The California Institute for Regenerative Medicine (CIRM) has awarded a $5.1 million grant to advance the development of stem cell-based therapy for blinding retinal conditions. Specifically, the proposed treatment will use patients' own skin cells to generate autologous induced pluripotent stem cells to derive retinal pigment epithelium cells, which are lost in many blinding eye conditions.
The recipients, scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and the Stein Eye Institute, plan to pursue the next critical step toward a clinical trial in humans by submitting an investigational new drug application to the FDA.
"We're grateful to CIRM and the people of California for this grant, which will allow our team to translate our research into a treatment for major causes of untreatable blindness such as macular degeneration," said lead researcher Dr. Steven Schwartz, Ahmanson professor of ophthalmology and chief of the retina division at the UCLA Stein Eye Institute. "This pivotal investment brings hope to countless patients and families suffering with blinding eye disease."
In the research, induced pluripotent stem cells will be differentiated into a large volume of the patient's own retinal pigment epithelium cells—between 50,000 and 500,000—which will then be surgically transplanted into their own retinas. Pre-clinical research suggests these new cells will integrate with the patients' remaining retinal pigment epithelium cells, rescue related eye tissue and spur regeneration of the surrounding tissue. Thus, these transplants may preserve or restore vision.
By creating retinal pigment epithelium cells from patient-derived induced pluripotent stem cells, the new method developed by Schwartz and his team should thwart the risk of immune system rejection, eliminating the need for immune suppression. Because many maculopathies are age-related, this key improvement may make Schwartz's novel treatment more accessible to the individuals who need it most. Another novel feature of the method is that the new retinal pigment epithelium cells will be delivered to patients suspended in liquid rather than on a scaffold. This makes it possible for the treatment to be administered in a minimally invasive transplant surgery, which reduces risks and provides faster recovery time.
These two advantages open up the possibility of administering the cell therapy earlier in the course of macular diseases, while some functional tissue remain. Early intervention could be critical to the success of treatments for maculopathies because when diseased retinal pigment epithelium cells are lost, neighboring and essential tissues such as photoreceptors are compromised or disappear completely.
Schwartz is developing this treatment in collaboration with a multi-disciplinary team comprised of stem cell biologists, ophthalmologists, retinal biologists, immunologists, transplant surgeons, bioengineers and regulatory experts.
Image: Patient-specific human induced pluripotent stem cell-derived retinal pigment epithelium cells. Image courtesy of Saravanan Karumbayaram/UCLA Broad Stem Cell Research Center.
See the full news release from UCLA.