Retinitis Pigmentosa Program

demac_imageThe Neural Stem Cell Institute and its parent non-profit organization the Regenerative Research Foundation, are committed to developing regenerative therapies for nervous system repair. We have a strong program focused on Retinitis Pigmentosa (RP), an important blinding disease for which current therapy is generally ineffective. The basis of our RP program is the discovery of a unique adult human retinal pigment epithelium stem cell (RPESC) that can be extracted from patients up to age 99 years of age, thereby providing a patient-matched cell for transplantation therapy and for screening drugs effective against RP. This basic research program received competitive peer-review funding from the New York State Stem Cell Research Board, the American Health Assistance Foundation and the Steinbach Foundation. Having discovered this novel stem cell, we are now using the RPESC to benefit RP patients in translational research.

RP is a complex disease in which loss of retinal pigment epithelial cells (RPE) causes loss of the overlying neural retina and diminished vision. Human RPESCs can be used for cell replacement therapy to restore supportive RPE cell functions, improve retinal viability and preserve vision. Promising results using other types of stem cell for replacement therapy of RP are limited by immune rejection of the implanted foreign cells. This immune problem can be overcome because RPESCs enable autologous transplantation. In this approach, RPESCs are taken from a patient, expanded in culture, and then transplanted into the same patient. Before this can be offered to patients, however, we need to investigate the effect of RPESC cells implanted into animal models of RP to determine if RPESC, like other types of stem cells, improve vision. Another exciting line of research made possible by RPESC is to create disease models of RP in the culture dish, models that can be used to find drugs that slow RP progression. These are defined and critical projects that we are poised to accomplish, and we ask you to help fund this important work.

The NSCI is the first independent stem cell research institute in the USA. This not-for-profit 501©3 organization was co-founded by Sally Temple, Ph.D. who helped discover and define nervous system stem cells. Dr. Temple has recruited highly talented scientists working to translate discovery research into new therapies for central nervous system repair, and Jeffrey Stern, Ph.D., M.D., a physician-scientist who carries out research and cares for patients with retinal problems. At NSCI, our staff of 30 highly talented researchers works in a state of the art facility to translate discovery research into therapy for central nervous system repair. We are uniquely positioned to make headway in this exciting research, and your gift will enable us to take the defined steps needed to generate greatly needed novel therapeutics.