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The cost of new gene-based sickle cell treatments isn’t the only barrier to access. Coming up with new ways to treat the whole disease—and person—could make treatment more equitable.

By Shobita Parthasarathy

Last fall, to great fanfare, US regulators approved two gene therapies for sickle cell disease, and the European Union and UK soon followed. Many people hope that these treatments will provide a “functional cure” for the genetic condition, which causes rigid, misshapen red blood cells that lead to anemia, episodes of extreme pain, blood vessel and organ damage, stroke risk and lower life expectancy. These sickle cell therapies also bring us closer to an age of “CRISPR medicine” in which new gene-editing tools could be used to fix a range of debilitating genetic diseases, including Duchenne muscular dystrophy and cancer.

“There is an urgent need for new methods for antibiotic discovery,” Dr. Luis Pedro Coelho, a computational biologist and author of a new study on the topic, said in a press release.

Coelho and team tapped into AI to speed up the whole process. Analyzing huge databases of genetic material from the environment, they uncovered nearly one million potential antibiotics.

The team synthesized 100 of these AI-discovered antibiotics in the lab. When tested against bacteria known to resist current drugs, they found 63 readily fought off infections inside a test tube. One worked especially well in a mouse model of skin disease, destroying a bacterial infection and allowing the skin to heal.

He added: “Their target: follistatin gene therapy. A pioneering technology with the potential to improve muscle and strength [and] slow the speed of aging and many more benefits.”

The millionaire explained that the procedure involves an injection in the stomach and in the buttocks.

It also costs $20,000, so not exactly cheap.

Scientists have achieved groundbreaking brain mapping using ultrasound, detailed in a May 2024 Science Translational Medicine paper. Led by Richard Andersen and Charles Liu, they developed a non-invasive method to monitor brain activity with unprecedented clarity. This innovation, employing an ‘acoustically transparent’ skull window, allows real-time observation of neuronal and blood flow dynamics. The technique promises new insights into brain function and potential advancements in treating neurological disorders, marking a transformative milestone in neuroscience.

The ability to communicate using only your thoughts might sound like the stuff of science fiction. But for people who don’t have the ability to speak or move due to injury or disease, there’s great hope that this may one day be possible using brain-computer interfaces (BCIs) that can “read” relevant brain signals and translate them into written or spoken words. A research team has made a preliminary advance in this direction by showing for the first time that a computerized brain implant can decode internal speech with minimal training.

In the new NIH-supported study, researchers implanted such a device in a brain area known to be important for representing spoken words called the supramarginal gyrus in two people with tetraplegia, a condition marked by full body paralysis from the neck down due to cervical spinal cord injury. The researchers found that the device could decode several words the participants “spoke” only in their minds. While we are far from using such a device to decode whole sentences or even phrases, and the exact mechanisms of internal speech are still under study, the findings, reported in Nature Human Behavior, are notable because it had been unclear whether the brain signals involved in thinking words could be reproducibly translated.

The findings come from a team led by Richard Andersen at the California Institute of Technology, Pasadena, CA, and Sarah Wandelt, now at the Feinstein Institutes for Medical Research in Manhasset, NY, and the study was supported by the NIH Brain Research Through Advancing Innovative Neurotechnologies® (BRAIN) Initiative Research Opportunities in Humans program. Though earlier research had shown that brain implants could decode vocalized, attempted, and mimed speech, it had yet to be seen whether internal speech could be similarly decoded.

Humanity is on the verge of entering a new era of space exploration, with the Artemis III mission planning to return humans to the moon in 2026, for the first time in 50 years. Not only will Artemis see a woman and a person of color walk on the lunar surface for the first time, but the week-long mission to the south pole of the moon will begin a period of longer and longer stays in space.

Study suggests heart rate may be a useful tool to determine where to stimulate the brains of individuals with depressive disorders when brain scans aren’t available.

New research suggests a common brain network exists between heart rate deceleration and depression. By evaluating data from 14 people with no depression symptoms, the team of researchers at Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, found stimulating some parts of the brain linked to depression with transcranial magnetic stimulation (TMS), also affected heart rate, suggesting clinicians may be able to target those areas without the use of brain scans that aren’t widely available. The findings were published recently in the journal Nature Mental Health.

Heart-Brain Coupling and TMS.