Through this so-called “in vitro clinical trial,” these HSCI scientists expect that they will be able to identify those compounds that work on the largest numbers of patients as well as identify those subsets of patients that respond best to selected drug candidates.
Mayo clinic stem cell treatment for ms skin#
Together, members of the Rubin and Eggan labs have pioneered a method to profile these candidate compounds, as well as prior clinical candidates, against a panel of sixty motor neuron populations derived from individual ALS patient and control skin cells. HSCI Executive Committee member Lee Rubin, PhD, has identified two small molecules and their target signaling pathways that promote survival of human ALS patient-derived motor neurons. In addition, HSCI’s investment in the Therapeutic Screening Center has made it possible to screen several drug candidates for ALS. His group found a toxic signal produced by accessory neuronal cells - called glial cells - that compromises the survival of motor neurons in patients with ALS, a previously unappreciated characteristic of the disease. Human motor neurons derived from ALS patients have been used by HSCI Principal Faculty member Kevin Eggan, PhD, to glean new insights into the development and progression of the disease. This achievement means that the disease can be studied in a laboratory culture dish filled with the cells responsible for this devastating condition, allowing HSCI scientists to identify new therapies for ALS. In a leap forward for the field, HSCI scientists have also derived human induced pluripotent stem cells - mature cells that are manipulated back to a stem cell state - from the skin and blood of ALS patients. The accelerated pace of advancements in stem cell biology at HSCI has enabled the production of millions of motor neurons generated from mouse embryonic stem cells derived from ALS genetic disease models, as well as normal human embryonic stem cells. Harvard Stem Cell Institute (HSCI) scientists are leaders in the search for an effective therapy for ALS.
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Furthermore, until recently, there was also no way to test if a drug would even work on motor neurons, the cells affected in ALS, because they couldn’t be obtained in large numbers. This diversity of potential causes means that any therapy would only be effective on a certain subset of patients. Only ten percent of ALS cases are caused by inherited forms of known genes. Ninety percent of cases are sporadic, which means they are caused by a combination of genetic mutations and/or presumed environmental variables. Several factors contribute to the difficulty in finding effective therapies for ALS.
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The cause of ALS is largely unknown and there is currently no effective therapy. Every year, over 5,000 Americans are diagnosed with ALS, which primarily affects people in their forties through seventies. People with ALS may experience weakness in their limbs followed by a rapid and progressive paralysis that leads to respiratory failure. Lou Gehrig’s disease, more formally known as Amyotrophic Lateral Sclerosis (ALS), is a neurodegenerative condition that involves the breakdown of motor neurons in the brain and spinal cord. Gehrig was soon diagnosed with a rare, late-onset, and fatal disease that would come to take his name. Known for his record-number home runs and perfect game attendance, the so-called “Iron Horse” was suddenly having trouble keeping his balance and catching fly balls. New York Yankee first baseman Lou Gehrig was having a lousy season in 1939.
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Harvard Stem Cell Institute scientists offer new hope for the 30,000 Americans living with a paralyzing and fatal disease with no known cause.