Pharmaceutical Compounds for the Treatment of Spinal Muscular Atrophy and Other Uses
Posted Sep 19 2011 8:00pm
Description of Invention: The SMA Project ( http://www.smaproject.org/programs.html ) was established by NINDS to identify new compounds with improved effectiveness, safety, and pharmacokinetic characteristics aimed at finding a new therapeutic treatment for Spinal Muscular Atrophy (SMA), a paralyzing and often fatal disease of infants and children. The result of the SMA Project medicinal chemistry optimization effort is a library of ~1400 indoprofren analogues with drug like properties. A lead pre-clinical candidate for SMA has been identified based on several factors, including its ability to increase SMN expression.
The mechanism by which these compounds affect ribosomal fidelity proves to be useful for many genetic CNS diseases. The ability of these compounds to read through nonsense stop codons, coupled with the ability to cross the blood-brain barrier and drug like properties, makes these compounds attractive as therapeutics for diseases such as Muscular Dystrophy and Cystic Fibrosis. Preliminary results in HIV and HPV assays show that these compounds potently inhibit viral replication, presumably via inducing ribosomal frame shift, suggesting potential for antiviral therapy. In addition, these compounds have been shown to be non-toxic and well-tolerated at high doses in rodents.
Applications: Broad applications based on mechanism of action —
Collaborative Research Opportunity: The National Institute of Neurological Disorders and Stroke is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize treatment for SMA. For collaboration opportunities, please contact Melissa Maderia at maderiam@mail.nih.gov .
For Licensing Information Please Contact: Charlene Sydnor Ph.D. NIH Office of Technology Transfer 6011 Executive Blvd. Suite 325, Rockville, MD 20852 United States Email: sydnorc@mail.nih.gov Phone: 301-435-4689 Fax: 301-402-0220
Description of Invention:
The SMA Project ( http://www.smaproject.org/programs.html ) was established by NINDS to identify new compounds with improved effectiveness, safety, and pharmacokinetic characteristics aimed at finding a new therapeutic treatment for Spinal Muscular Atrophy (SMA), a paralyzing and often fatal disease of infants and children. The result of the SMA Project medicinal chemistry optimization effort is a library of ~1400 indoprofren analogues with drug like properties. A lead pre-clinical candidate for SMA has been identified based on several factors, including its ability to increase SMN expression.
The mechanism by which these compounds affect ribosomal fidelity proves to be useful for many genetic CNS diseases. The ability of these compounds to read through nonsense stop codons, coupled with the ability to cross the blood-brain barrier and drug like properties, makes these compounds attractive as therapeutics for diseases such as Muscular Dystrophy and Cystic Fibrosis. Preliminary results in HIV and HPV assays show that these compounds potently inhibit viral replication, presumably via inducing ribosomal frame shift, suggesting potential for antiviral therapy. In addition, these compounds have been shown to be non-toxic and well-tolerated at high doses in rodents.
Applications:
Broad applications based on mechanism of action —
Advantages:
Development Status:
Inventors:
Jill E Heemskerk (NINDS)
Patent Status:
HHS, Reference No. E-050-2011/0
US, Application No. 61/475,541 filed 14 Apr 2011
Related Technologies:
US, Application No. 12/293,268 filed 16 Sep 2008, Reference No. E-133-2006/1 and foreign patent applications
US, Application No. 12/680,285 filed 26 Mar 2010, Reference No. E-187-2007/0 and foreign patent applications
US, Application No. 60/975,675 filed 27 Sep 2007, Reference No. E-187-2007/0
Collaborative Research Opportunity:
The National Institute of Neurological Disorders and Stroke is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize treatment for SMA. For collaboration opportunities, please contact Melissa Maderia at maderiam@mail.nih.gov .
For Licensing Information Please Contact:
Charlene Sydnor Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Email: sydnorc@mail.nih.gov
Phone: 301-435-4689
Fax: 301-402-0220
Ref No: 2317
Updated: 09/2011