Stem cell based clinical trials for retinitis pigmentosa (RP) begin enrolment in the USA

Image of Dr. Maria MeehanIn exciting news in stem cell technology, two companies have recently received approval to begin enrolment in the USA for their respective stem cell based clinical trials for retinitis pigmentosa (RP). The first trial is being conducted by Dr Henry Klassen of the University of Irvine, California under their start-up company Jcyte. This trial has already enrolled the first four individuals out of a total of 16. All participants in the trial will be injected with a single injection of retinal progenitor cells into one eye. The other clinical trial due to begin will be conducted by the company ReNeuron and will be co-ordinated by Dr Eric Pierce of the Massachusetts Eye and Ear. This trial will also inject human retinal progenitor cells, but they will be injected by sub-retinal injection (under the retina).

What are retinal progenitor cells?

Retinal progenitor cells are early descendants of stem cells that have already started down the differentiation pathway of becoming retinal cells. This form of experimental therapy is intended to preserve vision by treating at a time when degenerating photoreceptor cells in the patient’s retina can be protected and potentially reactivated. These trials will not address the more extreme challenge of replacing lost cells, which is still under development and has not yet moved to the clinic.

What do they hope to learn from these trials?

The most important measurements of these trials will be to test whether this approach is safe, and individuals will be followed up for 12 months post-treatment with safety monitoring. Vision measurements will also be taken and compared to the un-treated eye as a preliminary way of seeing whether there is any efficacy with these approaches. We will keep you posted on updates

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from both of these clinical trials as they are released in the future.

Maria Meehan, PhD. Research Manager


Dr Eric Pierce is one of the world renowned speakers at our Retina 2015 conference in November.  Dr Pierce is an ophthalmologist and scientist. He is focused on “gene hunting” – finding new disease genes associated with rare diseases such as retinitis pigmentosa and Stargardt disease, amongst others. He investigates how these disease genes lead to blindness and uses this information to develop gene and genetic therapies to prevent vision loss.