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jCyte Reports Results for Phase 1/2a Clinical Trial for Retinal-Cell Treatment

jcyteA retinal-cell treatment for people with retinitis pigmentosa (RP) has performed encouragingly in a Phase 1/2a clinical trial. Developed by jCyte, the treatment was evaluated for 12 months in 28 people at two sites in Southern California.

Side effects were minor in the safety-oriented trial. Those receiving the highest dose of the treatment had the best results. Their visual acuity, as measured using an eye chart, was nearly two lines (nine letters) better in their treated eye than in their untreated eye. Some participants reported increased light sensitivity, improved color vision, better mobility, and improved reading ability. Ultimately, 22 of the 28 participants had their second eye treated.jCyte has received authorization from the Food and Drug Administration (FDA) to launch a Phase 2b trial for the treatment. The study is enrolling participants with RP who have no better than 20/80 and no worse than 20/800 in their study eye. The FDA is encouraging jCyte to explore using higher doses of the treatment in the Phase 2b study.

Known as retinal progenitors, the cells used in the trial are similar to stem cells and haven’t yet fully developed into mature cell types, in this case, photoreceptors, which provide vision. The progenitors are injected into the vitreous, the soft, gel-like substance in the middle of the eye. Intravitreal injections have a good record of safety and are commonly administered for other conditions in a doctor’s office. The goal of jCyte’s treatment is to rescue and reactivate the recipients’ remaining photoreceptors before they die.

Henry Klassen, MD, PhD, jCyte co-founder and the study’s lead investigator, says the treatment is designed to work independent of the mutated gene causing the patients’ RP.

“We believe our therapy has the potential to help a broad range of RP patients as long as they have some remaining photoreceptors,” says Dr. Klassen. “Nearly 100 genes, when defective, can cause RP. So, it is important for the field to develop therapies that are broadly applicable across this population.”

RP affects about 100,000 people in the U.S. and millions worldwide.

“This is a very encouraging first step in the clinic for jCyte,” says Stephen Rose, PhD, chief research officer, Foundation Fighting Blindness. “jCyte’s report is good news for RP patients who have no options to save their vision.”

The Foundation Fighting Blindness funded Dr. Klassen during his fellowship studies.


93 Responses to 'jCyte Reports Results for Phase 1/2a Clinical Trial for Retinal-Cell Treatment'

  1. Alieu Kamara says:

    Wow! I cannot believe how fast scientist are moving in bringing blindness to an end. Only five years ago, none of us who suffers from RP had no actual hope of ever being able to live normal lives, that feeling has dramatically changed. Thank God for giving us Foundation Fighting Blindness. They have giving us reason to live.
    I would really like to be a part of Jcyte study.

    Alieu,

  2. Rodney Allen says:

    I was diagnosed with RP in Aug 17’and have been on a search to fine help with this condition. I am elated to here this news and anxious to discuss with my doctor. Hopefully it will result in the saving of my sight.

  3. Janette alberto says:

    I have my brother who has this condition. Do u guys soonsir patients to take the trials. Just like rare cases of a particukar disease. He is in the philippines Nd ww are oraying theres hope for him.

    • EyeOnTheCure says:

      Hi Janette,

      We are not aware of any trials in the Philippines. We suggest you search on http://www.clinicaltrials.gov to identify clinical trials for which he may qualify. Keep in mind that many are in Europe and the U.S. You need to contact them to see if they will accept people from the Philippines.

  4. SHIFALI GARG says:

    I am a patient in india. Female 26. Suffering from Rp.
    I wonder if there is any reliable way to find out my exact situation. I wouod like yo enroll. Pls consider.

  5. Ila says:

    My daughter has retinitis pigmentosa ,with no family history and no gene matched her,so doctor concluded it to be a case of mutation ,she has problem in night vision and no or very insignificant problem in Peripheral vision. Is ther chances of her to work efficiently at night as I don’t want her to make career choices due to this conditions.

  6. Guynella key says:

    Hi and hello my name is James Whitlaw I’m a 3generation recipient of retinatal pigmantosa my grandfather was diagnosed my dad and dads brother where diagnosed and I was diagnosed bout the age of 15 both my dad and grandpa died blind my grandpa tried a treatment where they removed his cataract witch only amplified the ultra violet rays my dad was a fool and just thought he was above it I on the the other hand have tried to stay out of the sun light yet still need to find work but most of my work is outside experiences I’m more than willing to try your treatments so that maybe I can not be afraid to pass this on to someone else so please get back to me

  7. Betty Jones says:

    My daughter has retinitis pigmentosa, no family history. She is 40 years old. Is there anything that could possibly help her. We are located near Wilmer Eye Clinic in Baltimore, MD. Is there anything that can be done for her?

  8. Alan Light says:

    My Wife cries often at the impending blindness that will rob her of seeing many joyous sights with our two young daughters as they grow, she is upset and shaken to her core each time she bumps into things or must rely on me for guidance and support in low light situations. My heart breaks for her. Please tell me how I can get her into the study?

    Alan

    • EyeOnTheCure says:

      Hi Alan,

      Visit http://www.clinicaltrials.gov. You can search on “retinitis pigmentosa” to find relevant trials. The jCyte trial has the following identifier: NCT03073733.

    • Dave says:

      My name is Dave, I was diagnosed with RP at the age of eight, I’ve recently graduated from the national Federation of the blind. A school that teaches people how to cope with and be OK with blindness. I have a job get around the city with buses taxis and Uber. Life is OK find this is OK I would encourage you to check into that situation thank you

  9. Ashok Kumar says:

    I am Ashok Kumar, 42 yrs from New Delhi, India. I am very happy to hear that your institution is about to touch the finish line towards the treatment of RP. Best of luck. Reach soon.
    I am a patient of RP diagnosed in 2007. I can feel light and darkness from my right eye whereas my left eye has about 2-3 degree vision field. But with my better eye too I cannot recognise faces, few colours, zig-zag areas.I also have floaters in my better eye. Time and again I also experience that something come from left side in my eye and make me blind for a second or fraction of second. When it occurs I experience it whole day in uncertain intervals and sometimes 2-3 days. Here in India reputed Doctors are also un-answerable to its causes and treatment. Sir kindly keep me on your rolls and please intimate me when there is stable treatment available at my e-mail ID : ashoksirsadhia@gmail.com. Thanks and lots of best wishes to you and your entire team.

  10. Fazal Amin beg says:

    Hi friends: Happy New Year 2018,
    First and foremost, I’d like to extend my heartfelt felicitation to the great scientists with regard to a landmark breakthrough in RP after decades of untiring endeavors and achievements so far. I’m highly touched by this inexpressible good news was shared in the official website of JCYTE with me by one of my good American friends , Dr. John Mock, affiliated with University of California (Santa Cruze).
    This is Fazal Amin beg of Gulmit within Hunza valley of the mountainous Gilgit-baltistan Region in the Northern Pakistan. I’m 49 years old and have suffered from RP severely at present. When I was in my teenage, I felt the weakness slightly but no issue in reading and writing . Even when I was in my 20 plus years of age, I would drive car and bikes occasionally. Gradual deterioration accompanied me like a strong and loyal partner, or otherwise, whatsoever you name it, even when I did my Masters in 1997. The glasses I began to use for the first time in 1989 when I was 19 years old.
    In 2006, I became day time dependent on second person’s in holding my hand, after getting my M. Phil degree with high efforsts (in Central Asian, Chinese and Russian Studies with specialization in socio-cultural anthropology). Since then I’ve been working as an independent field researcher as well as development consultant. In 2018, I did go to Delhi (India) for the treat of my eyesight opting for accupuncture . I spent over thee weeks there and a dramatic vision improvement I got. This phenomenon encouraged me signviantly and encouraged me towards my doctoral program in Pakistan. I thus opted for it. After qualifying my PhD coursework with high marks in Asian Studies in 2011, , my eyesight worsened/again abruptly. Although, issues persisted in easy mobility of mine during the coursework but on the screen of my laptop, I could see with less interruption and wrote my terms papers, other assignments and examination papers. It’s noteworthy that because of disabling environment within the universities and denial of employment within the organizations in Pakistan with regard to persons with disabilities like me led towards incompletion of my PhD dissertation. In 2014, I began facing critically even reding on the laptop screen and was highly concerned. In April 2014, I began training myself on a taling software called Job Access With Speech (JAWS). Thanks to the great scientists for enabling people like us who are visually impaired or blind. Since then, I work on my laptop, as am doing so right now while writing this mail to you people.
    At present, I cannot recognize any people or thing around me, or the texts on the screens of my laptop or cellular phone, which I could do so couples of years before. The lights within the rooms I can see not from my central vision (rather it’s blurr centrally) but can somehow see the light of bulbs fromm the peripheral /domains of my eyes. In the same manner, it becomes hard for me to walk in the sunlight without sunglasses, despite the fact I cannot see outside rather it seems dark cloud for me.
    Providing you such facts meant to know if my treatment could be taken up on priority if I offer myself for the trial, as Ive not become completely blind yet.

    Lookig forward to hearing from you.

    • EyeOnTheCure says:

      Hi Fazal,

      We recommend you visit http://www.clinicaltrials.gov. You can search on “retinitis pigmentosa” to find relevant trials. The jCyte trial has the following identifier: NCT03073733 and you can sign up for the trial and get more information.

  11. Swami says:

    I am a patient from India with RP, 76 years old. Is the trial going to include patients in Asia, India as well. If so, I would like to be a part of the trial. Please let me know

  12. Curt Johnson says:

    Hello-
    I was diagnosed with RP in 1991. I still have useful sight, but would like to be considered for the study.

  13. RITESH RAGHANI says:

    Hi All, i have my brother and sister both are suffering from RP since class 5th and now they feel very irrated when they have to see something which is far or even at 10 cm away.. due to which they started hating every1 and my brother has started cursing my parents which feels very weird. In our family there is no such case which has been found like this. I’d humble request to the top shorts who have discovered that RP can be cured. Please share me all details. How to step ahead with this?

    Kindly inbox me ritesh.raghani@gmail.com

  14. Lisa Bourg says:

    Hello,

    I am a 50 year old female from Louisiana. I have RP, and one of my four children has been diagnosed. My central vision is holding; however, my peripheral vision and depth perception are almost gone.

    I was surprised and excited to read about your research and work. I am emailing of behalf of my daughter. She would be an ideal candidate for your study. She is progressing slowly. At 23, she is a senior at Tulane in New Orleans, works to support herself as a student, and gives back to her community in service projects.

    I am not clear on the criteria set for
    consideration. Could you please share that criteria?

  15. Dennis Lupton says:

    I am a 52 year old male from South Carolina diagnosed with RP in 1997. I have lost my peripheral vision and beginning to lose center vision. I would like to be considered for your study. Thank you very much.

  16. Shane H says:

    I was diagnosed with RP at the age of 17 I am now 33 and it is impacted my life a lot these last few years I sure hope there is a chance this can be cured so I can live a life with a chance to see my kids prosper good luck fingers are crossed

  17. Hi to all,
    When my husband told me about this this morning, I instantly starting googling.

    When I found this site, I called my sister right away who was just waking up.

    My sister is 61 years old, was diagnosed with RP probably as long ago as 1977. She is in total darkness now. When I called her & told her about this, I welled up with tears & got full of goose bumps. What a great gift this would be for her (& all of the family actually).

    Is there possibly any financial assistance that she would be able to qualify for any procedures?

  18. Samir Shaikh says:

    Hi this is Samir Shaikh here 27 year male from Pune India.
    I was diagnosed with RP at age of 18 currently I can see at day time and partially in dark. Central vision is very good but sometimes feels like losing side vision. Can you please mail me detail procedure to enroll for same?

    Thanks and I Congratulate a team to give a breakthrough.

    • EyeOnTheCure says:

      Hi Samir, you can visit the following webpage for more information (including contact information) for the trial: https://clinicaltrials.gov/show/NCT03073733

      However, it may be difficult to get into the trial if you are in India. Here are four hospitals in India that may have doctors who provide care to RP patients, but I am not aware of which clinical trials they may be running:
      Shankara Netralaya, Chennai
      L. V. Prasad Eye Institute, Hyderabad – They have branches in different states
      Aravind Eye Hospital, Madhurai
      All India Institute of Medical Sciences- New Delhi

  19. Kamal Aggarwal says:

    Hi Sir,

    I am from new delhi and my cousin brother is suffering from RP. He was diagnosed 5-6 years back and since then is suffering.He is quite young and has whole life to discover Kindly provide me the treatment for this.We really need ur help

  20. Sharadkumar S Chandgothia says:

    Very happy to see the breakthrough in this field . My son and daughter both are suffering from RP ,I would request that please kindly enroll both of them for treatment.Please confirm..With kind regards.

  21. Oriola honeyman says:

    Great discovery and good news to all RP patients worldwide. I am a 47yrs old Nigerian diagnosed with this disease. I wish to be considered for this clinical program.

  22. Michael says:

    I have RP I’m 68 years old. I was diagnosed at age 15. I was told by age 25 I would probably be totally blind. That never happened. About every 10 years with an eye exam they kept telling me in 10 years I was going to be totally blind until I was age 55 and they said I might outlive it. I had cataract surgery and in my right eye they done laser surgery 5 years later because I was getting hazy Vision again. It is so bad that I get something that looks like looking through a frosted glass and I cannot make nothing out but if I look up and down quickly I can get it to clear to where I can see for a while until that bra straps right back over and blocks my vision. Up until lately I could read without glasses I still can at times when I’m looking downward. I was told nothing could be done for me I was going blind. But it just don’t make no sense to me how I can clear my vision and see again. Once again from age 15 until about age 62 or more my field of vision never changed never got better and it never got worse.

  23. Samantha says:

    Hi, great work. I am a 32 year old female with it from the uk. Could I please be considered for any trials.

  24. Rawan barakat says:

    Hi, is a 40 year old pharmacist who was diagnosed when I was 20 years old. At first I didn’t take things seriously because I still felt that I could see very well, however for the last four years my eyes have gotten much worse. My side vision is almost totally gone and my central is ok enough for me to continue working but I get very frustrated and I can not even see an entire screen or face without some parts missing. I’ve recently also begun feeling bluriness in my good eye and a very worried about the speed in which my sight is deteriorating.
    Is this study something you can tell me more about please? How would a person qualify and is the sooner to start treatment the Better?
    Thank you for all you do and for giving me hope!

  25. Zainab says:

    Hi my daughter 20yers she suffered RP last four years hope you sucsses very soon good is great in India no treatment many doctors I checked all say no treatment pleases any update information me

    • EyeOnTheCure says:

      We are aware of the following clinics in India, which may be helpful:
      Shankara Netralaya, Chennai
      L. V. Prasad Eye Institute, Hyderabad – They have branches in different states
      Aravind Eye Hospital, Madhurai
      All India Institute of Medical Sciences- New Delhi

      If you are interested in a clinic in a different location, contact one of these clinics and ask them for a suggested clinic in your region.

  26. Bgoswami says:

    My mother had suffered from RP since 1991.She had lost vision in both of her eyes since 1994.She lives in India. It would be so nice for her to see this world again. Kindly enroll her for treatment. Please confirm.

    • EyeOnTheCure says:

      We are aware of the following clinics in India, which may be helpful:
      Shankara Netralaya, Chennai
      L. V. Prasad Eye Institute, Hyderabad – They have branches in different states
      Aravind Eye Hospital, Madhurai
      All India Institute of Medical Sciences- New Delhi

      If you are interested in a clinic in a different location, contact one of these clinics and ask them for a suggested clinic in your region.

  27. Bhavya says:

    M a patient from India with RP. I hope I will soon get the benefit of this treatment. Praying and hoping for a better tomorrow.

  28. Joanne Glaude says:

    Hello, my vision is in the range noted when I am not affected by intense glare of the sun or fluorescent lights (as I ha cataract surgery 2 years ago with new lense implants). I am 63 yrs old and was born with a type of RP that 2 Labs have not been able to confirm what mutation I have (both gentic bloo test and saliva test). I have been searcing a long time for any hope and I have not found any help as I do not know m RP mutation type. I do not have tunnel vision. I do have some peripheral and some minor astigmatism still. The RP gives me fuzzy vision if I am exposed to any sunlight or fluourescent lights so I wear storng polarized glasses all the time. I have challenges in dim or dark lighting of course but in mornings and evenings in my home I can see quite well to function. I do hope to hear from you for any assistance; I am legally blind and on low income, living in Florida, caring for my elderly mom. Thankyou as I would like to know about treatments and possible help. In God we TRUST. JoAnne Glaude

  29. janina says:

    So encouraging that we might be able to cure this disease. My brother, who lives in Poland, now 62 years old, has been diagnosed with it since childhood. Unfortunately his eyesight has deteriorated significantly so I am hoping something could be done

  30. Larry Heath says:

    I was diagnosed with RP in 1967. I am now 62 years of age. My parents were told back then that I would be totally blind by age 18. I was able to graduate High School without assistive products and also to drive until I was ablut 30. I have 20/400 in my best eye and unknown as to the other because they cannot get a reading on it. I have 2 special grand-daughters and a fiancée that I would like to see. My situation is considered “light perception”. I use the JAWS adaptive computer software and have been able to work for the past 24 years. Would like to get into the trial.

  31. Christine Reynolds says:

    I’ve had our pee since I was born. I am now 46 I live in Ellensburg Washington. I would be interested in finding out a little bit more about this case study. I have some Vision left mostly what I look directly at is what I can see. I don’t say that I suffer from anything it’s an eye disease and eye condition it is what it is.

  32. Ruth Hopkins says:

    My brother lives in Las Vegas and has been dealing with this condition for 40 years. He says there is a doctor there that is treating with cell therapy. Is there a clinical trial there that he can be a participant in?? The therapy is too costly for him to do in his own.. Thank you for your consideration…

  33. Nitesh says:

    Hi I’m Nitesh, settled here in Dubai. Were there is no way, God brings light, and I infact we all see some hope. May God Bless us with positive results from the second trial.
    Please also do keep me posted through my email ID about the latest research and results for same.

  34. Kris says:

    I’m 38 and 5th generation rp. I have a 10 and 7 year old daughters. I’m so on board for any help I can get from this. I have already had glaucoma surgery. Both eyes cataract surgery and retinal surgery. I’m hoping that my rp is one that fits this genetic testing.

  35. Ricardo Robledo says:

    Hello,

    My dad suffers from RP. His vision has progressively gotten worse over time. My aunt, his sister, has the same disease. I would like for him to be a participant in this trial if possible. It would make his life a lot better. Please consider us for your research. Thank you

  36. Sophia Casasanta says:

    Hi, I’m 23 and have been diagnosed with RP for about 2 years now. My retinal doctor said that it might be caused by a genetic mutation called bardet bedal/Lawrence moon’s but I haven’t had any luck with getting genetic testing done. I thought there was no cure for RP but this gives me hope. Thank you. I would also be very interested in participating in this study.

    • EyeOnTheCure says:

      Hi Sophia, you need to be genetically tested in order to know what mutation is causing your disease. One of our programs, My Retina Tracker, offers free genetic testing to members in the registry, if you’re interested. To register and become a MRT member, visit: http://www.MyRetinaTracker.org

  37. Piyush Jain says:

    MY mon 59 is suffering from RP and would like to be part of thia program.

  38. Ingrid Olmo says:

    Hello,

    My Sister is 28 Years Old, and she was diagnosed with RP at the age off 11. She had a blurred cloud over her ,was how she explained it. After several visits to the Dr they finally told her she had RP. Over the years her vision got worse.She lost sight from her right eye, all she sees is shadows.I honestly want her to get cured. She has a child and I would hate for her to lose her vision.No one in our family has any problems with eyesight,so its quiet odd that she has this.I want her to participate in this study.

  39. Eva keshek says:

    My name is Eva Keshek and L am 52 yrs old . I have RP . I would love to be a part of the trial that you have . Please help and let me know . Thanks

  40. Marty Kiner says:

    My mother has RP and is almost completely blind, my brother has RP and his sight is diminishing quite a bit. He can no longer drive at night and entering indoors from the outdoors he must rely on assistance to get around. My sister has just been diagnosed with RP. Is there any room in your study for any of my family members? I will share this information with them

  41. Rakesh Agarwala says:

    Hi,

    It’s a great achievement indeed.
    My daughter, aged 27 years now, has been suffering from RP and her vision is declining. I would be obliged if she can be considered for treatment.

    Regards
    Rakesh

  42. John says:

    I have a 17yo therapy client with RP that is interested in participating in clinical trials. What is the process for participation? We are near Indianapolis, IN.

    • EyeOnTheCure says:

      Hi John, you can learn more about clinical trials for inherited retinal diseases by visiting:
      http://www.clinicaltrials.gov. The website has a search function to find trials relevant to you. Once you find a trial you’re interested in, you can contact the research facility closest to you about enrolling. Good luck!

  43. khaizar ziaee says:

    Hi I am a 27 year old man from india ,I have rp and would be really benefited from this study,can you please let me know what is the procedure and how to apply for opting to take the trial. my email id is khaizarz@gmail.com

  44. Larry Gordon says:

    Hello, I am a 69 year old man, diagnosed probably twenty years ago and still able to see, although at dark it is getting more difficult and even some during the day. I live in Southern California and would very much wish to be considered for this trial if it is open to do so, or if my participation would be of any benefit.

  45. Dennis Lupton says:

    I am a 53 year old male with RP. After leaving the retinal specialist today, I am discouraged. No clinical trials in my area-South Carolina. Please consider me for any future trials. Thank you.

  46. Prashant Raj says:

    Hi. My father was diagnosed with retinitis pigmentosa in 1997 and since then he has been suffering from this problem. His eyesight has deteriorated to a great extent and he’s unable to read, write or even go anywhere during the day. It’s almost 21 years since the diagnosis and I would request you to please contact me for trial.

  47. Raelene says:

    My daughter was diagnosed with Ushers Syndrome at a very early age. She is profoundly Deaf and has RP as a result of this syndrome. She is now in her 30’s and her sight is deteriorating rapidly. Can you tell me how close you are to bionics and or treatment.

  48. Arlene Rodriguez says:

    Hi, I have been diagnosed with RP since the eighth grade and I am now 30 years old and can feel that my eyes have gotten worse. I would love to be part of the trial not just me but my sister who has also been diagnosed with the disease. I prayed for this day to come and hope that one day I can regain my vision that I once had as a little girl. My mother and father do not have the disease so they do not know what i am going through. It is depressing and sad which makes me cry every night. Please provide me with any information that will help me be part of this trial as this is what I have been hoping and wishing for in a very long time. Thank you

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