CIRM funds clinical study to test safety, efficacy of new retinitis pigmentosa treatment

Apr 19 2017

Cell therapy company jCyte is launching a phase 2b clinical trial to test the safety and efficacy of its developmental retinitis pigmentosa (RP) treatment, jCell. The company is recruiting 70 patients for the single-masked study, which will begin enrollment this month. The multicenter trial is being funded by jCyte, which recently received an $8.3 million matching grant from the California Institute for Regenerative Medicine (CIRM).

The trial's primary goal is to assess changes in visual function following treatment with jCell, focusing on visual acuity, visual fields, contrast sensitivity and the ability to navigate a mobility course.

"We are encouraged by the therapy's excellent safety track record in early trials and hope to build on those results," said jCyte co-founder Henry Klassen, MD, PhD. "Right now, there are no effective treatments for retinitis pigmentosa. People must find ways to adapt to their vision loss. With CIRM's support, we hope to change that."

Granted orphan drug status by the Food & Drug Administration, jCell uses retinal progenitor cells (RPCs) to rescue diseased retinal cells and possibly regenerate new ones. The non-surgical treatment requires a simple intravitreal injection, which can be performed under topical anesthesia.

"One of jCell's strengths is its simplicity," said jCyte CEO, Paul Bresge. "Because no surgery is required, the therapy can be easily administered. The entire procedure takes minutes."

Trial participants will receive a single RPC injection (or a sham injection as a control) in their worst-sighted eye. Progress will be assessed periodically over the following 12 months. Patients in the control group will be allowed to join the test arm after they have completed one year of follow-up.

RP is a genetic condition that destroys retinal cells and usually strikes people in their teens. Many patients are legally blind by the time they reach 40. Worldwide, nearly 1.5 million people suffer from RP, making it the leading cause of inherited blindness.

After carefully considering the jCyte proposal, CIRM's grant review working group gave the therapy a powerful endorsement, with 11 of 15 members saying the application had "exceptional merit and warrants funding." In a statement released during their December 13 meeting, the working group added:

"The proposed treatment approach holds the potential to address a clear and urgent unmet medical need, improve the standard of care and offer a strong value proposition for this patient population. 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."

Source: http://jcyte.com/2017/04/powered-cirm-grant-jcyte-launches-new-clinical-trial/