Disease

• The FDA granted accelerated approval to a CRISPR-based gene therapy for severe sickle cell disease on April 20, marking the first approved genetic treatment editing patient blood cells to produce functional hemoglobin. • Clinical trials demonstrated 95% of treated patients experienced elimination or near-elimination of vaso-occlusive crises within 12 months, with durable responses observed for over two years post-treatment. • The therapy addresses a disease affecting approximately 100,000 Americans, primarily of African descent, offering potential cure for patients previously reliant on pain management and blood transfusions.

• McGovern Medical School at UTHealth Houston launched the Center for Innovation in Congenital Heart Disease, a multidisciplinary center bringing together experts in medicine, science, and engineering to transform understanding and treatment of the condition. • A major research focus involves recreating early human heart development using stem cells and bioprinted structures to study how congenital defects form. • The center bridges discovery and clinical care by advancing research, developing technologies, and translating innovations into patient treatment solutions across a lifetime of care.

• Thomas Jefferson University launched a multicenter clinical trial led by Manisha Verma, MD, and Victor Navarro, MD, at Jefferson Einstein Philadelphia Hospital to improve palliative care for advanced liver disease patients. • Advanced liver disease causes significant scarring and loss of function, leading to serious health challenges with limited quality-of-life support. • Funded by the Patient-Centered Outcomes Research Institute and published in JAMA Internal Medicine, the trial addresses a major care gap for ALD patients nationwide.

• A new VCU Health study indicates alcohol-related liver disease prevalence may be twice as high as previously estimated because of underreported alcohol consumption. • The research uncovers hidden scale of liver damage, challenging prior data on this growing public health issue. • Findings highlight need for better screening and awareness amid rising alcohol use disorders in the US.

• FDA has approved an investigational new drug application for synthetic CAR-T (sCAR-T) therapy targeting autoimmune conditions. • The clearance enables clinical trials to test this engineered cell therapy in US patients with refractory autoimmune diseases. • sCAR-T represents a novel approach adapting cancer immunotherapy for immune disorders, potentially offering durable remissions.

Charity advises replacing seed and nut feeders, where birds gather, with small amounts of mealworms, fat balls or suetGarden birds should not be fed seeds and nuts over the summer months, the RSPB has said, in an attempt to reduce the spread of avian diseases.Bird lovers are being urged to take down their bird feeders between May and October to help birds such as the greenfinch, whose numbers have plummeted after the spread of trichomonosis, a parasitic disease transmitted more easily when birds cluster around feeders in the warmer months. Continue reading...

Treatment reset wayward immune system of patient with life-threatening conditions, say scientists, in a world firstA woman who lived with three life-threatening autoimmune diseases for more than a decade has returned to a near-normal life after a cell therapy reset her wayward immune system.The 47-year-old had had nine different treatments, none of which had a lasting impact, before receiving the therapy last year at University Hospital Erlangen in Germany. At the time, she required daily blood transfusions and permanent blood thinning medication to control her illness. Continue reading...