In an Endpoints News article, jCyte CEO Paul Bresge explained the reasoning behind the approach.
“We enrolled a broad patient population in our Phase IIb trial. Participants had vision ranging from 20/80 to 20/800.
“We wanted to identify the patients most likely to respond to treatment.”
The treatment group achieved better functional vision and greater clarity than the sham group.
Researchers measured participants’ vision at the beginning of the trial and again after 12 months.
The placebo group improved by an average of 2.81 letters on an eye chart while wearing glasses.
Patients who received three million hRPCs improved by an average of 2.96 letters.
Those who received six million hRPCs improved by an average of 7.43 letters.
A specific patient subgroup showed even greater improvement.
Patients in that subgroup who received six million cells improved by 16.27 letters.
The rationale
In an Endpoints News article, jCyte CEO Paul Bresge explained the reasoning behind this approach.
“We enrolled a broad patient population in our Phase IIb trial. Participants’ vision ranged from 20/80 to 20/800.
“We wanted to identify the patients most likely to respond to treatment.”
The treatment group showed better functional vision and greater clarity than the sham group.
Researchers measured everyone’s vision at the start and again after 12 months.
The placebo group improved by an average of 2.81 letters on an eye chart while wearing glasses.
Patients who received 3 million hRPCs improved by an average of 2.96 letters.
Those who received six million hRPCs improved by an average of 7.43 letters.
A specific patient subgroup showed even greater improvement.
Patients in that subgroup who received six million cells improved by 16.27 letters.
Islands of vision
Dr. Henry Klassen, a jCyte co-founder, says the therapy preserves remaining photoreceptors and helps them recover.
“People typically view the disease as a gradual narrowing of peripheral vision. However, patients actually lose separate islands of vision.
“In our tests, we measure each patient’s vision islands at baseline. After treatment, we track whether those islands expand.
“This approach resembles tumor monitoring in oncology, although we seek the opposite result. We want the vision islands to expand.”
One trial participant experienced serious side effects but responded well to treatment.
The team plans to begin a Phase 3 clinical trial next year. They hope the trial will confirm the treatment’s safety and effectiveness.
The results could help the team obtain U.S. Food and Drug Administration approval. That approval would make the treatment available to those who need it.
Retinitis Pigmentosa (RP) is a rare genetic disorders that involves a breakdown and loss of cells in retinal cells. RP is one of the most common forms of inherited retinal degeneration. It involves multiple genes which are mutated. The common symptoms of people with RP include difficulty to see at night and loss of side (peripheral) vision. Gene therapy is not an efficiency tool to cure genetic disorders in eyes. A defective of a single gene to cause inheritance disease in human can be corrected by transferring of normal copy of DNA into cells. However, the ability of gene transfering vector to transduce the cells is limited by weakness of current technology. Therefore, stem cells transplantation by introducing the neural progenitor cells into retinal and growing them into retinal progenitor cells is an alternative choice of treatment. The early results showed the improvement of functional vision. Clinical investigation showed that transplantation of higher numbers of hRPCs (3 millions to 6 millions) can preserve the remaining photoreceptors in the eye and help patients to bounce back their vision. Stem cells are moveable, they interact with growth factors on other cells to enable them to growth and differentiate. The many cycles of growth and differentiation of progenitor stem cells are essential for immature cells to develop into mature and functioning cells. More hRPCs are introduced into retinal increasing the chances of growing progenitors to seed in the site of retinal. This may overcome the loss of stem cells which have been moved to other parts of tissues and organs. Therefore, the efficiency of hRPC treatment is very dependent on the number of hRPC seeding in retinal. The additional of essential growth factors may trigger hRPCs to speed up the time of maturation into functioning and healthy cells. The mature, healthy and functioning cells form and expand the island of vision. They are not only preserving photoreceptors but regenerating photoreceptors at the outer segment of hRPCs.
Hi, my wife has RP in Nigeria. How can she benefit from this novel treatment? She will willfully volunteer for the phase three clinical trials. Its such a distressing illness and we watch her sight deteriorate daily. She will be fourty by August 2021
Dear Dr. Hakeem, I’m so sorry to hear about your wife. I can only imagine how scary and challenging that must be for her and the whole family. The company running the trial is called jCyte and here’s the page on their website that has more information about the upcoming trial and how you can find out if your wife is eligible: https://www.jcyte.com/pipeline/#clinical-trials. I do hope that helps.
Yours
Kevin
Would this be considered for those that have RP due to Usher Syndrome?
Hi Mary, it’s possible it could be used for that in the future if it is approved to treat RP.
Would this results mean those people with better vision had photoreceptors regeneration ?
Not sure at this point Tom, more likely it rescued those that had not been completely damaged and protected others against destruction
This is exciting news but will it be affordable for the general public?
We hope so Sally
morning I am very interested in this treatement. My gradson was born with PHPV. I need your help. My name is Eunice Lindiwe Modiba from Pretoria in South Africa. My email address is eunice.modiba@smu.ac.za or modiba.eunice12@gmail.com and contact (027)828870072 or (027)0125215834. Your assistance will be highly appreciated.
regards
Dear Eunice, I am afraid none of the work we are funding would be of help to your grandson and looking online I didn’t find any clinical trials that might be appropriate for him. I’m sorry I cannot be of more help but I wish you, your grandson and the whole family all the best.
Great news! But will the treatment be able to restore vision to the ones that are almost blind from RP?
Hi Patricia, we hope so. In the first two phases it did help restore some vision to people who had been legally blind for some years, so that’s encouraging.
Dear Riaz, we don’t have any way of estimating when it could be available on a world wide basis. We think it is on track for approval here in the US but we would not be so rash as to say when.
Great news indeed!!
When Phase 3 goes to happen? And how can I participate for it?
Hello Joseph, the company behind the trial is called jCyte. Here’s a link to the page on their website that has details about the treatment and how you can apply to be part of the trial. http://www.jcyte.com/clinical-trials I hope that helps.
Hello Kevin,
I clicked on the trial web site but it is not working. Is there another web site.
Thanks
My apologies Lisa, they made a slight change to their web address that changed the address. Here’s the correct one: https://www.jcyte.com/pipeline/#clinical-trials
Great news!
Would this be considered for those that have RP due to CRB1 gene?
Hi Lisset, I don’t know. You can contact the company behind the trial and ask them, here’s the page on their website that has more information about the upcoming trial and how you can see if you are eligible to apply: http://www.jcyte.com/clinical-trials I do hope that helps.
My father and I both have RP and are very interested in participating in trials. My fathers vision is significantly more deteriorated than mine. We live in Kansas but are willing to travel!
Hello Tera, I’m so sorry to hear that both you and your dad have RP. Rotten luck. There is a company getting ready to start a new clinical trial for RP, here’s a link to the page on its website that has information about the trial and how you can see if you are eligible. http://www.jcyte.com/clinical-trials I hope that helps. Good luck.
Hi Kevin,
My genetic testing came back not able to identify genes causing my RP. When is the 3rd phase set to begin and how does one sign up to participate In the study ?
Hello Maddie, the company behind the therapy for RP is called jCyte. You can find out information about the trial, and also email or call them directly to try and get your name on a waiting list. Here’s the page with the information http://www.jcyte.com/clinical-trials I do hope that helps. Best wishes, Kevin
I have RP. I also have 2 brothers who have RP. We are at 3 different progressions, aging between 60-66 years old. Is there anything that we can do to find something that can help any of us. It seems we are all getting gradually worse.
Hello Larry, I’m so sorry to hear about you and your brothers. I can only imagine how challenging that must be for all of you. There is a company called jCyte that is about to start a clinical trial for RP. Here’s the page on its website that has information about the therapy and how you can apply to be part of the clinical trials. http://www.jcyte.com/clinical-trials. I do hope that helps.
Yours truly
Kevin
Hello, this treatment is suitable for Usher Syndrome ? and does this treatment only prevent losing more vision or it will treat the eyes fully ? finally, do you have any idea when this treatment will be available ?like 5 years ? or 10 years?
thanks.
Hi Adam
Right now it’s only being used to help people with RP. We’re hoping, if all goes well, that the FDA might approve it in the relatively near future, a few years rather than 5. But it all depends on the results. No one can predict this and the team behind the therapy definitely are not going to rush ahead, they’re doing all they can to move ahead safely.
Kevin
Could there be anything done for vision that was lost due to retinal detachment?
There may be surgical procedures that can help. Have you talked to your doctor about this?
How can I get on a waiting list or on the clinical study? I have RP!!! I would be very interested!!!!!!!
Hi Lisa, while we fund stem cell research, including programs targeting RP, we don’t run clinical trials or have the ability to keep a list of people interested in a particular therapy. You might try searching the http://www.clinicaltrials.gov data base for clinical trials targeting RP. Look for one being run by a reputable company or carried out at an academic institution, one that doesn’t charge you for the treatment. I hope that helps.
Will this treat any RP? I was told I have a dominant RP.
Hello Diego, that’s a question best directed at the jCyte team. Here’s the place to go: https://www.jcyte.com/pipeline/#clinical-trials
Great News JCyte, I applaud your effort to make RP treatable. Do you conduct trials on Zimbabweans affected by RP, I would appreciate if you would accept my brother who is affected for clinical trials.
Regards