Phase I Candidates
Athersys is also taking on AMI with Multistem, which is obtained from adult bone marrow or other nonembryonic tissue sources. In February Athersys completed patient enrollment in a Phase I trial. Results are expected mid-year upon completion of a four-month patient follow-up period and analysis of results. Athersys reports that it also received clearance to initiate a clinical trial in patients who have suffered an ischemic stroke. Multistem, which is an allogeneic stem cell-based product, is also being studied as an oncology treatment support as well as in a range of conditions involving immune system function.
StemCells is trying to tackle CNS disorders with neural stem cells. It uses a library of mAbs against cell surface antigens to identify, purify, and characterize the stem cells of interest, which are then sorted and tested in vitro or in vivo. Purified populations of stem cells are then expanded, banked, and retested in vivo.
The company says that its lead candidate, HuCNS-SC, can adapt in host brain environments and behave like host neural cells, dividing, migrating, and differentiating. And because cells are normal, genetically unmodified, and highly purified human CNS stem cells, they may prove safer and more effective than unpurified mixtures of cell types, it adds.
In November 2009, StemCells begun a Phase I study in Pelizaeus-Merzbacher disease, a myelination disorder that primarily affects young children. And on April 27, StemCells submitted a protocol to the FDA for initiation of a second clinical trial in neuronal ceroid lipofuscinosis, a brain disorder seen in children.