There is big news in the SMA world today. Here is part of the bulletin from Families of Spinal Muscular Atrophy.
CSC, a leading stem cell therapeutics company, has developed a stem cell-derived motor neuron transplantation therapy, MotorGraftTM, for the treatment of SMA Type I. Pre-clinical studies completed in collaboration with the Hans Keirstead Research Group at the University of California, Irvine have shown functional benefit and safety in animal models. CSC's MotorGraftTM was granted orphan drug status for treatment of SMA by the FDA in late 2009.
Filing of this application is the first step in a multi-phase clinical development pathway aimed ultimately at approval of a novel therapy. The approval process for cutting-edge therapeutic approaches such as cell products may present unique regulatory challenges compared to conventional drugs, so companies and the FDA must work in close partnership to ensure safety and efficacy of these first in-human products. A cautious regulatory approach has been the norm in cell therapy applications submitted to date in other disease areas.
This trial will study the safety of MotorGraftTM and the surgical procedure required to deliver these cells directly into the spinal cords of patients with SMA Type I and will enroll a very limited number of patients. This IND filing is a major milestone in the search for a treatment for SMA patients.
One more big step!
Love to you all,
8 Years an Angel
3 weeks ago