Progress In Gene-Based Therapies For Heart Attacks, Sickle Cell Disease
Gene-edited cells may be useful for repairing hearts damaged by heart attacks, according to new research. Separately, a "competitive biotech race" is underway to treat sickle cell disease. And gene therapy for tackling Duchenne muscular dystrophy is also reported.
The Washington Post:
Gene-Edited Cells Move Science Closer To Repairing Damaged Hearts
Scientists seeking to combat the nation’s No. 1 killer have discovered why experiments using cell transplants to repair damage from a heart attack wind up backfiring and causing life-threatening arrhythmias. A new study in the journal Cell Stem Cell points the way toward a possible solution, advancing medicine a step further toward the goal of regenerating the human heart. (Johnson, 4/28)
The Washington Post:
Gene Therapy For Sickle Cell Disease Treatment Brings Hope To Patients
After decades of neglect, stigma and underfunding, sickle cell is getting the equivalent of the red carpet treatment in science. It’s the target of a competitive biotech race, with scientists and companies using a crop of cutting-edge tools to try to cure the debilitating illness. ... It’s a dramatic about-face for sickle cell patients, who have often felt abandoned by the medical system. The rare disease afflicts about 100,000 people in the United States, most of them Black. Racism at both the institutional and interpersonal level has stymied funding and alienated patients, who are often treated as drug-seekers when they show up in emergency rooms in acute pain. (Johnson, 4/28)
In other pharmaceutical news —
Fox News:
Students Use AI Technology To Find New Brain Tumor Therapy Targets — With A Goal Of Fighting Disease Faster
Glioblastoma is one of the deadliest types of brain cancer, with the average patient living only eight months after diagnosis, according to the National Brain Tumor Society, a nonprofit. Two ambitious high school students — Andrea Olsen, 18, from Oslo, Norway, and Zachary Harpaz, 16, from Fort Lauderdale, Florida — are looking to change that. (Rudy, 5/1)
Stat:
Biotechs Aim To Leapfrog Ozempic In Red-Hot Weight Loss Market
For nearly a decade, Novartis aggressively pursued a drug candidate for muscle disorders, testing it on people with chronic inflammation, elderly people with frailty, hip surgery patients, and other groups. Time and again, the trials failed to show that the drug, bimagrumab, led to a significant enough improvement in patients’ physical function. But researchers noticed something else: The patients lost body fat. (Chen and DeAngelis, 5/1)
USA Today:
HIV In Remission: California Man Paul Edmonds 1 Of 5 Patients Cured
For the past two years, Paul Edmonds has been part of an extremely exclusive club with a membership that has reached five people. (Sasic, 4/30)
