A stem-cell transplant reversed sickle cell disease in adults, according to a study that offers a potential cure for the debilitating condition. Half of those who had the transplant, which involved the patient and a sibling, also were able to stop taking immunosuppressant drugs without experiencing rejection or having the donor cells attack their body, research released today in the Journal of the American Medical Association showed. People undergoing stem-cell transplants usually must take immunosuppressants for the rest of their lives. More than 90,000 people in the U.S. have sickle cell disease, a genetic disorder found mostly in people of African descent, according to the U.S. National Institutes of Health. The condition can cause severe pain, organ damage and stroke. Study author Matthew Hsieh said its too soon to say the researchers have found a cure as patients have been followed only for an average of 3 1/2 years, but he is optimistic. Theyre sickle-cell free for now, Hsieh, a staff clinician at the National Institute of Diabetes and Digestive and Kidney Diseases and the National Heart, Lung, and Blood Institute in Bethesda, Maryland, said today in a telephone interview. We are cautiously optimistic they are cured. Children with sickle … Continue reading →

CHICAGO (AP) Bone marrow transplants can reverse severe sickle cell disease in adults, a small study by government scientists found, echoing results seen with a similar technique used in children. The researchers and others say the findings show age need not be a barrier and that the technique may change practice for some adult patients when standard treatment fails. The transplant worked in 26 of 30 adults, and 15 of them were even able to stop taking drugs that prevent rejection one year later. "We're very pleased," said Dr. John Tisdale, the study's senior author and a senior investigator at the National Institutes of Health. "This is what we hoped for." The treatment is a modified version of bone marrow transplants that have worked in kids. Donors are a brother or sister whose stem cell-rich bone marrow is a good match for the patient. Tisdale said doctors have avoided trying standard transplants in adults with severe sickle cell disease because the treatment is so toxic. Children can often tolerate it because the disease typically hasn't taken as big a toll on their bodies, he said. The disease is debilitating and often life-shortening; patients die on average in their 40s, Tisdale … Continue reading →

(Leah Hogsten | The Salt Lake Tribune) A grow house at the Realm of Caring facility in Colorado Springs, Colo., in October 2013. Starting in July, a new law will allow some Utah families of children with epilepsy to import cannabis oil from Colorado. But children who use the oil may be disqualified from enrolling in an upcoming trial of pharmaceutical-grade cannabis oil at Primary Children's Hospital in Salt Lake City. Health Primary Childrens wins contract to study anti-seizure drug made from cannabis. Neurologists at Primary Childrens Hospital are one step closer to being able to prescribe pharmaceutical-grade cannabis to children and teens with severe epilepsy. Only those who apply to be part of a drug trial will be eligible 25 spots are available and its not known when the study will start. How to sign up for cannabis Children will be enrolled in the Epidiolex drug trial on the advice of neurologists, who will draw study participants from their patient pool at Primary Childrens Hospital. Starting July 8, Utahns with untreatable epilepsy, children and adults, will be able to apply for a hemp supplement registration card authorizing them to import whole-plant cannabidiol extracts from states such as Colorado. They … Continue reading →

WINSTON-SALEM, N.C. Families in North Carolina are pushing for insurance reform that would allow children to access what they call the golden standard of treatment for autism. House Bill 497, Autism Health Insurance Coverage, passed through the state House last year with a vote of 105-7. Advocates hope the Senate will hear the bill during the current short session. Kyle and Bobbie Robinson are from Greenville. Their son Samuel will be three years old in August. Samuel was diagnosed with autism at the UNC Center for Development Disabilities in April of 2013. The Robinsons immediately sought the best treatment for their son. Doctors recommended Applied Behavior Analysis (ABA therapy). I remember thinking will he ever say mama? Or will he ever say I love you? Bobbie explained. With this type of therapy, if we could somehow find a way to make it work financially, Samuel could possibly improve. The couple soon learned ABA therapy was hard to access in North Carolina and even harder to afford with treatments costingaround sixty thousand dollars peryear. Kyle still lives and works full time in Greenville. Bobbie took a medical leave from her teaching joband packs up every Sunday night to come to Winston-Salem … Continue reading →

Ann Edwards (right) listens as her husband, former South Carolina Gov. Jim Edwards, explains how doctors at MUSC last year removed a five-centimeter long blockage from an artery in his brain that was the cause of his stroke. Brad Nettles/staff What is a stroke? A stroke occurs when a blood clot blocks an artery (a blood vessel that carries blood from the heart to the body) or a blood vessel (a tube through which the blood moves through the body) breaks, interrupting blood flow to an area of the brain. When either of these things happen, brain cells begin to die and brain damage occurs. When brain cells die during a stroke, abilities controlled by that area of the brain are lost. These abilities include speech, movement and memory. How a stroke patient is affected depends on where the stroke occurs in the brain and how much the brain is damaged. Source: National Stroke Association How to check/respond to a possible stroke? Use the FAST test F: Face - Check ability to fully smile; look for drooping A: Arm - Can they raise their arms? Are they weak? Read the original post: A year after surviving a stroke, Gov. Edwards … Continue reading →

Contact Information Available for logged-in reporters only Newswise The David Geffen School of Medicine at UCLA is one of six institutions nationwide chosen by the National Institutes of Health to join their effort in tackling the most difficult-to-solve medical cases and develop ways to diagnose rare genetic disorders. Part of a $120 million initiative called the Undiagnosed Diseases Network, the four-year $7.2 million UCLA grant will enable comprehensive bedside to bench clinical research to support physicians efforts to give long-sought answers to patients who have been living with mystery diseases. Undiagnosed diseases take a huge toll on patients, their families and the health care system, said Dr. Katrina Dipple, a co-principal investigator of the grant with Drs. Stanley Nelson, Christina Palmer and Eric Vilain. This funding will accelerate and expand our clinical genomics program, enabling us to quickly give patients a firm diagnosis and clarify the best way to treat them. Despite extensive clinical testing by skilled physicians, some diseases remain unrecognized because they are extremely rare, underreported or atypical forms of more common diseases. An interdisciplinary team of geneticists at each of the network sites will examine and study patients with prolonged undiagnosed diseases. A vast number of children … Continue reading →