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BearNet News - Spring 2009



Children’s National Hosts Legislative Advocacy Day

Alan Korn, JD, Public Policy Director and General Counsel, Safe Kids Worldwide presenting on Child Injury Prevention Advocacy.
Alan Korn, JD, Public Policy Director and General Counsel, Safe Kids Worldwide presenting on Child Injury Prevention Advocacy.
On April 22, Children’s National Medical Center held its first Legislative Advocacy Day to educate faculty, staff, and board members about the importance of using their voices to affect positive change for children’s health. Throughout the course of the half-day event, nearly 100 faculty, staff, and BearNet members attended sessions on effective advocacy communications, child injury prevention, and health reform.

The day began with Advocacy Grand Rounds led by Peter Budetti, MD, JD, of the Department of Health Administration and Policy, College of Public Health of the University of Oklahoma Health Sciences Center. In his presentation,The Pediatrician in the Village: Treating the Child’s World as Well as the Child, Dr. Budetti reviewed the skills and approaches necessary to becoming an effective advocate for children. He also discussed ground-breaking efforts underway at the University of Oklahoma Health Sciences Center to refocus that medical center’s work from the individual to the community as the patient.

Participants were treated to a session on effective advocacy communications led by Brad Fitch, CEO and co-founder of Knowlegis, a knowledge management firm specializing in tools and services for enhancing communications with elected officials. In his session, Urgent Care Lessons in Advocacy: Secrets to Becoming a Powerful Citizen-Advocate, Fitch offered the latest research on effective advocacy strategies; provided step-by-step actions that activists can take to influence the policy process; and offered the “do’s and don’ts” for in-person meetings with members of Congress and staff.

Richard W. Snowdon III, Chairman of the Board, Children's National Medical Center, Dr. Joseph Wright, Senior Vice President of the Child Health Advocacy Institute and AJ Jones Former Policy Director of the Office of Majority Whip James Clyburn (D-SC)
Richard W. Snowdon III, Chairman of the Board, Children's National Medical Center, Dr. Joseph Wright, Senior Vice President of the Child Health Advocacy Institute and AJ Jones Former Policy Director of the Office of Majority Whip James Clyburn (D-SC)
Another session focused on child injury prevention advocacy at the state and federal levels and highlighted successful case studies involving all terrain vehicle safety, pool safety, and child passenger safety seats. The panel consisted of Dr. Joseph Wright, Senior Vice President of the Child Health Advocacy Institute, Alan Korn, public policy director and general counsel, Safe Kids Worldwide™, and Joel Rozner, partner in the law firm Rifkin, Livingston, Levitan & Silver, LLC.

Legislative Advocacy Day concluded with a keynote address on federal health reform by AJ Jones, former Policy Director for House Majority Whip James Clyburn (D-SC). Jones shared accounts of his first-hand interactions with the key decision makers on Capitol Hill and in the Administration, the principles that are driving the policy proposals, and where children’s needs fit into the debate.

The day was a great success that highlighted the important role that Children’s National, our nurses, clinicians and other pediatric healthcare professionals will play in a year that could produce the most significant changes in healthcare policy in generations.

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Federal Funding Helps to Advance Muscular Dystrophy Treatment

Thanks in large part to Congress’ investment in muscular dystrophy research, genetic researchers at Children’s National Medical Center and the National Center of Neurology and Psychiatry in Tokyo recently published the results of a study that holds promise for the first ever treatment for Duchenne muscular dystrophy (DMD). DMD is one of the most common lethal genetic disorders, striking one of every 3,500 boys born worldwide.

The study, published in March in the peer-reviewed journal of the American Neurological Association, Annals of Neurology, details the first successful application of “multiple exon-skipping” to curb the devastating effects of DMD in an animal larger than a mouse. Multiple exon-skipping employs multiple DNA-like molecules as “DNA band-aids” to skip over the parts of the mutated gene that block the effective creation of proteins. The study treated dogs with naturally occurring canine X-linked muscular dystrophy, a disease that is genetically homologous to the Duchenne muscular dystrophy. By skipping more than a single exon, this so-called DNA band-aid becomes applicable to between 80 and 90 percent of Duchenne muscular dystrophy patients, including the mutation found in dogs.

“This trial makes the much-talked about promise of exon-skipping a systemic treatment for Duchenne muscular dystrophy in humans a real possibility in the near term,” said Toshifumi Yokota, PhD, lead author of the study. “Of course this success has also introduced even more avenues for investigation, but these findings finally overcome a significant hurdle to our progress — we’ve solved the riddle of an effective system-wide delivery to muscle tissue, and seen promising results.”

“This study delivers the proof-of-concept that systemic anti-sense therapy can be done in a large organism in Duchenne muscular dystrophy or any disease,” says Eric Hoffman, PhD, a senior author of the study and director of the Center for Genetic Medicine at Children’s Research Institute , at Children’s National.

“Systemic treatment of the majority of Duchenne dystrophy will require multiple sequences to be delivered in the blood, and this study also is the first proof-of-principle of multiple exon-skipping in any organism,” said Shin’ichi Takeda, MD, another senior author. “In order to realize that promise in human trials, it also will be important to re-evaluate current measures of toxicity, efficacy, and marketing that ensure both safety for the patient, as well as rapid development and distribution of life-saving drugs.

The authors note that significant steps in this study remain. Successful systemic treatment with morpholinos requires large doses of the anti-sense molecules—and the technology is costly and difficult to obtain. Additionally, treatment in this study showed diminished success at curbing muscle deterioration of the heart, meaning that a more effective and specific delivery system is needed to rescue the organ’s delicate tissue in Duchenne muscular dystrophy patients. However, these early successes do show much promise for the exon-skipping method as an effective treatment for Duchenne muscular dystrophy and some other genetic disorders.

The study was funded by the Foundation to Eradicate Duchenne; the U.S. Department of Defense CDMRP Program; the Jain Foundation; the Crystal Ball Event of Hampton Roads and the Muscular Dystrophy Association USA; the National Center for Medical Rehabilitation Research; a collaborative grant from the U.S. National Institutes of Health Wellstone Muscular Dystrophy Research Centers; and several Grants-in-Aid from the Ministry of Health, Labour, and Welfare of Japan. We  hope that Congress will continue to fund this important research in order to advance it into clinical trials to treat this generation of boys with Duchenne Muscular Dystrophy.

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Health Reform Taking Center Stage: Speak Now for Kids

Consensus is growing for action on healthcare reform and many are speculating that this year could produce significant changes in healthcare policy. Negotiations are already underway on Capitol Hill; the President recently established a White House Office of Health Reform, and influential interest group coalitions have formed. How will children’s healthcare needs fit into the equation? That’s the question and challenge facing children’s hospitals and advocates. 

Situated in the nation’s capital, Children’s National is uniquely positioned to substantially influence the health reform debate and ensure that children’s unique needs are addressed. One way Children’s National will use our location, experience, and expertise to positively influence the policy making process for children is to participate in the national Speak Now for Kids in Health Reform Campaign. The purpose of this online campaign is to champion children's needs in health reform and to demonstrate the strength and reach of the pediatric community and its commitment to transforming children’s health care.

The Speak Now campaign, which will launch May 1 and conclude May 10, will urge families and pediatric health care providers across the country to tell Congress and the Obama administration what children need in health care. Visitors to the SpeakNowforKids.org web site will be able to upload their health care stories, impactful photos, and videos to create a multi-media collage of advocacy messages. The National Association of Children’s Hospitals, which is leading the campaign, will collect the submissions and send them to Congress and the Administration to remind our elected officials that children, families, and pediatric providers have an important stake in health reform.

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Local Leaders Support Children’s Hospitals Graduate Medical Education

In a strong show of support for the Children’s Hospitals Graduate Medical Education program (CHGME), 83 Members of the House of Representatives signed a letter to the chairman and ranking member of the committee that oversees CHGME funding urging full funding in FY 2010 at $330 million. The following Members of Congress from the Washington, DC metropolitan region signed the letter: Congresswoman Eleanor Holmes Norton (D-DC), Congressman John Sarbanes (D-Md.), and Congressman Chris Van Hollen (D-Md.). 

Of the 61 independent children’s teaching hospitals across the country that receive CHGME funding, Children’s National Medical Center is the fourth largest recipient, with more than $13 million annually. This funding is extremely important to Children’s National because it pays for the training of the next generation of pediatricians, pediatric specialists, and researchers. Adult teaching hospitals receive funding to pay for the costs associated with graduate medical education through Medicare. Because we treat children and not the elderly, Children’s National and other children’s hospitals are left out of the Medicare GME funding stream and therefore rely on the CHGME program to help sustain our teaching mission.

In fiscal year 2009, Congress appropriated $310 million for CHGME - the highest funding level in the program’s 10-year history. This year, Children’s National is working with the National Association of Children’s Hospitals and other children’s hospitals throughout the country to urge Congress to appropriate $330 million for CHGME.

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