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jCyte Stem-Cell Therapy Moves into Phase IIb Clinical Trial for RP

These are retinal progenitors.

These are retinal progenitors.

The stem-cell therapy company jCyte is launching a Phase IIb clinical trial of its therapy for people with retinitis pigmentosa (RP). The trial is taking place at University of California, Irvine, and Retina-Vitreous Associates Medical Group in Los Angeles. The 70-participant study is being led by Henry Klassen, MD, PhD. Participant enrollment is scheduled to begin this month.

The treatment involves intravitreal injection of retinal progenitor cells (RPCs), which are stem cells that have partially developed into the retinal cells that make vision possible. Based on lab studies, researchers believe the treatment can preserve and potentially rescue the patient’s existing photoreceptors, thereby saving and possibly restoring vision.

Administration of the treatment does not require surgery and can be performed in minutes in an outpatient setting. The RPCs are injected into the vitreous, the gel-like substance in the middle of the eye.

Twenty-eight patients were enrolled in the safety-oriented Phase I/IIa trial for the treatment, which began in June 2015. Dr. Klassen says safety results from that trial have been encouraging. “The cells have been well tolerated, without serious adverse events attributable to the treatment,” he says. “Also, we observed that the injected cells — which formed intravitreal grafts — survived for long periods, even more than one year. We hope to publish detailed results later this year.”

The Phase IIb study will be controlled, meaning that participants will either receive the injection of RPCs or a placebo treatment as the control. Study investigators will be evaluating changes in a number of measures of visual function including: visual acuity, visual field, contrast sensitivity, and the ability to navigate a mobility course.

“The new, Phase IIb trial is exciting because it is specifically designed to demonstrate efficacy, which of course is the question on many peoples’ minds,” says Dr. Klassen.

jCyte recently received an $8.3 million matching grant from the California Institute of Regenerative Medicine (CIRM). In a statement released in December 2016, CIRM’s grant-review working group said, “The proposed treatment approach holds the potential to address a clear and urgent unmet medical need…Further, the investigators leading this study are exceptional and have proposed a well-designed trial to generate useful data that will further inform clinical development of this treatment.”

The Foundation Fighting Blindness funded earlier lab studies of emerging stem-cell therapies that helped make jCyte’s RPC trial possible.

 


20 Responses to 'jCyte Stem-Cell Therapy Moves into Phase IIb Clinical Trial for RP'

  1. Raven Lexington says:

    I have a friend who is blind and was wondering about this procedure and all that, I just wish that she could see again. I want to see her look and smile at something that she herself can see… Please help me.

    • Blake says:

      That’s very caring of you to want something so meaningful for someone else. I would recommend she register with http://www.myretinatracker.org followed by genetic confirmation of the eye disease.

      From what I’ve researched, genetic data is required for participation in most clinical trials
      Spark Therapeutics’ ID Your IRD initiative is a great start. BTW, their gene testing panel is FREE.

      https://idyourird.com/?gclid=CPqH49T-0tMCFY9gfgodmeAOgA
      1-855-sparktx

      I hope this message finds you.

      I wish you and your friend the best!

  2. Jorgelina Castro says:

    Hello I have a daughter and she’s 12. She went blind two years ago after a R.D. And 9 surgeries. She was born with bilateral congenital retinal folds and lost sight from one eye when she was a baby. She had reduced sight with the other eye and manage her live pretty well, she could even read and write until a retinal detachment occurred.
    can this treatment help her? Thank you

    • Dr. Tim Schoen says:

      Dear Jorgenlina, At the present time, the JCyte stem cell trial is for individuals with retinitis pigmentosa. It is possible that in the future, JCyte or another company will conduct a stem cell trial for people affected by retinal detachment. The link to the following Medscape article is encouraging:
      http://www.medscape.com/viewarticle/747348_4

  3. Sunday Udele says:

    I have a son who just turned 3 in April . He was born early at 26weeks and has been diagnosed with stage 5 ROP. Presently he can’t but I am hopeful that this stem cell remedy will help restore his vision.

  4. Fernando De Benedictis says:

    Dear Sirs,
    I am a 69 years aged Italian affected by RP and degenerative myopia, which already resulted in an advanced retinal degeneration. Hence, I am interested in human retinal progenitor cells, in order to get the cure best fitted to me as soon as it will be available.
    For this purpose, I need to know some details regarding either my case and the progenitor cells. Please answer the following questions if you like,
    1. Do the preliminary results already show some cellular regeneration, or just rescue of existing cells?
    2. Considered that competitor hRPC are injected subretinally, which difference of outcome is expected between those, and intravitreally administered jCells?
    3. In case of pros and cons of the two procedures, like for instance better visual field for one and visual acuity for the other, would it be advisable a second injection of jCells, but done subretinally?
    4. Do jCells come from multiple donors or a unique one?
    5. Would it be granted unique donor’s cells supply, in the need of multiple transplantations?
    6. On the basis of current experimental or theoretical science, what can be said about the safety of simultaneous presence of jCells and autologous bone marrow MSCs in the eyes of people previously transplanted with MSCs?
    7. What kind of stem cells are jCells derived from?

    Thank you very much for your help, and best regards

    Fernando De Benedictis

    • Dr. Tim Schoen says:

      Dear Fernando, Please see my responses to your questions below:

      1. Do the preliminary results already show some cellular regeneration, or just rescue of existing cells?
      Answer: There are no published reports on the efficacy of the JCyte trial at the present time. The trial is currently in Phase 2/3 and it is likely that the efficacy data will not be released until the trial is completed.
      2. Considered that competitor hRPC are injected subretinally, which difference of outcome is expected between those, and intravitreally administered jCells?
      Answer: Subretinally-injected stem cells hold potential for differentiating in photoreceptor cells and restoring vision if synaptic connections are made. The intravitreally-injected cells in the JCyte trial most likely do not migrate into the retina. However, the vitreal stem cells are thought to release neuroprotective factors that could slow degeneration.
      3. In case of pros and cons of the two procedures, like for instance better visual field for one and visual acuity for the other, would it be advisable a second injection of jCells, but done subretinally?
      Answer: If the JCyte cells have the potential to differentiate into photoreceptor cells, a second, subretinal injection might be advisable. However, there is no published information available to help with this decision.
      4. Do jCells come from multiple donors or a unique one?
      Answer: It is my understanding that the JCyte cells come from a single donor. However, I am not positive about this.
      5. Would it be granted unique donor’s cells supply, in the need of multiple transplantations?
      Answer: The FDA would have to make this decision.
      6. On the basis of current experimental or theoretical science, what can be said about the safety of simultaneous presence of jCells and autologous bone marrow MSCs in the eyes of people previously transplanted with MSCs?
      Answer: I am not aware of any published data that could help answer this question.
      7. What kind of stem cells are jCells derived from?
      Answer: I don’t know the answer to this as it is not publicly available information.

  5. Deborah M Hadra says:

    My 26 year old daughter has RP, secondary to Carbohydrate Deficient Glycoprotein Syndrome. Are there any new treatments to help her save her vision or new trials that may possibility benefit her? With ber condition, she can see thingz close to her witb a lot of light. Her life is reading, tv, and playing with her Ipad and gameboy. PLEAZE HELP HER!!

    • Dr. Tim Schoen says:

      Dear Deborah, Unfortunately, I am not aware of any current treatments that may be able to help your daughter. However, as the scientific understanding of the mechanisms involved in this disease progresses, it is possible that genetic and pharmacologic interventions will be developed.

  6. Rogelio says:

    I have rp and slowly losing my vision. 29 young guy and in good shape and health other wise. Can i be put on the upcoming trials list??

  7. JW says:

    Hi Sir,

    I was told by my eye doctor I had RP last week. I did not realise how serious a problem until i did some research on RP. Then it sank in. I will continue losing my already bad vision and might eventually go totally blind. My whole world came crashing down. I’m 53 years old and was looking forward to spending more time with my family. Now all I feel is sadness, fear and uncertainty. I started looking for more information about the disease on the internet. Here are a few facts I gathered.
    1. There is no cure for RP.
    2. Best hope for a cure is by JClyte and ReNeuron. Clinical trials are on going and realistic availability of a cure, assuming trials are successful, is around 2020. Is this projection too optimistic?
    3. I can try to get into a clinical trial but chances are slim and that is not the answer to a cure.
    4. Until then there is nothing I can do. I just hope that my vision does not deteriorate drastically and become irreversible until the day a cure is available.

    Appreciate your comments on my points mentioned here.

    Regards
    JW

    • Dr. Tim Schoen says:

      Dear JW, I am sorry to hear about your recent diagnosis with RP. However, please don’t despair, there has been much scientific progress in the past 20 years that are now yielding effective treatments for retinal disease. The first step is to try and find out which gene is responsible for causing your RP. You should definitely consider genetic testing. If your gene is identified, you may be able to qualify for one of the gene therapy trials currently taking place. Additionally, if your gene is identified, you can go to online data bases like PubMed and find out which groups of researchers are studying your gene, some of which may be working on treatments. Please see the following web link to download a PDF document on genetic testing:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      You should also consider enrolling in FFB’s “My Retina Tracker”, a free registry that can help you 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 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.
      Regarding J-Cyte and ReNeuron, these two companies are trying to accomplish different objectives. J-Cyte’s stem cell treatment is injected into the vitreous with the objective of slowing retinal degeneration. On the other hand, ReNeuron’s stem cells are injected beneath retina with the objective of differentiating in new photoreceptor cells, making synaptic connections with the host retina and restoring lost visual function.
      Although not yet in human clinical trials, a stem cell researcher you might want to keep tabs on is Dr. David Gamm, at the University of Wisconsin-Madison. He is making great strides in stem cell research and will be headed to the clinic within the next year. Here is a link to a video interview with Dr. Gamm:
      https://www.youtube.com/watch?v=3LK62FC8Flg
      Also, here is a weblink to an FFB article on Dr. Gamm:
      http://www.blindness.org/foundation-news/researchers-move-closer-developing-transplantable-retinal-tissue-stem-cells

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

      • JW says:

        Dear Dr Schoen,

        Thank you for your reply. I read that stem cell treatments are really costly due to research costs and commercial objectives. As we wait anxiously for a treatment for RP, I hope the costs will not be out of reach for many when available.

        Regards
        JW

        • Dr. Tim Schoen says:

          Dear JW, The majority of clinical trials do not charge for treatment. However, you may have to pay for transportation to the trial site and room and board if needed. Both the Reneuron stem cell trial and the JCyte stem cell trial are free. Once the treatment has been approved by the FDA, it is difficult to say how much it will cost. However, I am hopeful that in the future, our government will regulate the pricing so that stem cell treatments are available for everyone that needs them.

  8. Paul Robinson says:

    Hi,

    My name is Paul Robinson. I am the father of Sandra Robinson, my four year old daughter. This week we received genetic results back on Sandra. She has Usher’s Syndrome. We are looking for specialists in the Greater Atlanta area. Any info on Doctors that would be wise to work with would be wonderful to know. She is Type One. Sandra was born Profoundly Deaf. We have worked closely with Dr. Todd ENT, Children’s Health Care of Atlanta. Sandra has both Left and Right Cochlear Implants, which work wonderfully. We believe our daughter is already experiencing night blindness. She bumps into everything because of her balance. But most importantly she is a very vivacious little girl and I pray that R.P. Usher’s Syndrome will become curable. Any would be helpful.

    • Dr. Tim Schoen says:

      Dear Paul, As you may already know, Usher syndrome is the most common condition that affects both hearing and vision. There are three general types of Usher syndrome. Individuals like your daughter that have Usher syndrome type 1 (USH1) are born completely deaf and experience problems with balance. With Usher syndrome type 2 (USH2), newborns have moderate to severe hearing impairment and in general, RP symptoms start shortly after adolescence. Usher syndrome type 2A is the most common cause of recessive RP. A rarer third type of Usher syndrome (USH3) involves only mild hearing impairment along with progressive RP. For more detailed information on Usher syndrome, please see the following web link: http://www.blindness.org/eye-conditions/usher-syndrome
      You will be happy to know that the Foundation Fighting Blindness is currently supporting a gene therapy trial for Usher type 1B. If your daughter has this type of Usher syndrome, she may be able to participate as they have reduced the age requirements for gene therapy trial participation. For more information on this clinical trial, please see the following link: http://www.blindness.org/foundation-news/usher-syndrome-gene-therapy-safe-thus-far-three-patients
      In order to participate in this gene therapy trial, individuals must first obtain a genetic confirmation of mutations in the myosin 7a gene. For information on genetic testing, please see the following link: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      You may also want to consider enrolling your daughter in “My Retina Tracker”, a free registry that can help you 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/
      Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  9. Patrizia says:

    I live in Italy.My daughter has 3 years old and has retinal distrophy.
    Please if you can inform us how we can help her With this treatment.
    Here in Italy there isn’t any cure or treatment.
    I ask you help for her.
    Thanks a lot

    PB

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