MDA advocacy in Washington
I have had the great privilege of witnessing some of the most transformative legislative moments in our MDA community’s history. While we have many heroes and legends among us, there are also many heroes whose names most will never know — people who, through their outreach to their elected officials, brought great champions to the cause of neuromuscular disease research and services.
I recently was reminded of just how powerful this relationship-building can be. A few weeks ago a team of organizational leaders from MDA and other muscular dystrophy advocacy groups met with representatives of Sen. Susan Collins (R-Maine), who has been a longtime champion for our MD community. Sen. Collins’ commitment stems from her personal connection to a young man who interned in her district office who had Duchenne muscular dystrophy (DMD), Matthew Denger.
During the meeting, we discussed how reauthorizing the MD CARE Act would benefit Matthew and his peer group by funding such things as transitions resources, and improved cardiac and pulmonary care. Sen. Collins’ staff understood the urgency of this request because they knew and cared about Matthew. At the end of that meeting, Sen. Collins offered her support of the bill and signed on as an original sponsor. Matthew — a young college student from Maine — had leveraged a powerful congressional relationship.
What none of us could have fathomed was that this congressional relationship would become a part of this young man’s legacy, as he died very suddenly from cardiac arrest just a few weeks later. Matthew taught all of us that you don’t need to have much range-of-motion to have expansive reach, nor muscles to move mountains.
Other senators and representatives who have worked hard to ensure our muscular dystrophy community remains a high priority in the U.S. Congress — Sen. Roger Wicker (R-Miss.), the late Sen. Paul Wellstone (D-Minn.), Sen. John Barrasso, (R-Wyo.), Rep. Eliot Engel (D-N.Y.), and so many more — each got involved because a local individual or family affected by a neuromuscular disease took the time to reach out and develop a relationship with them.
On a frigid Monday in February in Washington, D.C., this year, I watched in awe as people poured into a U.S. Food and Drug Administration (FDA) hearing. More than 200 members of our ALS community — men and women with ALS, parents, spouses, children, clinicians, researchers, advocacy organizations, industry, federal officials — had braved the weather to urge the FDA to consider the dire status of ALS patients when regulating ALS drug trials.
This was the first time the FDA had ever held a hearing of this type, urged on by an unprecedented joint appeal from MDA, the ALS Association and other ALS organizations. The testimony throughout the day was breathtaking. I was struck by how speakers with ALS seemed to be empowered by their words (many computer-generated) and by the opportunity to improve the current state of ALS drug development.
|In 2001, 13-year-old Ben Cumbo, who has DMD, asked a U.S. Senate subcommittee a pointed question resulting in a standing ovation.|
It reminded me of another cold February day in 2001, when another extraordinary segment of our community gathered, this time for a hearing before a subcommittee of the Senate Appropriations Committee to urge passage of the original MD CARE Act — historic legislation that would facilitate research and improve clinical care in nine forms of muscular dystrophy. Again, in what was a historic occasion of the muscular dystrophy community uniting, MDA and Parent Project Muscular Dystrophy packed the room that day with families, kids, researchers, clinicians and camp volunteers.
While there were many striking facts and comments presented by esteemed participants that day, the testimony that still resounds in my head came unscripted, unprompted and unplanned when senators were informally asking then-13-year-old Ben Cumbo of Upper Marlboro, Md., a bit about his life and interests. At the end of their brief exchange, Ben, who has DMD, was asked whether he had any questions. Ben asked, “If you can spend $2 billion on a single plane, don’t you think you can spend $100 million on muscular dystrophy?” The question drew a roar of applause, brought many audience members to their feet and drew smiles from senators. It was a memorable day.
The MD CARE Act was passed into law later that year (and reauthorized in 2008). The Act for which we all worked so hard has been a “game-changer” for our MD community in terms of clinical care and therapy development. Since 2001, there have been 67 clinical trials of drugs or therapies for muscular dystrophy, with 37 clinical trials currently under way. The MD CARE Act also has resulted in better knowledge about the disease, improved health outcomes and optimized clinical care interventions for people living with the various forms of MD.
As I mentioned at the outset, this year the MD CARE Act is again up for reauthorization. A great deal of work remains to be done, and increased federal support is needed to ensure that researchers continue to make progress toward a cure.
These are just two of the many federal partnerships in which MDA is engaged. In addition to the MD CARE Act and FDA ALS efforts, we are actively engaged in partnerships and advocacy efforts on behalf of every community we represent, including: better policies for teens and young adults with disabilities who are transitioning from childhood to adulthood; the ABLE Act (a financial planning vehicle) for all individuals served by MDA; the NIH Rare Disease Clinical Research Consortium for Charcot-Marie-Tooth disease and the NIH Rare Disease Clinical Research Consortium for Channelopathies; numerous spinal muscular atrophy efforts, including the SMA Project collaboration with NIH and the SMA Patient Advocacy Group; disease-specific research and policy collaborations with federal agencies; and much, much more.
We have many heroes to whom we are indebted for the strides we’ve made thus far. The people who fill the hearing rooms each time we secure such a forum. Those who respond to a public comment period in the Federal Register. Those who leverage personal connections by hosting a fundraising event, extending an awareness campaign or launching a legislative movement.
This spring, I urge you to follow Matthew Denger’s example, and the example of the hundreds of members of the ALS community who put on a show of strength for the FDA. Please take every opportunity to get involved in MDA’s advocacy efforts. This can be anything from emailing or calling your legislators, to making a visit to Capitol Hill, to submitting comments to the Federal Register — or, like Matthew, becoming the next “friend of the senator/representative” through visits and outreach to local congressional offices.
MDA Senior Vice President - Advocacy