FDA Gives the Go-Ahead for Phase I Trial of ALS Stem Cell Therapy

Researchers at Cedars-Sinai Regenerative Medicine Institute in Los Angeles, CA, have obtained FDA approval to initiate a Phase I clinical trial testing a new candidate stem cell therapy for ALS.   The stem cell therapy is based on transplantation of human neural progenitor cells (hNPCs) that are genetically engineered to secrete glial cell line-derived neurotrophic factor (GDNF), a protein with known neuroprotective effects. In ALS rat models, transplanted hNPCs expressing GDNF integrated into the spinal cord and improved motor neuron survival (Klein et al., 2005,  Suzuki et al., 2007). The planned safety trial will entail unilateral injection of stem cells into the spinal cord, allowing for direct comparison within each patient of the treated and untreated side. The trial is predicted to begin recruiting patients before the year’s end.

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