Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Leukodystrophy

Proteolipid protein 1
(PLP1)

Mouse and patient studies suggest human neural stem cells could help treat hypomyelination diseases. In a mouse model of hypomyelination, human neural stem cells transplanted into three different sites of neonatal or juvenile mouse brains differentiated into oligodendrocytes and myelinated axons. Brain slices from mice receiving transplants showed longer duration for action potentials than mice receiving vehicle, indicating functional myelination. In four juvenile patients with Pelizaeus-Merzbacher disease, a leukodystrophy caused by mutations in PLP1, transplantation of human neural stem cells into the frontal lobe white matter plus nine months of immunosuppression led to modest gains in neurological function and motor performance. Magnetic resonance spectroscopy indicated increased axon myelination at one year postsurgery. Next steps could include Phase II clinical testing (see Myelination from neural stem cells, page 6).

SciBX 5(42); doi:10.1038/scibx.2012.1120
Published online Oct. 25, 2012

Findings in both papers patented by StemCells Inc.; available for licensing

Uchida, N. et al. Sci. Transl. Med.; published online Oct. 10, 2012;
doi:10.1126/scitranslmed.3004371
Contact: Stephen A. Back, Oregon Health & Science University, Portland, Ore.
e-mail:

backs@ohsu.edu
Contact: Nobuko Uchida, StemCells Inc., Newark, Calif.
e-mail:

nobuko.uchida@stemcellsinc.com

Gupta, N. et al. Sci. Transl. Med.; published online Oct. 10, 2012;
doi:10.1126/scitranslmed.3004373
Contact: David H. Rowitch, University of California, San Francisco, Calif.
e-mail:

rowitchd@peds.ucsf.edu