July 1, 2005

Planning Underway for Initial Stem Cell Safety Trial in ALS

San Francisco Meeting of Stem Cell Society Highlights State of the Science

Roberta Friedman, PhD, ALSA Research Department Information Coordinator

In a preparatory step, ALSA funded researchers have submitted to the U.S. Food and Drug Administration a proposal for a safety study in ALS patients of a stem cell therapy that is still being tested in animals. A teleconference with the FDA in May clarified what tasks must be performed before that initial human safety study can begin. Significant work in large animals must take place successfully before the stem cell strategy can be tested for safety in people with ALS. The safety study will then have to determine that the stem cell treatment is safe, before testing to determine effectiveness in ALS can take place.

ALSA is encouraged that planning is underway for stem cell therapy to be tested in ALS. Other equally promising therapeutic avenues are also continuing to be explored in ALSA funded research. On a cautionary note, no published study has yet demonstrated that the stem cell technique proposed for human study has prolonged life or slowed disease progression even in rats modeling the disease.

ALSA Science Director and Vice President Lucie Bruijn, Ph.D., said she does not know the time frame for potential safety clinical trials, but ALSA will keep the community informed. Should a Phase I study successfully demonstrate the safety of the therapy, participants could be offered enrollment in a Phase II efficacy study if that takes place.

Bruijn attended the third annual meeting of The International Society for Stem Cell Research (ISSCR) in San Francisco, dedicated to all aspects of stem cell biology. Bruijn, who serves on the society’s policy and foundation committees, called the meeting a forum for scientists to understand the basic biology of stem cells with the hope that this knowledge will better prepare us for applications in the clinic as well as a chance to bring the research into the public eye. A video shown at the meeting provided an overview of stem cell research. ALSA has a primer on stem cell research available as well.

Leaders in the stem cell field focused discussions on how to nudge stem cells to become various cell types including neurons, how to grow cells free of contamination that would be appropriate for clinical use, and appreciation of the complexity of creating and caring for these cells in the lab. New technologies and approaches to understanding the biology were discussed.

Ian Wilmut, Ph.D., a Scottish researcher at the University of Edinburgh who gained attention by cloning the sheep Dolly, also reported at the meeting on his collaboration with an international group aimed at a better understanding of ALS. Wilmut and his colleagues will take skin cells of people with motor neuron disease, extract the genetic material and place it into donated egg cells, which will be grown for six days. Stem cells from the embryos will be prompted to develop into nerve cells. Because these cells could incorporate a genetic flaw responsible for the disease, studying the genes might give clues about how the disease developed.

The gathering of some 2000 stem cell researchers from around the world also featured a keynote speech by Robert Klein who played a leading role in the development of the California Proposition 71 that has led to that state’s $3 billion funding of stem cell investigations independent of the federal government’s restrictions on which lines of the cells can be studied with federal funding. Klein is now chairman of the institute created by passage of the stem cell initiative by the state’s voters in the November 2004 election. The headquarters of the institute was recently set to be in the city of San Francisco after a spirited competition among several of the state’s cities (see the CIRM web site).

Klein emphasized the value and importance of the international society and its meeting in bringing all the world experts together to help move the field forward.