Stem Cell Therapy for ALS
Clive Svendsen, PhD
University of Wisconsin, Madison, Wisconsin
As part of The ALS Association's commitment to funding research on stem cells, ALSA began funding a new project entitled "Stem Cell Therapy for ALS" on February 1, 2002. The study is led by Dr. Clive Svendsen in collaboration with Dr. Su-Chun Zhang, both at the Waisman Center, University of Wisconsin.
Dr. Svendsen will use human and rodent neural stem cells as a delivery system for trophic factors (nutrients important for the survival and differentiation of motor neurons). Although several laboratory studies have indicated that trophic factors such as glial-derived neurotrophic factor (GDNF) prevent or slow motor neuron degeneration, clinical trials using these trophic factors have been extremely disappointing. It is likely that these failures are due in part to the inability of the trophic factor to reach the damaged site. Dr. Svendsen will modify stem cells to release GDNF in a controllable manner and transplant them into a rat model of ALS. The rat model has recently been generated as part of the Lou Gehrig Challenge initiative and is an ideal model system for such studies.
The Waisman Center-University of Wisconsin has established a multi-disciplinary team of investigators with expertise in stem cell biology, tissue transplantation and gene delivery systems as part of its Stem Cell Research Program. Their mission is to understand the biology of stem cells and apply this knowledge to develop novel therapies for neurological disorders. Exploiting their collaborative and technical strengths, the team is well positioned to tackle the many challenging and unanswered questions to drive this field forward.
Dr. Su-Chun Zhang has a project* already being funded by ALSA to determine the factors that will stimulate stem cells to develop into and function as motor neurons. Zhang, together with Dr. James Thomson who first isolated human embryonic stem cells, have shown that human embryonic stem cells differentiate into specialized cells when transplanted into the brains of mice. These studies published in the December issue of Nature Biotechnology represent critical techniques that may be used in the future to repair brain and spinal cord tissue as a potential treatment for neurological diseases.
* Dr. Su-Chun Zhang's project, "Generation of Human Motor Neurons from Stem Cells," is funded by ALSA with support from the Jeff Kaufman Fund, The ALS Association Southeast Wisconsin Chapter, The Neil Brourman, M.D. ALS Research Fund, and Hope for ALS, Inc.
Studies published in the December issue of Nature Biotechnology.
