Last month we alerted you to when the FDA released its hold on the first trial of an embryonic stem cell based treatment for a spinal cord injury. Now it looks as though for the second time, the Food and Drug Administration approved a company’s request to test an embryonic stem cell-based therapy on human patients.
This is such a great news from the life extension perspective. The more successful clinical trials there are, the closer we get to comprehensive cell therapies that have a huge potential not only to alleviate the current health problems, but to rejuvenate our bodies.
Advanced Cell Technology (ACT), based in Marlborough, Massachusetts will examine the safety of a therapy for Stargardt’s Macular Degeneration, an inherited juvenile eye disease affecting an estimated 1 in 10,000 young people in the US. As the disease progresses, a layer of the retina called the retinal pigment epithelium (RPE) degenerates, causing vision loss. It’s hoped the new therapy would also work for other types of macular degeneration, a widespread cause of blindness, particularly in the elderly.
The treatment involves replacing diseased RPE cells with healthy ones generated from human embryonic stem cells, and hinges on a technique the company developed to create extremely pure concoctions of the cells. In the trial, 12 individuals at several US medical centers will receive injections of the cells directly into the eye to test the safety of the procedure. In a rat model, the group showed that the treatment could extensively rescue photoreceptors, thereby improving vision.
Robert Lanza, ACT’s chief scientific officer, is hoping that the company’s RPE cells do the same in the patients, since the approval has been a long time coming. The company had initially submitted its request to begin human trials with its RPE cells in November 2009, but has spent the past year addressing the FDA’s concerns about the safety of the embryonic stem from which the RPE cells are made.
According to Lanza, the current study will focus on establishing the safety of the RPE therapy, with any visual improvement to be considered as a welcome bonus. “We’re starting out with a safety study in those with advanced disease,” says Lanza. “But hopefully if we get in earlier in the progression of the disease we might see greater impact on visual improvement. What this approval shows is that the readiness of the FDA to work with researchers to move exciting new stem cell based therapies out of the lab and into the clinic.”