Children's Capitol Connections - March 2009
First Treatment for Muscular Dystrophy In Sight
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 this month 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 National Medical Center.
“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 do note that significant steps still remain. Successful systemic treatment with morpholinos requires large doses of the antisense 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 oft-discussed exon-skipping method as an effective treatment for Duchenne muscular dystrophy and some other genetic disorders. The post-treatment and non-treatment videos of the study are available on the Annals of Neurology web site.
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.
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Children's Research Institute Expands
Children's National Medical Center's research arm, Children’s Research Institute (CRI), has expanded to increase research space atop the main Children’s National building to 100,000 square feet. The expansion adds 20,000 square feet of research offices, conference rooms, and laboratories and allows researchers to better serve families with world-class bench to bedside care. The Centers for Neuroscience Research and Clinical and Community Research are now housed on this new floor, allowing the Centers for Cancer and Immunology Research and Genetic Medicine Research to expand their operations on the existing research floor.
The new research space features:
The new floor allows researchers from every center to work side-by-side, opening doors for greater collaborations with clinicians, taking research from bench to the bedside and into the community.
- Live video cell imaging
- Multiple onsite cell culture labs
- Three-dimensional cell recreation technology based on microscope imaging
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Children's National Opens Two New Operating Rooms
Children’s National Medical Center recently opened two new operating rooms (OR), increasing the hospital’s capacity to provide surgical services to children throughout the region and nationally. In addition to the ORs, Children’s also opened an expanded waiting area for families whose children undergo surgery.
The new ORs are 750 square feet each and bring the total number of operating rooms to 13. Each OR is equipped with state-of-the-art technology, including flat panel screens displaying clear views of radiology imaging and real-time images of the field of operation.
The new waiting area features expanded capacity, a quiet room for reading or meditation, and an educational kiosk with internet access. Members of the Family-Centered Care Committee were involved in making design decisions about the waiting area.
“The additional operating rooms are a real win for both our surgical staff and our patients,” said Kurt Newman, MD, surgeon in chief at Children’s National. “The large rooms provide the latest technology and sharpest imaging for our surgeons, while parents can wait in a comfortable environment surrounded by our surgical support team.”
One OR is designed specifically for orthopaedic surgery focusing on complex spine repair. It includes an O-Arm that provides real time, 3-D images. The Children’s National Orthopaedic Surgery team, led by Laurel Blakemore, MD, is one of the largest in the region, with a team of pediatric surgeons specializing in complex spine issues.
The other OR will be used by general surgery and uses the Condor System™, which optimizes the quality of images used in laparoscopic surgery. The ORs are equipped to display images of the surgical field alongside real-time and static medical imaging, such as CT scans, providing precise and clear views. The new ORs include the ability to broadcast the procedure to a nearby education room, further enhancing Children’s ability to train future generations of pediatric specialists.
The General and Thoracic Surgery team at Children’s National, led by Anthony Sandler, MD, estimates that more than 40 percent of procedures can now be done laparoscopically, including appendectomies and lung resections. Minimally invasive surgery is easier on patients because it results in a speedier recovery. Children’s surgical specialists are supported by a large team of fellowship-trained pediatric anesthesiologists. As the only freestanding children’s hospital in the region, Children’s National is the only hospital that can guarantee anesthesia administered by a fellowship-trained pediatric anesthesiologist around the clock. Children’s National performs more than 14,000 operations annually at Children’s Hospital and the Ambulatory Surgery Center in Montgomery County.
The new ORs are the first in a series of additions and renovations to the surgical services, which includes constructing additional ORs and renovating existing ORs.
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Children's National Named LeapFrog Top Hospital for Quality, Safety
Children’s National Medical Center is honored to be one of only seven children’s hospitals in the country named to The Leapfrog Group’s 2008 Top Hospitals, based on results from the Leapfrog Hospital Quality and Safety Survey.
“We are honored and proud to again be recognized as one of the top children’s hospitals in the country,” said Edwin K. Zechman, Jr., president and CEO of Children’s National Medical Center. “This selection is an important validation of our commitment to providing high quality care to families in our region, across the country, and around the world.”
“Once again, Children’s National has been singled out as one of the top children’s hospitals by the prestigious Leapfrog Group. This recognition underscores our consistent commitment to providing high quality care in a safe environment,” said Peter Holbrook, MD, chief medical officer at Children’s National Medical Center. “Our state-of-the-art intensive care units, the critical care physicians who staff them around the clock, and our use of computer physician order entry are some of the criteria by which we are judged. Families selecting a hospital for their child should ask for these hallmarks of quality and safety. The experts in the field define these as vital elements and we are pleased that our commitment to this caliber of care is recognized.”
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Donate Pints for Half Pints!
Children's National Medical Center's Edward J. Miller Blood Donor Center collects approximately 2,800 whole blood donations and 750 apheresis platelet donations each year. Children with cancer, blood diseases such as sickle cell, and children undergoing certain surgeries are among the many patients who require fresh blood or special rare blood. Every unit donated may help three or more patients, but as the number of patients treated at Children’s National continues to grow, the need for blood donations is greater than ever.
To donate blood, visit the online appointment center or call 202-476-KIDS (5437).
Located on the second floor of the hospital in room 2302, the Blood Donor Center’s hours of operation are:
Donors not able to travel to Children’s can donate through the Bloodmobile, a 40-foot vehicle that travels throughout the greater Washington, DC, area to businesses, schools, organizations, and places of worship for sponsored blood drives. The Bloodmobile is available every Tuesday and Thursday. Call 202-476-6524 to schedule a blood drive or for more information.
- Mondays, Tuesdays, and Thursdays from 7 am to 4 pm
- Wednesdays from 7 am to 2:30 pm
- Fridays from 7 am to 3:30 pm
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