CURRENTLY AVAILABLE TECHNOLOGIES
California State Polytechnic University, Pomona
Technology Transfer Office and Industry Clinic
Contact: G. Marie Talnack, (909) 869-3771
gmtalnack@csupomona.edu
The following is a listing of some of the technologies currently available for licensing or followon technology maturation opportunities at California State Polytechnic University, Pomona (one
of the 23 campuses of the California State University system):
TREATMENT AND DIAGNOSIS OF HUNTINGTON’S DISEASE
Patent Number: U.S. 61/927,645–filed January, 2014
Description of the Technology:
This invention relates to the discovery of novel methods for the treatment and diagnosis
of Huntington’s disease. Huntington’s disease (HD) is a degenerative disease, characterized by
debilitating motor and cognitive defects. Presently, no effective treatment exists for patients with
HD. The cognitive and motor deficits associated with HD have led the majority of the scientific
research to focus on the neurological component of the disease. This invention discloses and
focuses on the muscular component of HD. This invention addresses the physiological properties
of HD muscle and provides novel methods for the treatment, diagnosis, and prognosis of HD.
Current treatment regimens are aimed at delaying the progression and severity of the disease
symptoms from a neurological perspective. Traditional therapy is a drug that targets the brain
and suppresses dystonia or reduces muscle rigidity. They act as an antidepressant or mood
stabilizing drug. This approach aims to reduce the number of HD symptoms and/or slow the
progression of HD symptoms. Unfortunately, the current therapy is moderately effective at best.
There also remains an unmet need to develop treatment regimens targeted at the muscular defect
component of the disease-rather than using neurological drugs to indirectly treat abnormal
movement disorders.
Contrary to the existing belief that HD is purely a neurological disease rooted in neurodegeneration, this invention approaches HD as a muscle disease. Specifically, it demonstrates
that HD skeletal muscle possesses chloride and potassium channel dysfunction, which greatly
affects skeletal muscle physiology. The defects in the chloride and potassium currents in HD
skeletal muscle can help explain the movement disorders of HD and offer novel therapeutic
targets.
It approaches the diagnosis and treatment of the disease by detecting a reduced expression of
Clcn1 mRNA or an increase in Clcn1exon 7 coupled with a reagent that binds nucleic acids in
the diseased muscle as compared to normal muscle. It focuses on the administering of a muscle
relaxant to patients.
Features:
-Muscle relaxants of this invention can be administered in combination with a traditional HD
therapy that targets the brain.
-The methods can be used to detect the disease before a patient manifests physical symptoms and
monitor the progression of the disease.
-Covered treatments can slow the expression of physical symptoms and the number and severity
of symptoms.
-A medical professional can readily prescribe the effective amount of the pharmaceutical
composition required.
-The treatment utilizes therapeutics already approved for other disorders that can be administered
orally or intravenously (injections, syringes, or saline drip system).
-The treatments can also be administered in a kit containing necessary reagents with
accompanying instructions, labels, warning and information.
Market Potential: More than a quarter of a million Americans have HD or are "at risk" of
inheriting the disease from an affected parent. The annual direct economic burden of HD is
substantial and increases with disease progression. Besides the direct medical costs associated
with the disease there are significant indirect costs such as time spent and working days lost
caring for the patient.
Notice: This invention was made with Government support of Grant No. NIH 1SC3GM096945,
awarded by the National Institute of Health, and by a CSUPERB New Investigator S12 Grant
awarded by the California State University.