Encouraging Update on Clinical Trial for Retinitis Pigmentosa

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Patients describe their experience as “stepping out of darkness into light” after joining a CIRM-funded clinical trial.

The trial targets retinitis pigmentosa, or RP, a rare form of vision loss.

Clinical trial

jCyte, the company conducting the trial, announced 12 month results for its stem cell-based RP treatment.

RP is a genetic disorder that affects about one in 40,000 people and 1.5 million people worldwide.

The disease destroys photoreceptors, the light-sensing cells at the back of the eye.

Patients often develop vision loss during their teenage years. Many eventually become legally blind by middle age.

No cure currently exists for RP. However, stem cell therapies might slow the disease’s progression.

In a normal retina, photoreceptors appear healthy. In a person with RP, those cells show damage. (Source: National Eye Institute.)

jCyte is a leader in developing cell-based treatments for RP.

UC Irvine scientists founded the company under Dr. Henry Klassen’s leadership.

The company is testing jCell, a treatment made from progenitor cells derived from pluripotent stem cells.

These cells can develop into photoreceptors.

When doctors transplant jCell into the back of the eye, the cells may release growth factors. These factors could protect the retina’s remaining cells from further damage.

The cells might also integrate into the retina and develop into new photoreceptors. In doing so, they could improve patients’ vision.

Photoreceptors look healthy in a normal retina (left). Cells are damaged in the retina of an RP patient (right). (Source National Eye Institute)

jPositive Results

Patients received a single cell injection in their worse eye. Researchers then compared vision between the treated and untreated eyes.

Patients who received 500,000 cells read one additional eye-chart letter with the treated eye.

Those who received three million cells read nine additional letters.

Importantly, no patient experienced significant treatment-related side effects.

At the Annual Ophthalmology Innovation Summit in November, jCyte announced results from its Phase 1/2a trial.

The 12-month study tested two cell doses in 28 patients.

The company reported a favorable safety profile and possible benefits for patients’ vision.

According to jCyte’s news release, patients gave especially encouraging feedback.

Many patients reported better vision, greater light sensitivity, improved color perception, and improved reading ability.

Some patients also reported better mobility.

In addition, 22 of the 28 patients received treatment in their other eye through a follow-up extension study.

Rosie Barrero participated in the trial. Earlier this year, she told us that the treatment improved her vision and gave her hope.

Watch her video

Next Steps

These results suggest that the jCell therapy is safe (at least at the one year mark) to use in patients and that larger doses of jCell are more effective at improving vision in patients. jCyte CEO, Paul Bresge commented on the trial’s positive results:

Paul Bresge

“We are very encouraged by these results. Currently, there are no effective therapies to offer patients with RP. We are moving forward as quickly as possible to remedy that. The feedback we’ve received from trial participants has been remarkable. We look forward to moving through the regulatory process and bringing this easily-administered potential therapy to patients worldwide.”

Bresge and his company will be able to navigate jCell through the regulatory process more smoothly with the product’s recent Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA). The FDA grants RMAT to regenerative medicine therapies for serious diseases that have shown promise in early-stage clinical trials. The designation allows therapies to receive expedited review as they navigate their way towards commercialization.

jCyte is now evaluating the safety and efficacy of jCell in a Phase2b trial in a larger group of up to 85 patients. CIRM is also funding this trial and you can read more about it on our website.

 

3 thoughts on “Encouraging Update on Clinical Trial for Retinitis Pigmentosa”

  1. The dedication of the researchers at Jcyte and CIRM to the RP community is unparalleled and thank you seems so insufficient. Nevertheless, thank you! This study’s results truly give new hope that the encroaching darkness may stop.

    Reply

    • Hi.i was diagnosed with rp in2016 at the age of 28.am eagerly waiting for new treatment to stop its progress.if you need I will be part of your research.am a medical student.now persuing my MD pharmacology.please inform further development in treatment.

      Reply

  2. Hope that Phase 3 will be success and it will end the suffering of millions of people across the world. Fingers crossed

    Reply

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