"The energy in the room was high, and the mood was optimistic," said Annie Kennedy, MDA's senior vice president of advocacy, speaking about the Congressional briefing on reauthorization of the MD-CARE Act that she attended and helped to organize. The briefing was co-hosted by MDA and other muscular dystrophy organizations in Washington, D.C., on Feb. 25, 2014, and was attended by some 250 people — Senate and House staffers, researchers, advocates and members of the muscular dystrophy community.
Kennedy emphasized that the legislation is an example of a highly successful partnership between public and private entities.
"We're cautiously optimistic that reauthorization of this landmark legislation is possible," Kennedy said, adding that it's imperative to keep the momentum going by encouraging legislators to sign on to reauthorization of the Paul D. Wellstone Muscular Dystrophy Community Assistance Research and Education Act Amendments (MD-CARE Act) of 2014 (H.R. 594/S. 315).
"The MD-CARE Act has leveraged significant breakthroughs in many of the nine muscular dystrophies," Kennedy said, "including research discoveries, therapeutic advances and quality-of-life improvements for those living with muscular dystrophy."
The Feb. 25 congressional briefing highlighted three major MD forms — Duchenne muscular dystrophy (DMD), myotonic muscular dystrophy (MMD, or DM), and facioscapulohumeral muscular dystrophy (FSHD), with testimony by patients, parents, experts in the field, and representatives of advocacy organizations, including MDA CEO Steven M. Derks.
In addition to MDA, Parent Project Muscular Dystrophy (PPMD) and the Federation to Eradicate Duchenne (FED) helped organize the briefing.
Among the presenters were California residents Lisa Harvey and her daughter, Kayla Vittek, both of whom have MMD. Kayla was born with the severe congenital form of the disease and required a ventilator to breathe when she was born. At age 4, she was using American Sign Language to communicate. Today, she's in third grade, is able to speak and read her own testimony at the briefing.
"Today, families living with this disease have a real reason to have hope, as later this year the first clinical trials will begin for myotonic dystrophy," Harvey said in her testimony. "This hope sustains us all and motivates us to do all we can for our families. I speak for the entire community of families struggling with muscular dystrophy when I say that the MD-CARE Act has already saved lives, and with this needed reauthorization, it has the potential to save millions of lives in the future."
Government funding is a critical catalyst
"What’s novel here is that we're not asking the government to do it all alone," Kennedy said. "The federal government is helping to support the same laboratories and medical institutions that have long been supported by MDA and other private organizations. But we cannot — and should not — have to carry this funding load alone."
A key provision of the original legislation was the establishment of centers of excellence in MD research. John Porter, a program director at the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH) (a U.S. government agency), was present at the briefing and described these so-called Wellstone centers, which were originally co-funded by MDA and NIH. They're named after the late Senator Paul D. Wellstone of Minnesota.
There are now six Wellstone centers, five of which have been designated and are listed below. A sixth center will be named in the near future.
The five named Wellstone centers are:
To learn more about the MD-CARE Act Reauthorization of 2014 and see a list of its current House and Senate sponsors, go to MD-CARE Act 2014: Take Action Now. To find your elected officials and convey the importance of the MD-CARE Act, go to Find Your Elected Officials.
"We have already moved the needle in muscular dystrophy care with lifesaving therapies in development because of the momentum of the MD-CARE Act," Kennedy said. "We need all members of the MDA community to urge their members of Congress to support the reauthorization of this bill. The MD-CARE Act is a model of how advocates, organizations, government, clinicians and researchers are stronger together. We must ensure that the therapeutic promise that lies on the horizon is realized, and that this bill is reauthorized this year."