NOVEL THERAPY FOR TREATMENT OF CHRONIC DEGENERATIVE BRAIN DISEASES

BACKGROUND:  There are many types of neurological diseases that affect infants and children. Although the frequency of individual disorders is not high, together they are a significant group of disorders with a collective frequency of 1 in 18,000 live births.  Unfortunately, neurologic disease is seldom curable.  Thus, strategies for the treatment of these debilitating and often fatal diseases frequently focus primarily on palliative measures. Attempts at curing neurological disease have also been proposed. These treatments have included enzyme replacement therapy, gene therapy, and allogenic bone marrow transplantation. Sadly, however, these treatments typically do not improve the condition nor alter the ultimate outcome of the disease, leaving a desperate need to develop effective therapies.

TECHNOLOGY:   UCSF scientists have determined for the first time that the use of neuroactive steroids is effective for the treatment of degenerative brain diseases and central nervous system disorders.   The effectiveness of these neurosteroids has been proven in the Niemann-Pick Type C mouse model, as well as other mouse models of neurological disease caused by lipid storage defects.

APPLICATIONS:  This invention would provide effective therapy for the treatment of a wide range of neurological diseases, including congenital lipid storage diseases, inflammatory diseases, and chronic degenerative brain disease.

STATUS OF IP:  PCT publication # WO 2006/037016

CASE NO:  SF2004-017 and SF2008-067

If you would like to receive further information about this technology and potential licensing opportunities, please contact:

If you would like to receive further information about this technology and potential licensing opportunities, please contact:

Ellen S. Kats, Ph.D.
Licensing Associate
(415) 514-8210 phone
(415) 348-1579 fax
ellen.kats@ucsf.edu

Reference: OTM Case #SF04-017