dose of vitamin A because of an
increased risk of birth defects on high-dose vitamin A supplementation; whether
lutein supplementation increases the risk of birth defects is not known, but as a
precaution we would advise pregnant women not to take lutein. Patients age 49 and
older should monitor their bone health because of the slight (0.5 -1%) increased
risk of hip fracture among patients taking long-term high-dose vitamin A
supplementation. Some concern has been raised that long-term lutein
supplementation is associated with an increased risk of lung cancer among smokers
older than 50 years; therefore, the recommendation for lutein 12 mg/day is limited
to adults with typical retinitis pigmentosa who do not smoke.

We must emphasize that our conclusions are based on group averages and we
cannot provide assurance that this regimen will benefit a specific patient. We did
not include patients with best corrected vision of 20/200 or less in both eyes,
patients with less than 10 degrees diameter central frelds in both eyes, or patients under
age 18, and, therefore, we cannot make any formal recommendation for such
patients. Patients with less common or atypical forms of retinitis pigmentosa such
as Usher syndrome, type I (retinitis pigmentosa with profound congenital
deafness), Bardet Biedl syndrome, Leber congenital amaurosis, and unilateral,
paravenous, pericentral, or sector forms of retinitis pigmentosa were not included,
nor were patients with allied night blinding disorders such as choroideremia or
retinitis punctata albescens; therefore, we also cannot make any formal
recommendation of lutein and vitamin A for these patients.

We estimate that the average patient with typical retinitis pigmentosa on
vitamin A and an oily frsh diet who starts lutein supplementation by age 40 would
retain midperipheral visual field sensitivity 3 to 10 years longer than the average
patient who does not take this supplement. Calculations leading to this estimate are
presented in the Archives of Ophthalmology, 128:403-411, 2010.

Both lutein and vitamin A are available without prescription. Lutein is
available as a 6-mg capsule or softgel; we advise two 6-mg capsules or softgels per
day preferably with breakfast. Some sources of lutein are attached. Current sources
of vitamin A palmitate 15,000 IU are also attached and we continue to advise one
tablet or gelcap per day.

Please be reminded that this information alert applies to patients who are
taking vitamin A or who have taken vitamin A in the past. For non-smoking adult
patients who will be starting vitamin A for the first time, we advise vitamin A
palmitate 15,000 IU/day, lutein 12 mg/day, and docosahexaenoic acid (DHA) 600
mg twice each day for 2 years. After 2 years they should stop the DHA and begin
to eat 1-2 three-ounce servings of oily fish per week; they should continue the
vitamin A and lutein. Evidence exists that DHA supplementation for 2 years
shortens the interval for vitamin A to have its benefit among patients taking
vitamin A for the first time.

Eliot L. Berson, M.D.
ELB/lb

Attachments: Current sources of lutein and vitamin A

(l) Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco CW, Brockhurst RJ,
Hayes KC, Johnson EJ, Anderson EJ, Johnson CA, Gaudio AR, Willett WC,
Schaefer EJ. Clinical trial of lutein in patients with retinitis pigmentosa receiving
vitamin A. Arch Ophthalmol, 128 403-411, 2010.
(2) Berson EL, Rosner B, Sandberg MA, Hayes KC, Nicholson BW, Weigel-
DiFranco C, Willett W. A randomized trial of vitamin A and vitamin E
supplementation for retinitis pigmentosa. Arc:h Ophthalmol, lll:761-772, 1993.
(3) Sibulesky L, Hayes KC, Pronczuk A, Weigel-DiFranco C, Rosner B, Berson
EL. Safety of less than 7,500 RE/day (25,000 IUiday) of vitamin A in adults with
retinitis pigmentosa. Amer J Clin l{utr,69:656-663, 1999.
(4) Berson EL, Rosner B, Sandberg MA, Weigel-DiFranco C, Moser A,
Brockhurst RJ, Hayes KC, Johnson CA, Anderson EJ, Gaudio AR, Willett WC,
Schaefer EJ. Further evaluation of docosahexaenoic acid in patients with retinitis
pigmentosa receiving vitamin A treatment: Subgroup analyses. Arch Ophthalmol,
122:1306-1314, 2004.

Sources of Lutein 6 mg -April 2010

Consult with your doctor before taking two (2) 6 mg capsules or softgels per day for retinitis
pigmentosa. (Note: a single 12 mg capsule, softgel, or tablet is not currently available.)

Sources of Lutein 6 mg (for adults with retinitis pigmentosa)

Nature’s Bounty Lutein (Natural Carotenoid)
6 mg. #003482
110 Orville Drive
Bohemia, NY I 1716
www. naturesbounty. com
1-800-433 -2990
50 softgels $6.99
Available online, in drugstores, and at major retailers.
Shipping & Handling may vary

Source Naturals Lutein
6 mg #SN0051
23 Janis Way
Scotts Valley, CA 95066
www. sourcenaturals. com
1-800-815-2333
Fax: 1-831-438-7410
45 capsules $9.50
90 capsules $17.98
Available online, in drugstores, and at major retailers.
Shipping & Handling may vary

Vitamin World Eye Guard Lutein
6 mg #003481
105 Orville Drive
Bohemia, NY 11716-2599
www.vitaminworld.com
1-866-836-7050
Fax: 1-866-667-9236
100 softgels $13.99
Member’s price $10.99
Shipping & Handling $1.95

Puritan’s Pride LUTIGOLD tm LUTEIN
6 mg #003481
Attn: Order Department
1233 Montauk Hwy
P.O. Box 9001
Oakdale, NY I 1769
www.Puritan.com
1 -800-645-1030
100 softgels -Buy I get 1 Free
(2 for $8.99)
Shipping & Handling $4.95

The Vitamin Shoppe Lutein
6 -mg #VS-1269/1042934
Customer Care Department
2l0l 91st Street
North Bergen, NJ 07047
www. vitaminshoppe. com
1-866-293-3367
Fax: 1 -800-852-7153
100 softgels $11.49
Available online and at local “The Vitamin Shoppe” store locations
Shipping & Handling may vary

Disclaimer: The products described above have not been tested or evaluated to determine their safety or
effectiveness in children or adolescents with RP. The listing of these suppliers and their products should not be
misinterpreted as a recommendation or indication of proprietary interest in any of these cornpanies

Sources of Vitamin A Palmitate 15,000 Iu -April 2010

Consult with your doctor before taking 15,000 international units of vitamin A per day for
retinitis pigmentosa.

Sources of Vitamin A Palmitate 15,000 IU (for adults with retinitis pigmentosa)

Freeda Vitamins, Inc.
47-25 34th Street, 3rd Floor
Long lsland City, NY 11101
l-800-777-3737 (outside NY) or
I-718-433-4337
Fax: l-718-433-4373
100 Tablets $6.36/bottle (with discount)
250 Tablets $12.72/bottle (with discount)
Shipping & Handling $5.95
Will ship outside USA for extra postage

J. R. Carlson Laboratories, Inc.
15 College Drive
Arlington Heights, IL 60004-1985
I -888-234-5656 (outside Chicago) or
1-847-255-1600
Products #1101 and 1102A -Palmitate -15
120 Gelcaps $8.90/bottle
240 Gelcaps $15.50/bottle
Shipping & Handling $10.00
Free shipping for orders over $40.00
Will ship outside USA for extra postage

Disclaimer: The products described above have not been tested or evaluated to determine their safety or
effectiveness in children or adolescents with RP. The listing of these suppliers and their products should not be
misinterpreted as a recommendation or indication of proprietary interest in any of these companies.
Related Health Topics

Written by sassynknowit18

Foundation Fighting Blindness (FFB) proposes to expand its existingconsortium of the world’s leading researchers in stem cell therapies to accelerate advances in finding treatments and cures for retinal degenerative diseases.Stem cell therapies hold great promise for restoring vision to individuals whohave lost their sight, or are rapidly losing their sight, due to death ofphotoreceptor cells—rods and cones—located in the retina. A stem cell is a cell whose job in the body hasn’t yet been determined. Every cellin the body “stems” from this type of cell. There are many types of stem cells that have different names based upon how specialized the cells have become. Goingfrom the least specialized to the most specialized, these are: embryonic,progenitor/precursor, umbilical, and adult stem cells. As a stem cell becomesmore specialized, there are fewer cells that it can ultimately become. For instance, retinal adult stem cells can only become cells in the retina, not cells ofthe skin or other body part. However, no matter what stem cell is used as thestarting material, the goal of any stem cell therapy is to repair a damaged tissuethat can’t heal itself. As the science and efficacy of stem cell replacement therapy advances, it holdsgreat promise to individuals who are losing their sight due to retinitis pigmentosa,advanced macular degeneration, Leber congenital amaurosis, and other retinaldiseases that result in photoreceptor cell death.

Photoreceptor cells are those cells which turn light into electrical impulsestransmitted to the brain, where “seeing” actually occurs. When thesephotoreceptor cells die prematurely, the only way to restore their function is toreplace them with “new” cells through transplantation or injection in the eye.In recent years, Foundation-funded researchers from all over the country havemade enormous strides in advancing stem cell therapies to treat the entirespectrum of retinal degenerative diseases. For the first time, scientists havegenerated functional photoreceptors from developing progenitor/precursor stemcells (stem cells that already have begun to follow a specific path but aren’t attheir final identity), and at the same time, facilitated their integration into the hostretina. These advances bring us much closer to the reality of human clinical trialsof stem cell therapies to save and restore vision. The anticipated five year budget for this program is $11.5 million. II. Research Goals — Human Phase I Clinical TrialFFB will build on the results of studies it has supported to accelerate the use ofembryonic stem cell transplantation to replace diseased retinal tissue, such asphotoreceptor cells (rods and cones) and retinal pigment epithelial (RPE) cells, tosave and restore vision. Research will use existing and new rodent models and,importantly, non-human primates (e.g., monkeys) as a “proof of principal” tosupport filing an Investigational New Drug Application (IND) with the FDA for ahuman phase I clinical trial within five years. Specifically, the primary anticipated outcome of the FFB research program will beto rapidly accelerate the progress of emerging stem cell therapies to clinical trials. We expect that stem cell therapies will:

(1) Arrest photoreceptor and retinal pigment epithelial cell degeneration, theprimary cause of vision loss from retinitis pigmentosa and maculardegeneration; and, (2) Reverse the degeneration by replacing the diseased photoreceptors andretinal pigment epithelial cells with cells manufactured using adult,umbilical cord blood, progenitor/precursor, or embryonic stem cells as thestarting material. The research program will define and optimize the cell production, delivery, andtransplant procedures. This is necessary to enable human clinical trials to beconducted in the safest and most effective manner, thereby optimizing everyopportunity for success. Rationale: Retinitis pigmentosa afflicts between 100,000 and 200,000 peopleannually in the U.S. alone, and several million around the world. More than ninemillion people in the U.S. have many types of degenerative retinal diseases,including Stargardt and Usher diseases that primarily affect children. Age-relatedmacular degeneration (AMD) is a leading cause of blindness in the U.S. and thedeveloped world. The National Eye Institute, a part of the U.S. National Institutesof Health, estimates that 1.7 million Americans have vision loss from advancedAMD, and another 7.3 million face the risk of vision loss from AMD. Given theaging of the baby boomer generation, the government estimates that by 2020,the number of Americans with vision loss from advanced AMD will increase by50% to approximately 3 million.

The Foundation Fighting Blindness, the world’s largest source of non government grants for retinal disease research, has supported pioneering investigations that have yielded important new insights into causes, preventions,and treatments for retinitis pigmentosa and other retinal diseases. One example of the impact of the Foundation’s support is found in a 2006publication by an FFB-supported researcher which states that transplanation of developing photoreceptor cells (i.e., developing precursor/progenitor stem cells)into mice with RP led to some evidence of vision restoration. The research findings were reported in Nature magazine and were the focus of major news stories in The Washington Post and The Boston Globe. The FFB is now poised to leverage its previous support to move this landmark research to its next stage — a platform for developing clinical trials. The FFB proposes to invest $11.5 million over five years to support this important research project. If U.S. government support for this project is obtained, research supported with these funds would adhere to U.S. governmental regulations for stem cell research. Success of this project will move stem cell therapies closer tothe promise of new therapies to treat the entire spectrum of retinal degenerative diseases. III. Research Overview Goal 1: Develop cells to sustain existing photo receptors and visual capability:(A) This phase of the research will explore several stem cell types, including but not limited to: i) federally “approved” human embryonic stem cells, one type of which has already been shown to become photoreceptor cells when transplanted into diseased retinas in rodents; ii) new, “non approved”human embryonic stem cells; iii) all other stem cells, including specialized retinal or brain progenitor stem cells, and umbilical or adult stem cells; and, iv) modified human stem cells (“approved” and “non approved”)that secrete specific neuro protective proteins that preserve photo receptors and RPE cells. (B) Once the safety and efficacy of the cells are proven, selected cells will be prepared for further development at a facility that meets FDA Good Manufacturing Practices (GMP) and additional safety studies will be conducted. In March 2007, PLoS ONE reported that human neural progenitor cells (hNPCs)were used as effective retina rescue agents.

Researchers used two types ofh NPCs: one type is naturally occurring, while the second type is engineered to contain an additional, non-neural growth factor. Both cell types migrated to appropriate and advantageous locations within the retina, and produced no evidence of unwanted effects. Two members of this research group are part of the FFB Stem Cell Consortium. Goal 2: Develop strategies to repopulate the photoreceptor cells and RPE: Stem cells for replacing the diseased cells will be derived primarily from human embryonic stem cells. Only cells that have demonstrated the capability to produce cell types with retina-specific markers (i.e.,photoreceptor precursor cells) will be used for further testing and gene and protein analysis. Therefore, a series of cell-surface markers will be characterized to enable identification and development of these cells from the mixed stem cell populations. A set of animal models suitable for sustaining and repopulating photo receptor cells will be assembled to cover a wide range of retinal degenerative diseases.FFB provided funding for a landmark research project, published in the November 9, 2006 issue of Nature, to investigate using transplanted stem cells to restore vision in laboratory mice with retinitis pigmentosa. The study has shown that transplanted cells could become functioning retinal cells and integrate into the host retina. In addition, the March 1, 2007 issue of Stem Cells reported that human umbilical cord tissue cells were used to rescue vision in a specific rodent model of retinal degenerative disease, a fact that may help treat patients with retinitis pigmentosa. This work was done by an FFB-funded researcher, in collaboration with Centocor, a biomedical company. Goal 3: Define the most appropriate stem cell delivery method using non-human primates to support a FDA Investigational New Drug application (IND) for a human clinical trial: These studies will define and optimize the cell delivery procedures [sub retinal injection, transplantation on a matrix substrate (a growth “skeleton”), etc.], appropriate dosage, and safety in non-human primates. Sub retinal cell injections will be given to healthy primates, and the outcomes will be studied and evaluated. This phase will include generating the development of an acquired outer retinopathy in primates, designed to test the efficacy of the above-mentioned photo receptor replacement techniques. Goal 4: Final preclinical safety studies using GMP manufactured stem cells: This will involve a series of safety studies in addition to those detailed above, including examining the potential for developing tumors, and containing immune reactions, as well as preparing the best cell lines for GMP production. IV. Elements Critical to the Success of This Program• The entire research effort has been built around the collective expertise of an FFB-supported group of investigators, each of whom has a commitment in this area of research and a proven track record of success.• The project will be developed with a rigorous timeline and set of deliverables that will be actively monitored. • The project will be conducted with close ties to clinical programs which can share findings and knowledge, ensuring the preclinical studies progress naturally toward human clinical trials.• Multiple rigorous and clinically relevant safety and efficacy studies will be used to validate each potential stem cell treatment.FFB is actively pursuing relationships with industry for translating the outcomes to clinical trials, and establishing safety and GMP capability. FFB is also meeting with multiple companies committed to stem cell therapy for retinal degenerative diseases. V. Timelines Years 1-2: Newly-identified and presently-existing stem cells will be usedto test the efficacy of either encouraging the survival of existing photo receptors or replacing them in mouse models of specific retinal degenerations and related diseases. This phase might be extended beyond the two year period, based on results, to explore whether the infusion of additional “helper” cells, or the inclusion of a growth “skeleton”to organize the infused cells, will improve efficacy. Years 2-5: Take “best” cells to GMP level of production. Years 3-5: Compare relative efficacy of cells in appropriate rodent models of retinal degenerative diseases and nonhuman primates. Year 5: Prepare and submit regulatory documents for human Phase I clinical trial. VI. Budget The total five-year cost of this program is estimated to be $11.5million. Ideally, the Foundation would need $4 million in commitments to undertake the project, and would then need to meet target goals of raising an additional $2.5 million per year in each of the next three years to meet the goal and timeline. Fund raising costs are estimated to be $500,000. Cost summary of the program:• Manufacture appropriate cells: $4 million• Test efficacy: $3 million• Examine the feasibility of implanting manufactured cells in primates: $1 million• Explore issues of safety and GMP production: $2 million• Cost of Fund raising: $500,000 (4.5%)TOTAL: $11.5 million Project leader: Dr. Stephen Rose, Chief Research Officer, Foundation Fighting Blindness The Foundation Fighting Blindness: A Proven Leader in Retinal Research The Foundation Fighting Blindness has raised more money for retinal degenerative disease research than any non-governmental in the United States.Since our inception in 1971, we have raised more than $260 million. The Foundation funds the world’s top scientific and clinical research experts at hundreds of prominent institutions. In the current fiscal year, the Foundation is funding 110 total research grants to115 investigators who are located at 48 institutions around the world. To promote collaborations between basic and clinical researchers and accelerate treatments to the clinic, the Foundation funds 16 collaborative national and international research centers. These include the Southwest Regional Center for the Study of Retinal Degenerative Diseases (Retina Foundation of the Southwest, University of Texas, and University of Oklahoma) and the Wilmer Eye Institute Research Center for the Study of Retinal Degenerative Diseases (Johns Hopkins University). In 2003, the Foundation established the National Neuro vision Research Institute to accelerate the translation of promising research into clinical trials, and make more treatments (including stem cell therapies) commercially available to the people who need them. As a result, in 2004, the Foundation established the FFB Stem Cell Consortium—a nationwide collaboration of stem cell experts—to quickly advance emerging stem cell treatments for retinal degenerative diseases. Over the past year alone,Consortium scientists have been recognized internationally for making numerous, extraordinary advancements in stem cell research for retinal diseases.

Spending long hours in front of computers, laptops, or televisions has become a part of modern man’s life today. There is no single profession that does not require use of computers. When at office, one has to spend long eight to nine hours, if not more, in front of the systems. But this rendezvous of modern man with his computer does not end here. Back home, once again he gets hooked to is laptop or television set for some entertainment. Working for late hours on computers is not uncommon either. In another words, there is no escape from computers nowadays.

All these take a heavy toll on the health of modern man, not to mention the eyes. And eyes being the most sensitive organs of our body, get very badly affected by these stress. Result- vision related problems and eye diseases. Here are some useful eye care tips for computer personnel, which if followed religiously will give long-term results:

Work for 20 to 30 minutes on computer at a stretch. Then look at a distant object and blink slowly several times. Do not stare at your computer. This can lead to Sjogren’s syndrome or dry eyes. Make a conscious effort to blink frequently, at least 12 to 15 times every minute. Once you start doing this, it will soon become a habit.

Exercise your eye balls at frequent intervals. Close your eyes and role them underneath the eyelids in clockwise and anti clockwise direction. While you do this this, breathe in deeply and breathe out. Finally, open your eyes very slowly.

Take a small piece of soft linen, say, your clean handkerchief, fold it in a square and puff on it till it is warm. Now, cover your eyes with the handkerchief for about a minute. It helps in relaxing and soothing your eyes. You can also do this with your palms, if you find your handkerchief not clean enough. Take small breaks at frequent intervals, and splash water on your face. This helps to cool your eyes. Take a brisk walk down the floor every time you take a break. Walking not only relaxes your whole body but also increases blood supply to your eyes.

Get an anti-glare screen fixed on to your computer monitor, or use your own pair of anti-glare glasses while working on computers. Also adjust the brightness of the monitor in such a way that the harmful glare from the computer screen is minimum.

Here

You are on your final approach.  The runway is clearly in sight.  At this moment you glance down at your instrument panel and find that it’s just a wee bit fuzzy.  You lean forward to get a closer look and find that this makes it even more difficult to see.  You have been noticing  changes like this since your 40th birthday, but have been overlooking them because  you’ve only been considering these changes to be related to “eye strain.” When looking at maps prior to planning your next flight, you have noticed that you have had to hold the map out somewhat further from you than in the past.  In fact, on some occasions you have found yourself “cheating” a bit with a hand magnifier for some of the finer details.

You begin discussing this topic with a number of your contemporaries and hear a myriad of “solutions.”  Reading glasses appear to be the most popular solution, but although they certainly provide easy viewing of reading material, it makes the instrument panel in the cockpit almost impossible to see.  You have heard that some individuals resort to “monovision” where one contact lens, if necessary, is used for distance, and the contact in the other eye is used for close work. While this once again helps with reading, it still does not help with the instrument panel, but most importantly contributes to a slight decrease in depth perception.  This has not been recommended for pilots coming in for a landing.

Bifocal lenses would help both distance and near, but a blended bifocal or trifocal would be necessary to see distance, instrument panel and reading (far, intermediate, and near.) But what if you have found that you just can’t wear glasses or contact lenses, or at least prefer not to if at all possible?   Many have been experiencing nearly perfect vision all their lives, but now after the age of 40, are recognizing that near projects are beginning to become more and more difficult without any optical aid whatsoever.

There is now a solution that can actually restore the natural ability to focus once again.  The act of changing focus from distance to near is called accommodation.  The prescription in the eye does not change, but the ability to allow the lens in the eye to see things clearly up close requires the use of the “accommodating muscles” of the eye.  A new surgical procedure can actually turn back this clock 25 years and allow you to do your close work like you used to be able to do, when you were much younger.

Because the natural lens continues to grow throughout life, it eventually (at around 40 years of age) comprises the ability of the “muscles of accommodation” to function.  By placing four tiny implants just beneath the surface of the white of the eye overlying these muscles, natural “accommodation” is restored.  This outpatient procedure is done under topical anesthetic where you do not have to be put to sleep or stay overnight in the hospital.  It restores your natural ability to see at near.  There are no patches needed for the eyes once the procedure is completed.  Immediately after completing the surgery, you may be able to pick up a newspaper and read.

Not everyone is nearsighted (myopia), not everyone is farsighted (Hyperopia,) and not everyone has astigmatism—but everyone who lives beyond the age of 40 experiences presbyopia, the inability to see at near without additional optical assistance.  Recent technology in refractive surgery in ophthalmology has allowed us to reduce or eliminate nearsightedness, farsightedness, and astigmatism.  The last frontier is to “cure” presbyopia.  All methods of treating presbyopia—to date—are merely “compensation.” If you wear reading glasses, bifocals or bifocal lenses, you still have the same problems at near when these are removed.  If you wear “monovision” contacts or undergo “monovision” refractive surgery, only one eye is clear at near, and depth perception is decreased. Until this new procedure was developed, there was no ability to allow both eyes to see clearly both at a distance and at near without any additional assistance.  This procedure takes you back to your days of high school when you could look at the blackboard and then look at your notes without any additional assistance.
The estimated duration of the effectiveness of this procedure is projected to be between 25 and 30 years.  At that time, if necessary, it may be possible to repeat the procedure to provide additional effect.

So, if you’re coming in on your final approach and are starting to notice difficulties and have the runway clearly in sight, but are having trouble in seeing the instrument panel or you watch, then you may wish to consider pursuing information regarding a new technique called The Surgical Reversal of Presbyopia.

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Written by Wiggy
12 December 2009

We are pleased to provide the results of a clinical trial that indicate a specified dose of vitamin A can potentially save years of sight for some people with retinitis pigmentosa (RP). Although this is not a cure, it is a first step in managing RP. Equally important, this trial shows it is possible to influence the course of RP. lncluded is a comprehensive list of questions and answers regarding the vitamin A palmitate treatment. This six-year, $5 million study was conducted at the Berman-Gund Laboratory for the Study of Retinal Degenerations of Harvard Medical School with funding by The Foundation Fighting Blindness and National Eye lnstitute, National lnstitutes of Health.

Published in the June 1993 issue of Archives of Ophthalmology, the study concludes that most adult patients with RP should take 15,000 lU of vitamin A daily and avoid high doses of vitamin E. However, be sure to consult with your
ophthalmologist before beginning this treatment. While vitamin A may help many, it is not a cure. The Foundation Fighting Blindness
continues an accelerated research strategy to pursue other treatments -and ultimately, cures -for RP and other retinal degenerative diseases.

Sincerely,
The Foundation Fighting Blindness
Communications Department
lnformation & Referral

ln 1993, Dr. Eliot Berson and colleagues at The Foundation’s Research Center, the Berman-Gund Laboratory for the Study of Retinal Degenerations of Harvard Medical School, published results from a clinical trial, which found that a daily dose of 15,000 lU of vitamin A palmitate on average slowed the course of common forms of retinitis pigmentosa (RP) including Usher syndrome type ll. This clinical trial heralded vitamin
A as the first sight-saving treatment for RP.

Recently, Dr. Berson and colleagues published results of a 12-year follow-up study evaluating the long-term safety of vitamin A in 146 patients with RP who participated in the original vitamin A clinical trial. These patients were otherwise healthy and between the ages of 18 and 54. The daily doses of vitamin A palmitate for this group of patients ranged between 16,349 lU and 24,918 lU.

After 12 years of supplementation, vitamin A blood levels increased by 18 percent. However, all the patients in this follow-up study had blood levels of vitamin A within the normal range. The patients also showed no clinical symptoms or signs of liver toxicity attributable to vitamin A. The study authors conclude, “Prolonged daily consumption of less than 25,000 IU of vitamin A can be considered safe for that time and amount in
this age group.”

While the long-term safety of vitamin A palmitate in healthy adults is now better established, Dr. Berson recommends that before beginning the vitamin A therapy, patients consult their physician to review their medical history and current health status. As a precaution, patients should also have blood tests to measure vitamin A levels and liver function tests prior to beginning the treatment and annually thereafter.
lf liver function tests reveal any abnormalities, cessation of vitamin A would be expected to prevent any possible complications due to vitamin A toxicity. This long-term safety study, published in the April 1999 American Journal of Clinical Nutrition, now provides patients and doctors with more complete medical information regarding the safety of the vitamin A treatment for RP.

lf you have questions regarding the vitamin A treatment, The Foundation publishes a free booklet entitled, Vitamin A treatment for Retinitis Pigmentosa. The Foundation also distributes a list of mail order companies that provide vitamin A palmitate in
15,000 lU capsules.

The Foundation Fighting Blindness
11435 Cronhill Drive
Owings Mills, MD 21117-2220
800-683-5555 800-683-5551 (TDD for deaf)
410-568-0150 410-363-7139 (TDD for deaf)
www.blindness.org

STUDY SHOWS VITAMIN A SLOWS RP

Most adults with blinding retinitis pigmentosa (RP) should take a daily 15,000 lU vitamin A palmitate supplement and avoid high dose vitamin E to help prolong their vision, based on results from a large randomized clinical trial published in the June 1993 issue of Archives of Ophthalmology. This recommendation is the first from a well-designed clinical trial indicating that people can be treated for RP. Alan M. Laties, M.D., chairman of The Foundation Fighting Blindness’ Scientific Advisory Board, said of these findings, “This treatment can be a benefit, a very real one, for people who have RP. Although not a cure, it will improve quality of life, potentially adding many years of useful vision. The
discovery that vitamin E is of no use, and in fact potentially harmful, is not only important on its own, but further justifies this extensive study.”
Eliot L. Berson, M.D., the study’s principal investigator and Professor of Ophthalmology at Harvard Medical School, said that adults who supplemented their diets with 15,000 lU of vitamin A palmitate daily had on average about a 20 percent slower annual decline of remaining retinal function than those not taking this dose. Based on this finding, the investigators estimated that an average patient in the
study, who started taking a daily supplement of 15,000 lU vitamin A palmitate at age 32, would retain some useful vision until age 70, whereas a patient not on this dosewould lose useful vision by age 63.

The investigators also recommended that adults with RP should avoid taking high- dose vitamin E supplements. ln the study, the disease appeared to progress faster on average in patients on a daily 400 lU vitamin E supplement than in those taking a trace amount of the vitamin. However, the study showed no evidence that normal dietary or small supplemental amounts of vitamin E have an adverse effect on the
disease. RP is a group of inherited diseases that usually starts with night blindness, followed by progressive loss of peripheral vision. Over a period of years this results in “tunnel vision,” and may lead to total blindness. lt causes the progressive deterioration of specialized, light-absorbing cells in the retina, the paper-thin tissue that lines the back of the eye like film in a camera. The majority of people with RP are legally blind by the age of 40, with a central visual field of less than 20 degrees in diameter. An estimated 1.5 million people worldwide are affected by RP.
The carefully designed and monitored clinical trial involved 600 patients with RP between the ages of 18 and 49, who were at different levels of visual function. The six-year study was conducted at a cost of $5 million at the Berman-Gund Laboratory for the Study of Retinal Degenerations at Harvard Medical School, with the support of The National Eye lnstitute and The Foundation Fighting Blindness. The investigators stressed that adults considering vitamin A palmitate supplementation should first:

* Consult with their doctor about vitamin A palmitate supplementation.

* Have fasting blood levels of vitamin A measured and liver function
tests administered before starting treatment. People with certain preexisting
medical conditions may not be eligible for this treatment.

* Take vitamin A palmitate, since this form of the vitamin was used in the
study. Beta-carotene, a natural precursor of vitamin A, is not a
predictable source of vitamin A and therefore is not a suitable
substitute for vitamin A palmitate.

* Make a point of eating a balanced diet, without selecting foods that are
especially high in vitamin A.

* Avoid taking high-dose vitamin E supplements.

Commenting on the results of the vitamin A study, Dr. Berson stated, “One of my biggest concerns is that people will make the mistake of thinking that vitamin A supplementation in excess of the 15,000 lU recommended will provide even greater benefit. We have evidence in fact that supplementation of a regular diet with greater than 15,000 lU of vitamin A does not provide greater benefit. Moreover, a daily vitamin A intake exceeding 25,000 lU over the long-term can be toxic in adults and may cause side effects such as liver disease’ “Our interpretation of the study results is that the course of the common forms of retinitis pigmentosa is slower on average among adult patients on a regular diet who
take a daily 15,000 lU vitamin A supplement in the palmitate form compared with the course of those patients not on this supplement.”
Because the study involved adults between the ages of 18 and 49, no formal recommendations can be made for patients under the age of 18. Also, women with RP should not take 15,000 lU vitamin A palmitate supplements during the time leading up to planned pregnancy and during pregnancy, because high doses of vitamin A have been linked to birth defects.

Questions and Answers About Vitamin A Palmitate Supplementation as a Long-Term Treatment for RP

1. ls this a treatment for RP?

Based on the results of a six-year study of 600 patients with typical retinitis pigmentosa (RP), researchers concluded that, on average, the course of retinal degeneration was slower among patients receiving a daily vitamin A palmitate supplement (15,000 lU) than was the disease course among those not taking this dosage. There was about a 20 percent slower rate of decline of remaining retinal function (8.3 percent) per year in the 15,000 lU dose group versus the control (trace dose) group (10.0 percent), as monitored by electroretinographic (ERG) amplitudes.
The results also suggested that the course of disease mignt be faster on average among patients receiving a daily high dose supplement of vitamin E (400 lU) than among those receiving a trace dose of vitamin E. lt is recommended that most adult patients with the common forms of RP take a daily 15,000 lU supplement of vitamin A palmitate under the supervision of a doctor and avoid the use of high dose
supplements of vitamin E, such as 400 lU.

2. ls this a cure?

This treatment may decrease the total loss of vision over a lifetime and thereby delay blindness. Adults who use this treatment have on average about a 20 percent slower annual decline of remaining retinal function than those not using this treatment. While this does not represent a cure, it does represent a first step in treating typical RP. The patient must realize that this study did not find improved vision from one year to the next, and did not find that vision loss was completely arrested. Research aimed toward the development of additional treatments, a cure,
and prevention actively continues.

3. What types of RP may be helped by this treatment?

The clinical treatment trial of vitamin supplementation studied patients with typical forms of RP, including the inheritance types of X-linked, autosomal dominant, and autosomal recessive, as well as isolated RP and Usher syndrome type ll. Several other forms of RP were not included in the study because there was no preliminary evidence to suggest that vitamins could modify the course of these forms. Diseases
not included in this study were Usher syndrome type l, sector RP, cone-rod dystrophy, unilateral RP, Bardet-Biedl syndrome, choroideremia, gyrate atrophy, Leber congenital amaurosis, Refsum disease, and other less common syndromes that involve RP. Also, patients were not evaluated in this study if they had very advanced RP (less than 8 degree visual field with a large test light; if they were below the fifth percentile of normal with respect to body weight for a given age, sex, and height; if they had impaired liver function; or if they were under age 18. Therefore, formal recommendation with respect to vitamin A palmitate supplementation cannot be made for these groups of patients. They should be
assessed individually by their doctors.

4. Was any type of macular degeneration included in this study?

No. This study was limited only to typical RP. Macular degeneration, whether the juvenile onset or late onset age-related forms, was not included in this trial. ln fact, because macular degeneration has different symptoms than RP, it might also be caused by different disease processes than RP. Recommendations regarding vitamin supplementation for patients with macular degeneration will have to await completion of other studies now being conducted through other large multi-center projects supported by the US government.

5. Can children who have RP be given supplements of vitamin A palmitate?

Because patients under the age of 18 were not evaluated in this study, no formal recommendation can be made. Patients under 18 years of age should consult with their ophthalmologist and pediatrician for possible vitamin A palmitate treatment with a dose that takes into account the age, weight, general health status, and published guidelines for the safe use of vitamin A palmitate. A 15,000 lU daily supplement of
vitamin A is well above a child’s recommended daily allowance and could be toxic. Your child’s doctor might recommend that your child take a daily multiple vitamin capsule that contains the recommended daily allowance for children.

6. Tell me more about this study on vitamin A. Why was it done?

This study was a prospective, double-masked clinical trial, meaning neither patient nor clinician knew to which treatment group the patient had been randomly assigned. lt was designed to assess the effectiveness of vitamin A and E supplements in halting or slowing the progression of RP. An independent data and safety monitoring committee provided advice to the investigators and monitored the
accumulating data on a regular basis for evidence of harm or benefit to study participants. A total of 600 patients affected with typical RP from across the United States and Canada were enrolled in the study. All were in good general health, between the ages of 18 and 49, and entered the study with different levels of retinal function.

Following an examination, participants were assigned randomly to one of four treatment groups:

1) 15,000 lU vitamin A plus 3lU vitamin E
2) 75 lU vitamin A Plus 3 lU vitamin E
3) 15,000 lU vitamin A plus 400 lU vitamin E
4) 75 lU vitamin A plus 400 lU vitamin E

Thereafter, the researchers examined each patient annually, with an average follow-up of 5.2 years. The electroretinogram (ERG) was used as the primary measure to follow the course of the disease. The ERG is a light-evoked electrical response from the retina that can be accurately measured. Much like an electrocardiogram measures heart function, an ERG provides an objective measure of retinal funition.

7. where can I get a copy of the full report on this study?

The report from this clinical treatment trial was published in the June l993 issue of the medical science journal Archives of Ophthalmology, pages 761-772. You and your doctor should refer to this article for a complete presentation and discussion of the results. This journal is available through medical school and university libraries. Your local library may be able to obtain a copy of the article for you. An abridged version
of this study is reprinted in the back of this booklet.

8. Do the results of this study imply that vitamin A deficiency or an excess of vitamin E was the cause of my RP?

No, vitamin A and E levels present in the blood of unaffected and affected individuals do not differ significantly. Scientists have discovered that the actual cause of most typical RP is a mutation in one of several important genes, which then produces a defective protein in photoreceptor cells (rods and cones). lt is well known that vitamin A is critical to normal retinal function. However, the precise
benefit vitamin A provides is unknown.

9. What is vitamin A?

Vitamins are relatively complex organic substances that are not made by the human body. They are required in small amounts from a balanced diet in order to sustain normal metabolism and good health. Diseases caused by extreme vitamin deficiency can usually be cured when the lacking vitamin is supplied. Vitamin A is essential to normal cell growth and development. lt plays an important role in visual function. Severe nutritional deficiency of vitamin A can cause disease (especially of the eye) and degeneration of mucous membranes.

10. How does supplemental vitamin A palmitate preserve vision for RP patients?

We don’t know the answer to that question yet. One well-defined function of vitamin A is in vision. lt is intimately involved in the cascade of events triggered by light reaching the retina and culminating in delivery of an image to the brain. A second major role of vitamin A is during the developmental stages of life when simple cells change into specific types of cells like those in the retina. A characteristic of the typical forms of RP is death of the specific, light-sensing cells in the retina, the photoreceptor cells called rods and cones. lt is reasonable to hypothesize that high
levels of vitamin A preserve these dying cells in some way. lt is also possible that some patients with RP have a reduced capacity to retain vitamin A in the retina due to the condition of the sick, degenerating rods and cones. Likewise, such cells may have an abnormal carrier protein causing vitamin A to be transported from the blood to the retina with lower efficiency. Whatever the explanation, it appears that vitamin A supplements may provide partial protection against cell degeneration. Research will continue to seek an understanding of this effect, with the hope of further
enhancing its positive actions.

11. Why should I avoid high dose supplements of vitamin E?

On average, study patients who took 400 lU of vitamin E daily as a supplement appeared to experience close to a 20 percent faster rate of visual function decline per year than those who received only a trace amount of vitamin E (11.8 percent compared to 10.0 percent). ln other words, the average patient taking 400 lU of vitamin E could expect to lose visual function several years sooner. lf vitamin E supplements, without vitamin A, were started by the average patient at age 32, legal blindness would be accelerated by as much as five years. However, the study found
no evidence that normal dietary or small supplemental amounts of vitamin E have an adverse effect on RP.

12. How could vitamin E cause a negative effect on RP?

It is possible that a daily high dose of vitamin E might affect the course of RP at least in part by inhibiting the absorption or transport of vitamin A. ln the study, it was observed that patients receiving 400 lU vitamin E had slight but significant decreases in blood vitamin A concentration compared with those not receiving that dose of vitamin E.

13. What if my doctor advised me to take vitamin E for other reasons?

There is some early information from nutritional studies suggesting that supplemental vitamin E could be helpful in preventing heart disease. lf your doctor has advised you to take vitamin E for that reason, you should ask to discuss the relative benefits and risks of taking vitamin E, or taking vitamin E along with vitamin A, given your general health and your eye disease.

14. What is the benefit of vitamin A palmitate for the “average” individual who has RP or Usher syndrome type ll?

ln this clinical treatment trial, patients were examined annually for four to six years. Visual function was assessed by an ERG (electroretinogram). Those who took daily vitamin A supplements of 15,000 lU had close to 20 percent slower loss of remaining visual function each year than others in the study. lf the average patient started vitamin A supplements at age 32, legal blindness could be delayed by as much as seven years. However, researchers cannot provide assurance that every patient will benefit from treatment with vitamin A palmitate.

15. To what extent might my vision be saved?

Because the results of this study are based on group averages, individual outcomes cannot be predicted. There are considerable differences in the severity of the disease among individuals at the same age, even within the same family. This study pooled the information from 600 patients and conclusions were based on “group averages.” Actually, there are probably very few patients who exactly fit the “average” description. Some will have better vision and some will have worse vision than the average study patient. lt isn’t possible to tell you the exact extent of benefit
you will receive from this treatment. Some patients may not be helped at all; some may be helped more than others.

16. But what does that say about me?

The best we can answer is that you may have a high likelihood of benefiting from vitamin A treatment. This treatment slows the rate of a degenerative process, but does not stop it. For some patients it may prevent total blindness, while others may
not be helped at all.

17. Will I notice any change in my vision when l take vitamin A?

It is very unlikely that you will notice an improvement in your vision compared to any time in the past. The degenerative process will continue, although at a slower rate. Based on the results of this study, it is predicted that patients taking vitamin A over the long-term would have a higher probability of retaining the capacity to perform certain daily activities at a given age than patients not taking the vitamin A
treatment.

18. lf I take vitamin A long enough, or start taking vitamin A as a young adult, will I be cured?

Vitamin A supplementation is not a cure for RP. “Cure” implies that health has been restored to normal, and a cure for RP would imply restoration of lost vision. Vitamin A supplementation does slow the rate of visual function loss, therefore it is expected that supplementation begun in early adulthood may be more beneficial in the long-term than if begun later. For some patients, this might result in some amount of
retained central vision for life.

19. Should l take vitamin A if l am already legally blind?

This study did not evaluate patients with extremely low-vision because ongoing measurements of change in vision would have been difficult to assess in those patients. lf you now have very advanced RP, you should consult with your ophthalmologist for advice about the possible benefit of vitamin A for you.

20. What should I do to begin vitamin A treatment?

Consult with an ophthalmologist, a medical doctor who specializes in eye care. Do not start taking vitamin A supplements on your own. Your ophthalmologist will want to administer blood tests to measure your fasting blood levels of vitamin A and to assess your liver function. Other standard laboratory tests may also be done before starting your treatment. These tests are needed to assure that you do not have a
pre-existing medical condition that would prevent you from taking this treatment. lf blood tests indicate you already have abnormally high levels of vitamin A, your doctor may need to adjust your vitamin A intake accordingly. lf you are not going to an ophthalmologist now, we recommend that you find one who is willing to advise you regarding your eye care.

21. How often will I need to be examined while taking vitamin A?

It would be a good practice to visit your doctor annually. Annual blood tests to measure fasting levels of vitamin A will help to insure your are receiving the appropriate intake of vitamin A as required by the study over the long-term. Liver toxicity is a potential complication of daily doses of vitamin A in excess of 25,000 lU, so blood tests to reassess liver function should be done annually. As reported in the study, no significant chronic or acute toxicity of any kind was found in the groups taking vitamin A. Surveys of the medical literature find no reported toxicity among
patients in good general health who are taking daily doses of 15,000 lU of vitamin A.

22. Is it safe to take 15,000 lU of vitamin A for many years?

No evidence of systemic illness or toxicity attributable to the daily intake of vitamin A palmitate capsules (15,000 lU) used in the clinical treatment trial could be established during the six-year study based on blood tests, urinalyses, patient responses to a symptom questionnaire, and, in some cases, examination by a consulting internist. Nevertheless, it is extremely important that your own condition be monitored by a physician on a regular basis while you are taking vitamin A for extended time periods that will probably exceed the six year term of this study. We
cannot exclude the possibility of the development of side effects. Therefore, to monitor treatment, we recommend that patients have a yearly evaluation by their doctor.

23. What are the possible side effects of taking too much vitamin A?

For normal, healthy adults the US Recommended Daily Allowance (US RDA) for vitamin A is 5,000 IU. The recommended daily dose for most adult patients with RP is 15,000 lU from capsule supplements, in addition to a regular balanced diet, which typically provides 3,000-4,000 lU per day. This totals approximately 18,500 lU per day of vitamin A. Regular long-term intake in excess of 25,000 lU has been
associated with liver toxicity. Side effects and complications from very high doses include liver toxicity, persistent headache, joint or bone pain, appetite loss, weight loss, nausea, vomiting, unusually dry skin, increased hair loss, and fatigue. When taken during pregnancy, high-dose vitamin A has also been associated with birth defects.

24. Should I be concerned with alcohol consumption while taking vitamin A?

It is well known that excessive alcohol consumption can cause liver toxicity. However, no systematic studies have been done in patients taking 15,000 lU of vitamin A to see if excessive alcohol consumption would be more likely to produce liver toxicity. lt is therefore advisable to limit alcohol consumption to no more than two drinks per day (one drink is equivalent to 12 oz. beer, 4 oz. of wine, or 1 ounce
of hard liquor).

25. What about cigarette smoking and vitamin A?

A recent study found that current and former heavy smokers (i.e., two packs a day for 25 years) who took 25,000 lU of vitamin A and 30 mg of beta-carotene had 28 percent more lung cancers and 17 percent more deaths than those current and former heavy smokers who did not take these supplements. This percentage increase in lung cancers is equivalent to 2 additional cases per year for every 1000 smokers. During the 4 to 6 year course of the vitamin A clinical trial for RP, there were no reported cases of lung cancer among study subjects who took 15,000 lU of
vitamin A. lf you are a current or former heavy smoker, please share your smoking history with your physician. lt should be stressed that there are no reports in the medical literature of vitamin A increasing the risk of lung cancer in non-smokers.

26. Should I continue this treatment if I become pregnant or plan to become
pregnant?

Women who are pregnant or are planning to become pregnant should not take high doses of vitamin A because of the potential for birth defects. Women should discuss the impact of vitamin A treatment on childbearing with their doctor. You should stop taking vitamin A before a planned pregnancy. Women who become pregnant should immediately discontinue this dose of vitamin A palmitate during the entire
pregnancy. However, you should take prenatal vitamins as prescribed by your obstetrician. Patients should consult with their doctor about taking this dose while breast-feeding.

27. If l’m planning to become pregnant, when should lstop taking vitamin A?

There is no scientific data to tell us when you should stop, but if you are planning to become pregnant, you should discontinue this regimen.

28. How can I find a doctor who is familiar with RP?

The Foundation Fighting Blindness sponsors 18 Research Centers, many of which are in the US and can provide access to ophthalmologists who specialize in retinal degenerative diseases. The Foundation also offers a nationwide list of retinal specialists to help individuals who are outside the geographic range of The Foundation’s Research Centers. This referral list was compiled by contacting all vitreo-retinal specialists who are members of the American Academy of Ophthalmology. All those listed indicated interest in seeing individuals with inherited retinal degenerative diseases. Please be aware that The Foundation has not screened these ophthalmologists to judge their training, experience or standing in
the medical community and cannot recommend one in preference to another. Also, The Foundation cannot be held responsible for any services given by referral specialists or for the fees that they may charge. ln conjunction with this list, you may wish to consult with your general ophthalmologist, optometrist or family doctor for his or her recommendation. You may also wish to consult with your state department of
rehabilitation, local medical society, university medical school, or major hospital ophthalmology clinic for a referral to a retinal specialist.

29. Will my health insurance pay for annual appointments and tests

You will need to check with your individual health insurance carrier. Coverage of costs will depend on the individual insurance plan being used. Medicaid and Medicare coverage might be available, but will probably vary from state to state. When checking with your health insurance carrier, you should indicate that you are receiving treatment based on the results of a clinical trial endorsed by the National Eye lnstitute.

30. What do I need to know in order to buy the right capsules of vitamin A?

For normal healthy adults the US RDA of vitamin A is 5,000 lU, while the recommended daily dose based on the results of this clinical treatment trial is 15,000 lU vitamin A palmitate per day for most adults with RP. The designation “lU” stands for lnternational Unit. “lU” is a standardized measurement that quantifies the amount of the specific substance present in the capsule. When you look for such a supplement, labels should be read very carefully to be sure the capsules contain vitamin A palmitate, and not another component like beta-carotene. Beta-carotene is a natural precursor of the active form of vitamin A. Very high levels of beta-carotene are needed to achieve activity equivalent to 15,000 lU of vitamin A palmitate. The final activity could vary from person to person because people metabolize beta- carotene with different efficiencies. Therefore, beta-carotene is not a predictable source of the vitamin and is not recommended in context to this study. Vitamin A in the palmitate form was used in this clinical treatment trial and the recommendations derived from the study apply specifically to this form.

31. Do I need a prescription to get vitamin A supplements?

No.

32. where can l obtain capsules of 15,000 lU vitamin A palmitate?

The Foundation keeps an updated list of vitamin A palmitate suppliers.

33′ Why can’t I simply take three “one-a-day” multiple vitamin capsules each day? Wouldn’t I get the right dosage of vitamin A?

Because these capsules contain many vitamins and other components at 100 percent of the US RDA, three one-a-day multiple vitamin capsules a day are not recommended. ln addition, each of these multiple vitamins includes the US RDA of vitamin E, which appears to interfere with the beneficial action of vitamin A for RP taken in high doses.

34. Can I adiust my daily dose so that I can make use of the vitamin A capsules of lower or higher dosages that are sold at my local stores?

At present in the US, locally available preparations of vitamin A are provided in 10,000 lU and 25,000 lU capsules as either a water soluble base, in the acetate form, or concentrated from fish liver oil. These vitamins cannot be recommended because they were not part of the study, do not contain the appropriate dosage and may not contain the vitamin in the palmitate form. ln the study, optimal benefit occurred among patients with a total daily intake of 15’000 lU of vitamin A palmitate in capsule form and 3,000 lU vitamin A from diet. Lower intake gave less benefit and higher intake up to 25,000 lU provided no greater benefit. Toxic side effects have been associated with long-term intake of 25,000 lU and therefore, capsules of 25,000 lU should be avoided. Combinations to achieve an average of 15,000 lU per day over several days (for example, 3 capsules
of 10,000 lU every two days) are not recommended because it would be hard to take three capsules evenly spaced over a two day period and you could risk temporarily high blood levels of the vitamin. Remember, more vitamin A than the recommended dose is not better. Moreover, daily vitamin A intake exceeding 25,000 lU over the long-term may be toxic in adults and may cause side effects such as liver disease.

35. when should I take the capsules and how should I store them?

You might find it useful to develop a routine of taking one capsule each day right after breakfast. These vitamin A capsules have more than a one-year sheli life. On the advice of your doctor, you could order a full year supply (four-bottles of 100 each) and store them at room temperature. Keep capsules out of the reach of children.

36. Should I be concerned about getting the right amounts of vitamin A and vitamin E in my diet?

The average American diet provides approximately 3,000-4,000 lU of vitamin A per day. ln the clinical treatment trial, the actual average intake for the patients was 3,OOO lU per day. This daily dietary amount was taken into account for the final recommendation of a supplement of 15,000 lU vitamin A palmitate per day as a treatment for RP. You should maintain a regular, balanced diet, following the standard guidelines for good nutrition developed by the US Department of Agriculture (USDA). You should NOT try to specifically select foods that are high in vitamin A content. Also, you should NOT try to eliminate foods that are high in vitamin E content.

37. ls there anything else I can do besides maintaining a balanced diet and taking vitamin A to preserve my vision?

At this time, no other approaches for preserving vision affected by RP have been proven useful and safe through a carefully controlled clinical treatment trial. Some doctors advise their patients io protect their eyes from bright light. As a precaution, individuals with RP are encouraged to protect their eyes from long-term exposure to bright sunlight until more is learned. Good quality sunglasses are useful for bright
days outdoors.

38. Should I be encouraged by the discovery of this treatment?

This breakthrough is a concrete reward for the many years of research that preceded it. Daily vitamin A supptementation is the first discovered means to partially control RP that has proven useful and safe for most patients. This clinical treatment trial has demonstrated that ways to slow vision loss can be proposed and tested, and is an extraordinary example of carefully designed work. lt was done under close monitoring for appropriate benefit and avoidance of risk to patients. lt will serve as a model for future studies. Meanwhile, until a cure is found, some years of vision may be saved for some patients affected with RP’

39. Will other research on RP stop now that this treatment has been found?

No. This treatment may preserve visual function in certain patients with RP while the search for additional ahd improved treatments and the means to cure or prevent RP continues. Studies on nutritional supplements represent only one aspect of the overall scientific strategy. Currently, funding is provided by The Foundation for over 100 research projects done at 13 dedicated RP Research Centers and other universities and research institutions worldwide. The National Eye lnstitute devotes additional federal funds to this topic. This disease must be understood at the most basic cellular level in order to understand how it is caused and how it can be stopped. Research in biochemistry and cell biology, as well as molecular genetics and clinical studies, will lead to the design and testing of other treatments that may stop the progression of the disease and ultimately prevent it from ever occurring.

40. Can I volunteer to participate in a ctinical treatment trial in the future?

The Foundation maintains a computerized National Registry to identify and collect medical and family histories on all people in the US with retinal degenerative diseases. Participants who complete a Registry questionnaire may give permission to The Foundation to release their names to researchers for Foundation-approved studies. Many patients asked to participate in future clinical studies will be identified through the National Registry. Registry forms are available through The Foundation. ln addition, patients seen regularly at a Foundation Research Center may be
included in certain clinical studies at that center.

Disclaimer

Retinal degenerations are very difficult diseases to describe because each case is
so different. That,is why the information presented in this booklet is offered only as
general information and as referral to other sources for complete information. lf you
suspect you have retinal degeneration, or any other eye problem, it is best to consult
with an ophthalmologist, a physician who specializes in the care of the eye. lf you
think long-term treatment with 15,000 lU vitamin A palmitate may apply to your
condition, you should consult with an ophthalmologist before taking such high dose
supplements. The Foundation Fighting Blindness can not be held responsible for
any individual’s decisions or actions regarding eye health care.

Vilamin A Supplementation for Retinitis Pigmenosa Eliot L. Berson, M.D.
Summary of paprer published in Archives of Ophthalmology, June 1993 Page I of 3

Randomized Trial of Vitamin A and Vitamin E Supplementation
for Retinitis Pigmentosa

Study Design
Randomizcd, controlled, double-masked tial

601 adult Patients, ages 18-49

Facorial desisn -four treatment groups:
15,000 IU of A + 3 IU of E (Group A)
75 IU of A + 3 IU of E (Group Trace)
15,000 IU of A + 400 IU of E (Group A+E)
400 IU of E + 75 IU of A (Group E)

Mean outcome variable: 30Hz Cone ERG

Two pre-treatment examinations at 6-week interval:

Screening to establish eligibility; baseline to determine intervisit variability
Average of screening and baseline visits to provide pretreatment values

Groups balanced with respect to genetic type and pretreatment intake of A and E

Annual follow-up: ERGs,visual fields,visual acuities
Compliance: serum, capsule counts, calendars
Outside Data safety and Monitoring Committee appointed by the National Eye Institute

EligibilityCriteria

Common forms of retinitis pigmentosa; ages 18-49; one/family
Visual Acuity >20/100; visual field 28o diameter to V4e, white test light
30 Hz ERG>0.12microvolt or 0.5 Hz ERG > 2.5microvolt (Norms: 30Hz>50microvolt; 0.5Hz > 350microvolt)
Total intake: vitamin A <11,500IU/day; vitamin E<40IU/day
Normal serum retinol ( Not pregnant or planning to become pregnant
Weight above lower fifth percentile for age, sex, and height
Agree not to know content of supplepent or group assignment

Masking

All members of the staff in contact with the patients were masked as to the treatment groupassignment of each Patient

Each ocular examination and ERG was performed without review of previous records
All serum samples were analyzed without knowledge of treatment group assignment
Patients did not know the contents of the supplement or their group assignment
Patients agreed not to know the course of their retinal degeneration until the end of the
study

Written by JamesM2070

Neurotech SA announced positive results from an open-label Phase I clinical trial (03-EI-0234) of its lead product, NT-501. NT-501 uses Neurotech’s patented Encapsulated Cell Technology (ECT) as a device to deliver ciliary neurotrophic factor (CNTF) to eyes of visually impaired patients with retinitis pigmentosa (RP). Neurotech is a biotechnology company specializing in the development of novel therapeutics to treat diseases of the eye. The company is headquartered in Paris.

Results of the latest clinical trial confirm that CNTF can be safely delivered into the vitreous of patients with RP and that the ECT device was well tolerated by all patients. Futhermore, some patients experienced more than one-line of improvement in their visual acuity score. These Phase I results were presented at the ARVO annual meeting and the trial was conducted at the National Eye Institute (NEI), Bethesda, USA. Neurotech has confirmed that it will now progress to a multi-center Phase II trial.

ECT, a technique developed and patented by Neurotech, allows for genetically-engineered specific protein delivery without manipulating the patient’s genetic information or transferring new genetic information into the target tissue. The Phase I study of NT-501 involved 10 patients with late-stage RP. The study was designed as an open-label safety and tolerability evaluation. Two doses of CNTF (5-fold difference in dose) were evaluated. Phase Ia treated 5 patients with a lower dose; Phase Ib treated 5 patients with a higher dose. The ECT device was implanted in one eye per patient and removed after six months. All explanted devices contained viable cells that continued to produce CNTF.

Commenting on the positive results, Weng Tao, MD, PhD, CSO and VP of R&D with Neurotech said:

“These safety and tolerability results are extremely encouraging and strengthen our confidence in pursuing NT-501 for treating RP and other retinal degenerative diseases. I would like to thank the National Eye Institute for conducting this milestone study and for its continued involvement with this technology.”

Al Reaves, PhD, VP of Clinical Development with Neurotech confirmed: “This study represents the first use of ECT in human eyes and it is reassuring that the devices were safe and well-tolerated. We are planning Phase II development with well-designed and controlled multi-center clinical studies to help understand the role that NT-501 will play in treating patients with retinitis pigmentosa and other retinal degenerative conditions.”

In this trial, the small ECT device was surgically-implanted into the vitreous cavity through the pars plana in one eye per patient. The primary inclusion criteria for the study eye included visual acuity of 20/100 or worse, central visual field diameter of 40 degrees or less, and flicker ERG amplitude of 2 �V or less. The first 2 patients were also required to have visual acuity of 20/400 or worse. After surgical implantation of the device, each patient was followed for six months after which the device was explanted. Safety and tolerability was monitored by an independent Data and Safety Monitoring Committee. All 10 patients completed the study as planned and the devices have been explanted. The ECT devices were well tolerated during the 6 months of implantation and the surgical procedure resulted in minimal or no observed inflammatory reaction. No serious adverse events were reported and in the untreated fellow-eye, there was little change from baseline in the visual acuity score during follow-up. In the treated study-eye, however, the visual acuity score was more variable during follow-up: while some treated eyes showed little change from baseline, some patients experienced more than one-line of improvement in their visual acuity score.

In addition to NT-501 for the treatment of Retinitis Pigmentosa, Neurotech is applying ECT technology to deliver other protein factors for the treatment of other ophthalmic diseases, including anti-angiogenic factors for the treatment of the wet form of age-related macular degeneration (AMD) and diabetic macular edema (DME), and anti-inflammatory factors for the treatment of posterior uveitis.

1. About Retinitis Pigmentosa (RP)

RP is a group of inherited retinal diseases that affects about 100,000 Americans and 1.5 million people worldwide. It causes the progressive deterioration of specialized, light-absorbing cells in the retina, the paper-thin tissue that lines the back of the eye like film in a camera. As these cells slowly degenerate, people with RP develop night blindness and a gradual loss of peripheral vision. By about age 40, most have tunnel vision, although many may retain good central vision. Between the ages of 50 and 80, however, they typically lose their remaining sight. The extent of vision loss in people of the same age with RP may be different.

2. About Encapsulated Cell Technology (ECT)

ECT is a unique technology to overcome drug delivery problems into the back of the eye. ECT enables the controlled, continuous, long-term delivery of therapeutic proteins directly to the retina. In addition, the implants can be retrieved, providing an added level of safety as well as the ability to reverse or adjust therapy, if needed. ECT relies on the use of an immunoisolatory hollow fiber membrane technology to allow the implantation of genetically engineered cells that continuously produce the therapeutic protein at the site of implantation. The cells are loaded into the interior volume of the hollow fiber membrane and attach to a proprietary supportive matrix within this space. The genetically modified cells remain captive within the ECT device thus avoiding the risks associated with traditional gene therapy. Long term protein delivery (18 months) in the vitreous cavity of the eye has consistently been achieved when ECT devices containing human retinal pigmented epithelial (RPE) cells genetically engineered to secrete CNTF have been implanted in a highly disparate mammalian species (rabbits).

3. About Neurotech

Neurotech is a biotechnology company specializing in the development of novel therapeutics to treat diseases of the eye. The company’s initial focus is on chronic diseases affecting the back of the eye, especially the retina, because retinal diseases represent the greatest unmet medical need and therefore offer the largest market opportunities in ophthalmology. The Company has one product (NT-501) in development for the treatment of retinitis pigmentosa and other degenerative diseases of the retina, and is evaluating other neurotrophic factors and agents that can be used with ECT for treating other retinal diseases. The company is headquartered in Paris with an American subsidiary, Neurotech USA, Inc., located in Lincoln, RI, south of Boston. Neurotech is supported in its scientific and business strategies by world experts in ophthalmology and by a group of international investors led by Apax Partners and Merlin Biosciences.

Reference URL

http://www.neurotech.fr

http://www.alphagalileo.org

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