Advanced Cell receives green light from independent monitoring board to move forward with US trials

Advanced Cell Technology (OTCBB:ACTC) said Wednesday that it has received approval from the independent Data and Safety Monitoring Board to move forward with enrolling and treating the next set of patients in each of its two US stem cell clinical trials.

In July, surgeons treated the first patients in the company’s clinical trials for Stargardt’s Macular Dystrophy (SMD), a degenerative eye disease seen in young people, and Dry Age-Related Macular Degeneration (Dry AMD), a degenerative disease causing vision loss in older people.

In both cases, eventually, severe vision loss and even blindness results from photoreceptor loss associated with degeneration in a layer of the retina, called the retinal pigment epithelium (RPE).

The company’s treatment uses retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs), which can potentially be used to supply an unlimited amount of healthy RPE cells to treat degenerative retinal diseases.

Each patient received an injection of 50,000 hESC-derived RPE cells in one of their eyes. Both the Dry AMD and SMD clinical trials are dose-escalation studies involving twelve patients in each, and are designed to evaluate the safety and tolerability of the injected RPE cells.

The study design involves four cohorts of three patients, each being treated at a pre-determined dose of RPE cells, ranging from 50,000 to 200,000 cells.

Based on the results of the first patient in each study, the independent board authorized the company to move forward with the next two patients in the studies. Each of these additional patients will also be treated with 50,000 RPE cells, the company said.

“The unanimous recommendation of the DSMB to proceed with the next four patients represents a significant milestone for our clinical programs,” said chief scientific officer, Dr. Robert Lanza.

“We hope that the positive trend in clinical results derived from treating the first patients with our RPE cells will continue.”
Chairman and CEO of Advanced Cell, Gary Rabin, added: “The outcome of this in-depth safety review by an unbiased team of experts is very reassuring.

“The DSMB, which includes independent experts in macular degeneration, has recommended that the study continue at the present dosage of cells. We hope that in the near future we will be able to share both safety and physiological data we are continuing to obtain in these trials.”

Last week, the company announced that health authorities in the UK gave the go-ahead for its stem cell trials to treat patients with Stargardt’s, the first time such studies have been approved in Europe. The UK trials will begin in December at the prolific Moorfields Eye Hospital in London, the oldest and largest eye hospital in the world, under Professor James Bainbridge, consultant surgeon and Chair of Retinal Studies at University College London.- Deborah Sterescu

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