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Foundation Investing in Drug to Slow Many Forms of RP

Sometimes, fighting blindness means helping people save the vision they have, or at least slowing disease progression enough so they can maintain useful vision for all of their lives.

That’s the idea behind a promising, emerging drug for retinitis pigmentosa (RP) known as N-acetylcysteine-amide (NACA). The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has announced an investment of up to $7.5 million to advance the potential therapy into and through a Phase II clinical trial. In several animal models, including previous FFB-funded lab studies of rodent models at Johns Hopkins University, NACA slowed retinal degeneration.

Nacuity Pharmaceuticals, Inc., a start-up company in Fort Worth, Texas, owns the rights to NACA for ophthalmology and will be developing the drug with support from FFB-CRI. As part of its agreement with Nacuity, upon commercialization of NACA, FFB-CRI will be entitled to royalty payments from future NACA sales.

“Based on the knowledge we’ve gained in lab studies, we are excited about NACA’s potential for saving vision,” says Patricia Zilliox, PhD, chief drug development officer at FFB-CRI. “Oxidative stress causes cell degeneration and vision loss in virtually all forms of RP, so we are hopeful that NACA, with its anti-oxidative properties, can benefit most people with RP, regardless of the gene mutation causing their disease.”

NACA is derived from N-acetylcysteine (NAC), a drug approved by the U.S. Food & Drug Administration (FDA) for the treatment of acetaminophen overdose by lessening hepatotoxicity. The benefits of NACA over NAC are believed to include greater lipophilicity, or ability to dissolve in fats, and tissue penetration therefore increasing anti-oxidative properties in the retina.

The drug is designed to slow vision loss by protecting retinal cells from oxidative stress, a process that accelerates and exacerbates degeneration in many inherited retinal conditions including RP. When the cells in the body process oxygen, they produce byproducts known as free radicals, which can be harmful if the body is unable to neutralize them. In certain diseases and conditions, cells have a difficult time neutralizing free radicals, and damage occurs.

“We are excited to make a significant investment in NACA, because the drug has an opportunity to help a substantial number of people with RP, a blinding retinal disease affecting 100,000 people in the U.S. and more than 2 million worldwide,” says William T. Schmidt, chief executive officer at FFB. “The fact that NACA is derived from an FDA-approved drug also boosts our confidence that it can make it through the clinical development process and out to the people who need it.”


45 Responses to 'Foundation Investing in Drug to Slow Many Forms of RP'

  1. sajad says:

    my son age23 has RP.please help mefor cure.

    • Dr. Tim Schoen says:

      Dear Sajad,

      There are three inherited forms of RP: recessive, dominant and X-linked. If you are not sure which type your son has, you 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 his RP. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/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/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf

      Your son should also consider enrolling 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/

      Finally, you and your son may find it 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. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  2. where can we get this medication?

    • Dr. Tim Schoen says:

      Dear Russell,

      N-acetyl cysteine (NAC) is available without a prescription from multiple on-line vitamin stores as well as most pharmacies. Before taking any nutritional supplement, you should discuss it with you primary care doctor. Please remember that the best way to get your antioxidants is to make sure you’re getting them from whole foods, not from supplements, which are often isolated synthetics rather than the readily bioavailable versions.
      Here is a list of the top 10 Highest in Antioxidant Foods
      Goji berries: 25,000 ORAC score.
      Wild blueberries: 14,000 ORAC score.
      Dark chocolate: 21,000 ORAC score.
      Pecans: 17,000 ORAC score.
      Artichoke: 9,400 ORAC score.
      Elderberries: 14,000 ORAC score.
      Kidney beans: 8,400 ORAC score.
      Cranberries: 9,500 ORAC score.

  3. Alaric Anthony says:

    I would like to know more about this drug because I have RP. When do this drug come on market.

    • Dr. Tim Schoen says:

      Dear Alaric,

      N-acetyl cysteine (NAC) (not the amide form) is available without a prescription from multiple on-line vitamin stores as well as most pharmacies. Before taking any nutritional supplement, you should discuss it with you primary care doctor. Please remember that the best way to get your antioxidants is to make sure you’re getting them from whole foods, not from supplements, which are often isolated synthetics rather than the readily bioavailable versions.
      If you have not done so already, you should consider genetic testing to try and identify the mutant gene responsible for causing your RP. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a genetic diagnosis, you may qualify for one of the gene therapy trials currently being conducted. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      You should also 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/
      Finally, you may find it 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. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  4. Phil Ranalli says:

    I’m a 60 year old man that has RP and I’m very interested to know more about NACA. Please add me to any email list that may provide updates or availability on NACA.

    Thank You
    Phil Ranalli

    • Dr. Tim Schoen says:

      Dear PHil,

      N-acetyl cysteine (NAC) is available without a prescription from multiple on-line vitamin stores as well as most pharmacies. However, before taking any nutritional supplement, you should talk with you primary care doctor. Please remember that the best way to get your antioxidants is to make sure you’re getting them from whole foods, not from supplements, which are often isolated synthetics rather than the readily bioavailable versions.
      Here is a list of the top 10 Highest in Antioxidant Foods
      Goji berries: 25,000 ORAC score.
      Wild blueberries: 14,000 ORAC score.
      Dark chocolate: 21,000 ORAC score.
      Pecans: 17,000 ORAC score.
      Artichoke: 9,400 ORAC score.
      Elderberries: 14,000 ORAC score.
      Kidney beans: 8,400 ORAC score.
      Cranberries: 9,500 ORAC score.

  5. douglas says:

    I have a good feeling that this drug would go through clinical trial with flying colors. Thank you FFB for everything you have done over the years and may God Bless You.

    Douglas

  6. Kelli Bull says:

    My husband has RP, please keep me informed on any availability or updates on this drug for RP.

  7. Nilam says:

    My and my father are affected by RP.

  8. Nilam says:

    What is the cost in india for genetic testing for RP

  9. The NACA (N Acetly Cysteine Amide) “drug” is basically NAC (N Acetyl Cysteine), a nutritional supplement that can be purchased online and in most health food stores. You can also get NAC in eye drop from in a product called “CAN-C.” NAC boosts glutathione levels, which is a powerful antioxidant that may help slow RP in patients who have elevated free radical activity and oxidative stress. As we already know, carotenoid antioxidants like lutein and zeaxanthin can be beneficial for RP as well – in my opinion even more so then NAC in terms of retinal neuro-protection. Talk to your doctor if you are thinking of starting a regiment of NAC supplementation.

    • Steve says:

      Dr. Rosenfarb, thank you for your post. I would like to talk to my doctor about this. When I do, what dosage should we be considering?

  10. A. Wilmers says:

    My doctor retired, and after 20 years as a volunteer for R.P. research, I am wondering what happens to all the data collected on me. Is it dropped, or does someone continue where they left off. I can’t imagine the study ending, as no cure has yet to be found. I also need to find a doctor who is as informed as my previous MD. Unsure what direction to take.

  11. I am 81 years old man whit RP I am working .

  12. Busabakorn vattasingh says:

    My dear friend has RP , , 67 years old. His vision is deteriorating .

    How can he get this medicine??

    Thank you

    • Dr. Tim Schoen says:

      Dear Busabakorn, N-acetyl cysteine amide (NACA) is available only by prescription. N-acetyl cysteine (NAC) (without the amide) is available without a prescription from multiple on-line vitamin stores as well as most pharmacies. I understand that there is also an NAC eye drop that is available. Please note that until a double blind clinical trial with placebo controls is conducted, it will be impossible to determine the efficacy of NAC or NACA in slowing retinal degeneration. As always, before taking any nutritional supplement, you should talk with you primary care doctor.

  13. Virginia Sblendorio says:

    I have RP. I am interested in more info.

  14. Karen arrington says:

    I am 63. Was diagnosed with rp around mid 30 s.
    Would like info on a doctor that is knowledgeable
    On the latest research,drugs,procedures,etc.
    I would like to preserve the vision I have.

    Thanks
    Karen arrington

    • Dr. Tim Schoen says:

      Dear Karen, Since you still have vision left, you may be able to benefit from gene therapy. 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 (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a genetic diagnosis, you may qualify for one of the gene therapy trials currently 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/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      You should also 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/
      Finally, you may find it 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. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  15. Flora Jackson says:

    I was diagnosed with RP in 1971. How does one know if they are a candidate for NACA.I volunteered and raised money for FFB until my vision got so bad I couldn’t drive. I only have about 7° of vision left of vision left in my eyes. However what is corrected to 20/20 and 20/30. I go to NIH yearly where they track my progress. Prior to that I went to Johns Hopkins and participated in a couple of their research projects with no benefits from the project. Am I a candidate for NACA? How can I find out if I would be a candidate for it?

    • Dr. Tim Schoen says:

      Dear Flora, There is no way to know if you will qualify for the NACA (N-Acetyl Cysteine Amide) trial until the exclusion and inclusion criteria are published on http://WWW.ClinicalTrials.gov. Most likely the trial will include individuals like yourself that still have some vision left. You 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/
      Finally, you may find it 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. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  16. andy says:

    I am a 49 year old man with RP.
    How can I participate in clinical trial?

    • Dr. Tim Schoen says:

      Dear Andy, If you still have some vision left, you should consider genetic testing to try and identify the mutant gene responsible for causing your RP. With a genetic diagnosis, you may qualify for one of the gene therapy trials currently 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/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      You should also 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/

      If you have lost a substantial number of photoreceptor cells to disease and are at the light perception or hand motion level, you may be able to benefit from several new sight restorative technologies that are currently being testing in human clinical trials. Currently, there are three promising technologies for sight restoration. They are: Visual Prosthetics, Optogenetics and Stem Cell photoreceptor replacement therapy. Visual prosthetic devices such as the ARGUS-2, developed by Second Sight, provides electrical stimulation of the retina to induce visual perception in blind individuals. To learn more about the ARGUS-2, please see the following weblink: http://www.secondsight.com Optogenetics is an exciting new technology that is able to convert non-light sensing cells in the retina into photoreceptor-like cells. Currently, Retrosense is conducting a human clinical trial in blind patients with RP. For more information, please see the following weblink: http://www.retrosense.com/development.html
      Stem cells hold much promise for sight restoration. However, like optogenetics, the safety and efficacy of stem cell treatments are still being evaluated in human clinical trials. ReNeuron is currently conducting a clinical trial to evaluate the safety and efficacy of a stem cell treatment to restore visual function in blind individuals with retinitis pigmentosa. For more information, please see the following weblink: http://www.reneuron.com/products/hrpcs-for-retinitis-pigmentosa/

      Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  17. The article states this is entering Stage II of FDA approval. How many stages are there and what is the spectrum of approval time? (4 months, 4 years etc.) Is there an FFB article related to FDA approval?

    • Dr. Tim Schoen says:

      Dear Nick, The FDA requires all drugs as well as medical treatments such as gene or stem cell therapy to undergo rigorous testing for safety as well as efficacy before they can be approved. The process is broken down into three different phases.
      A phase I clinical trial tries to show that people can safely use a new drug or treatment. Doctors collect data on the dose, timing, and treatment safety. At this point, the drug or treatment has already been shown to be safe for animals. People who join phase I clinical trials are often the first to get a new therapy or combination of therapies. In phase I clinical trials, doctors slowly increase the dose of the drug being studied. The goal is to find the dose that works best without causing severe side effects. Doctors keep testing until they find the highest dose with the least side effects. Phase I clinical trials often last several months to a year. They usually have only 10 to 30 people.
      Phase II clinical trials give doctors more information about the treatment’s safety and how well it works. Phase II clinical trials may last as long as 2 years. They usually have 30 to 120 people.
      Phase III clinical trials start with a new treatment that’s worked well in a small number of patients. In a Phase III trial, data from large numbers of patients, typically more than 300 are analyzed for efficacy. Phase III clinical trials are usually randomized so that neither the doctor nor the patient knows if they are getting a treatment or placebo. Once the research shows that the treatment works, doctors can apply for FDA approval. If data from the clinical trials meet FDA standards, the FDA approves the treatment for a specific use.
      Sometimes doctors prescribe a drug for a use not specified by the FDA. This may be because studies in peer-reviewed journals show that the treatment works for other diseases, conditions, or symptoms. Drug companies call this “off-label” use. However, such “off-label” use of drugs is not always covered by insurance companies.

  18. Nicole Reed says:

    My 4 year old granddaughter Marlaya has L.C.A. she’s currently waiting to be chosen for one of the clinical trials and although she’s learning to adjust every day seems to be a new struggle if this new drug could help her we would like some information on it.shes seeing less now then she did a few months ago . Please if this is a safe option for her we would like to try it
    Thankyou for your time

    • Dr. Tim Schoen says:

      Dear Nicole, There is published scientific data in RP animal models that indicate that N-acetylcysteine (NAC)is able to slow photoreceptor cell death. However, until a controlled human clinical trial is conducted we really don’t know how efficacious the treatment is.

  19. Jurga says:

    Hello,

    I have an RP. I am 40. Working, driving and looking forward to positive news saving the sight through my life.
    Please keep me updated as here in North of Europe no information or support is provided by ophthalmologists so far.

  20. neeraj says:

    I lived in India…I m patient of rinetine pigmentosa ….please suggest me if my rp not increase… Please suggest me….my WhatsApp. Number+918005184544

  21. C Hernandez says:

    Today I took my 14 year old daughter to the ophthalmologist for an exam to get reading glasses. He requested my family history which included my aunt with MD and my grandmother with RP. He requested for dilation and he checked her. He then told me that he saw patterns/activity in both eyes which looked like it’s RP related. The referral read “inferior peripheral mottling, nasal bone spicules” I’m way too familiar with that terminology…… We are seeing a retina specialist tomorrow. I’m in prayer for her– but am concerned it’s RP and she is so so young … I’d like more information if this comes about.
    Thanks

  22. Kilic says:

    I have rp how we can get this medicine?

    • Dr. Tim Schoen says:

      Dear Kilic, Although there is published scientific data in RP animal models indicating that N-acetylcysteine (NAC)is able to slow photoreceptor cell death, until a controlled human clinical trial is conducted we really won’t know how efficacious the treatment will be. It is possible to buy NAC from many nutritional supplement companies. However, you should first discuss with your personal physician before taking any supplement.

  23. Yvonne korstange says:

    Hubby has Rp, aling with 3 other siblings. This would be awesome for the family.

  24. Valarie Thornton says:

    Thank you for this information. My son is 42 and one of my daughters is 39, and both have RP.

  25. Deb says:

    I have RP and my vision is deteriorating. I would be interested in more info about this medicine and when available.

    • Dr. Tim Schoen says:

      Dear Deb, Make sure that you are on the Foundation Fighting Blindness’ mail list so that you can be kept up to date on new treatments. 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 (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a genetic diagnosis, you may qualify for one of the gene therapy trials currently 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/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      You should also 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/

  26. pete sessions says:

    Thank God for FFB and researchers who are helping to cure blindness . Pete Sessions

    • Dr. Tim Schoen says:

      Dear Congressman Sessions, Thank you for all you are doing to raise awareness and generate funding to support the development of safe and effective treatments for degenerative retinal disease.

  27. Nicole Maer says:

    It’s great that the FFB-CRI plans to invest $7.5 million for studies on NACA. Retinitis pigmentosa is among the most common genetic diseases of the retina and affects hundreds of thousands of people in the US. The blind spots caused by RP can hamper regular lifestyle.

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