U.S. approves trial of using human embryonic stem cells to treat eye diseases

The US Food and Drug Administration has approved a second clinical trial involving cells derived from human embryonic stem (hES) cells. A biotechnology company called Advanced Cell Technology (ACT), based in Marburg, Massachusetts, announced on November 22 that it had obtained permission to treat macular dystrophy in Stargardt's disease-a type of gradual loss of vision in children Rare diseases-patients' retina cells are tested.

U.S. approves trial of using human embryonic stem cells to treat eye diseases

U.S. approves trial of using human embryonic stem cells to treat eye diseases

There are several reasons why cell therapy for this disease seems promising. Robert Lanza, chief scientific officer of ACT, pointed out that first of all, the eyeball is an organ with privileged immunity, so the researchers hope that patients will not have to be treated with anti-rejection drugs after transplantation. Second, Lanza said that since the retina can be observed at the single-cell level, it should be able to follow the performance of the transplanted cells very accurately.

U.S. approves trial of using human embryonic stem cells to treat eye diseases

U.S. approves trial of using human embryonic stem cells to treat eye diseases

The company said it plans to treat 12 advanced patients with the disease in clinics in Portland, Oregon, Worcester, Massachusetts, and Newark, New Jersey. Lanza said the patients will receive up to 200,000 retinal pigment epithelium (RPE) cells from hES cells, which will be transplanted directly into the eye.

Experiments conducted in animal models have always been promising. Mice with a Stargardt disease preserved their vision after transplanting human RPE cells, but the untreated control group eventually became blind. Lanza said the researchers found no side effects. He said that the company has filed an application to try to treat age-related macular degeneration, a disease with similar characteristics that affects 30 million people in the United States and Europe.

ACT submitted its Stargardt disease trial application a year ago, and hopes that it may become the first company to test hES cells in patients. It has not been able to do this completely. In October this year, researchers from Jielong Biopharmaceuticals and the University of California Irvine began the first clinical trial of using hES cells to treat patients with spinal cord injury.

ACT is one of the first companies involved in embryonic stem cell research, but it has experienced ups and downs and has been closed for several times. Lanza said: "Our phone has been closed several times. But we did it. This is some very difficult work and is the result of cooperation."

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