The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene editing, make it possible to repair almost any defect in the genetic code. At the same time, there is a clear rationale for intervening before birth, Tippi MacKenzie, professor of surgery at UCSF’s School of Medicine, told attendees of the third International Human Genome Editing Conference in London on March 7.

Whether as primary tumors or metastases, brain tumors remain stubbornly intractable to the progress that has occurred in many other tumor types. As Igor Vivanco, who is a senior lecturer in the Institute of Pharmaceutical Science at King’s College London, noted in his talk at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) meeting in Paris this week, the last win in glioblastoma was the addition of temozolomide to the radiotherapy standard of care in 2005. And temozolomide’s benefit is measured in months, not years.

Performing experiments and potentially manufacturing products in space offers some unique advantages in a near-zero gravity environment. Space changes buoyancy, hydrostatic pressure and convective heat flow. Researchers are studying how those changes affect cells, but also looking to take advantage of the changes to create products in manufacturing processes that wouldn’t be possible on earth.

HIV research is a winding road where one obstacle leads to another, slowing down success. The first barrier to getting the cure starts before one can even talk about it. “Cure may be too powerful and promising a term. Remission is probably better,” said John Mellors, whose work led to the universal use of plasma HIV-1 RNA and CD4+ T-cell counts in HIV-1 infection.

“Cure means maintaining an undetectable viral load off antiretroviral treatment. That means you cannot transmit it to people. Within that definition, there are people that have complete eradication of every single virus. And then, you have people that have a low level of virus that are able to keep under control without drugs,” Sharon Lewin told BioWorld. “Remission is maintaining a viral load less than 50 copies per milliliter in the absence of any retroviral. But there is still virus detectable,” she explained. Lewin is the director of The Peter Doherty Institute for Infection and Immunity in Melbourne, and the president of the International AIDS Society (IAS).

In the larger picture, the fight against HIV has been a triumph of modern medicine. A patient diagnosed with HIV in the 1980s had a remaining life expectancy of 1 to 2 years. In 2023, they can expect to live another half century. But so far, an HIV vaccine has remained elusive. In the newest phase III failure, Janssen Pharmaceutical Cos. of Johnson & Johnson closed down its Mosaico trial more than a year ahead of schedule, following a data and safety monitoring board’s (DSMB) report saying the study was not expected to hit its primary endpoint.

Psychedelic drugs may exert their effects at intracellular serotonin receptors that serotonin itself, which does not cross cell membranes, cannot reach. The findings were published in the Feb. 17, 2023, issue of Science by researchers from the University of California at Davis. An accompanying editorial by Evan Hess and Todd Gould at the University of Maryland School of Medicine called them “a key achievement in the understanding of the mechanism of action of psychedelics” and “an important step forward for a rapidly ex­panding and much-needed field of study.”

Research led by Indiana University School of Medicine and the University of Notre Dame shows a new treatment for peanut allergy is effective in a mouse model. The therapy, a covalent heterobivalent inhibitor, differs from most allergy treatments in that it is more of a preventative therapy rather than a drug to treat immediate acute symptoms. “Essentially, in the model, we can treat once and then the mice seem to be protected for several weeks from challenge with peanut,” lead researcher Mark Kaplan, a professor at Indiana University School of Medicine, told BioWorld.

Metabolic health is at an odd juncture. With the advent of glucagon-like peptide (GLP-1) agonists, pharmacologically induced weight loss has matured into a viable therapeutic option at long last. GLP-1R agonists, which are also called incretin mimetics and GLP-1 analogs, are likely to continue their success across multiple areas of medical care. Already, the class has transformed diabetes care, making a splash in weight management, and it may yet do the same for other indications.

Researchers at Stanford University School of Medicine have developed a method to measure several thousand metabolites, including proteins, metabolites, inflammatory markers such as cytokines and, to a degree, lipids. “It’s like Theranos, except it works,” corresponding author Michael Snyder, director of the Center for Genomics and Personalized Medicine at Stanford Medicine, told BioWorld.

A psychiatric disorder rarely comes alone. More than half of all individuals who meet the diagnostic criteria for any psychiatric disorder are diagnosed with more than one condition. That high degree of comorbidity is often viewed as a consequence of the heterogeneity of psychiatric disorders – and as evidence that psychiatric diagnoses poorly reflect the underlying brain biology. Data published in Nature Human Behaviour on Jan. 12, 2023, has identified another likely contributor to the high degree of overlap between different psychiatric disorders.