The US regulator approved the first drug for spinal muscular atrophy that works on the muscle itself

The rest of the day

9 more stories on this beat.

Each with its own sources. None of these is a link to the story above.

1. 02

   A lung cancer drug outlives Keytruda

   Akeso, a Chinese drugmaker, and its US partner Summit Therapeutics said people with lung cancer lived a median 30.8 months on their drug ivonescimab, against 22.6 months on Merck's Keytruda. [8] Keytruda is the immunotherapy that much of lung cancer care is built around. [8] Ivonescimab blocks two targets, PD-1 and VEGF, where Keytruda blocks one. [8] The trial, HARMONi-2, had already shown the drug held cancer back for longer, and the survival figures came out at a lung cancer conference in South Korea. [8]

   Why it matters — Analysts have doubted whether trials run in China carry over to patients elsewhere, and the trials that will decide approval outside China are still running. [8] One analyst noted that Merck's Sac-TMT cut the risk of death by 45% in a similar group, against 27% for ivonescimab. [8]

2. 03

   AstraZeneca's new breast pill fails a wider test

   AstraZeneca said on Friday that its pill Etcamah, also called camizestrant, failed a major trial called SERENA-4. [7] Given with a common drug to people newly diagnosed with advanced breast cancer, it did not hold the cancer back significantly longer than the standard pairing. [7] The US regulator had approved it earlier this month for patients whose tumours develop ESR1 mutations, which help cancer resist treatment. [7] AstraZeneca has not released the numbers. [7]

   Why it matters — The result could limit how widely the pill is used. [6] AstraZeneca executives had talked of it one day earning $5 billion a year. [7]

3. 04

   US health department pushes AI that prescribes

   The New York Times reported on Monday that the US health department is speeding up projects in which AI programs diagnose patients, offer therapy and prescribe medicine. [15] Some officials in the department worry the change is moving too fast, with too little evidence that it is safe and works. [15] Vinod Khosla, a billionaire investor whose son runs a health AI company, has been influential with top officials, people close to the matter said. [15] Those officials include Mehmet Oz, who runs Medicare and Medicaid, the US government's health insurance for older and poorer people. [15]

   Why it matters — Prescribing is the step where software stops helping a doctor with paperwork and starts choosing a patient's treatment. [15] Khosla told a start-up event in July that AI would replace human doctors. [15]

4. 05

   A blood test chose who got harder chemotherapy

   After colon cancer surgery, 135 patients at 11 hospitals in Italy and Spain had their blood tested for DNA shed by leftover tumour. [11] Patients with no tumour DNA got a single chemotherapy drug instead of the usual combination. [11] The 26% who had it got a stronger combination, stepped up again if the DNA stayed. [11] Three years on, 83% of those without tumour DNA were free of cancer, against 58% of those with it. [11] The results were published on Monday in Nature Cancer. [11]

   Why it matters — The trial missed its own goal: 88% of the test-negative patients stayed free of relapse at two years, below the 92% set in advance. [11] Against a matched group from an older trial, results were similar with much less nerve damage, and the authors say a proper randomised trial is still needed. [11]

5. 06

   Roche's lung cancer drug lands mid-pack

   MediLink, which licensed the drug to Roche for sale outside China, reported a final-stage trial of tam-peli in small cell lung cancer. [9] Tam-peli is an antibody that carries a cancer-killing drug to cells showing a protein called B7-H3. [9] In the trial, run in China, patients lived a median 13.3 months, against 9.4 months on the chemotherapy drug topotecan. [9] GSK's rival drug reported 18.5 months in its own trial, and Merck and Daiichi Sankyo's reported 12 months in a smaller, earlier-stage study. [9]

   Why it matters — Comparing separate trials run in different places can be unreliable, so the three companies are also competing on side effects. [9] Roche says it will start a worldwide final-stage trial quickly. [9]

6. 07

   Stool transplants calmed immunotherapy's gut damage

   Checkpoint inhibitors are cancer drugs that free the immune system to attack tumours. They commonly cause diarrhoea and colitis, an inflamed bowel. [12] At MD Anderson Cancer Center in Texas, 13 patients got a transplant of donor stool as the first treatment for it. [12] Ten improved, and among them the typical wait was a day and a half. [12] All 13 had stopped their cancer drug because of the gut damage, and some were able to restart it. [12]

   Why it matters — The study had no comparison group, and the authors say some patients might have recovered with ordinary care or by stopping the drug. [12] They want trials against steroids, the usual treatment, which dampen the immune system. [12]

7. 08

   Cellectis gives up on donor cell therapy

   Cellectis, a gene-editing company built on technology from France's Institut Pasteur, said it will seek partners for its two cell therapies for leukaemia and lymphoma and move into heart disease. [10] It had worked to make these therapies from donors' cells, as a simpler option than CAR-T treatments custom-made from each patient's own cells. [10] New medicines have changed how blood cancers are treated, leaving fewer patients for each therapy and slowing its trials. [10] It will now work on two heart-disease gene-editing treatments not yet tested in people. [10]

   Why it matters — Both heart treatments aim at targets, APOC3 and PCSK9, where approved medicines already exist. [10] That leaves Cellectis facing an uphill battle in a crowded field. [10]

8. 09

   A nose-spray bird flu vaccine protected mice

   Researchers built a vaccine for H5N1 bird flu on VSV, a virus that does not cause disease in people, by swapping in the bird flu virus's surface genes. [13] They changed the carrier's genes to make it safer and then gave mice two doses into the nose. [13] Every vaccinated mouse survived a dose of H5N1 that would otherwise have killed it. [13] A vaccine built on the same kind of carrier is already sold against Ebola. [13] The study was published on 9 September. [13]

   Why it matters — H5N1 bird flu is treated as a pandemic threat, and the nose spray raised defences in the lining of the airways as well as in the blood. [13] This is a mouse result, and most things that protect mice never reach people. [13]

9. 10

   Servier licenses an AI to guide cancer work

   Owkin, a company that builds AI for drug research, licensed its system K Pro to Servier, a French drugmaker, for cancer discovery and trial design. [14] The deal also opens Owkin's MOSAIC collection to Servier's scientists. [14] MOSAIC maps tumour samples by their molecules and by where each cell sits in the tissue. [14] The two companies first worked together in 2023, and Servier signed an $888 million cancer research deal with Insilico Medicine in January. [14]

   Why it matters — Servier says the value is having patient data and the analysis in one place. [14] Owkin has earlier signed deals with AstraZeneca, Merck and Sanofi. [14]