MEMPHIS, Tenn. – Scientists at St. Jude Children’s Research Hospital comprehensively identified genes directly regulated by a protein associated with high-risk pediatric leukemias. High-risk leukemias, particularly MLL-rearranged (MLL-r) leukemia, often overexpress the homeodomain transcription factor HOXA9 protein, which cannot currently be targeted with drugs. This study provides a foundation for...
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FLORISSANT, Mo. – After a viral video on social media, one St. Louis girl got to live her dream of owning a bakery for the day. On top of that, customers raised enough money for her to have a once in a lifetime treatment, that increases her ability to live....
Guangzhou, China – Researchers from the South China University of Technology identified a novel approach to effectively silence the expression of transthyretin (TTR), with potential implications for innovative therapeutic approaches in TTR amyloidosis, including transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The proposed strategy involves stabilizing the G-quadruplex structure—a fundamental genomic structural feature—and...
The burden from activating KIT proto-oncogene (KIT) mutations in patients with advanced systemic mastocytosis (advSM) can be relieved with KIT inhibitors, improving the clinical trajectory of the disease. The protocols for determining staging of SM require updating since these drugs have become available, according to a review published in the International...
Stanford, Calif. – An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a phase 1 clinical trial has shown. While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease that makes standard stem cell transplant...
Stanford, California – Pancreatic cancer is a particularly aggressive and difficult-to-treat cancer, in part because it is often resistant to chemotherapy. Now, researchers at Stanford have revealed that this resistance is related to both the physical stiffness of the tissue around the cancerous cells and the chemical makeup of that...
Stanford, CA – Cell therapies for cancer can be potentially enhanced using a CRISPR RNA-editing platform, according to a new study published Feb. 21 in Cell. The new platform, Multiplexed Effector Guide Arrays, or MEGA, can modify the RNA of cells, which allowed Stanford University researchers to regulate immune cell metabolism in...
Palo Alto, Calif. – Handgrip strength was associated with breathing muscle strength in adults with cystic fibrosis (CF), according to a new study, suggesting the simple test may help complement standard lung function tests in clinical care. The research also found that lung function and breathing muscle strength were related to...
Stanford, Calif. – Researchers have identified 22 pesticides consistently associated with the incidence of prostate cancer in the United States, with four of the pesticides also linked with prostate cancer mortality. The findings are published by Wiley online in CANCER, a peer-reviewed journal of the American Cancer Society. To assess...
SOUTH SAN FRANCISCO, Calif. — Star Therapeutics, a late clinical-stage biotechnology company focused on the discovery and development of life-changing therapies for diseases with significant unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) and Breakthrough Therapy designation (BTD) to...