First Human Trial For Stem Cell Based AMD Therapy By NIH
Age-related macular degeneration – AMD is a widely occurring disease that affects more than 1 million people a year in India. It is characterized by loss of the center of the field of vision. Retinal pigment epithelium (RPE) is a thin cell layer that nourishes the photoreceptors lack of which leads to the loss of vision in AMD. Scientists at NIH’s National Eye Institute (NEI) have developed a stem cell-based therapy to prevent blindness in AMD cases, which have shown promising results from animal-based data and now is all set for Human-based clinical trails.
The AMD therapy designed by NEI works by replacing prone to die RPE cells thus preventing vision loss.
Blood samples were collected from AMD patients and were converted into Induced pluripotent stem cells iPSCs which were further directed to become RPE tissue. The iPSC-derived RPE cells were further grown to replicate the way it occurs naturally – in a single layer. The RPE was grown on on a biodegradable scaffold designed to promote integration once they reach the retina.
While experimentation of the therapy in pigs and rats models, the lab-grown RPE cells successfully integrate with the host body retina within 10 weeks after the RPE were implanted, the researchers said
and the degeneration of retina’s in the animal model was reversed. The expression of the gene RPE65 by The RPE cells upon Immunostaining confirmed that the cell layer had reached a level of maturity necessary for regenerating visual pigment within the photoreceptors. Even the electric pulses recorded from the photoreceptors showed positive results.The concept of reversing a damaged tissue into a healthy one via stem cell-based therapy has been used by researchers worldwide, ranging from Eye diseases to malignant cancers conditions.
iPSC’s therapeutic use in humans has been a major concern lately due to a fear that it can propagate uncontrollably and give rise to cancerous cells. But the NIE researchers have confirmed that their therapy is designed specifically to such avoid cancer-causing mutations.
Kapil Bharti, Ph.D., head of the NEI Unit on Ocular and Stem Cell Translational Research and the lead investigator said that if the phase 1 trial moves ahead it would be the first ever to test a stem cell-based therapy derived from induced pluripotent stem cells for treating a disease.
