Gene Therapy, a Cholesterol Pill, and Microplastics in the Brain

A day of clinical follow-up, regulatory action, and public-health vigilance — from encouraging two-year durability data on a gene therapy with particular relevance to Cyprus, to a new Phase 3 result that could reshape cholesterol treatment, to an EU childhood obesity plan, and twin warnings on drug-resistant infections and microplastics accumulating in the human brain.

Wednesday, 26 August: Casgevy Gene Therapy for Beta-Thalassaemia Holds Strong at Two Years

A two-year follow-up of patients treated with Casgevy (exagamglogene autotemcel), the first CRISPR-based gene therapy approved for transfusion-dependent beta-thalassaemia, shows that 93% of participants remain transfusion-free, according to data published in the New England Journal of Medicine. The therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, works by reactivating foetal haemoglobin production in the patient's own stem cells, effectively bypassing the defective adult haemoglobin gene. The treatment requires a single administration after a bone-marrow conditioning regimen, and these 24-month data extend the durability picture beyond the pivotal trial's 12-month primary endpoint — though longer follow-up is still needed to confirm whether the effect is permanent.

The finding carries particular weight for Cyprus, which has one of the highest beta-thalassaemia carrier rates in Europe — roughly one in fourteen people carries the trait — and has operated a national screening and counselling programme since the 1970s. Most patients eligible for the therapy are being evaluated at specialist haematology centres; Casgevy received EMA approval for beta-thalassaemia in early 2024.

Wednesday, 26 August: An Oral Drug That Lowers Cholesterol Like an Injectable Completes Phase 3

Merck announced positive top-line results from MACADAM-3, a 12-month Phase 3 trial of MK-0616, an oral inhibitor of PCSK9 — the enzyme that degrades LDL receptors in the liver. Among 3,800 patients with established cardiovascular disease or familial hypercholesterolaemia who were already on maximally tolerated statin therapy, MK-0616 reduced LDL-cholesterol by 61% versus placebo, with data published in the New England Journal of Medicine. The drug is taken once daily as a tablet.

Current approved PCSK9 inhibitors — alirocumab (Praluent) and evolocumab (Repatha) — are injectable and expensive, limiting uptake even among high-risk patients. An oral option could substantially expand access. The safety profile in MACADAM-3 was consistent with earlier Phase 2 data, with no new safety signals and only rare liver-enzyme elevations. Merck has indicated it will file for regulatory approval with the FDA and EMA before the end of 2026. This is a Phase 3 result, not yet an approval; regulatory review typically takes six to twelve months.

Tuesday, 25 August: Europe Bans Junk-Food Advertising Aimed at Children Under 16

The European Commission formally adopted its Childhood Obesity Prevention Framework, the centrepiece of which is a ban on cross-border digital and broadcast advertising of foods high in sugar, saturated fat, or salt to audiences under 16. Member states must transpose the measure into national law by mid-2027. The framework also sets mandatory reformulation targets for school-meal suppliers — sugar in school lunches must fall 15% by 2028 — and requires clear front-of-pack nutritional labelling on ultra-processed foods.

Cyprus faces particular pressure to act: the European Health Report places its childhood overweight and obesity rate at approximately 38%, among the highest in the EU and above the Mediterranean regional average. The WHO European Office welcomed the move as a meaningful first step, while cautioning that advertising restrictions are one lever among many and that national action on school food environments and physical activity will be equally important.

Tuesday, 25 August: WHO Flags Drug-Resistant Gonorrhoea Cluster Across the Eastern Mediterranean

An interim report from WHO's Global Antimicrobial Resistance and Use Surveillance System (GASP) documents a cluster of extensively drug-resistant (XDR) Neisseria gonorrhoeae cases in Greece, Cyprus, Lebanon, and Türkiye. The strains are resistant to ceftriaxone — the current first-line injectable treatment — as well as azithromycin and ciprofloxacin, leaving clinicians with very limited options. The European Centre for Disease Prevention and Control (ECDC) has issued a rapid risk assessment and is working with national health authorities to strengthen contact-tracing capacity across the affected countries.

WHO's recommended response for confirmed XDR gonorrhoea cases points to zoliflodacin, a novel antibiotic developed specifically for drug-resistant gonorrhoea whose Phase 3 data were strong but whose EMA and FDA submissions remain under review. Clinicians in Cyprus are advised to send all gonorrhoea isolates for culture and susceptibility testing rather than relying on syndromic treatment, and to report confirmed XDR cases promptly to the Cyprus Ministry of Health.

Monday, 24 August: Microplastics Found in the Human Brain at Record Concentrations

A post-mortem study of 200 brain tissue samples, published in Nature Environmental Health, found microplastic particles in every sample examined — at concentrations roughly tenfold higher than a decade ago and higher than in any other organ studied, including liver and kidney. The samples, collected between 2020 and 2025, showed the highest concentrations in the prefrontal cortex and hippocampus. Particle sizes ranged from 10 to 500 micrometres; the most common polymer types were polyethylene and polystyrene.

The study is observational and post-mortem: it cannot establish whether microplastics cause neurological harm in living people or account for individual confounders. The authors call for prospective cohort studies linking plastic body burden to cognitive outcomes, and urge regulatory agencies to accelerate restrictions on single-use plastic food packaging. The National Institute of Environmental Health Sciences (NIEHS) described the findings as a strong signal that demands further investigation. For Mediterranean residents, where seafood consumption is high and plastic pollution in the eastern Mediterranean Sea is among the highest globally, the findings add urgency to ongoing dietary and environmental guidance.

Monday, 24 August: WHO and EMA Align on Autumn 2026 Combined COVID and Flu Booster

WHO's Strategic Advisory Group of Experts on Immunisation (SAGE) and the European Medicines Agency have issued joint guidance recommending that adults aged 60 and over, immunocompromised individuals, and healthcare workers receive both an updated COVID-19 booster (formulated against XBB.1.5/JN.1 descendants circulating this summer) and a quadrivalent seasonal influenza vaccine from September onwards, and that the two can be given at the same clinical visit. Multiple studies have confirmed that co-administration does not reduce immunogenicity to either vaccine.

The joint guidance is designed to simplify autumn vaccination campaigns for health systems still managing staff shortages and appointment backlogs. Cyprus's Ministry of Health typically launches its autumn vaccination programme in October; the EMA guidance gives national authorities flexibility on timing based on local epidemiology. Both vaccines remain available without charge to eligible groups across EU member states under existing procurement frameworks.

The health content above is journalistic in nature and is intended for general public information only. It does not constitute medical advice. Readers should consult a qualified healthcare professional before making any changes to their treatment, medication, or health routine.