110th Congress

SMA Treatment Acceleration Act

H.R. 3334, S. 2042

Background

Spinal muscular atrophy (SMA) refers to a group of inherited disorders that cause the death of nerve cells in the spinal cord, which results in weakness and muscle wasting.  The most severe form of SMA causes paralysis and death in infancy.  A milder form affects adults.  Although SMA is the most commonly identified single-gene cause of death in infants, it is a rare disease and not among the most common causes of infant mortality.  The severe form of SMA affects an estimated 200–300 infants each year in the United States.

In 2003, the National Institute of Neurological Disorders and Stroke (NINDS) launched “A Collaborative Program to Accelerate Therapeutics Development for SMA,” or the SMA Project, to develop candidate drugs for the disease.  In 2008, after developing and testing more than 1,000 potential new drugs in laboratory models, the project has advanced the most promising drug candidates to preclinical testing in animals.  If the results are favorable, further preclinical safety studies could lead to clinical testing within about 1 year.  Because drug development is an extremely high-risk process, the project has begun to develop two new alternative chemical classes of drugs to increase the chances of success. The National Institutes of Health (NIH) Institutes coordinate their SMA activities, with each Institute taking the lead in the areas of its greatest expertise.  The National Institute of Child Health and Human Development (NICHD), for example, has been supporting the development of both newborn and carrier screening tests for SMA, which will be critical for the success of clinical trials in infants and, ultimately, will ensure that interventions can be administered as soon after diagnosis as possible.  NICHD also supports research on quantitative testing of muscle strength in SMA, which will be a crucial outcome measure for clinical trials of therapies for adults and children.

SMA has been of interest to Members of Congress such as Representative Patrick J. Kennedy (D-RI) and Senators Tom Harkin (D-IA) and Arlen Specter (R-PA) for several years.  In both the fiscal year 2008 House and Senate reports, there is report language regarding SMA for both NINDS and NICHD.

Provisions of the Legislation/Impact on NIH

On August 2, 2007, Representative Kennedy introduced H.R. 3334, the SMA Treatment Acceleration Act.  On September 12, Senator Debbie A. Stabenow (D-MI) introduced S. 2042, a virtually identical companion bill, in the Senate.  The aim of these bills, according to the sponsors, is to advance treatments for SMA, enabling investigators to conduct national clinical trials to demonstrate treatments that are safe and effective.  The bills would also mandate more active engagement by NICHD, the National Center for Research Resources, the Centers for Disease Control and Prevention (CDC), the Health Resources and Services Administration, and the Food and Drug Administration (FDA).

Specifically, H.R. 3334 would do the following:

• Require Federal support for a national clinical trials network for SMA supported by NINDS and NICHD
• Require the establishment of a joint Data Coordination Center supported by NINDS and NICHD
• Require the Secretary of Health and Human Services (HHS), acting through CDC, to provide Federal support to enhance the existing SMA patient registry and for expanded research on the epidemiology of SMA
• Require the Secretary to establish an SMA Coordinating Committee that includes Federal agencies, SMA researchers, and SMA families
• Require the SMA Coordinating Committee to conduct a study to identify current and potential future barriers to the development of drugs for treating SMA and other similar genetic disorders and provide a report to Congress on the results of the study, together with recommendations for legislation or administrative action as the Committee deems appropriate
• Require the Director of NIH to establish a trans-Institute research collaboration to ensure that all relevant NIH Institutes are contributing to and collaborating on SMA research
• Require the Secretary of HHS, in collaboration with FDA, to study and report to Congress on ways to improve existing incentives to promote SMA drug development among private industry as well as to recommend additional incentives
• Require the Secretary to establish a program to provide information and education on SMA to health professionals and the general public

Status and Outlook

H.R. 3334 was introduced by Representative Kennedy on August 2, 2007, and was referred to the House Committee on Energy and Commerce Subcommittee on Health.  No further action has occurred on this legislation. 

S. 2042 was introduced by Senator Stabenow on September 12, 2007, and was referred to the Senate Committee on Health, Education, Labor and Pensions.  No further action has occurred on this legislation.

June 2008