|
|
|
| |
News - Anson Community Hospital, Wadesboro North Carolina USA |
|
|
|
|
|
|
| Anson Community Hospital |
| CMC Discovery Holds Hope for MD Patients |
| Date : - 16/09/2008 |
|
A medical research team at Carolinas Medical Center (CMC), headed by Qi Long Lu, M.D., Ph.D., has made a discovery that holds promise to restore muscle function in patients with Duchenne muscular dystrophy (DMD).
Dr. Lu’s findings are summarized in an article that will be published Sept. 30 in a highly respected research journal called “Proceedings of the National Academy of Sciences of the U.S.A.” He and his colleagues have been utilizing a medical approach called “gene manipulation therapy” that – if ultimately proven safe and effective for humans – would offer new hope to DMD patients.
DMD patients suffer an affliction in which gene mutations prevent the body from producing a normal supply of dystrophin, a protein that helps voluntary skeletal muscles (such as those that move the limbs and trunk) and heart muscle to function normally.
Dr. Lu’s research uses an approach called “exon skipping.” Exon skipping is intended to help the DMD patient's body again produce dystrophin that is at least somewhat functional, so that muscle cells will not break down and die over time, the way they do currently in patients afflicted with DMD. In recent years, the life expectancy of DMD patients has increased from roughly 20 years of age to roughly 30 years of age, due to better medical management and care. Currently, however, the effects of the disease cannot be stabilized or reversed.
Muscular dystrophy research efforts have been high profile in the United States, largely due to fundraising efforts by the Muscular Dystrophy Association (MDA), including the Jerry Lewis Labor Day Telethon and other awareness initiatives. MDA currently spends some $ 40 million dollars annually on biomedical research.
Dr. Herbert Bonkovsky, Vice President of Research at CMC, said that Dr. Lu’s work, in collaboration with scientists at other research centers, has great potential for benefit if experimental results in mice can ultimately be safely adapted for humans.
Dr. Lu, who came to work at CMC’s McColl-Lockwood Laboratory for Muscular Dystrophy Research in September 2004, said that his team is looking forward to the next step in DMD research. His current article explains in detail the laboratory trials that have produced measurable improvement in mice. “Toxicity has been tolerable,” he said, “with no deaths, weight loss or adverse effects on the blood or on liver or kidney function.”
If effectiveness holds up in subsequent research, scientists would eventually move to large-scale clinical trials in humans. “Our eventual goal,” he said, “would be a treatment regimen for human patients that would rely on intravenous injections every couple of weeks.”
Dr. Bonkovsky said the therapeutic concept being pursued by Dr. Lu is about 10 years old, but that therapeutic efficiency has “always been quite low until now.” He said the exon skipping approach is only a few years old, and that he and his colleagues have been greatly encouraged by the recently observed successes in mice. These successes include the restoration of protein in all muscles, including the heart muscle, leading to improved muscle function.
Michael Rose, President of the Carolinas HealthCare Foundation, said he was pleased to see Dr. Lu’s work publicized in such a well respected academic journal. “It is significant when research findings hold up to such a rigorous process of peer review,” he said. “DMD is a dreaded disease that impacts an estimated 10,000 patients in our country. It is heartening to see new developments that offer hope to patients who have one of the most common and severe forms of MD.”
Rose noted that Dr. Lu’s research effort is being funded by three sources, the Carolinas Muscular Dystrophy Research Endowment at Carolinas HealthCare Foundation, the MDA, and the U.S. Army Medical Research Office, Department of Defense.
“This work would not be possible without a dedicated base of donors who are strongly committed to finding better treatments and hopefully a cure,” he said. “I am glad that the team at CMC has been given the support needed to assume an international leadership role. Dr. Lu was recruited to CMC from a prestigious position at the Royal College of Medicine in London, where he developed a global reputation in this highly specialized field. It speaks well for our community that we have him here.”
|
|
| Reference : - www.carolinashealthcare.org |
| Back To List |
|
|
|
|
|
| Copyright © 2024 Medical Tour Information All Rights Reserved |
|