In a closely-watched clinical trial, StemCells said it has transplanted the company’s purified human neural stem cells into the first five patients in the final cohort of its 16-patient phase I/II trial for the advanced or dry form of age-related macular degeneration (AMD). 

They are part of an eight-member group that will receive a dose of one million stem cells injected into the space behind the retina of the most affected eye. The outpatient procedure takes about one hour.

“The premise behind our therapeutic approach is that it is a one-time surgical transplantation of stem cells and the patient goes home the same day,” said Stephen Huhn, M.D., vice president of CNS clinical research at StemCells. “We will release interim data on the study at the International Society of Stem Cell Research in Vancouver, Canada on June 18.”  

StemCells is the first company to conduct stem cell research in AMD directly in the diseased eye.

An estimated 1.75 million people in the U.S. have AMD, which typically strikes adults in their 50s or early 60s and gradually progresses to destroy central vision. It results in the loss of photoreceptors (rods and cones) from the macula, the central part of the retina. The dry form accounts for 85% of patients with AMD, often referred to as “geography atrophy.” An estimated 10 million people in the U.S. either have the dry or wet form of AMD or are at substantial risk for developing it, according to the Foundation Fighting Blindness.

Huhn said there is no “successful” FDA-approved therapeutic treatment for the dry form, which is why his company is pursuing a stem cell strategy. He said the trial, studying the safety and preliminary efficacy of purified human neural stem cells over a one-year period, is enrolling patients in five centers, including three in Texas and one each in New York City and Palo Alto, Calif. Participants will be followed for an additional four years in a separate observational study.

“We view the eye as just another part of the central nervous system and recognize the photo receptors are a type of neuron that can be protected by stem cells,” said Huhn. “We think we can protect the eye for a very long time and preserve vision, based on our animal model. Also, the eye is particularly amenable to measuring the effects of stem cell transplantation.”

An estimated 20 stem cell/spinal cord phase I trials are listed on clinicaltrials.gov from various sponsors seeking patients.