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Isis Pharmaceuticals Licenses Intellectual Property Created at the University of Massachusetts and Funded by Families of Spinal Muscular Atrophy, to Develop Drugs to Treat Spinal Muscular Atrophy.
March 9, 2010.

ISIS recently announced that it had added a SMA drug candidate to its development pipeline, called ISIS-SMNRx.  ISIS-SMNRx is designed to treat SMA, a neuromuscular disorder and the leading genetic cause of infant mortality.

ISIS is developing ISIS-SMNRx as part of its strategy to discover and develop antisense drugs against neurodegenerative diseases.  ISIS' SMA program is part of its collaboration in neurodegenerative disease with Dr. Adrian Krainer at Cold Spring Harbor Laboratory and Genzyme, pursuant to which Genzyme has an exclusive option to license ISIS-SMNRx from ISIS.  Click here to read more about this announcement.

The antisense therapeutic approach for SMA involves the use of a short, chemical structure, called an antisense drug, to increase the production of the protein, SMN.  SMN protein is reduced in SMA and is associated with normal motor function.  SMN2 is a closely-related gene that normally produces a truncated and low-functioning form of SMN protein.  Isis designed an antisense drug that binds to the SMN2 RNA and drives the production of SMN protein.  Using this approach, Isis hopes to provide therapeutic benefit to patients with SMA.  Click here for a detailed explanation of how the process works.

Families of SMA funded the early research for this program with three grant awards to Dr. Ravindra Singh and his colleagues between 2003 and 2006, originally in collaboration with Dr. Elliot Androphy at the University of Massachusetts.  Dr. Singh is now at Iowa State University.  The grants were given through the FSMA basic research program, which is governed by our Scientific Advisory Board.
The FSMA funded projects were designed to gain a greater understanding of how the splicing of  SMN2 is regulated and controlled in cells.  Click here to read more about Dr. Singh’s research.

SMA is a terrible disease and the leading genetic cause of infant mortality.  The ability of our drugs to specifically target RNA and drive the production of SMN may be able to compensate for the underlying genetic defect and offer some therapeutic benefit to patients with SMA.  We are grateful for the support provided by Families of SMA in funding early research on SMA, which has significantly expanded the basic science of SMA and provided a roadmap to developing the first targeted therapy to treat SMA,” said Frank C. Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals.

The strong commitment of Families of SMA to basic research has been very foresighted for finding novel drugs and drug targets for SMA.  I began submitting transformative research proposals with apparent high risks.  FSMA kept supporting my basic research that finally resulted in discovery of a novel drug target for antisense-based therapy of SMA.  I take this moment to commend Families of SMA for its vision of promoting basic research,  dividends of which have started to pay off”, said Ravindra Singh, Ph.D.

This program is a perfect illustration of how basic research can directly lead to new therapeutic avenues to treat SMA.  This idea is one of the main tenets of the FSMA research strategy”, said Jill Jarecki, Ph.D. Research Director of FSMA.
Click here to read more about our research strategy.

About Families of SMA:
Families of SMA funds and directs the leading SMA research programs.
Our successful results and progress from basic research to drug discovery programs to clinical trials provides real hope for families and patients.
-Families of SMA has funded 5 multi-center clinical trials for existing drugs that have potential for SMA.
-FSMA has funded 5 leading new drug development programs for therapies specially designed to treat SMA.
We have raised and funded over $50 million for SMA research.  Our support comes from generous individual donations and numerous fundraising events held by volunteer families and our Chapters.

We are a non-profit, 501(c)3 tax exempt organization.  Today we have 27 Chapters throughout the United States and over 65,000 members and supporters.  We are a collaborative organization where families and friends and researchers are all working together towards the same goal.

Both Isis and Genzyme were recent sponsors at The Annual SMA Conference.


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