The first – and positive – results of an in-human stem cell transplant to treat retinitis pigmentosa (RP) have been released by regenerative medicine company jCyte.
The transplants are safe and well tolerated six to 12 months after injection of the immature, undifferentiated retinal cells into one eye, the company said. The next step, a phase IIB clinical trial, could get underway as soon as next year, co-founder of jCyte, Dr Henry Klassen, told OT.
A number of patients received the stem cells, which were allogeneic or derived from an unrelated donor, in the trial funded by the California Institute for Regenerative Medicine. The research was conducted at the University of California, Irvine and the Los Angeles-based clinic, Retina Vitreous Associates.
Dr Klassen highlighted that: “To enrol in this initial safety trial, the patients had to be severely impacted visually and at a relatively late stage of disease progression.”
The first patients to receive the transplants had very low vision left – some were only able to see hand motions – though later patients with better vision were then also offered the treatment, once initial safety data first became available, he said.
Dr Klassen, said the initial results are pleasing, adding: “RP is an incurable retinal disease that first impacts people’s night vision and then progressively robs them of sight altogether. This is an important milestone in our effort to treat these patients.”
The phase IIB trial will look at how effective the transplants are, he said.
“The goal will be to show statistically significant benefits for the treatment, versus the control, group in terms of specific, clinically relevant visual functions.
“The timeline is yet to be determined, but [the trial] could get underway during the next calendar year, 2017,” Dr Klassen concluded.
Image credit: Christian Hamel