Biotech & Longevity

The US regulator cleared a lung cancer pill for untreated patients, on 69 people and no comparison group →

Sevabertinib was already allowed for lung cancer that had grown through other treatment. On 9 September the US drug regulator allowed it as the first treatment, on a single-arm trial of 69 patients in which 75% of tumours shrank.

The US drug regulator stopped new patients joining an epilepsy drug trial over a chemical found in rats. The drug was licensed for $795m nine days earlier. →

A substance rats made while breaking down Biohaven's epilepsy drug was enough to pause enrolment, and nobody can yet say whether it matters in people. Across the day, four more decisions were taken on evidence that has not settled.

A gene editor rewrote every copy of a cholesterol gene in human embryos. Delivered a second way, it stopped them developing. →

A team at Columbia University changed one DNA letter in human embryos without cutting the strand, and the embryos grew on to the stage just before implantation. Sending the same tool in as instructions instead of as a finished protein arrested many of them, and the authors say none of this can be used in reproduction.

A drug for the world's third-biggest killer cut attacks by 30%, and it was tested in the patients the other new drugs leave out →

AstraZeneca said tozorakimab cut sudden worsenings of COPD by about 30% over a year in two final-stage trials. The number matters less than who was in them: the trials enrolled people who cannot take the other new lung drugs, and that group is where the money is.

A pig kidney kept a man off dialysis for 271 days, then came out so a human one could go in →

Tim Andrews lived nine months on a kidney from a gene-edited pig, the longest anyone has managed on an animal organ. It failed, was removed, and he then took a human kidney that is still working - which is the result the field had been waiting for.

Dengue has reached all 64 districts of Bangladesh, and one month could put 30,000 people in hospital →

Bangladesh's dengue outbreak is speeding up in hospitals already full of measles patients, and a researcher there says spraying cannot work while the breeding sites are left alone. Plus 29 more stories from the day in medicine and biology.

A mushroom compound given before chemotherapy prevented its worst lasting side effect. In mice. →

Most people on platinum chemotherapy end up with burning, numb hands and feet, and nothing treats it once it arrives. Two doses of psilocybin beforehand stopped it completely in mice, without weakening the chemotherapy, and human trials start this month.

Novartis lowered the blood particle that genes tie to heart attacks. In 8,323 people, the heart attacks did not fall. →

Pelacarsen cut lipoprotein(a), a fatty particle set largely at birth and long treated as a proven cause of heart disease. The people taking it had no less cardiovascular risk than the rest. Two rival drugs that cut the particle far deeper are still being tested.

These drugs block nothing. They hold two proteins together and let the cell do the rest. →

The first medicine that destroys a protein instead of blocking it was approved this year, and Johnson & Johnson paid $3.05bn for a company with no approved drug at all.

Old mice on a weight-loss drug lived 12% longer. Mice simply fed less got most of the same. →

Researchers in the United States gave semaglutide to healthy mice at an age close to a 60-year-old person. The treated mice lived a median 834 days against 742. Mice fed exactly as little as the drugged ones chose to eat lived about as long -- but did worse on memory and blood sugar.

Two people died of measles in Pennsylvania. The US national count still says zero. →

The new head of the US disease agency ordered her staff to challenge a state's finding, breaking a rule that has stood for decades. The agency has no investigators at either bedside, so its review can only add the deaths or leave them out.

Denmark offered half a million adults an RSV shot at random, then counted the hospital beds →

A trial of 511,535 people found the vaccine cut hospital stays from one winter virus by 70%. It also found that total hospital stays and total deaths barely moved.

Eight drugs had failed them. A cancer therapy left three of six needing none. →

Doctors in Berlin gave six people with severe rheumatoid arthritis a treatment designed for blood cancer. Every one improved, and three ended up on no arthritis medicine at all. Plus measles deaths in Pennsylvania, a one-shot cholesterol edit, and the day's other biotech news.

A heart drug failed because most patients were already on one that works →

AstraZeneca and Ionis explained a 1,400-person failure on Friday. Eight in ten volunteers were already taking a rival drug, and there was little harm left to prevent.

A patch of muscle under the skin, attached to nothing, did much of what exercise does →

Mice given a self-contracting graft of their own muscle cells gained lean mass, bone density, grip and stamina - and the graft moves nothing. Plus 37 more stories from the beat's day.

The first drug to lengthen life in pancreatic cancer is approved, at $39,800 a month →

The FDA cleared Rasonque six and a half months early for a cancer that had almost nothing to offer. Two thousand people were already taking it free.

A gene therapy halted over spots no child can feel, in a week medicine spent looking harder →

The FDA has paused Regenxbio's Hunter syndrome gene therapy after extra scans found small masses on the spines of five treated boys. None of them has symptoms, and nobody has ever routinely scanned the spines of untreated children with this disease - so there is nothing to read the finding against.

The first blind test of AI antibody design, and the week biology checked its own tools →

Twenty-nine organisations committed 511 AI-designed antibodies before a lab made a single one. On the ranking task all but one model lost to picking clones at random - and days later a sleuth found 54 ageing papers built on an antibody that binds the wrong organism.

The immune cell that multiplies after 100, in a week spent at the far edge of life →

A Japanese team found killer immune cells growing, not shrinking, in people past 110 - a week when longevity science, ageing clocks and the rarest-disease drugs all came down to how few people you can actually study.

The gene therapy that fixes a child's liver and leaves the family history alone →

A one-time infusion reaches children with an inherited sugar disorder, two children die in gene-editing trials abroad, and a new study finds a mother's age leaves a mark that is not written in DNA.

A cancer vaccine finally clears a phase 3, in a week of four approvals →

Merck and Moderna say a personalised mRNA shot kept melanoma from coming back in a 1,137-patient trial. Four FDA clearances landed alongside it, two gene-therapy deaths surfaced in China, and a quieter thread ran through the week: the handedness of a molecule.

One eye chart, a billion dollars: the week the yardstick decided everything →

EyePoint lost nearly a billion dollars in an hour because a vision drug missed on the measure the trial had picked, while winning on the one patients complain about. It was that kind of week: an approval built on a brand-new stand-in for remission, a Duchenne filing rewritten around a different body part, and one company that measured the emergencies themselves.

The switch, not the gene: a week spent on what a cell is allowed to read →

A company raised $90m for a drug that silences a gene without cutting DNA, and four journals reported on cells locked into the wrong state. Plus a big lung-cancer failure and two undisclosed deaths in China.

The deadliest Ebola outbreak on record, and a vaccine built for the wrong virus →

Congo's outbreak is on course to pass 2014-16 as the worst ever, against a species with no vaccine and no drug. Elsewhere: engineered cancer-fighting cells still alive ten years on, the first whole-body treatment tested for vitiligo, and blood immune cells found moving into the ageing brain.

A new class of cancer drug wins approval by changing a protein's shape →

The FDA cleared iberdomide for multiple myeloma on 13 August, the first of a class that works by reshaping a protein's surface rather than blocking it. The same week brought a map of what else that trick could reach, a cystic fibrosis pill that failed to fix a broken fold, and fresh work on the folds that go wrong in the brain.

One dose, twelve weeks: an LSD-based drug clears a second phase 3 for anxiety →

A 214-patient trial found a single psychoactive session still beating placebo on anxiety scores three months later - the first real movement in anxiety drugs since 2007. Much of the rest of the week was an argument about the opposite question: how long a drug should be taken, and what a body does to itself while it waits.

A vaccine order lands as measles hits a 35-year high, and the math of the crowd →

Trump ordered fewer childhood shots and the MMR vaccine split into three, as US measles reached its worst level since 1991 - a week where the science of collective protection ran straight into policy.

A deafness gene therapy reaches a child, in a week ruled by the delivery problem →

Getting a working drug to its target, past the body's own barriers, is often harder than inventing it - and this week's biotech news kept proving the point.

A blood test that hunts 50 cancers reaches the FDA, and the math that decides what a positive means →

US regulators will review Grail's Galleri, a single-draw blood test for more than 50 cancers, on September 23. It is 99.6% accurate at clearing the healthy, yet a big UK trial found it did not catch cancer earlier overall, because when a disease is rare, even a near-perfect test throws off false alarms.

The weight-loss pill arrives, and it rewrites how often you dose →

Britain became the first country in Europe to clear a once-daily obesity pill that rivals the weekly injections, as a busy week added a narcolepsy drug, the first mRNA flu shot, and a self-copying melanoma virus.

The gene that decides who suffers side effects from antidepressants →

A 114,000-person study found one liver-enzyme gene predicts who gets sick on common antidepressants, sharpening a bigger truth this week: the same gene can help one person and harm another depending on the setting it sits in.

A diabetes drug keeps protecting organs it was never aimed at →

New pooled trial data shows semaglutide guards the kidney, on top of the heart and the waistline. It is the reach of a drug that hits one receptor the body keeps in many places at once.

A living cancer drug: the FDA clears an engineered virus that copies itself inside tumors →

After two rejections, regulators approved Replimune's Tudriqev, a virus reengineered to multiply inside melanoma and burst it from within - part of a two-approval week that also cleared the first mRNA flu shot.

A vaccine for inherited cancer risk, and a week that redrew who's really at risk →

Britain began the first trial of an mRNA vaccine to prevent cancer in people born with Lynch syndrome, while two other studies reshaped who counts as genetically at risk.