In the short time I’ve served as MDA’s President and CEO, I’ve seen up close how progress in fighting muscle disease is being fueled by MDA’s mission…and leadership. Today, those affected by neuromuscular diseases experience longer life spans and improved quality of life. Decades of MDA research and clinical care investment are paying off, with more breakthroughs on the horizon.
About 300 MDA research projects were underway in 2012, with $4,200 spent every hour on the quest for better treatments and cures. More new drugs are planned in the next five years than were seen in the past 50. MDA families monitor groundbreaking clinical trials daily, some taking direct part in them.
Last year, 3,700 children attended MDA summer camps, supported by nearly 4,000 volunteer counselors…with 20,000 hot dogs served! More than 56,000 MDA clinic visits took place, while some 4,000 items were provided at no charge through MDA's equipment program.
While MDA is still in a state of financial rebound after several challenging years, we continue to rally strength impressively in local communities. In 2012, more than 30,000 participants on 5,000 teams took part in Muscle Walks, with that program’s income up 40 percent from 2011. The 300,000 members of the International Association of Fire Fighters (IAFF)—our nation’s fire fighters—surpassed their prior year “Fill the Boot” efforts by a remarkable $600,000. New partner Jiffy Lube beat its $1 million goal by a healthy margin. And our national and regional sponsor partners, including Lowe’s, CITGO Petroleum Corp. and Harley-Davidson, stayed strong for MDA families.
Whether consulting with our scientific and medical advisers, brainstorming new marketing and fundraising initiatives with our sponsors, testifying in our nation’s capital on FDA research protocols, or spending privileged time with the amazing children and adults we serve, I am deeply struck by the vibrancy and intimacy of our mission…and the passion of those who put their hearts and souls behind it.
Humble thanks are due the public who lend support throughout the year, giving online, buying Shamrocks, taking part in MDA Lock-Ups and donating to our MDA Show of Strength Telethon. MDA raised $159 million in income in 2012, the same compared to the prior year. We have big plans to grow and diversify this portfolio further, while strengthening our operational efficiencies and effectiveness.
Yes, your support of our life-enhancing and lifesaving mission helps fuel this progress we all so desperately seek to accelerate. Thank you for continuing to support MDA as we race toward breakthroughs, our sights set on a future in which muscle health, not muscle disease, will prevail.
Steven M. Derks
MDA research has arrived at a stirring juncture. Encouraging progress continues to unfold while we actively pursue breakthroughs. None of this is happening by accident. It’s the result of our rock-solid adherence to principles such as peer review, encouragement of young investigators at early stages, and most critically, our long-term investment in scientific and clinical studies.
Thanks to this consistent investment, the barriers that stand between our MDA families and lifesaving treatments for neuromuscular diseases are beginning to fall.
One strategy being pursued is gene therapy, in which working copies of genes are given to people with flawed genes. We learned early this decade that some children with Duchenne muscular dystrophy (DMD) who received new genes for the muscle protein dystrophin didn’t tolerate the new protein very well.
In May 2012, MDA and our French counterpart, the AFM, co-hosted a symposium on overcoming obstacles to gene therapy, and we’re now confident we’re on our way to figuring out this challenge.
Another strategy—one we’re now pursuing for DMD but which has potential to treat other genetic disorders—is exon skipping, which encourages cells to block, or “skip,” certain regions of a gene, with the goal of ensuring that a needed protein will be produced. MDA has funded basic research in exon skipping since the 1990s and continues to do so. A human trial of the exon-skipping drug eteplirsen is currently underway, showing much promise.
Several years ago, MDA began a fruitful partnership with the ALS Therapy Development Institute. With MDA support, ALS TDI identified a specific part of the immune system that appears to go wrong in ALS (Lou Gehrig’s disease). Based in part on that discovery, ALS TDI is now testing Gilenya—a drug that’s already approved for multiple sclerosis—in people with this disease.
Not so long ago, inserting new genes into muscles, changing the way cells process genes, or pinpointing the part of the immune system that contributes to a disease were far-fetched ideas. But after careful review by MDA’s world-class advisory committees, we took a chance on these projects. Investments are paying off, and we now have promising drugs in development to treat what were once thought to be untreatable diseases.
Finally, having a strong staff complement to our volunteers is essential. The Board was delighted to retain Steve Derks as our new President and Chief Executive Officer in December 2012. He and his capable leadership team will drive us toward a better tomorrow, one that builds on our rich heritage for the families we serve.
R. Rodney Howell, M.D.
This is a remarkable time of progress.
That’s both a reflection of how far we have come in fighting stubborn, lifethreatening neuromuscular diseases. It’s also an organizational imperative to move faster as we set our sights on building muscle health. In the early 1950s, MDA was founded to help families living with severely progressive neuromuscular diseases. Medical understanding up to that moment had been meager.
Today, hope and progress have transformed the landscape. Children and adults are living longer, staying stronger. Years of research investment are paying off, with more new treatments on the horizon than we’ve seen in over six decades of work leading to this moment.
“If we can find a cure, we can save people’s lives,” said MDA National Goodwill Ambassador Bryson Foster when he appeared on the 2012 MDA Show of Strength at age 12. Bryson, who lives in Concord, N.C., has Duchenne muscular dystrophy, one of a number of muscle diseases currently targeted in human trials of potential therapies.
As the world’s leading nonprofit agency dedicated to fighting these diseases, we know there’s never been a more hopeful time for the families and individuals we serve.
MDA’s recent activities in research, service and community engagement are documented in this report. In addition to funding scientific and clinical studies, MDA provides comprehensive health care services and support as we rally communities to fight back through advocacy, fundraising and local engagement.
Progress continues at a rapid pace, thanks to our extended family of researchers, medical professionals, MDA staff and volunteers … not to mention the generous public and our dedicated sponsors, without whom MDA could accomplish nothing.
“Every dollar has given me hope,” said MDA National Task Force Member Angela Wrigglesworth, a teacher from Houston, Texas, who’s fighting spinal muscular atrophy (SMA). “Every minute at MDA camp has taught me lessons. I live independently. I work. All of this is because of MDA.”
MDA is more strongly committed than ever to making progress that can move muscles, find treatments and save lives. We stand on the sturdy shoulders of investigators and supporters who have gotten us to this point with the confidence in knowing that our best days are still ahead.
Progress. Our mission requires it. Our donors demand it. Our families deserve it.
The goal: lifesaving treatments and ultimately cures for people affected by any of the rare, neuromuscular diseases under our umbrella.
Real and significant progress has been made toward this goal, thanks to a comprehensive strategy that:
• nurtures research discovery;
• constantly brings new minds to the problem; and
• facilitates the movement of the most promising therapeutic strategies through the drug development pipeline.
This “discover-and-develop” approach has resulted in clinical trials of several treatments with the potential to turn incurable, life-shortening diseases into chronic, manageable conditions. If that potential can be realized, boys with Duchenne muscular dystrophy may experience milder progression with improved life span. Babies born with spinal muscular atrophy (SMA) won’t die in infancy from severe respiratory weakness. Adults living with Friedreich’s ataxia won’t suffer from a debilitating lack of muscle control. Those with limb-girdle muscular dystrophy and ALS (Lou Gehrig's disease) will have effective treatments available. These scenarios are the hope-filled visions that drive our immediate and urgent work.
In the 1990s and early 2000s, MDA gave separate grants to two researchers working on an unknown strategy for Duchenne muscular dystrophy (DMD) called “exon skipping.” This treatment coaxes cells to “skip over” specific genetic instructions and create usable dystrophin protein, which is missing in this disease.
MDA’s initial funding helped researchers develop exon skipping to the point that it attracted the interest of biotech firms and the pharmaceutical industry, which today are investing millions to bring an effective and safe exon-skipping drug for DMD to market. A phase 2 clinical trial of an exon-skipping drug has found it to significantly slow the natural decline in walking ability in boys with DMD.
But we didn’t stop there. To ensure such potentially lifesaving treatments are reviewed and approved as quickly as possible, in 2012, MDA’s advocacy program pushed to include new language in the Prescription Drug User Fee Act that creates an accelerated approval pathway for drugs for rare diseases. The amended act was signed into law in July 2012.
Using infrastructure to speed research
Having access to a database of clinical information can enhance quality of life, improve survival and speed research. MDA launched an ambitious new disease registry in late 2012 through our national clinic network. Some 25 pilot clinics and three diseases—ALS, DMD and spinal muscular atrophy—were included in our initial launch; more clinics and diseases will be added over time.
Gene therapy trial wins award
Five out of six people with type 2D limb-girdle muscular dystrophy (LGMD) who received injections of alpha-sarcoglycan genes into a foot muscle produced the needed protein, a very encouraging result for LGMD gene therapy. Whole-limb gene therapy is planned as a next step. The MDA-funded research team that conducted the study received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.
Helen Keller once observed, “Alone we can do so little; together we can do so much.”
In the race to save lives, we take that advice to heart.
Our clinical research networks leverage the expertise of MDA’s national clinic network to conduct disease studies and trials. MDA launched a new myotonic dystrophy clinical research network in 2012.
— Alan Pestronk, Professor in the department of neurology at Washington University School of Medicine, and a former mentor for a training grant recipient
Conferences and symposia bring together the top minds in the world to share
knowledge, solve problems and advance science. More than 500 physicians, allied
health care professionals and MDA staff attended MDA's 2012 Clinical Conference.
Our biennial scientific conference, held in April 2013, drew more than 500 participants from the academic, corporate drug development and clinical arenas. Both events fostered sharing across diseases and specialties.
The search for therapies and cures must be accompanied by the cultivation of the best and brightest researchers. As we enter the era of therapy development, MDA’s unique “Bridge-to-Industry” grant, launched in 2012, trains new researchers to effectively bridge the gap between academic research and industrial drug development in search of lifesaving treatments for progressive muscle diseases.
The path from “important discovery” to “approved drug” is long, expensive and littered with failures. It’s estimated that in 2005, the development costs of one successfully approved drug reached $1.3 billion. Meanwhile, only about 20 percent of drugs now in phase 1 trials will ultimately see approval.
MDA employs a number of strategies to navigate these high-stakes waters and successfully reach our goal of muscle health.
The first step is to fund rigorous research with a high potential for drug development. The most enticing of these projects attract the interest of the drug development industry. Next, we support selected industry partners as they complete the extensive preclinical work necessary to file an investigational new drug application with the government.
MDA advocates also play a role in this process, lobbying for faster and more efficient government review of experimental drugs for life-threatening rare diseases.
In 2012, MDA’s translational research program directly supported the development of potentially lifesaving drugs for ALS, several forms of muscular dystrophy, myasthenia gravis and spinal muscular atrophy, while advisers helped projects in many more diseases move forward efficiently.
• expert medical care;
• practical and emotional support;
• summer camps for kids;
• empowerment through advocacy and education;
• information resources; and
• assistance with durable medical equipment.
Exciting experimental treatments may be making their way through the drug development pipeline, but their promise is in the future. MDA is committed to helping today, so that individuals with muscle diseases and their families can lead healthy, active and fulfilling lives.
Thanks to improved medical care (especially for the heart and lungs) and advances in technology (like better airway clearing devices and power chairs that enable activity), many people with neuromuscular diseases are living longer, and pursuing personal and professional goals.
Clearly, in the fight to optimize well-being, the multidisciplinary MDA clinic is the front line of defense. Teams of experts prescribe respiratory and cardiac care, therapy and durable medical equipment, flu shots, genetic counseling and more. A visit to an MDA clinic means getting comprehensive care.
What’s the difference between MDA’s national network of neuromuscular clinics and standard neurology offices? A lot.
MDA clinicians are experts in muscle and nerve diseases that many neurologists rarely, if ever, have diagnosed or treated. They recognize the subtle differences between, say, polymyositis and limb-girdle muscular dystrophy—two diseases that can resemble each other at onset, but which have radically different underlying causes and standards of care. MDA’s health professionals know what tests to order, what treatments to try and what interventions have the best chance of enhancing quality of life.
Expert, comprehensive care is so important in neuromuscular disease that the American Academy of Neurology’s guidelines in ALS advises patients to enroll as soon as possible in a specialized multidisciplinary ALS clinic such as an MDA/ALS center. MDA’s ALS centers are set apart by the team’s level of expertise in managing ALS, and by the amount of ALS research conducted there.
As MDA makes progress toward muscle health, independence and quality of life are part of its mission—and camp is a big part of that.
Camp is a week of silliness, messiness, laughter and adventures. Aided by one-on-one volunteer counselors who are by their sides day and night, campers max out on the super-fun things regular life doesn’t always allow them to do.
Little do they know they’re also learning about being more resilient, independent and self-confident. Such qualities are vital for children with neuromuscular diseases, who are living into adulthood in unprecedented numbers.
And little do they know that their parents also are benefiting from their free week at camp.
“I love my son and, believe me, taking care of him and meeting his challenges is one of the most wonderful things in my life,” confided one parent of a 13-year-old boy with spinal muscular atrophy. “However, it’s really nice to know that once a year my husband and I can ‘escape’ the day-to-day grind and have an opportunity to relax and simply focus on ourselves and each other. We generally plan a getaway and enjoy the time that he’s at camp.”
Durable medical equipment can range from a high-tech power wheelchair to a simple plastic shower chair. The value of the equipment is not in its technology, but in its ability to preserve health and enhance independence.
MDA made life a little easier for individuals and caregivers in 2012 by helping families locate the right equipment for their needs; providing financial assistance with repairs; and maintaining an inventory of gently used equipment to provide to families at no cost.
Staying in the know
The MDA community was kept well-informed about research, health care, advocacy and daily living strategies in 2012. Information was presented in a wide variety of formats, ranging from print to digital to in-person presentations.
Nearly 10,000 people a day visit with us at mda.org—next to our clinics, our main channel with our families and the public. Families also get resources from us directly, like Quest magazine and our electronic newsletters, so they can stay abreast of the latest news and disease information. Other information vehicles include webinars, blogs and educational conferences to inform and empower families.
When someone has a rare and debilitating disease, it helps a lot to know you’re not alone.
MDA actively empowers families and their communities to fight back against the destructive effects of muscle disease through engagement in awareness-building and fundraising events where they live.
• MDA’s national advocacy program amplifies the voice of the MDA community in matters of public policy and research advancement.
• Community engagement activities unite volunteers, sponsors, corporate partners, individuals and families at fun events that raise awareness of muscle disease, and funds for research, camp and clinics.
Thanks to progress in medicine and technology, children and adults with neuromuscular diseases are living longer, fuller lives—and recent research discoveries promise even better times ahead. But challenges remain.
People with neuromuscular diseases continue to encounter barriers to accessing necessary resources and support. And potentially lifesaving experimental therapies can become mired in regulatory requirements that add years to the time it takes for a treatment to become available.
In response to these concerns, MDA worked with legislative and policy partners to accelerate research advances and treatment discovery, while expanding the resources available to those affected by neuromuscular disease.
In 2012, MDA and its community of advocates continued to push for the passage of critically important legislation, such as the MD CARE Act Reauthorization and the Achieving a Better Life Experience Act, and would carry this work forward in 2013.
Cost of illness study
What does it cost to have a neuromuscular disease? Because such data is useful in influencing drug development and social and economic policies, MDA and the Lewin Group (a health care consulting firm) studied three diseases—ALS, Duchenne and myotonic dystrophy. The study found average annual per-patient costs to be: ALS—$63,692; DMD—$50,953; and MMD—$32,236. The combined estimated cost to the nation of these three diseases is $1.07 to $1.37 billion per year—but the study authors noted that this estimate is likely very conservative.
At a standing-room-only public hearing with the U.S. Food and Drug Administration (FDA) in February 2013, people with ALS and their families, researchers, clinicians and others urged the agency to address the unique needs of those with ALS—for whom time is of the essence—when considering therapies in development for this disease. Working with other ALS organizations, MDA played a leadership role in securing the first-ever FDA hearing on ALS, amplifying the ALS community's voice.
Supporting young adults through Transitions
As children with pediatric diseases live into adulthood in unprecedented numbers, new opportunities and challenges arise—education, employment, independence. Through a national survey and its active Transitions program, MDA identified the multifaceted needs of this “new generation”—and expanded the MDA Transitions Center, an online community and resource for young adults.
Through its National Transitions Summit, MDA brought together leaders from
federal agencies and experts in the disability community to explore ways in which
we collaborate with our federal partners to work through the resource barriers
that currently prohibit transitional freedom for young people with neuromuscular disease. The yield was a robust transitions policy that has been internationally recognized for its innovation.
MDA’s premier Labor Day Weekend event received a new name—the MDA Show of Strength—and moved to a three-hour, pre-taped format. The show sparkled with the gifts of talented and generous entertainers and presenters, stories of strength from the MDA community, and expressions of support from our national sponsors. Musical artist Pitbull ended his performance by emotionally informing MDA’s 12-year-old National Goodwill Ambassador Bryson Foster, “We need you guys more than you need us. You’re the ones that are strong.”
MDA’s mission is possible because of the generous support from our steadfast national sponsors, local groups and retailers, individual donors and relentless army of passionate volunteers.
Thanks to their fundraising and awareness-building efforts, research into devastating neuromuscular conditions has raced ahead, and scientific knowledge has grown significantly.
To keep this exciting momentum going, a large part of MDA’s role continues to be strengthening our mission through community involvement—whether it’s people dropping dollars into fire fighter boots, exhilarated crowds gathering at Muscle Walks, or a single caring person buying a Shamrock at a local retail check-out counter.
Across the country, community events and cause-marketing campaigns have provided essential support to the fight against muscle disease. While our nation’s economic recovery was far from complete in 2012, MDA programs persevered.
In only its third year of operation, MDA’s community walking event, Muscle Walk, raised $6.7 million—an increase of 40 percent over 2011, with some 30,000 walkers and 5,000 teams taking part.
With its more than 300,000 members strong, the International Association of Fire Fighters (IAFF) has been going to remarkable lengths to help MDA since 1954 and remains our No. 1 national sponsor. In 2012, an estimated 1,700 IAFF locals took part in 2,000 events, including IAFF’s signature campaign for MDA, Fill the Boot. Total IAFF fundraising in 2012 was an extraordinary $25.1 million, a 3.2 percent increase over the previous year. Fire fighters continue to be heroic figures for all who care about MDA’s mission.
MDA Shamrocks achieved their greatest year to date, raising $24.5 million and beating the 2011 total by $1.6 million. Some $7.6 million was accounted for by Lowe’s Home Improvement. More than 15,000 retailers across the country joined in the festive St. Patrick’s Day fun, including CITGO Petroleum Corp., Burger King, Kroger, 7-Eleven and Corner Stores.
MDA rallied local communities in another way as local VIPs went “behind bars for good,” taking part in our jail-themed MDA Lock-Ups and contributing to a program total of $23.8 million in 2012.
High society galas and other signature events raised $12.3 million in 2012, up nearly $2 million from 2011. The two most successful signature events, Tradition of Hope and the Bash, both generated income for MDA’s Augie’s Quest, supporting fast-track research seeking a cure for ALS. By sponsoring outstanding social and golf events around the country, CITGO Petroleum Corp. continues to bring help and hope to MDA families while building stronger relationships with their participating business partners.
In May 2012, more than 950 motorcyclists from Pennsylvania, New Jersey, Maryland, Delaware, New York and Virginia participated in the 25th Annual Eastern Harley-Davidson Dealers Association Ride for Life. Engines roared and the hearts of MDA families soared as this magnificent and historic Harley-Davidson event raised nearly $1 million to benefit MDA.
Each year, thousands of preschools, day care centers and elementary schools across the country are taken over by hopping students who take part in MDA Hop-a-Thons. Hop-a-Thons helped spread disability awareness while raising $1.2 million for MDA in 2012.
MDA’s Aisles of Smiles program, which is driven by the dedicated efforts of sponsor partner Acosta Sales & Marketing, not only helps MDA but has proven to be a valuable component in consumer marketing efforts. While driving sales, Aisles of Smiles raised $2.7 million to benefit MDA, an increase over the previous year with the Safeway family of stores leading the country with the top fundraising cause-marketing campaign.
A welcome new addition to MDA’s family of sponsors, Jiffy Lube, aimed to raise $1 million through its first annual “Muscle Up” savings book campaign in 2012, generating return business while actually amassing more than $1.15 million for MDA.
National sponsor partners such as CITGO Petroleum Corp., Lowe’s, IAFF, The Safeway Foundation and Harley-Davidson lent an extra dimension of support above and beyond the call of duty by helping out in person at MDA summer camps.
Along with the partners mentioned above, the National Association of Letter Carriers, 7UP, Valero and Tall Cedars of North America were recognized and thanked on the 2012 MDA Show of Strength for their phenomenal support of MDA. MDA remains grateful to all of its partners, whose dedicated support has helped the quest for treatments and cures advance in leaps and bounds.
At MDA, we’re grateful to our sponsor partners, volunteers, and the members of the public who generously support our mission. MDA invests donor dollars wisely, with 77 cents of every dollar going directly to fund our mission.
Thanks to the support we receive, we’re making a profound difference in the lives of an estimated 1 million Americans who fight life-threatening muscle disease.
Designated a “Top-Rated Charity” by the American Institute of Philanthropy, MDA is the first nonprofit to receive a Lifetime Achievement Award from the American Medical Association “for significant and lasting contributions to the health and welfare of humanity.”
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.
Visit mda.org for more information or to add your support.