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Blue Dogs: We Have Agreement With House Dems On Health Bill
Work on the House health care reform bill is slowing as Rep. Henry Waxman of California and fiscally conservative Blue Dog Democrats have come to an agreement on Medicare payments, The New York Times reports.

NICE Approves Use Of Lenalidomide In Patients With Multiple Myeloma Who Have Received Two Or More Previous Therapies
Around 2000 multiple myeloma sufferers in the UK could have their lives extended by around three months after a decision by The UK National Institute for Health and Clinical Excellence (NICE) to approve lenalidomide in those patients who have received two or more previous therapies-provided that the cost of cycles beyond the 26th cycle of treatment are met by the drug manufacturer. A summary of the NICE decision is published in a Special Report Online First and in the July edition of The Lancet Oncology.
News of the day
Experts Urge Increased Education About Diabetes During Pregnancy
The American College of Obstetricians and Gynecologists and the American Diabetes Association next month plan to launch a joint campaign designed to raise awareness about pregnancy-related risks from diabetes, USA Today reports. An increasing number of women are either beginning pregnancies with existing Type 1 or Type 2 diabetes -- known as pre-gestational diabetes -- or developing gestational diabetes, according to Sue Kirkman, vice president of clinical affairs for ADA. Kirkman said that diabetes increases the risk for miscarriage, delivery complications, maternal health problems and birth defects. However, these risks can be reduced through preconception counseling, controlling blood sugar and maintaining a healthy weight, she said. According to the Centers for Disease Control and Prevention, almost five of every 1,000 women ages 18 to 44 have diabetes. The majority of these women have Type 2 diabetes, which is linked to obesity, USA Today reports. Denise Charron-Prochownik, an associate professor of health promotion at the University of Pittsburgh Medical Center School of Nursing and Graduate School of Public Health, said that pre-conception education for women with existing diabetes should begin as early as age 13 but that "it"s not happening."Helain Landy, chair of the Department of Obstetrics and Gynecology at Georgetown University Hospital, said that gestational diabetes occurs in about 4% of pregnant women. Landy said, "From an epidemiological standpoint, that is a lot." Florence Brown, co-director of the Joslin Beth Israel Deaconess Medical Center"s Diabetes and Pregnancy Program, said that many women with gestational diabetes are unaware that they are more likely to develop cardiovascular disease later in life and have children who develop diabetes (Brophy Marcus, USA Today, 7/6).
Endocrinology

Genetic Of Muscular Dystrophy Neutralized

Researchers at the University of Rochester Medical Center have found a way to block the genetic flaw at the heart of a common form of muscular dystrophy. The results of the study, which were published in the journal Science, could pave the way for new therapies that essentially reverse the symptoms of the disease. The researchers used a synthetic molecule to break up deposits of toxic genetic material and re-establish the cellular activity that is disrupted by the disease. Because scientists believe that potentially all of the symptoms of myotonic dystrophy the most common form of muscular dystrophy in adults flow from this single genetic flaw, neutralizing it could potentially restore muscle function in people with the disease. "This study establishes a proof of concept that could be followed to develop a successful treatment for myotonic dystrophy," said neurologist Charles Thornton, M.D., the senior author of the study and co-director of the University of Rochester Medical Center"s Wellstone Muscular Dystrophy Cooperative Research Center. "It also demonstrates the potential to reverse established symptoms of the disease after they have developed, as opposed to simply preventing them from getting worse." Myotonic dystrophy is a degenerative disease characterized by progressive muscle wasting and weakness. People with myotonic dystrophy have prolonged muscle tensing (myotonia) and are not able to relax certain muscles after use. The condition is particularly severe in the hand muscles and can cause a person"s grip to lock making it difficult to perform rapid, repeated movements. Currently there is no medication to halt the progression of the disease. Toxic RNA Holds Proteins Hostage Although the genetic flaw that causes myotonic dystrophy was discovered in 1992, researchers studied the defect for many years before they had a clear understanding of the molecular events that ultimately produce the symptoms of the disease. Over time it became apparent that a central player in myotonic dystrophy was RNA, a versatile molecule that is very similar to DNA. RNA serves a vital function by relaying the genetic information from the nucleus the protected area of the cell that houses DNA out to the main body of the cell, where the instructions are used to build proteins. Every gene produces its own RNA, usually in multiple copies, and every RNA is a genetic blueprint of its parent gene. The surprising aspect of myotonic dystrophy was that the genetic defect leads to production of a toxic RNA the first example in human genetics in which RNA was cast in the role of molecular perpetrator. The errant RNA has a toxic effect because it grabs onto and holds hostage certain proteins, preventing them from carrying out their normal functions. For example, the capture of a protein called "muscleblind" causes the locking grip phenomenon that is a hallmark of the disease, a sign of faulty electrical control in muscle cells. Over time, the toxic RNA is produced in abundance and the captive proteins accumulate in deposits or inclusions that are visible in the cell"s nucleus. "An unexpected byproduct of research on myotonic dystrophy was that we were forced to change our ideas about the role of RNA in genetic disease," said Thornton. "Once we adjusted to this new concept, we realized that the prospects for developing treatment might be unusually good. No essential component of muscle is missing, but some important proteins are in the wrong place, stuck on the toxic RNA." New Tools to Tackle Genetic Flaws The Rochester team used a synthetic molecule called an antisense morpholino oligonucleotide that mimics a segment of the genetic code. In this case the morpholino was specifically designed to bind to the toxic RNA and neutralize its harmful effects by releasing the captured proteins. When injected into the muscle cells of mice with myotonic dystrophy the molecule found its way to the cell nucleus, broke up the deposits of toxic RNA, freed the captive muscleblind proteins, and ultimately improved the function of the muscle cells. The researchers specifically observed a restoration of proper electrical control in the cells, which is a convenient way to monitor the condition. However, because the hostage proteins play a role in a myriad of other cellular functions, they believe that this treatment will ultimately alleviate other aspects of the disease as well. "Based on our current understanding we would predict that by releasing the proteins held hostage, many of the symptoms of the disease may potentially be corrected by this approach," said URMC neurologist Thurman Wheeler, M.D., co-author of the study. These genetic tools are relatively new and have provided researchers with a heretofore unprecedented ways to precisely target and manipulate genetic activity. "The current textbooks for medical students do not have chapters on antisense oligonucleotides, but this will change in the near future," said Thornton. "As compared to conventional drugs that work on proteins, antisense oligonucleotides work on RNA. They have been around for 20 years, but only recently is their full potential being realized. They provide great flexibility and they can be developed rapidly." The authors are quick to point out that major hurdles must be overcome before this compound can be tested in humans. Specifically, a better delivery system must be developed to get this or a similar compound to where it needs to go in the body, and the potential side effects must be carefully analyzed. However, having established a general concept of what a treatment for myotonic dystrophy may look like, researchers believe that the next steps in developing an effective drug should go faster. Other authors on the study include University of Rochester Medical Center scientists Krysztof Sobczak, Ph.D., Robert J. Osborne, Ph.D., Xiaoyan Lin, Ph.D., John Lueck, Ph.D., and Robert Dirksen, Ph.D. Funding for the study was provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Neurological Disorders and Stroke, the Muscular Dystrophy Association, and the Run America Foundation. University of Rochester Medical Center


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