The 10 most exciting new medical discoveries (and what they mean for patients)

Below are ten of the most clinically meaningful breakthroughs from roughly the past 12–18 months, explained in plain English with why they matter for care.

1. A CRISPR cure reaches the clinic for sickle cell disease
   The first CRISPR/Cas9 therapy, Casgevy (and another gene therapy, Lyfgenia), won FDA approval for people ≥12 with severe sickle cell in late 2023. It edits a patient’s blood stem cells so they produce healthy hemoglobin and can prevent pain crises—offering the possibility of a one-time, durable treatment instead of lifelong transfusions and hospitalizations. U.S. Food and Drug Administration+1
2. Alzheimer’s: donanemab (Kisunla) approved
   In July 2024 the FDA approved donanemab, an anti-amyloid antibody for early Alzheimer’s (MCI or mild dementia). In trials it slowed cognitive and functional decline, giving patients and families more time at earlier levels of independence. It’s a monthly IV with MRI monitoring for ARIA risks. U.S. Food and Drug Administration+1
3. A simple blood draw for colon-cancer screening
   The FDA cleared Shield, the first blood test authorized as a primary screening option for average-risk adults 45+. It looks for tumor DNA (and epigenetic signals) in the bloodstream—an option that could bring many screening-averse people into care and catch cancers earlier. Positive tests still need a follow-up colonoscopy. U.S. Food and Drug Administration+2FDA Access Data+2
4. Twice-yearly HIV prevention
   In June 2025 the FDA approved lenacapavir (Yeztugo) as the first every-6-months PrEP shot. Phase 3 trials reported near-perfect protection—transformative for people who can’t (or don’t want to) take daily pills or frequent injections. Global guidelines and EU authorization are following. AP News+3Gilead+3New England Journal of Medicine+3
5. GLP-1 medicines protect the kidneys, not just weight and sugar
   The FLOW trial (NEJM, 2024) showed semaglutide cut the risk of major kidney outcomes and CV death in type 2 diabetes with CKD—meaning fewer dialysis starts, slower eGFR decline, and better survival. This moves GLP-1s squarely into kidney- and heart-protection, not only weight or glucose. New England Journal of Medicine+2PubMed+2
6. First drug treatment for obstructive sleep apnoea
   In December 2024 the FDA approved tirzepatide (Zepbound) as the first medication for moderate-to-severe OSA in adults with obesity. By driving substantial weight loss, it reduced apnea severity—important for patients who struggle with CPAP or are waiting for surgery. U.S. Food and Drug Administration+1
7. Xenotransplantation steps forward
   In March 2024 surgeons performed the first transplant of a gene-edited pig kidney into a living person at Mass General; follow-up reports and additional porcine organ milestones (including a temporary pig liver support case) underscore rapid progress toward easing organ shortages. Patients on long waitlists could ultimately see new, life-saving options. The Times of India+3Massachusetts General Hospital+3Harvard Medical School+3
8. Gene editing as a one-time infusion for heart and amyloid diseases
   Two in-human “in vivo” gene-editing programs advanced: NTLA-2001 entered Phase 3 for transthyretin amyloidosis (a fatal protein-buildup disease), and VERVE-101 base-editing showed durable LDL-cholesterol lowering by turning off PCSK9 in the liver (with active safety monitoring and program adjustments). If successful, these could replace lifelong pills or infusions with a single treatment. Reuters+4CGTlive+4Somatic Cell Gene Editing+4
9. CAR-T therapy moves beyond cancer into autoimmune disease
   Early clinical reports in 2025 show CD19-directed CAR-T cells can induce deep remissions in severe, refractory systemic lupus erythematosus, pointing to a future in which short-course cell therapy “resets” the immune system and reduces years of steroids and organ damage. Larger trials are underway. Nature+1
10. Non-invasive ‘ultrasound helmet’ for precise brain stimulation
    A UK team unveiled a 256-transducer ultrasound helmet that can modulate deep brain circuits with MRI guidance (Nature Communications, 2025). It could offer a future alternative to brain surgery for conditions like Parkinson’s, depression and tremor—opening neuromodulation to far more patients. Nature+1

A bonus “watch this space”

A blood test for ME/CFS: researchers from the University of East Anglia and Oxford Biodynamics reported a highly accurate epigenetic blood test for ME/CFS (Oct 2025). It’s early and needs independent validation before routine clinical use—but if confirmed, it could end years-long diagnostic odysseys. The Guardian+1

What this means for patients—practically

• More conditions treated once, not forever: Gene editing (for SCD, ATTR, possibly high LDL) and CAR-T for autoimmunity are pushing care from chronic management to single-episode interventions.
• Earlier, easier detection: Blood-based screening (Shield) and future multi-cancer/ME-CFS tests mean diagnosis with a simple blood draw, improving uptake and stage at detection.
• Prevention gets simpler: Twice-yearly PrEP could dramatically reduce new HIV infections—especially where daily pills are hard to sustain.
• Common diseases, fewer complications: GLP-1s are now disease-modifying for kidneys and hearts, not only weight.
• Access could still be a hurdle: Many of these advances are costly or specialized. Ask about clinical trials, patient-assistance programs, and whether a center near you offers these options.