Use of JNK Signaling and JNK Inhibitors for the Treatment of Spinal Muscular Atrophy


Current State of the Art:

SMA is a devastating neurodegenerative disorder with progressive muscle weakness and loss of motor control due to the loss of neurons in the spinal cord. SMA affects approximately 4 out of every 100,000 people. Children born with SMA Type I, die within 2 years of birth. Those diagnosed with Type II and III can live on into adulthood with strictly managed care.


Treatments currently undergoing clinical trials for SMA therapy are Olesoxime (Trophos), Quinazoline495 (Repligen), Stem Cells (California Stem Cells), ISIS-SMNrx (Isis Pharm), SMN Gene Therapy (OSU), Indoprofen Analogues (NINDS), tetracycline derivatives (Paratek) and Small Molecules from SMA/PTC and Novartis.

Drugs currently being used off-label to treat SMA include Albuterol, Hyrdoxyurea, Riluzole, Somatotropin, Sodium Phenylbutyrate and Valproic Acid.


Disadvantages with the Current Art:

Therapeutics are not readily available for SMA that have been shown to treat the debilitating and sometimes lethal disease. Patients are offered off-label use of FDA approved drugs while other targeted therapies await FDA approval.


Advantages of Invention:

The inventor has identified a promising therapeutic regimen that would prevent loss of motor neurons in the spinal cord of an animal model system for neural degeneration:

·         Compounds and method of use identified

·         Analyzed in an animal model system for SMA

·         Compounds prevented neurodegeneration in spinal cord neurons

·         Candidate for orphan status


Patent Status: PCT Filed


Inventor: Laxman Gangwani, PhD


Case Number: GHSU 2009-036

Patent Information:
For Information, Contact:
Augusta University
Laxman Gangwani
Muscular Atrophy
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