Lena Marshall
136 stories · Page 2 of 6
The Data on the First Sanfilippo Gene Therapy Were Ready Last Summer. The FDA Spent the Next Year on the Factory.
The FDA said Ultragenyx's clinical data were robust in July 2025, then rejected its Sanfilippo gene therapy over manufacturing questions. Approval came fourteen months later, for children who lose skills and die on a clock.
CancerYounger Generations Are Testing Biologically Older, and Early Cancer Is Tracking the Gap
By a nine-marker blood panel, Americans born in the 1990s scored biologically older than those born in the late 1960s did at the same age, and Washington University researchers found that accelerated aging tracked a higher risk of early-onset cancer.
Drugs & FDAThe FDA's New Pilot Lets a Drug Sponsor Choose the Institution Helping Build Its First-in-Human Application, and Reviews the Pieces as They Arrive
The agency will select 8 to 10 sponsor-QRI pairs to co-develop first-in-human trial applications it reviews on a rolling basis, and its stated reason is that China and Australia get there faster.
Brain & AgingSame Genes, Different Fold: A Fly Wing Regrows Only When Distant DNA Touches
Disturb the way the DNA folds after an injury, and the fruit fly still builds a normal wing but loses much of its power to repair one. The instructions to heal weren't only in the genes. They were in the shape.
CancerThe Women Low on Vitamin D Were Three Times as Likely to Wake From a Mastectomy in Serious Pain
Ninety-two breast cancer patients went into a mastectomy deficient in vitamin D; they were three times as likely to be in serious pain afterward and drew far more from the tramadol pump than the women whose levels were fine.
CancerThe Study Breaking Pancreatic Cancer's 'Shield' Was Co-Written by the Drug's Maker
The 'breakthrough' bought engineered mice eleven extra days, its headline human numbers come from a post-hoc subgroup, and one author is the chief scientist of the company that makes the drug.
Drugs & FDAThe FDA Approved a Breast Cancer Drug Its Own Advisers Had Just Rejected, 6 to 3
An FDA advisory panel voted 6 to 3 that AstraZeneca had not shown its breast cancer drug delivered a meaningful benefit. The agency approved it four months later anyway.
InvestigationsThe Escape Hatch in Trump's Drug Deal: 26 Companies Signed Their Way Out of the Price Cuts
Twenty-six drugmakers have signed voluntary White House agreements that exempt them from the proposed mandatory Medicare price cuts, and an analysis finds the deals could erase as much as 80 percent of the projected savings.
CancerMD Anderson Made the Case for Radiation Cleanup After Immunotherapy. Then Its Own Trial Undercut It.
In the oligometastatic subgroup the strategy was built for, small and underpowered, the patients who added radiation trended the wrong way: median survival of 42 months against 75.8 for the drugs alone.
CancerUCLA Can Grow Thousands of Cancer-Fighting Cell Doses in Six Weeks. Every Patient So Far Has Been a Mouse.
The lab put a $5,000-a-dose price on an off-the-shelf cancer therapy grown from cord blood that controlled tumors in mice with ovarian cancer and melanoma. No human has received it.
Drugs & FDAThe 'Bone-Building Switch' Is Back in the Headlines. It Was Tested in Mice, and Only Mice
A publicly funded Leipzig compound rebuilt bone in ovariectomized mice, 8 per treated group. More than a year later the same experiment is still being sold to readers as a way to keep their bones strong for life.
CancerSummit's $500 Million Lung Drug Split the Curve Patients Can't Feel
Ivonescimab delayed tumor progression by 2.4 months in EGFR-mutated lung cancer, but its overall-survival curve never cleared statistical significance and serious treatment-related adverse events nearly doubled.
Brain & AgingStatins and Dementia: A Benefit the Registries Keep Finding and the Trials Never Do
A Danish registry of 132,000 diabetics reports early statin use lowers dementia risk by 15 percent. The randomized trials that could confirm it, covering tens of thousands of patients, found nothing.
CancerThe Breast Cancer Cells That Hide From Chemotherapy Were Already There Before the First Dose
British researchers mapped where dormant breast-tumor cells hide, inside protective pockets of immune and connective cells, and found cells resembling therapy-resistant survivors already parked there before treatment begins.
CancerTwenty People, No Control Arm, and a Startup on the Side: Reading the Pancreatic Cancer Vaccine's Fine Print
Ninety percent of twenty volunteers made KRAS-specific T cells. None was shown to avoid cancer. Two of the investigators co-founded the company that licensed the shot from Johns Hopkins, which stands to collect royalties.
Drugs & FDAThe FDA Approved a Breast Cancer Drug Four Months After Its Advisers Voted No
On April 30 the FDA's oncology advisers voted 6 to 3 that AstraZeneca's camizestrant hadn't shown a clinically meaningful benefit. On September 4 the agency approved it anyway, conceding in its own approval that the benefit is "not yet confirmed."
CancerDecades of Cancer Warning Signs, and Still No Proof They Save Lives
England's cancer-awareness campaigns sent more people to the doctor for a decade. The reviews still can't show they caught a single cancer earlier or cut deaths, while the cancers that kill fastest stay silent until it's already too late.
Drugs & FDAChina Runs More Drug Trials Than America. The FDA Says It Can No Longer Trust Data From Sites It Can't Inspect.
China went from under 8 percent of the world's drug trials in 2010 to passing the United States by 2020. After three deaths in Chinese studies surfaced late, the FDA says it has sometimes been denied access to the very sites whose data underpins American approvals.
Nutrition & FoodBeef-Level B12 in Spirulina: The Study Measured the Tank, Not a Human
An Icelandic supplement maker's light-tuned spirulina tested at beef-level active B12. The reading was taken from the biomass in a reactor. No one has yet fed it to a person to see whether the body can absorb it.
Brain & AgingA Stanford Lab Deleted One Immune Receptor and Its Old Mice Barely Aged
Stanford says it found one big reason we age. The proof is a genetically engineered mouse, no such drug exists for people, and the senior author already holds patents on inhibiting the receptor she wants blocked.
InvestigationsOn the Study's Cleanest Test, the Doctors' $12 Billion AI Only Tied Google's Free Search Box
In June a Nature Medicine paper crowned general chatbots over the specialized AI that 40 percent of U.S. doctors use. Its biggest margins came from a public exam and a benchmark OpenAI built. On the one test made for the comparison, the expensive tool only matched a free Google box.
CancerHigh Blood Sugar Hands Cancer Cells a Cloak the Immune System Can't See Through
Grown in a fluid built to mimic the human body, cancer cells used excess glucose to thicken a sugar coating that hid them from immune cells, an effect that never showed up in the standard lab dishes used across cancer biology.
Drugs & FDAThe Pancreatic-Cancer Pill That Doubles Survival: $39,800 a Month, and a Fast Lane Congress Never Approved
Rasonque roughly doubles median survival in metastatic pancreatic cancer. Revolution Medicines priced it at $477,600 a year, more than double Keytruda, and the FDA cleared it early through a voucher program lawmakers say Congress never authorized.
InvestigationsGSK Bankrolled the Study That Turned Its Shingles Vaccine Into a Dementia Headline
The academics who best isolated a shingles-vaccine effect on dementia used a natural experiment and no industry money. The study behind this month's headlines used a claims database and GlaxoSmithKline's checkbook.