Neurological disorders (spinal cord injury, stroke, cerebral trauma, demyelination diseases and related pathologies) are treated by stimulating the migration of neural cell precursors or their neurite extensions to a region where the pathology is present. The migration is accomplished by increasing the presence of hydrated macromolecules (e.g., polysialic acid (PSA) both in the area of interest and along the path linking neuronal stem cell reservoirs or neurites with such region of interest. These hydrated macromolecules loosen up cell-cell interactions, facilitating cell migration and neurite axon extension. The therapy involves increasing the activity of polysialyltransferases (PSTs) or increasing the presence of the polysialylated form of the neural cell adhesion molecule (PSA-NCAM) on the cell membrane of the cells along the path extending from a source of corticospinal neurons or neural stem cells to the area where, or across which, the pathology is present. Therapeutics that may be utilized include, but are not limited to, a PST polynucleotide, polypeptide, or peptide, or its analogs, derivatives, fragments, or homologs; a conjugate of a moiety that targets a component of a neural cell surface and a hydrated macromolecule; or a compound that increases PST activity.
There is no effective curative therapy for spinal cord injury or stroke.
Therapeutic drug, therapeutic process
Proof of concept obtained in mice
Dr. Urs Rutishauser
World-wide patent protection available
Lisa Placanica, PhD
Tel: 212-639-6181; Fax: 212-717-3439
E-mail: placanil@mskcc.org