New stem cell treatment gains approval to enter US clinical trials
Biotechnology company Blue Rock Therapeutics has been given clearance in the USA to go ahead with in-human testing of a new type of stem cell therapy for retinal conditions like RP.
Unlike gene therapies, which aim primarily to slow or stop further deterioration, some cell-based therapies offer a chance to repair part of the retina and potentially restore some degree of vision. Many of the researchers and companies exploring this approach are using cell lines originally derived from human embryonic stem cells, but Blue Rock is pioneering the use of stem cells created from adult skin or blood.
In 2006, Japanese scientists discovered a technique that enables reprogramming of adult cells, turning them back into cells similar to those found in embryos. These “induced pluripotent stem cells”, or iPSC, can then be coaxed into developing into any type of specialized body cell, including photoreceptors and other retinal cells.
The adult cells used for iPSC generation can be easily and cost-effectively obtained, and iPSCs are now used routinely to create retinal tissue as a model for laboratory-based research. However, Blue Rock’s Investigational New Drug clearance from the US Food & Drug Administration paves the way for an iPSC-based therapy for retinal disease to be tested in people for the first time.
Blue Rock hopes that by using cells derived from healthy adult donors, they will be able to efficiently manufacture the therapeutic product at scale and with a high degree of consistency. In order to achieve repair, the cells will most likely need to be injected behind the retina during surgery. The first phase of clinical testing will focus almost entirely on safety, involving a very small number of people in the US, and will probably take around 12 to 18 months.
Meanwhile, thanks to your generous support, Retina UK is funding two projects that will support the development of better cell-based treatments in future. Prof Linda Lako at Newcastle University is creating stem cells that are “invisible” to the recipient’s immune system, making them less likely to cause inflammation and suffer subsequent damage. Prof Jane Sowden at UCL is developing a tiny bioengineered scaffold for implanted cells to sit in, with the aim of protecting them during surgery and maintaining them in the correct orientation. To find out more, visit Research grants – Retina UK