Radically different
Nature Neuroscience, Published online: 04 June 2026; doi:10.1038/s41593-026-02338-5
Androgens — hormones that are generally present at higher levels in males than females — regulate intestinal transit, but their cellular targets and mechanisms of action are unclear. We identify the neurons that mediate androgen-dependent gut motility and reveal that androgen reactivation by a bacterial enzyme in the gut lumen is necessary for this vital neuroendocrine axis.
Americans who have high-risk exposures to Ebola in the current outbreak in Central Africa will have access to an antibody treatment that has shown great promise in animal testing but hasn’t yet undergone a clinical trial to show whether it is efficacious in people, the Department of Health and Human Services confirmed Thursday.
The antibody treatment, known as MBP-134, is made by San Diego-based Mapp Biopharmaceuticals, with funding from the Biomedical Advanced Research and Development Authority, an agency within HHS that helps develop medical countermeasures for rare and emerging diseases, and biological threats.
WASHINGTON — Leaders at the Food and Drug Administration on Thursday listened to criticisms and recommendations for how to move forward with a speedy drug review program put in place by former FDA commissioner Marty Makary.
The listening session, held on the FDA’s White Oak Campus, featured 17 speakers representing patient groups, drug companies, and academic organizations. Some had positive feedback, particularly those whose drugs have already been approved through the program. But most asked the agency to pause the program, and then bring it back through normal regulatory procedures that require public feedback.
Makary launched the Commissioner’s National Priority Voucher program about a year ago, offering one- to two-month FDA reviews to companies that could prove their drugs “align with national priorities.” The priorities, which included addressing health crises and delivering innovative cures, were vague. Critics worried the process was vulnerable to political interference.
Why did oncologists give a standing ovation to a data presentation on Revolution Medicines’ pancreatic cancer drug, daraxonrasib? Why did biotech stocks perform so badly this week? And are concrete beaches better than normal beaches?
We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast.
Otsuka’s Voyxact slowed the loss of kidney function after one year in patients with a chronic autoimmune kidney disease, but the benefit was less than expected and left room for competing treatments to perform better.
In a Phase 3 study, patients with IgA nephropathy, or IgAN, who received injections of Voyxact saw their kidneys lose function at an annualized rate of 3 points over one year compared to an annualized function loss of 7.6 points over one year for patients receiving a placebo, the Japanese drugmaker reported Thursday.
Kidney function was assessed with a lab test, called eGFR, that measures how well kidneys filter waste from the blood.
Changes in the brains of babies shortly after birth could help explain why people go on to develop Parkinson’s disease (PD) and why this is particularly common in people with autism spectrum disorder (ASD).
The findings, in Science Advances, suggest a reason why humans are vulnerable to neurodegenerative diseases in a way that is not seen among other primates.
Researchers were surprised to find increased expression in genes associated with PD among humans that was not found in chimps or rhesus monkeys.
Human changes in regulatory DNA operated across two cell lineages, one linked with autism and the other with PD.
“Patients with ASD are also more likely to develop PD, a link that is currently not understood,” commented the researchers, led by Menno Creyghton, PhD, from Erasmus University Medical Center in the Netherlands.
“Our analysis linking the two diseases through a common network could provide insight into common biology between the two diseases.”
Human brains have several differences to those of primates, including a larger volume, more projections between outermost areas, and greater complexity of neurons in the upper layer.
The researchers note that, soon after birth, babies experience protracted brain development that coincides with periods of extensive synaptic modeling.
To investigate differences with our close evolutionary relatives, they conducted a single-cell transcriptomic and epigenomic analysis of human, rhesus macaque, and chimpanzee brain tissue during early postnatal development.
Through this, the team identified human infant–specific programs and cell states that were not observed in adults and correlated these early life processes with susceptibility to PD in aged neurons.
Specifically, Creyghton and co-workers identified human gene expression changes in immature oligodendrocytes enriched in both ASD risk genes and gene expression losses in patients with ASD.
There were also gene expression gains in human babies enriched in PD risk genes for and among genes dysregulated in PD patients.
Both transcriptional programs were part of a core network that contained human-specific sequence changes in regulatory DNA. This lacked cell lineage specificity, operating in both the oligodendrocyte and neuronal lineages, with different downstream targets for each.
In the oligodendrocyte lineage, the downstream target genes were overrepresented in autism risk genes whereas in the neural lineage, PD risk genes were enriched.
“It is therefore possible that these pathologies could be maintained in the human lineage by complex antagonistic interactions across developmental stages,” the authors speculated.
They noted the “unexpected” correlation between human evolution and Parkinson’s disease.
“It has been suggested that humans are exceptionally vulnerable to neurodegenerative diseases including PD as similar disease pathology has not clearly been observed in other primate species,” the team pointed out.
“We show that the human infant–specific expression changes that are overrepresented in PD-deregulated genes are enriched in synaptic developmental genes.
“Imbalances in synaptic function can strongly affect neural vulnerabilities, especially in larger projection neurons. As such, pathological deregulation of such a program may play a role in PD susceptibility.”
The post Infant Brain Development Sheds Light on Parkinson’s Link with Autism appeared first on Inside Precision Medicine.
The Supreme Court unanimously ruled that Hikma Pharmaceuticals did not infringe patents held by Amarin in a decision that makes generic drugmakers less vulnerable to lawsuits over so-called skinny labels.
The ruling overturned a lower court decision that sided with Amarin. Generic drugmakers had argued that, if the Supreme Court also ruled in favor of Amarin, they would be discouraged from making and selling lower-cost versions of brand-name medicines, which would maintain higher prices for prescription drugs.
At issue is skinny labeling, which refers to moves by generic companies that seek regulatory approval to market a medicine for a specific use, but not other patented uses for which a brand-name drug is prescribed. For instance, a generic drug could be marketed to treat one type of heart problem but not another. In doing so, the generic company seeks to avoid lawsuits claiming patent infringement.