Listen to this page using ReadSpeaker

VISIONS 2015 – Dr. José Sahel Receives Foundation’s Most Prestigious Research Honor

Dr. SahelI’ve known Dr. José Sahel for more than a decade, and every time I’m with him, I’m impressed by his humility and graciousness. He’s not much for rhetoric or small talk, but is always polite and insightful. Dr. Sahel is also very soft-spoken, but I think that’s his secret weapon. He forces you to really listen to what he’s saying.

You’d never imagine that someone with such a pleasant demeanor would be leading one of the most productive and innovative retinal-disease research centers on the planet, the Institut de la Vision in Paris. Not only am I surprised that he keeps an even keel with all he has going on; I can only wonder when he finds time to eat or sleep.

So, I was privileged to present Dr. Sahel with the Foundation’s highest research honor, the Llura Liggett Gund Award, at VISIONS 2015, the Foundation’s annual conference. As Ed Gollob, the president of FFB’s board of directors said during the presentation, “The recipients of the award are the best of the best.” Dr. Sahel is only the ninth investigator to receive the award during the Foundation’s 44-year history.

When Dr. Sahel isn’t eating or sleeping, he’s leading approximately 250 investigators and overseeing 25 clinical trials, including one for UshStat®, a gene therapy for Usher syndrome type 1B, and another for StarGen™, a gene therapy for Stargardt disease.

Dr. Sahel is also a scientific founder of Pixium, a company developing three bionic retinas, one of which is in a human study, and another more advanced device, still in preclinical development. Known as the PRIMA, it’s designed to provide high-resolution vision, including facial recognition.

If that isn’t enough, Dr. Sahel is also a scientific founder of the company GenSight. It’s developing optogenetic treatments — innovative gene therapies which bring light-sensitivity to retinas that have otherwise stopped working.

Also, Dr. Sahel, Dr. Thierry Léveillard and his staff are developing a neuroprotective gene therapy that shows promise for keeping retinas affected by a wide range of diseases alive and functioning. Known as rod-derived cone viability factor, or RdCVF, it works like a drug factory to provide sustained release of a vision-saving protein to the retina. This project is a long-standing collaboration between Drs. Sahel and Léveillard that the Foundation has supported for many years.

The secret to Dr. Sahel’s ability to manage so much research is his talent for fostering collaboration and motivating his colleagues. He leads selflessly. His focus is always on the project. For those of us supporting the drive for vision-saving treatments and cures, he’s exactly the type of person we want on our team.


26 Responses to 'VISIONS 2015 – Dr. José Sahel Receives Foundation’s Most Prestigious Research Honor'

  1. Respected dear sir
    I am currently working in bahrain
    my father is suffering retinitis pigmentosa so I would like to know about its latest treatment
    thanks best regards
    nadim
    0097333081690
    kingdom of Bahrain

    • EyeOnTheCure says:

      The Foundation Fighting Blindness continually monitors the research on degenerative retinal disease and new developments are posted on the FFB website. Please also see the response to Akshay above.

  2. niloufar Yazdanbakhsh says:

    Dear Sir:

    Please help me to contact with Dr. Jose Sahel to save my eye. I have both RP and usher syndrome. I wear both hearing aids. I am a low vision. How can I help myself to do stem cell therapy.

  3. Ravi Dhungel says:

    Hi,
    I am a 26 year old opthalmic patient from Nepal.
    I am possessing Retinitis pigmentosa since my childhood. I have undergone diagnosis in Nepal and India(Sankara Netralaya, chennai).
    They told me it is basically a genetic disease. But Interestingly my younger sister from my same parents is possessing another type of retinal disease called Stargardt’s defect.I would like to know about its latest treatment for me and my sister.

    thanks best regards

    • EyeOnTheCure says:

      You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. It is interesting that your sister has Stargardt disease which is caused by mutations in the ABCA4 gene. Mutations in this gene can cause either Stargardt, cone-rod dystrophy and RP. The ABCA4 gene is also thought to be involved in AMD.

      For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing provides information on what the study is about, the requirements for participating and contact information.

  4. patricia muskrat says:

    my mother has retinitis pigmentosa. I would like to know if there is anything out there to help her.

    • EyeOnTheCure says:

      Your mother should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing provides information on what the study is about, the requirements for participating and contact information.

  5. I am 67 years old woman, having diagnosed RP since 30 years of Age. My brother is also having same problem.
    My vision is ~ 10%.
    Dr, can you advise if there is any way to stop the degeneration or improve my sight.

    • EyeOnTheCure says:

      Regardless of your age, I think you should still consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  6. sheila scott says:

    Greeting,
    Are there any trials for Stargarts disease in Illinois?

    Thank you in advance for you help.
    S. Scott

    • EyeOnTheCure says:

      There are currently no Stargardt trials in Illinois. Please check the website: http://WWW.CLINICALTRIALS.GOV for the most recent information on clinical trials.

    • Sujata Banwari says:

      Hi ,
      My son is 26years old and have RP so he is now ok but he is really worried about the progress of the disease in future and what advice you will give me so that I can help my son and what are the prognosis of the research in the gene therapy ?
      how effective it will be.

      • EyeOnTheCure says:

        Your son should know that there are three inherited forms of RP: recessive, dominant and X-linked. If he is not sure which type he has, he should ask his ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance: http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf
        Your son should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
        Whether the disease gene is identified or not, your son should consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
        https://www.myretinatracker.org/
        It may also be helpful for your son to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

    • Rosa Capone says:

      Doctors in the US are saying my 17 year old Ap student who loves to play Ice hockey has stargardts disease please, if Dr. Jose Sahel can help my baby. Please contact me Asap. Long lsland New York

      • EyeOnTheCure says:

        The Foundation Fighting Blindness is partnering with Sanofi to conduct a gene therapy clinical trial for Stargardt disease in Oregon and Paris, France. For more information on this trial, see the following link:
        http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2

        Before one can participate in any gene therapy trial they must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document:
        http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

        “My Retina Tracker” is a free registry that can help people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

        To learn more about the progression of Stargardt disease, The Foundation Fighting Blindness is also conducting a natural history study, referred to as PROSTAR. For more information on PROGSTAR, please see the following link: http://progstar.org/

        You may be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where you can communicate with other families affeced by Stargardt disease. Here is the link:
        https://www.facebook.com/groups/Stargardts/
        Other Pharmaceutical Companies Developing Treatments for Stargardt Disease:
        Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

        Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
        *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

        Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
        Visum Therapeutics, (http://www.visumtherapeutics.com) Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future.

  7. sajad says:

    My son age 21 is suffering from retinits pegmentosa .He has a little vision left.I did acupancture and other therphy .but nathing has hapend.please help me.

  8. ameen says:

    hello ,

    i was wondering which phase are the trials for stargardt’s disease?

    me and my twin are 19 years old with the disease since childhood which didn’t appear until we were 8 years old

    we’d really appreciate future information about the trials and how soon to expect a cure

    kind regards

    • EyeOnTheCure says:

      I am sorry to hear of you and your brother’s diagnosis. However, you should be pleased to know that the Foundation Fighting Blindness is currently partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 Keep in mind, to participate in this or any other gene therapy trial you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      I also highly recommend that you and your brother enroll in “My Retina Tracker”, a free registry that helps people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      Your daughter may also be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where she can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/
      Just to show you how far we have come, below is a list of pharmaceutical / biotech companies that are developing pharmaceutical treatments for Stargardt Disease:
      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.
      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.
      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Visum Therapeutics, (http://www.visumtherapeutics.com) Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future. I hope you find this information helpful. Thank you for your support that is enabling the development of new treatments for degenerative retinal disease.

  9. Christopher Ritchie says:

    Hi My Son Christopher is 26yrs and has Stargardt. I would like to know if there is any thing that you could tell us about this condition and what trials are going on in Alberta Canada . he is 20/200. Thanks

    • EyeOnTheCure says:

      Your son will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 However, before your son can apply to participate in any gene therapy trial she must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      I also highly recommend that your son enrolls in “My Retina Tracker”, a free registry that helps people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      He may also be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where she can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/
      Finally, below is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Visum Therapeutics, (http://www.visumtherapeutics.com) Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future.

      I hope you find this information helpful. Please feel free to contact me if you have any other questions or concerns.Thank you for your support that is enabling the development of new treatments for degenerative retinal disease.

  10. judith says:

    my son aged 20 was disgonised with stargardts at the age of 11. Can you let us know if there is any cure found for stargardts

    Judy
    Mumbai – India

  11. Varsha(mum) says:

    I am very interested in finding treatment for my daughter who was diagnosed with stagardts about 3 years ago. she is a teacher now and her and determined to make a difference by being in the education world. I am very interested in any treatment that is available to help her continue in her pursuit

    • EyeOnTheCure says:

      You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) completed Phase 3 trials for dry AMD in June 2016. Unfortunately, the drug failed to slow lesion growth in over 500 patients with geographic atrophy, a form of dry AMD. Because of a strong demonstration of efficacy in the Stargardt mouse model, Acucela is considering initiating a study to explore potential benefits of Emixustat in patients with Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

Leave a Reply

Your email address will not be published. Required fields are marked *

*