Medical progress this week
Top 5 advances: 19th to 25th May
Every week there are incremental steps forward in our understanding and treatment of disease. Here are my top 5 picks for this week.
An eosinophil-lowering antibody for COPD (FDA-approval)
A new antifibrotic slows progression of lung fibrosis (Phase 3 trial)
The world’s first bladder transplant (First in human)
An antisense oligonucleotide in a subtype of ALS (First in human)
Towards malaria eradication with anti-parasite nets (Pre-clinical)
1. An eosinophil-lowering antibody for COPD (FDA-approval)
The background
COPD is the most common lung condition - it’s usually caused by smoking or air pollution
It’s the 4th leading cause of death worldwide, of any condition
~20–40 % of people with COPD have high eosinophil levels, driven largely by interleukin-5 (IL-5)
The advance
In a phase 3 trial, the IL5-blocking antibody, Mepolizumab, cut COPD exacerbations and hospitilisations - when added to standard-of-care (triple therapy), in the subset of patients with high eosinophils
Safety profile pretty much matched placebo
It was FDA-approved this week - it joins the anti-IL4R antibody, Dupilumab, which is also approved for eosinophilic COPD
The limitations
There was no improvement to quality-of-life or lung function
Only patients with high eosinophils benefit
The future
No treatments can halt or reverse the structural lung damage in COPD - yet
Prevention beats treatment
Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2413181
2. A new antifibrotic slows progression of lung fibrosis
The background
Idiopathic pulmonary fibrosis is scarring of the lungs with no clear cause (it’s ‘idiopathic’)
There are two approved antifibrotics medications: nintedanib and pirfenidone
But fibrosis usually progresses despite these treatments
The advance
Nerandomilast is a PDE4-B inhibitor -it’s anti-inflammatory and anti-fibrotic
This is a phase 3 trial - Nerandomilast slowed the decline in lung function
A comparable trial for a different subtype of pulmonary fibrosis (progressive pulmonary fibrosis) had similar positive results
The main side-effect was diarrhoea
The limitations
Whether this reduces hospitilisation/death isn’t yet known
It slowed but did not reverse the decline in lung function
The future
Regulatory filings expected in 2025
Better treatments to prevent or reverse fibrosis needed
Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2414108
3. The world’s first bladder transplant
The background
A patient in California had most of their bladder removed by surgery for cancer
This would typically be managed by an ileal-conduit (where urine passes through a repurposed part of the small intestine into a stoma), but this carries risks
The advance
After an 8 hour operation - a combined kidney and bladder transplant - urine flowed immediately and kidney function improved
It’s only been 3 weeks since the surgery but the patient’s doing well - it’s the first time he can pee in 7 years!
The limitations
It’s a complex surgery
There’s a limited supply of donors
It requires lifelong immunosuppression
The future
Four more transplants are planned as part of a clinical trial
Gene-edited or decellularised bladders are being explored to avoid rejection risk
Source: https://www.nytimes.com/2025/05/18/health/bladder-transplant-human.html
4. An antisense oligonucleotide in a subtype of ALS
The background
The mutant protein accumulates as aggregates and causes death of motor neurons
Inherited mutations in the FUS gene cause ~1–3 % of ALS
Symptoms usually start in teenage years or 20s
There are no approved treatments
The advance
This is a small, single-arm trial of monthly intrathecal injections with Jacifusen - an antisense oligonucleotide that triggers degradation of the FUS mRNA
This reduced death of motor neurons (measured by CSF NfL)
And seemed to stabilise or even improved function in a few participants when started early
The limitations
It’s an open-label study, small, and uncontrolled
Most patients continued to decline despite the therapy
It requires repeated lumbar-puncture dosing
The future
A global phase 1–3 double-blind RCT is underway to further explore safety and efficacy
Source: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)00513-6/fulltext
5. Towards malaria eradication with anti-parasite nets
The background
Malaria deaths have halved since the year 2000, but progress is slowing
Insecticide-treated nets are an effective measure against malaria, but mosquitoes are evolving resistance to the insecticides
Targeting the parasite rather than the mosquito itself might be an alternate option
The advance
Drugs targeting two independent sites in the same essential malaria parasite protein were embedded into nets
All (100%) of the parasites in mosquitos that came into contact with the anti-parasite-coated nets were killed
Even in insecticide-resistant mosquitoes
The limitations
No data from the field yet (no evidence that this can actually reduces malaria infections)
The future
45 countries have already eliminated malaria
Combined with the other anti-malarial therapies, including emerging vaccines, can malaria be eradicated worldwide?
Source: https://www.nature.com/articles/s41586-025-09039-2