Clinical Trial of Neural Stem Cell Treatment for Dry AMD Authorized by the FDA

February 07, 2012
lab equipment
A groundbreaking treatment for retinal degenerations is poised to become the second-ever stem cell therapy to move into a Phase I/II clinical trial. The company StemCells, Inc. (SCI), has received authorization from the FDA to launch a clinical trial of its neural stem cell treatment for people with the dry age-related macular degeneration (AMD).

SCI’s announcement came 10 days after Advanced Cell Technology (ACT), which launched the first stem cell treatment clinical trials for retinal degenerations in 2011, announced encouraging safety and efficacy results for its first two study participants, one with dry AMD, the other with Stargardt disease. SCI is planning to launch a 16-participant clinical trial for dry AMD. Additional details of the study are forthcoming.

SCI’s neural stem cell treatment is designed to preserve vision by protecting the rods and cones, the cells that provide vision, from degeneration. The therapeutic cells, which will be injected beneath the macula, the central region of the retina, are designed to continually release proteins that keep rods and cones healthy.

In a research paper published on January 30, 2012, in the European Journal of Neuroscience, researchers reported that the neural stem cell treatment was safe and effective over the long term in a rodent model of retinal degeneration. The stem cells prevented vision loss without causing any immunological reactions or other safety issues.

Dr. Stephen Rose, chief research officer at the Foundation Fighting Blindness, says that SCI’s treatment differs from ACT’s therapy in one key respect: SCI’s neural stem cells are transplanted in their native form to play a protective role. In contrast, ACT derives retinal pigment epithelial (RPE) cells from embryonic stem cells. The new RPE cells are transplanted to replace and supplement the patients’ own RPE cells, which are affected by conditions such as macular degeneration.

“SCI’s stem cells works like a drug factory, providing continuous delivery of proteins to keep the retina healthy and preserve vision,” he says. “ACT, on the other hand, has the goal of replacing RPE lost to disease. RPE play a critical support and maintenance role in the retina, providing nutrients and waste disposal for rods and cones, and ACT is hoping its transplanted RPE provide those functions as well. Hopefully, both approaches work. They have implications for treating a wide range of retinal conditions, including retinitis pigmentosa and Usher syndrome.”

Dr. Raymond Lund, professor emeritus at the Oregon Health & Science University, and an investigator in the SCI rodent study, says, “None of this would have happened without direct Foundation support to me and my group, as well as the environment the Foundation has created to motivate scientists to be cognizant of the needs of people and their family members losing their sight to retinal diseases. I'm optimistic this effort will deliver sight-saving results.”

Funded by the Foundation for various projects for nearly 20 years, Dr. Lund is a renowned expert in stem cell research for retinal diseases, and also played a leading role in helping ACT move its therapy into the clinic.

SCI has clinical trials underway at the University of California, San Francisco, for its neural stem cell treatment in Pelizaeus-Merzbacher disease, a fatal neural disorder in children, as well as a clinical study in Switzerland for spinal cord injury. It is also pursuing preclinical studies of its therapy in Alzheimer's disease.

The Foundation will post additional information about SCI’s clinical trial for dry AMD, including recruiting contacts and inclusion and exclusion criteria, to its Web site when it becomes available.