Therapy

• 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.

• Elijah Dottery, diagnosed with sickle cell anemia as a baby, became the first U-M patient to receive the newly FDA-approved stem cell therapy. • The treatment uses patient's own stem cells edited via CRISPR to produce functional hemoglobin, administered at Michigan Medicine. • This breakthrough matters for over 100,000 U.S. sickle cell patients, offering potential cure where prior therapies only managed symptoms.

• Texas A&M researchers developed a nasal spray therapy that reverses brain aging by healing inflammation and restoring memory function. • The treatment targets age-related cognitive decline, turning 'brain fog to brain focus' in preclinical models. • Announced on April 14, 2026, it reshapes future therapies for neurodegenerative diseases.

• 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.

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...

• NIH awarded $500 million in grants on April 7, 2026, to 15 US institutions for CRISPR-based gene therapies addressing 100,000 Americans with sickle cell disease. • Phase 2 trials at Boston Children's show 94% pain crisis reduction; total funding supports 200 patients over five years. • Initiative responds to 17% US mortality drop goal by 2030; 'Breakthrough for underserved communities,' per NIH Director.

• Combination therapy of ixekizumab and tirzepatide yields better results in psoriatic arthritis patients compared to ixekizumab alone, per recent medical research. • This finding advances treatment options for the chronic inflammatory condition affecting joints and skin. • Updated clinical approaches could enhance patient quality of life and disease management.

• A Philadelphia cancer patient who had exhausted conventional treatment options was kept alive using a novel T-cell therapy developed and administered at Rutgers Health. • The case represents a breakthrough application of immunotherapy, offering hope to patients facing terminal cancer diagnoses with limited options. • The success demonstrates the therapeutic potential of advanced cellular therapies and highlights Rutgers Health's role in pioneering cutting-edge cancer treatment innovations.

• The MajesTEC-3 clinical trial led by UAB researchers demonstrates that a new two-drug immunotherapy regimen can lead to long-lasting remission for multiple myeloma patients. • Over 83 percent of patients enrolled in the trial remain alive and progression-free three years after therapy, supporting approval of this potentially curative treatment. • The findings represent a significant advance for multiple myeloma, a blood cancer with limited treatment options historically.

• The U.S. Supreme Court ruled 6-3 on April 1, 2026, that state bans on conversion therapy for minors violate counselors' First Amendment rights. • The decision overturns California's pioneering 2012 law and similar measures in over 20 states prohibiting practices aimed at changing sexual orientation. • Chief Justice Roberts wrote the majority opinion, emphasizing licensed professionals' speech protections in private counseling.

• A Phase 2 clinical trial led by Memorial Sloan Kettering Cancer Center demonstrated that engineered CAR-T cells targeting mesothelin showed a 52% overall response rate in 89 patients with advanced pancreatic and ovarian cancers. • The trial, results published March 19 in Cancer Cell, showed durable responses lasting over one year in 68% of responding patients, compared to historical response rates below 20% with conventional chemotherapy. • CAR-T therapy has revolutionized blood cancer treatment but remained ineffective against solid tumors until recent engineering advances, potentially opening a new therapeutic avenue for 400,000 Americans diagnosed with pancreatic and ovarian cancers annually.

• Creative Biolabs announced an upgraded next-generation drug discovery pipeline integrating AI-driven antibody engineering in Shirley, New York. • The platform accelerates development of CAR-T therapies and antibody humanization using advanced deep learning techniques. • This innovation targets faster drug discovery in healthcare and pharmaceuticals, enhancing precision medicine applications.

The American Council on Science and Health lists 16 landmark public health achievements, including George Washington's 1777 order for Continental Army smallpox inoculations that saved forces from a disease killing 10 soldiers per battle death. Polio cases sharply declined after Jonas Salk's 1955 vaccine was deemed safe, with Albert Sabin's 1960s oral version enabling community-wide protection. CDC identified initial AIDS clusters in 1981 among young men; by 1996, NIH-supported combination antiretroviral therapy turned HIV into a manageable condition, bolstered by US companies like Gilead and PEPFAR in 2003. These advances revolutionized medicine from antibiotics to organ transplants and birth control.