Families of SMA Research Funding

FSMA invests in three areas of research:  Basic Research, Drug Discovery, and Clinical Research. 

Basic research into Spinal Muscular Atrophy biology tells us what causes the disease. Understanding what causes SMA reveals new and more effective ways of making SMA drugs.  Pre-clinical drug discovery programs then convert basic research ideas into novel types of SMA drug candidates.  Establishing clinical trial infrastructure provides the means to test the drug candidates in humans. Continued investment in all three research areas is essential to finding an effective SMA treatment and ultimately a cure. 

Basic Research:
Basic research projects identify the most effective new strategies for SMA drug discovery.  FSMA has been funding this type of research since 1984, and invested almost $20 million in basic research during its first 20 years.  Since 2004, the FSMA Scientific Advisory Board has awarded 66 grants for over $8 Million in funding.  The grants have been awarded to 33 different institutions. 
Click here to see the Basic Research grants funded by Families of SMA since 2004.

Drug Discovery and Therapeutic Development Programs:
The ultimate goal at FSMA is to find an effective treatment and cure for SMA. Traditionally, it has been difficult to attract major pharmaceutical companies to conduct research for orphan diseases like SMA, which have small patient populations with small potential for profit. Therefore, FSMA has taken the strategy of providing seed funding to encourage biotech and pharmaceutical partners to engage in SMA drug research.   FSMA has invested $17 Million in pre-clinical SMA drug development since 2000. This includes investments in five distinct programs:
Click here to see the Quinazoline Program funded by Families of SMA.
Click here to see the Stem Cell Program funded by Families of SMA.
Click here to see the Tetracycline Program funded by Families of SMA.
Click here to see the Oligonucleotide Program funded by Families of SMA.
Click here to see the Gene Therapy Program funded by Families of SMA.

Clinical Trials and Research:
In 2001 Families of SMA established the clinical trial network Project Cure SMA and has solely funded the network since its inception. The network first worked on developing the necessary clinical infrastructure and drug testing protocols required to assess candidate drugs for SMA and later to actually conduct clinical trials. To date Project Cure SMA has conducted five clinical trials with an investment of over $6 Million by Families of SMA.
Click here to see the Clinical Trials funded by Families of SMA.

International SMA Research Group Meeting:
The International SMA Research Group Meeting is the largest SMA research conference in the world.  It is the only open venue for annual communication between all SMA researchers.  Families of SMA has been hosting this conference for the past 15 years by organizing the content and by financially underwriting the meeting (covering hotel, travel, registration for all research presenters, a $1,000 investment per person).   Since its inception it has grown from 40 researchers to almost 250 researchers attending each year.  In 2011, researchers representing 11 countries, 70 academic and government and non-profit institutions, and 14 biotech and pharmaceutical companies attended.   To date, Families of SMA has invested over $1.5 Million in the research conference.   Click here to see more about the SMA Research Group Meeting.