Stem Cell Therapy

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Stem cells are notable cells that are produced in the body and have the amazing potential to transform into many different cell types during growth and early life.

There are two main different types of stem cells that are most likely to be used for stem cell therapy:

  • Embryonic
  • Induced pluripotent (iPS cells). These cells are engineered in the lab.

Stem Cell Clinical Trials for Stargardt’s Disease

Ocata Therapeutics (formerly Advanced Cell Technology, ACT) have conducted a clinical trial in patients with advanced Stargardt's disease using human embryonic stem cell derived retinal pigment epithelial (RPE) cells. These cells were injected under the retina. RPE cells whose role is to support and nourish the photoreceptors (rods and cones) in the retina, degenerate in Stargardt's disease, often before photoreceptor cell loss. The idea is that by replacing healthy RPE cells, this will stop or slow down photoreceptor cell loss and thereby slow / halt vision loss.

Early results have been promising with a successful safety trial, conducted in the USA and UK.

Ocata Therapeutics has since been taken over by Astellas Pharma – with a new Phase I/II trial in Stargardt's disease of an improved RPE cell line being developed.

Opsis Therapeutics are developing a clinical trial where a patch consisting of retinal cells derived from stem cells will be placed at the back of the eye with the hope to restore vision. Dr. David Gamm, MD, PhD at the University of Wisconsin-Madison is leading this study with a team of researchers.

Dr Anai Gonzalez-Cordero, in association with Professors Robin Ali and Rachael Pearson, at UCL Institute of Ophthalmology, is working on a type of stem cell called an induced pluripotent stem cell or iPS cell. iPS cells are generated in a lab from tissue samples donated by patients. By taking a sample of tissue, such as blood or skin, these cells can be reprogrammed and turned into stem cells, which can subsequently be turned into photoreceptors for potential transplantation in the future.

Dr Anai Gonzalez-Cordero is currently working on producing retinal cells from patients with Stargardt's disease. By studying these cells, it is hoped we can have a better idea of how the faulty Stargardt's gene causes retinal cells to be damaged and subsequently be lost. It can also help with developing potentially new treatments as these cells can be tested with drugs or viral vectors (gene therapy) to explore the safety and any beneficial effects of these approaches.