Contact: Leonard N. Karp
215-575-3720
lkarp@philadelphiamedicine.com
April 25, 2007
For immediate release:
In this month’s edition:
- Children’s Hospital To Open World’s First Comprehensive Medical Unit for Mothers Carrying Fetus with Known Birth Defect
- Fox Chase Cancer Center Offers da Vinci Robotic-Assisted Laparoscopic Surgery for Gynecologic Cancers
- Jefferson Scientists in Nationwide Trial to Study Creatine for Parkinson’s Disease
- Penn: First Demonstration of Muscle Restoration in an Animal Model of Duchenne’s Muscular Dystrophy
Editors note: Research, new techniques and improved facilities by Philadelphia International Medicine hospitals and physicians may lead to new ways to treat some of our most challenging diseases. Below are just some examples from our hospitals.
Children’s Hospital To Open World’s First Comprehensive
Medical Unit For Mothers Carrying Fetus with Known Birth Defect
Philadelphia – We’re expecting - and The Children’s Hospital of Philadelphia could not be prouder to announce it will launch the world’s first comprehensive medical unit for mothers carrying a fetus with a known birth defect. Set to open in January 2008, the Garbose Family Special Delivery Unit will offer comprehensive medical care, from prenatal diagnosis through delivery to mother-infant care.
The new eight-bed labor and delivery unit will allow Children’s Hospital’s experts in fetal medicine and surgery to provide comprehensive, seamless care from diagnosis, delivery, necessary interventions through postnatal follow-up. The hospital’s Center for Fetal Diagnosis and Treatment team expects the unit to improve outcomes for children with fetal abnormalities, as well as for their mothers. The Garbose Family Special Delivery Unit will feature a welcome center, concierge service, private rooms, and round-the-clock care provided by obstetricians, nurse-midwives and the most highly experienced nurses.
"This Special Delivery Unit, the first of its kind in the world, will allow mothers carrying babies with birth defects the best treatment possible from the most experienced team of fetal experts in the world; this is truly a unique concept," said N. Scott Adzick, MD, surgeon-in chief and director of the Center. "Approximately one in every 33 babies born are diagnosed with a birth defect and now the team with the greatest experience and expertise
will have the premier facility for treating those babies and their mothers."
This is the first delivery unit at Children’s Hospital and the first special delivery unit in a children’s hospital dedicated to high-risk pregnancies. Dr. Adzick believes the new unit will provide an environment more conducive to developing and perfecting fetal treatments to cure disease.
"We know that babies with special needs require special care before and after birth and now babies from across the nation and around the world can be cared for in one place dedicated specifically to the care of these babies and their mothers," said Lori Howell, RN, MSN, executive director of the Center for Fetal Diagnosis and Treatment. "The SDU is located near the hospital’s world-class Newborn/Infant Center and Cardiac Intensive Care Unit, and the multitude of Children’s Hospital’s renowned pediatric subspecialists are available at a moment’s notice."
The Special Delivery Unit is named for the Garbose Family in recognition of a major gift from William and Lynne Garbose to support Children’s Hospital’s Center for Fetal Diagnosis and Treatment.
"When I was carrying a baby diagnosed with a rare and complicated heart defect, I was unable to deliver my baby at the pediatric institution where she was to be treated. I was tremendously anxious about the logistics of transporting my critically ill newborn and, once transferred, I was pained by the physical separation between us," said Lynne Garbose, a member of Children’s Hospital’s Board of Directors. "The Special Delivery Unit will enable babies to receive treatment immediately and allow mothers to be just ‘down the hall’ rather than in another location."
As one of only a few centers in the world of its kind, the Center for Fetal Diagnosis and Treatment at The Children’s Hospital of Philadelphia offers a wide range of comprehensive fetal surgery services that support patients from the evaluation stages to follow-up care. The Center was established in 1995 and has received over 7,000 referrals from all 50 states and 46 countries and has performed over 400 fetal surgery operations including more than 70 EXIT procedures.
Fox Chase Cancer Center Offers da Vinci Robotic-Assisted
Laparoscopic Surgery for Gynecologic Cancers
Surgery is often a necessary step in the treatment of women with gynecologic cancers, but traditional open surgery requires a four- to five-day hospital stay and an extended recovery at home. To reduce the recovery time and improve other outcomes, Fox Chase Cancer Center now offers these women da Vinci robotic-assisted laparoscopic surgery.
Fox Chase offers da Vinci robotic surgeries for cancers requiring the removal of the uterus (partial hysterectomy), uterus and cervix (total hysterectomy), fallopian tubes and/or ovaries. The da Vinci system also can be used to help determine the stage of the cancer.
Performing da Vinci surgery usually requires only three to four small incisions, each less than an inch in size. The smaller incisions, advanced optics and superior instrumentation can mean less blood loss and a more precise surgery.
"Using da Vinci technology, we’re able to offer a less invasive alternative to open surgery," said Mark A. Morgan, M.D., F.C.O.G., chief of gynecologic surgical oncology at Fox Chase. "This minimally invasive approach replicates the capabilities of an open surgical procedure, without the six- to 10 inch-incision."
Morgan says the "robot" is superior to traditional laparoscopic surgery because it improves visualization, dexterity, precision and control. In addition to less bleeding, the benefits of robotic surgery compared to open surgery include reduced pain, shorter hospital stay (one to two days instead of three to five days), quicker recovery (one to two weeks versus four to six weeks) and an earlier return to normal activity.
Not all patients are appropriate candidates for minimally invasive gynecologic surgery, Morgan noted. It may not be suitable for women who have had prior pelvic radiation or surgery. Open surgery also remains the best option for most women with ovarian cancer or large tumors that would require larger incisions to remove.
Jefferson Scientists in Nationwide Trial to Study Creatine for Parkinson’s Disease
Thomas Jefferson University Hospital in Philadelphia has been named one of 51 medical centers in the United States and Canada to participate in a large-scale, National Institutes of Health (NIH)-supported clinical trial to learn if the nutritional supplement creatine can slow the progression of Parkinson’s disease (PD).
The double-blind, placebo-controlled, phase III study is one of the largest PD clinical trials to date. The trial – which is expected to enroll 1,720 people with early-stage PD – is the first large study in a series of NIH-sponsored clinical trials called NET-PD (NIH Exploratory Trials in Parkinson’s Disease).
"The study is important because it is the first time that the NIH has undertaken such a large scale, long-term study of a possible Parkinson’s therapy aimed at slowing the course of the disease," notes Jefferson principal investigator Jay Schneider, PhD, professor of pathology, anatomy and cell biology and neurology at Jefferson Medical College and director of the Parkinson’s Disease Research Unit at Thomas Jefferson University.
At the same time, Jefferson has garnered another distinction, being named one of only 15 centers nationwide that are participating in a consortium to develop strategies aimed at reversing a woeful lack of participation of ethnically diverse groups in PD trials. Few minority groups, such as African Americans, Hispanics and Asian Americans, for example, participate in Parkinson’s trials. The NET-PD group hopes to develop strategies to increase minority participation, recruiting and retaining diverse populations into the current trial.
PD is a progressive, degenerative disorder of the brain in which patients develop symptoms such as tremor, slowness of movements, and stiffness of muscles. It affects at least one million people in the United States. Although certain drugs, such as levodopa, can reduce the symptoms of PD, there are no proven treatments that can slow the progressive deterioration in function.
Studies have suggested that creatine can improve the function of mitochondria, which produce energy inside cells. It also may act as an antioxidant that prevents damage from compounds that are harmful to cells in the brain.
The study will enroll people who have been diagnosed with PD within the past five years and who have been treated for two years or less with levodopa or other drugs that increase the levels of dopamine in the brain. Many of the symptoms of PD result from the loss of dopamine, a neurotransmitter that helps to control movement. Half of the participants will receive creatine and half will receive a placebo. Neither the participants nor their doctors will know which treatment they receive. The investigators will measure disease progression using standard rating scales that measure quality of life, ability to walk, cognitive function, and the ability to carry out other activities of daily living.
As enrollment only now begins for this large creatine trial, Dr. Schneider hopes that the experimental educational program will increase recruitment of racially and ethnically diverse participants into the study.
"The problem of recruiting diverse populations into clinical trials is not unique to Parkinson’s disease trials," says Dr. Schneider. "However, it is important to have different populations represented in evaluations of new treatments. This new intervention study is aimed at increasing community awareness about the NET-PD trial and fostering cooperation between neurologists and other physicians in the community who have minority Parkinson’s disease patients and the NET-PD researchers.
"We need to do a better job educating the lay public as well as community physicians about the value of having diverse populations participate in these types of clinical trials," he says. "We know from other studies, particularly with some heart medications, that not all treatments work equally well for all groups. The hope is that if the strategy we use to increase minority enrollment is effective, it could become a model for use in future studies."
First Demonstration of Muscle Restoration in an Animal Model of Duchenne’s Muscular Dystrophy
Implications for Treating Many Types of Genetic Diseases
Using a new type of drug that targets a specific genetic defect, researchers at the University of Pennsylvania School of Medicine, along with colleagues at PTC Therapeutics Inc. and the University of Massachusetts Medical School, have for the first time demonstrated restoration of muscle function in a mouse model of Duchenne's muscular dystrophy (DMD). The research appears ahead of print in an advanced online publication of Nature.
"This new class of treatment has the potential to help a large number of patients with different genetic diseases that have the same type of mutation," says senior author H. Lee Sweeney, PhD, chair of the Department of Physiology at Penn. This genetic flaw causes from 5 to 15 percent (and in a few instances up to 70 percent) of individual cases of most inherited diseases, including DMD, cystic fibrosis, and hemophilia.
The new drug, developed by the South Plainfield, NJ-biotech firm and called PTC124, binds to the ribosome, a cellular component where the genetic code is translated into proteins, one amino acid at a time. The drug allows the ribosome to read through a mistake in the genetic code called a premature stop codon in order to properly make whole proteins.
In DMD, patients are missing dystrophin, a protein that helps keep muscle cells intact. About 15 percent of DMD patients do not make dystrophin because of the mutation. DMD eventually affects all voluntary muscles, as well as heart and breathing muscles.
PTC124 attaches to ribosomes in all cell types within the MD mouse model, overriding the mutation in the dystrophin gene that tells it to halt production of the protein. Instead of stopping, the full-length dystrophin protein is made. The drug enables enough protein to be made to correct defects in the muscle of the DMD mouse, and at the same time the drug does not prevent the ribosome from reading correct "stop" signals in the genetic code to make other necessary proteins.
"Enough dystrophin accumulated in the muscles of the MD mice so that we could no longer find defects in the muscles when we examined them," says Sweeney. "For all intents and purposes the disease was corrected by treatment with PTC124." The drug allowed dystrophin to be made in cells in which it was previously absent, to be delivered to the proper location at the cell membrane, and to induce restoration of muscle function in rodent muscles.
Co-first author Elisabeth Barton, PhD, worked on this project as a postdoctoral fellow in the Sweeney lab, and continued as a collaborator when she became an Assistant Professor in Penn’s School of Dental Medicine.
The study was supported in part by the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy.
PTC124 is presently nearing the end of a Phase II multi-center clinical trial in DMD patients, of which Children's Hospital of Philadelphia is a major accruing site.
Dr. Sweeney directs a Paul Wellstone Muscular Dystrophy Cooperative Center, sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. He is also on the Scientific Advisory Board of PTC Therapeutics Inc.