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A Cancer Therapy Is Being Aimed at Lupus. Nine Patients In, the Early Signal Is Real and Very Small
Researchers reported results this summer from a trial of CAR T-cell therapy in severe lupus, using a cell therapy originally developed for leukemia. Nine patients received a single infusion of engineered cells targeting CD19, the marker carried by the
A Blood Test for Alzheimer's Has Reached the Family Doctor. Its Real Value Is the Answer Nobody Expects
Blood tests that detect the protein changes of Alzheimer's disease have moved out of research settings and into ordinary clinical use, and the study that matters most is the one that put them in front of family doctors
The Warning Label on Hormone Therapy Changed This Year. The Underlying Decision Did Not Get Simpler
In February the FDA approved labeling changes for menopausal hormone therapy products, removing the boxed warnings about cardiovascular disease, breast cancer and probable dementia that had sat at the top of those labels for more than two decades. Six products
The First Real Test of Lowering Lipoprotein(a) Has Reported, and It Did Not Go the Way the Field Expected
Novartis released topline results this month from Lp(a)HORIZON, the first large outcomes trial of a drug designed to lower lipoprotein(a). The trial enrolled 8,323 people with elevated Lp(a) and established cardiovascular disease, and it did
The Weight Number Was Never the Point. A New Trial Puts the Argument on the Record
A phase 2 trial published this year in Nature Medicine tested what happens when a muscle-preserving antibody is added to a GLP-1 drug. In 507 adults with obesity, the combination produced more total weight loss than the GLP-
The Glucose Monitor on Your Arm Is Producing Data Nobody Has Shown How to Use
A review of continuous glucose monitoring published this year in JAMA Internal Medicine reached a conclusion that sits awkwardly against how these devices are now marketed. In people who do not have diabetes, the authors found no scientific evidence that
Cellular Reprogramming Reached Its First Patients This Year. It Started in the Eye
In June, a company working on epigenetic reprogramming dosed the first patient in a phase 1 trial of a therapy called ER-100, aimed at two optic nerve conditions: open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy. The
A New Gene Therapy Approval Shows What Accelerated Pathways Actually Ask For
In August the FDA granted accelerated approval to a one-time gene therapy for glycogen storage disease type Ia, a rare inherited disorder in which the body cannot release stored glucose properly and patients depend on carefully timed doses of
Federal PFAS Water Rules Are Shifting. The Household Layer Is Unchanged
In May, the EPA proposed two changes to the federal drinking water rule for PFAS compounds. One would extend the compliance deadline for the limits on PFOA and PFOS by two years, to 2031, for eligible water systems. The other
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