SMA Treatment Acceleration Act

The SMA Treatment Acceleration Act is a bill originally introduced in 2007 in the United States Congress "to authorize the Secretary of Health and Human Services to conduct activities to rapidly advance treatments for spinal muscular atrophy, neuromuscular disease, and other pediatric diseases, and for other purposes." [1]

Contents

Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA) is an inherited motor neuron disease that destroys the nerves controlling muscle movement, affecting crawling, walking, head and neck control, swallowing, and respiratory function. It is the leading genetic cause of death for children under the age of two. Approximately one in 40 people, or approximately 7.5 million Americans, carry the gene mutation that causes SMA. Each child of two carriers of the mutant gene has a one in four chance of being afflicted by the disease.[2]

SMA research and the National Institute of Health

In the past few years, researchers have identified the gene responsible for SMA, called SMN 1, as well as a disease modifying “back-up” gene, called SMN2, that has resulted in new treatment pathways.[3]

Because of the advanced genetic understanding of the disease, the National Institute of Health (NIH) identified SMA as the disease closest to treatment out of more than 600 neurological disorders. The NIH, a component of the Department of Health and Human Services, is a $29 billion Federal agency that has primary responsibility for funding much of the nation's basic medical research through grants to institutions of higher education and research facilities throughout the country.[4] Although the disease is the leading genetic cause of death among children under two years of age, currently the NIH funds just $11 million in SMA research annually.[5]

Features of the Act

Specifically, "The SMA Treatment Acceleration Act" provides for the following:

  1. Federal support for a national clinical trials network for SMA.
  2. Federal support to enhance the existing SMA patient registry and for expanded research on the epidemiology of SMA.
  3. Establishes an SMA Coordinating Committee to include federal agencies, SMA researchers, and SMA families, which shall study barriers to development of SMA treatments.
  4. Establishes a trans-Institute research collaboration at NIH under the Director to ensure that all relevant Institutes are contributing and collaborating on SMA research.
  5. Requires the Secretary of the U.S. Department of Health and Human Services (HHS) to study and report to Congress on the use of incentives to promote SMA drug development among private industry.
  6. Provides for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public.[6]

History of the 2007 version of the Bill

H.R. 3334, “The SMA Treatment Acceleration Act”, was introduced in 2007 in the U.S. House of Representatives by Congressman Patrick Kennedy (D-RI) and Congressman Eric Cantor (R-VA), as H.R. 2149/S. 1158. The Senate version of the bill, S. 2042 was introduced by Senator Debbie Stabenow (D-MI) and Senator Johnny Isakson (R-GA). Although by the close of the 110th Congress on January 3, 2009, the SMA Treatment Acceleration Act had 85 cosponsors in the House of Representatives and 21 cosponsors in the Senate, or approximately 20 percent of each legislative body, Congress failed to pass the measure.[7]

Status of the 2009 version of the Bill

After the 110th Congress’ failure to pass the 2007 version of the bill, the act was reintroduced in the 111th Congress in April 2009 as H.R. 2149 and S. 1158. Currently, responsibility for the bill in the House falls under the United States House Committee on Energy and Commerce, chaired by Representative Henry Waxman (D-CA).[8] In the Senate, the bill has been read twice and referred to the United States Senate Committee on Health, Education, Labor, and Pensions, chaired by Sen. Thomas Harkin (D) of Iowa. After the bill’s reintroduction, various activist groups in the SMA community began a renewed drive to find cosponsors. As of February, 2010, 57 House members had signed on as cosponsors of the bill. Fifteen Senators had initiated or renewed their status as cosponsors. In February, 2010, Congressman Kennedy, who introduced both versions of the bill with Congressman Cantor, announced that he will not seek reelection after his term expires.[9]

Prominent cosponsors

Currently, the 2009 version of the bill has received bipartisan support, including Sen. John Kerry (D-MA), Sen. Saxby Chambliss (R-GA), Sen. Kay Hagan (D-NC), Sen. Roger F. Wicker (R-MS), and Sen. Arlen Specter (D-PA). While still in the Senate, Barack Obama served as a cosponsor of the first version of the bill.

House cosponsors

Senate cosponsors

[10]

Grassroots support

An online petition to push for the passage of the SMA Treatment Acceleration Act was developed in 2009 by Bill and Victoria Strong. SMA organizations "Fight SMA" and "Families of SMA," backed by affected families, have also been involved in lobbying efforts.

External links

References

  1. ^ H.R.2149: SMA Treatment Acceleration Act of 2009 – U.S. Congress – OpenCongress
  2. ^ Spinal Muscular Atrophy Type 1 – Welcome to MDA | Muscular Dystrophy Association Helping Jerry's Kids
  3. ^ For example, see a list of current SMA related clinical trials posted by the Muscular Dystrophy Association at http://www.mda.org/research/ctrials.aspx
  4. ^ Spinal Muscular Atrophy – Families of SMA – Research Support Hope
  5. ^ Families of Spinal Muscular Atrophy – Appropriations
  6. ^ The text of the 2007 version is located on the Library of Congress’s database, THOMAS. See http://thomas.loc.gov/cgi-bin/query/z?c110:H.R.3334:
  7. ^ A Letter to the SMA Community on the SMA Treatment Acceleration Act
  8. ^ http://www.fightsma.org/blog/index.php?tag=sma-treatment-acceleration-act; Also see http://thomas.loc.gov/cgi-bin/query/D?c111:1:./temp/~c111wpaPsg::
  9. ^ Patrick Kennedy won't seek reelection – John Bresnahan and Jonathan Martin – POLITICO.com
  10. ^ A complete, updated list of cosponsors can be found at http://www.fightsma.org/blog/index.php?tag=sma-treatment-acceleration-act