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When Research Offers More Than Just Hope

John and Pam CorneilleI’ve lost a lot of my sight to retinitis pigmentosa, or RP, and as Dr. Edwin Stone is conducting exciting stem cell research focused on the disease, I began the process of scheduling an appointment with him last summer. Dr. Stone’s home base is the University of Iowa, which is just a two-and-a-half-hour drive from my house in Illinois. When an appointment date was finally offered, I found it a little odd that it was for New Year’s Eve, but I was eager to go, so I said yes.

I should point out that Dr. Stone is a longtime recipient of FFB funding, and that he received its highest research honor, the Llura Liggett Gund Award, in 2012. So, as I was driven to Iowa that cold and snowy morning, I was anxious, to say the least.

I arrived at Dr. Stone’s facility at 7 a.m., and testing began at 7:30—the “typical” tests for those of us with RP, i.e. visual fields, visual acuity, etc. His staff was friendly and professional, and made me feel very much at home. I think I was their first patient that day, as the office was almost eerily quiet when I arrived. By mid-morning, though, the waiting room was buzzing with activity. It felt as if it was any ordinary day, not one of the biggest party days of the year.

Around noon, it was time to meet with Dr. Stone. Because I was at VISIONS, the Foundation’s annual conference, when he was given the Gund Award, I was familiar with Dr. Stone’s work, but I’d never met him. Instantly, I knew I was in good hands, as he unconditionally gave of his time and expertise.

He told me about the work he and his team are doing, specifically the creation of new, healthy photoreceptor cells to replace the defective ones in a typical RP patient. They do that by turning the blood or skin cells of a person into stem cells, which, in turn, can be transformed into whatever cells are needed. Dr. Stone also described a new research facility being built on the university’s campus. Due to be finished in March, it will be of “NASA quality,” in terms of equipment and scientific standards, he told me.

Before I left, a member of Dr. Stone’s staff extracted blood from me to confirm the prior identification of the gene mutation that causes my RP. She also took some skin from the back of my right arm to begin the stem cell process.

While there’s no guarantee that Dr. Stone’s work will result in the restoration of sight, it was impossible to ignore the optimism in his and his colleagues’ voices. His goal is to be able to place healthy photoreceptor cells in patients’ eyes in less than 10 years, but hopefully much sooner.

Personally, the experience helped put the work of FFB, including the invaluable assistance of our volunteers, into perspective. Those of us associated with the Foundation try to pay attention to the latest research news, but until I’d made the trip to Iowa, everything was a bit “abstract.” I couldn’t really grasp how it all fit together.

My visit with Dr. Stone changed that. It made me feel that the money we raise for the cutting-edge work done by countless researchers—many of them moving potential treatments toward clinical trials—really does make a difference. And when that chunk of skin was removed from my arm, it really hit me that great things are actually happening.

It’s more important than ever that the Foundation increase its efforts to raise money for research. The experience I had in Iowa is happening, in similar ways, in research facilities around the world, thanks to those who’ve contributed thus far. And because FFB has a topnotch Scientific Advisory Board, which thoroughly examines every grant request, it can ensure that the money raised goes to those who will do the most good.

I always encourage my fellow Foundation members to stay in tune with the phenomenal research going on through FFB funding and otherwise, and to do what they can to promote awareness in their communities so that we can continue to expand our donor base and the FFB family. The Foundation has always been, and will continue to be, a volunteer-driven organization. And the money it raises will enable Dr. Stone and many others like him to move us closer and closer to our ultimate goal.

Picture, above: John Corneille and his wife, Pam, at a recent FFB event.


36 Responses to 'When Research Offers More Than Just Hope'

  1. Muhammad Ashraf says:

    My wife is suffering from RP. Please confirm treatment is available we can come from Pakistan for treatment

  2. sajad says:

    I hope something will come out soon .whole my life is destroyed because my son is sufring from retinits pegmentosa.

  3. sivaprakash says:

    i am very happy to read these kind of research is going for RP , i am waiting for the clinical trials

  4. Shubha says:

    I Hope people like us to be cured very soon, the research seems promising. We have to make so much adjustments and compromises that normal people won’t even understand. We just want to break the barrier and be free. I Hope, Wish and Pray to get THE CURE Very Very Soon, till then be strong and hope for better tomorrow.

  5. Leonard Canter says:

    Is any research being done for “Rods and Cone Dystrophy”? 15 years ago a specialist in Miami said “Macular Dystrophy”…(My 93 year old mother lost her vision due to Macular Degeneration). 7 years ago in Palm Harbor, FL another specialist said “Retinitis Pigmentosa”. 4 years ago a vision check at Bascom-Palmer, Miami, FL showed decreased peripheral vision. Last year a specialist at Shands Medical Center, Gainesville, FL said “Rod and Cone Dystrophy” causing the loss of peripheral vision and “No Research Presently being done”. Would Stem Cell Research Help?

    • EyeOnTheCure says:

      Leonard,
      It is not unusual for doctors to have a difficult time precisely diagnosing inherited retinal degenerative diseases. Ultimately, genetic testing is the only way to get a definitive diagnosis. If you are able, we recommend that you visit a research center that is familiar with retinal degenerations. Bascom Palmer is such a research center, but given that you visited there 15 years ago, we suggest you go back for a follow-up and discuss genetic testing. The retinal degeneration research field has changed dramatically over the past 15 years – we have much better diagnostic tools and considerably more knowledge. If you call us at 1-800-683-5555, we can give the name(s) of doctors at Bascom Palmer or other research centers that might be convenient.

  6. Muhammad Tahir Bashir says:

    I am suffering from RP.my eye sight is constantly deterioating.need help

  7. Paul Sheldon Foote says:

    Dr. Harvey Uy has reported that he has used the skin of patients (cultured by the German firm TICEBA) to treat at least ten RP patients at St. Luke’s Medical Center in the Philippines. It is difficult to understand why Dr. Stone is predicting ten more years until he can offer a similar treatment.

  8. Meka says:

    I really hope they come up with a breakthrough very soon. I have been dating a guy for the past 3 years who wishes he can get some of his eye sigt back. We want to get married, but he feels incomplete to marry be because of his RP. It doesnt matter to me, I love him with or without eye sight. But it would be nice to come up with a cure :)

  9. Simin Saiidi says:

    My husband is suffering from RP disease. Is there any treatment or anything that help him to stop the progress of the disease. Please let me know.

  10. Ronald Coe says:

    I am a 59 year old male that has been on vitamin A treatment for several years. My vision is quickly deteriorating. Are there any trials available? I live in New Jersey and am willing to travel to try and slow down the progression of this machine.. I lost my wife to cancer four years ago and my family really needs a parent – I’m the last one in the family.

    • EyeOnTheCure says:

      Thanks for your comments Ronald.

      We would recommend that you visit http://www.ClinicalTrials.gov. We fund many clinical trials but do not operate the studies ourselves and so reaching out them directly may be the best way for you to find out more about where they are happening and if participating is an option.

      On twww.ClinicalTrials.gov, which is maintained by the National Health Institute, you are able to search for Stargardt clinical trials by searching based on your disease. Each clinical trial listing will tell you more about what the study is about and how you can contact them to see if they are recruiting and what the participant criteria may be.

      We hope this information is helpful and encourage you to contact us for additional help at info@fightblindness.org for further information if needed.

  11. kristie says:

    ok so i have rp ive had it sense i was born an i would love more than anything for a cure to come me my dad an my brother have it but my sister dont an my my dont i just dont understand why me i have panic attacks because of my eyes an depression because of them i hate it so much i just dont no how to to let the anger go because i no that my eyes are so bad an one day i might not see again an it scares me so bad but people also tell me there probably want be a cure in my life time i am now 36 an steel praying that one day soon i will be able to see what other people see at nite an see what the sky full of stars looks like an to not run ove people an stuff because i cant see it or them its so embarrising when that happens i cant even go in to a dim lit restursnt because i cant see its to dark u no u miss out on a lot of life because of rp an its sad but all i can do is pray i dont even ty to work because no matter where i am im always bumpig in to some one running over something or even running over a kid im on ssi but thats not alot of money an i cant disibility because they say i have not worked enough tpo pay in but how when its so hard to see to work well any way i do hope an pray for a cure soon

  12. Bernadette Sanchioni says:

    My son has rp and is deteriorating fast. His eyes go black when he stands up. Is help almost available.Praying

  13. patricia arnold says:

    my mother suffers from an advanced case of rp. she is currently 76 years old and can only at times see small amounts of light but this is not consistent. my question is…that if a treatment such as this does become avaliable would she be a canidate? vision is so precious and any help would be greatly appreciated. she has met each phase of this disease with such grace but my daily pray remains for her sight to be restored in any capacity. thank you for your site and time. god bless those who fight the good fight and search for treatments and cures.

    • EyeOnTheCure says:

      Thanks for your comment Patricia.

      We would recommend that you visit http://www.ClinicalTrials.gov. We fund many clinical trials but do not operate the studies ourselves and so reaching out them directly may be the best way for you to find out more about where they are happening and if participating is an option.

      On twww.ClinicalTrials.gov, which is maintained by the National Health Institute, you are able to search for RP clinical trials by searching based on your disease. Each clinical trial listing will tell you more about what the study is about and how you can contact them to see if they are recruiting and what the participant criteria may be.

      We hope this information is helpful and encourage you to contact us for additional help at info@fightblindness.org for further information if needed.

  14. Kris says:

    Praying for a cure

  15. okeke prisca says:

    my sister is suffering from Rp diseases.Is any a treatment or anything that can help her to stop progress of the disease and she also diabetic. thanks

  16. Kathy says:

    I am also interested in Dr. Uy’s research at St. Luke’s in the Philippines. His approach appears to be similar to what the US docs are saying will be within <1-3 years before clinical trials begin. Has anyone at the FFB taken a look at his research? I would be very interested to hear of a non-partial report on his results. He apparently has been doing stem cell injections since 2011.

    Thanks.

  17. Barbara Rein-Weiner says:

    Thank goodness for the knowledge and wisdom of all working to find a cure. I am a 51 year old woman who was diagnosed with RP 25 years ago. At this point I have no side vision and have a large amount of blind spots in my central vision. I am now in school updating my technology skills. I would very much like to be involved in the studies that are upcoming. Please contact me; I can travel where ever necessary.
    I look forward to hearing from you soon.
    Barbara

    • EyeOnTheCure says:

      The Foundation Fighting Blindness funds many clinical trials but does not manage the studies and so reaching out to them directly may be the best way for you to find out more about where they are happening and if participating is an option. The website: http://WWW.CLINICALTRIALS.GOV 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.

  18. Lucy Spicer says:

    I have a 67 year old first cousin with RP she lives in Augusta Ky. She was told about this stem cell procedure she is in great need of help. She lives by herself and doesn’t want to move to a care faculty. Could you please let me know if it is being done anywhere yet, and if so where. Thank you so much.

  19. Lucy Spicer says:

    I have a 67 year old female cousin with RP she lives by herself and wants to keep her independence. She has heard of stem cell treatment and us praying to get to try it. She lives in Ky and I would like to to hear from you about any progress in making this available to the public. Thanks so much I appreciate all research and the hard work involved

  20. Karen Mollica says:

    My Aunt lives in Florida and has the wet type of macular degeneration and losing vision quickly. She would be so grateful to participate in any studies that become available. We’re praying for a cure. Thank you for your efforts toward a cure.

    Karen

  21. niloufar Yazdanbakhsh says:

    I am suffering from RP. I am hoping to find a cure for my eyes. I have two young children that I need to take care of them. I am a single mom tool please help me to find the cure for RP.

  22. Angela Quail says:

    My partner has RP, he can’t work anymore
    Or drive, he was diagnosed 8 yes ago but it’s going fast now, night vision gone totally snd only a touch of central vision left he’s a lovely man and the depression is upon him too now, it’s so hArd for us all, I really hope there research is a success in a dark and lonely world for some, we are in England and I know a few rp sufferers

  23. Catherine Smith says:

    Would all the information I received in 2014 be able to be updated to 2017? This website was helpful when my son was first diagnosed but we need to be updated on what is happening now. Thanks for anything you can tell me.

    • Dr. Tim Schoen says:

      Dear Catherine, Thank you for your support. The Foundation Fighting Blindness continually updates it web page as new information becomes available. Each article is dated and so when you are reading a specific article or Blog, you should check the date to make sure it is current.

      If your son has not done so already, he should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, he may be able to 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

      Your son should also consider participating in FFB’s “My Retina Tracker”, a free registry that can help him identify clinical trials that are recruiting. 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. Each listing provides 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.

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