Sickle Cell Gene Therapy Holds at Two Years; Europe Tallies Its Deadliest Summer
Casgevy's CRISPR cure stays durable; Lecanemab holds at three years; ECDC counts 8,100 heat deaths across the Mediterranean.
By Dr. Asher Knippel
Two years into the gene-therapy era for sickle cell disease, durability data are holding. Elsewhere, the close of a record-hot Mediterranean summer arrives with a mortality bill, and three trials report steady progress against Alzheimer's disease, resistant hypertension, and early-stage cancers.
Casgevy Gene Therapy Shows 97% Freedom from Vaso-Occlusive Crises at Two Years
Two-year follow-up data from the CLIMB-SCD-121 pivotal trial — published in the New England Journal of Medicine — show that 28 of 29 participants (97%) who received Casgevy, the CRISPR-Cas9-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics, remained entirely free of severe vaso-occlusive crises (VOC) through month 24. The single remaining participant experienced one mild crisis at month 18 — substantially below that person's historical average of four annual crises. Casgevy acts by reactivating foetal haemoglobin, sidestepping the defective adult haemoglobin gene that causes the disease; the two-year data suggest the edit is stable and durable. The therapy received US FDA approval in December 2023 and EMA conditional marketing authorisation in early 2024 and is now available in 14 specialist centres across Europe. Cyprus patients are eligible for GeSY referral to the Athens-based Laikon General Hospital haematology unit, which has treated three Cypriot patients since authorisation. Full unconditional EMA approval is anticipated before end 2026. Sickle cell disease affects approximately 300,000 newborns annually, disproportionately in sub-Saharan Africa and in communities with southern Mediterranean and Middle Eastern ancestry.
Lecanemab Sustains 31% Slowing of Alzheimer's Decline at Three Years
A prespecified three-year extension of the CLARITY AD trial, reported in JAMA Neurology, finds that lecanemab (Leqembi; Eisai/Biogen) continues to slow clinical progression on the CDR-Sum of Boxes scale by approximately 31% relative to placebo — a benefit that has not eroded between year one, year two, and year three. The amyloid-targeting monoclonal antibody received full US FDA approval in July 2023 and conditional EU approval in September 2024; roughly 25,000 people are now receiving treatment globally. Safety data remain stable: amyloid-related imaging abnormalities (ARIA) occurred in 21% of trial participants, the vast majority asymptomatic and resolving within months. The European Medicines Agency had requested additional real-world safety registry data before considering a label update; the three-year extension readout will be submitted to support that application. Lecanemab is indicated for early-stage Alzheimer's disease, which requires PET or cerebrospinal-fluid confirmation of amyloid pathology — a diagnostic step that remains limited in lower-resource settings across the eastern Mediterranean.
ECDC: European Summer 2026 Claims More Than 8,100 Heat-Related Deaths; Cyprus Records Its Hottest August
The European Centre for Disease Prevention and Control has released preliminary excess-mortality data for summer 2026, estimating 8,140 heat-attributable deaths across 20 EU member states between June and August — the second-highest summer toll on record after 2022. Mediterranean countries carried the greatest burden: Italy (approximately 2,100), Spain (1,800), and Greece (1,400) together account for more than half of attributable deaths, concentrated among adults aged 75 and over living alone or without air conditioning. Cyprus recorded its hottest August since instrumental records began, with Nicosia reaching 43.8 °C on 14 August; island-wide excess mortality for the month is under active review by the Cyprus Ministry of Health and the pan-European EuroMOMO network. ECDC is calling on national health systems to expand cool spaces, integrate heat-risk into geriatric care planning, and scale heat-health alert systems ahead of the 2027 season. The summer data will feed directly into the EU Climate Adaptation Health Strategy, expected to be presented to the European Parliament in October 2026.
QDENGA Dengue Vaccine Shows 81% Real-World Effectiveness in European Traveller Study
A prospective cohort study of 7,400 adults vaccinated with QDENGA (TAK-003; Takeda) in Germany, France, and Spain — published in The Lancet Infectious Diseases — reports 81% vaccine effectiveness against confirmed dengue fever over 12 months of follow-up. Participants were adults planning travel to dengue-endemic regions, matched to unvaccinated travellers by destination and age group. Protection was consistent across all four dengue serotypes (DENV-1 through 4), including serotype 3, which was less well represented in the original phase-3 trials. On the basis of these data, the ECDC this week upgraded its travel medicine guidance to classify QDENGA as a recommended (rather than merely optional) vaccine for travel to high-transmission destinations. The distinction is clinically meaningful: healthcare providers in GeSY-registered travel medicine clinics are now expected to proactively discuss the vaccine with eligible patients, rather than offering it only on request. Dengue is the world's fastest-spreading vector-borne viral disease; the ECDC recorded 69 locally acquired cases in EU/EEA countries in summer 2026, predominantly in southern Italy and Madeira.
Baxdrostat Cuts Resistant Hypertension by 11 mmHg in Phase-3 HALO-2 Trial
Full results of the HALO-2 phase-3 trial for baxdrostat — an aldosterone synthase inhibitor developed by AstraZeneca — show a statistically significant 11.4 mmHg reduction in office systolic blood pressure versus placebo in adults with treatment-resistant hypertension (TRH), defined as uncontrolled blood pressure despite three or more maximally tolerated antihypertensives including a diuretic. The 1,240-person trial ran across 18 countries; secondary endpoints including 24-hour ambulatory blood pressure, left ventricular mass index reduction, and urinary albumin-to-creatinine ratio were all met. Potassium monitoring was required throughout given the drug's mechanism; clinically meaningful hyperkalaemia occurred in 4.2% of participants, consistent with phase-2 data. AstraZeneca has announced plans for a simultaneous FDA and EMA regulatory submission in Q4 2026. Treatment-resistant hypertension affects an estimated 10–15% of all adults with hypertension — roughly 100 million people globally — and remains one of the most significant modifiable risk factors for stroke, heart failure, and chronic kidney disease. Baxdrostat's mechanism differs from existing mineralocorticoid-receptor antagonists such as spironolactone and eplerenone, and researchers are already designing combination protocols.
Multi-Cancer Early Detection Blood Test Reaches 87% Sensitivity in 10,000-Person NHS Validation
The largest prospective validation of a circulating tumour DNA (ctDNA) methylation-based multi-cancer early detection (MCED) test to date — conducted across 23 NHS trusts in England and Scotland with 10,040 participants aged 50–77 — reports 87% sensitivity for stage I–II cancers across 12 cancer types, including lung, colorectal, pancreatic, ovarian, and several haematological malignancies. Specificity was 99.3%, corresponding to a false-positive rate below 1 in 100. The study, published in the New England Journal of Medicine, is the first MCED validation in a representative, unselected NHS screening population. The UK National Screening Committee is reviewing the results; a positive recommendation could enable phased NHS rollout to adults aged 50–77 from 2028. The most transformative opportunity lies in pancreatic and ovarian cancers, which are typically diagnosed at stage III or IV and carry five-year survival rates below 20%; detecting them at stage I–II would substantially alter prognosis. Cyprus is a participating site in the parallel European MCED consortium study, coordinated through the Bank of Cyprus Oncology Centre in Nicosia.
This article is a journalistic summary produced for informational purposes only and does not constitute medical advice. Readers should consult a qualified clinician before making any change to their treatment, medication, or health management plan.