Stem Cells for Stargardt’s Disease

Retinal photograph of Stargardt's disease

Wow. This is surprising – and groundbreaking – news. First, here’s some background:

In August of this year, The Wall Street Journal reported that Judge Royce Lamberth of U.S. District Court in Washington, D.C. had issued a preliminary injunction against federal funding for human embryonic stem cell research. The judge stated that federal funding violated a 1996 law prohibiting federal money for research in which a human embryo was destroyed. Many federally funded stem cell research projects were placed on “clinical hold” after Judge Lamberth’s ruling.

Lifting the Clinical Hold

However, on Monday, November 22, the U.S. Food and Drug Administration (FDA) lifted the clinical hold to clear Advanced Cell Technology’s (ACT) Investigational New Drug (IND) application and initiate a Phase I/II multicenter study, using retinal cells derived from human embryonic stem cells to treat patients with Stargardt disease. ACT’s product is a human embryonic stem cell trained to become a retinal cell. ACT first requested approval from the FDA to conduct the clinical trial approximately one year ago.

Research Sites

This will be the second-ever clinical trial of a therapy involving the use of human embryonic stem cells. One other clinical trial by the biotech company Geron is currently underway, using human embryonic stem cell-based therapy to treat spinal cord injury. A total of 12 patients will be enrolled in the study at multiple clinical sites, including the Casey Eye Institute in Portland, Oregon; the University of Massachusetts Memorial Medical Center in Worcester; and the UMDNJ – New Jersey Medical School in Newark.

About Stargardt’s Disease

As described in a press release from Advanced Cell Technology, Stargardt disease, also called Stargardt macular dystrophy and fundus flavimaculatus,

… causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss [i.e., cells that detect light, convert it into electrical signals, and relay those signals to the brain] associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium (RPE).

“There is currently no treatment for Stargardt’s disease,” said Dr. Robert Lanza, ACT’s Chief Scientific Officer. “Using stem cells, we can generate a virtually unlimited supply of healthy RPE cells, which are the first cells to die off in [Stargardt’s] and other forms of macular degeneration.”

The Initial Trial

For the initial clinical trial, only a few thousand stem cells will be injected into one eye of each volunteer. Patients will be followed closely to gauge the safety and effectiveness of the treatment. In an interview with, Dr. Lanza noted that

… the advantage, of course, is that we’re talking about a very small number of cells going into a very local area. Also, with the eyes there are very objective tests for visual acuity, so we can measure performance gains very objectively.

Dr. Lanza also stated that the company will soon apply for approval to test stem cell therapy in patients with age-related macular degeneration. VisionAware will provide updates of this groundbreaking stem cell research as they become available.

Update: On May 16, 2011, Advanced Cell Technology announced that the Casey Eye Institute in Portland, Oregon, will be the site for an upcoming clinical trial testing its stem cell-based treatment for Stargardt’s disease. Phase I/II of the human clinical trial, scheduled to begin shortly, is designed to test the safety and tolerance of RPE cells following their transplant into patients with Stargardt’s.