Stem cell treatment als3/6/2023 To improve efficacy of the treatment and decrease unwanted side effects such as bleeding, researchers further investigated the drug dosing and administration.Ĭlinical trials are now underway for peripheral administration of APC for ALS. It is previously approved by the FDA, proven safe and is currently being given to patients for another condition. Identifying an FDA approved drug for slowing progression of ALSĭon Cleveland's team, in partnership with Professor Berislav Zlokovic at University of Rochester, demonstrated that administration of a serum protease known as C(APC) slowed disease progression in mice that mimic an inherited form of the fatal paralytic disease ALS.ĪPC is the chemical cousin of a drug currently used to treat sepsis. The researchers will study two methods of administering progenitors in animal models and test the safety and efficacy of these approaches, with the goal of providing proof-of-principle and laying the groundwork for clinical trials by 2014. This project is being funded by an $11.5 million California Institute for Regenerative Medicine (CIRM) "disease team" grant for stem cell research. Previous research indicates that transplanting healthy glial cells into patients could be a possible treatment for ALS, and animal studies have shown that astrocytes possess particular promise. In ALS, the decay of astrocytes and other cells eventually causes neurons to malfunction and die, leading to a host of debilitating and ultimately fatal consequences. The particular job of astrocytes is to help with neurotransmissions and neuronal metabolism. Researchers at UC San Diego, along with co-principal investigator Sam Pfaff, Ph.D., a professor in the Salk Institute's Gene Expression Laboratory, hope to develop an ALS therapy that uses human embryonic stem cells to create astrocyte precursors that would be transplanted into patients where they would mature into new and healthy astrocytes that could halt - and perhaps reverse - the progressive ravages of ALS.Īstrocytes are glial cells, a family of cells that support the proper functioning and insulation of neurons. Current Projects in ALS & Motor Neuron Disorder Research Developing a cell replacement therapy for ALS
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