Stem Cell Research

University of Utah’s Nobel Prize-winning geneticist Dr. Mario Capecchi successfully treats mental disorder with adult bone marrow stem cell transplant

Scientists were lead to the conclusion that the immune system has a direct correlation to behavior with the discovery of a lab mouse suffering from trichotillomania, a condition where one pulls their own hair out, was cured by a bone marrow transplant. Traditionally these kinds of conditions were treated with drugs to alter the chemistry of the brain.

University of Utah’s Nobel Prize-winning geneticist Dr. Mario Capecchi tried a new approach on a lab mouse by treating it with a bone marrow transplant normally used on cancer patients. Dr. Capecche’s team found that people and animals afflicted with behavior disorders have deformed microglia cells. So, instead of treating mental illness the way doctors traditionally have — with medication to alter brain chemistry — they tried a new approach by treating the immune system.

“Microglia , immune cells originating from adult stem cells in the bone marrow and migrate from the blood into the brain, were believed to be “scavenger cells” that would clean up damage in the brain, but are now believed that microglia are much more sophisticated and are actually controlling behavior and they do it by interacting with the nerve cells in your brain,” Capecchi says.

This discovery could offer hope to parents of children afflicted with behaviour disorders such as autism.

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The Multiple Sclerosis Research Center of New York (MSRCNY) and the International Cellular Medicine Society (ICMS) announced that the ICMS Institutional Review Board has approved the first study to use autologous brain-like or neural stem cells for multiple sclerosis.

“This initial stem cell treatment strategy opens up new avenues of treatment options focused on repair and regeneration that didn’t exist before,” said Dr. Saud A. Sadiq, neurologist and director of the MSRCNY.

The study will investigate a regenerative strategy using mesenchymal stem cell-derived neural progenitor cells harvested from the patient’s own bone marrow. These stem cells will be injected into the cerebral spinal fluid surrounding the spinal cord in 20 participants with a confirmed diagnosis of progressive MS.

This will be an open label safety and tolerability study where all participants will be enrolled through the Multiple Sclerosis Research Center of New York (MSRCNY).

All study activities will be conducted at the MSRCNY and affiliated International Multiple Sclerosis Management Practice (IMSMP).

Participants in the three-year study will undergo a single bone marrow collection procedure, from which the neural progenitor cells will be isolated, expanded and tested prior to injection. Participants will undergo three rounds of injections at three month intervals. Safety and efficacy parameters will be evaluated in all participants through scheduled follow-up visits.

MS is a chronic human autoimmune disease of the central nervous system (CNS) that leads to myelin damage and neurodegeneration. Stem cell transplantation has long been regarded as a viable treatment option for patients with neurodegenerative disorders. The clinical application of autologous neural progenitors in MS is the culmination of almost a decade of basic research conducted at the MSRCNY, which has found that the injection of these cells may decrease inflammation in the CNS and promote myelin repair or neuroprotection.

The ICMS IRB reviewed the treatment protocols, informed consents and the inclusion/exclusion criteria for the study at its November meeting.