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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.


27 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

  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?

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