Turning back cellular clocks, in an eye

From STAT's Jason Mast: Life Biosciences said yesterday that three glaucoma patients have now received a single injection of its experimental cellular reprogramming drug, with no serious adverse events.

Life’s drug is the first human test of an idea that has electrified scientists, biotech executives, and billionairess over the last decade: that giving a precise cocktail of proteins could rejuvenate cells, reverting them to a younger and healthier state. Academics have reported these cocktails can be used to extend the lives of mice. But there have been concerns the approach, if applied incorrectly, could cause cancer.

Life Bio aims to deliver a cocktail of three proteins — as opposed to the usual four — to minimize such risks. The company, presenting the data at the American Academy of Ophthalmology's annual meeting in New Orleans, said the injection was well-tolerated after 56 days, with early signs of efficacy in two patients. The company will now test a higher dose.

podcast

NIH funding uncertainty thwarts U.S. researchers

How has the National Institutes of Health been spending its budget? Why are biotech investors feeling gloomy? And what is the most quintessential Boston movie?

We discuss all that and more on this week's episode of “The Readout LOUD,” STAT's biotech podcast. We bring on STAT reporter Anil Oza and Heather Pierce, senior director of science policy at the Association of American Medical Colleges, to discuss the how the NIH distributed its money in the latest funding cycle and the effects on U.S. biomedical research.

We also chat about the shuttering of a CRISPR CAR-T biotech, the Nobel Prize in medicine, and biotech investors' current mood.

Listen here.

autoimmune disease

Argenx stumbles as Vyvgart fails Sjögren’s trial

Argenx hit an unusual speed bump this week, announcing that its blockbuster autoimmune drug Vyvgart failed a Phase 3 trial in Sjögren’s disease. The study was halted early after an independent monitoring committee determined it was unlikely to meet its primary endpoint, sending shares down 12%, STAT’s Jason Mast writes.

The setback is notable for a biotech that has enjoyed a remarkable string of clinical successes, and potentially costly: William Blair analysts had pegged peak annual sales in the indication at $1.4 billion. Still, analysts had warned that the trial was risky, given the notoriously high placebo responses among Sjögren’s patients. Argenx has other shots underway, however — as it announced at the same time that its Phase 2 celiac disease drug performed well.

opinion

Fifty years of IL-2 and unexpected discoveries

Fifty years after helping discover interleukin-2, pioneering virologist Robert Gallo reflects in a First Opinion essay for STAT on the scientific breakthroughs that followed — making a case for the importance of basic research.

The discovery of IL-2, a protein that allows T cells to grow, paved the way for identifying the first human retroviruses. This includes HTLV-1 and eventually HIV — but the work also laid the groundwork for modern cancer immunotherapy. None of that was the original goal, Gallo writes. His team was simply trying to figure out how to grow human blood cells in culture.

“IL-2 has led to many important milestones, but to me, its larger lesson is about the nature of scientific discovery,” Gallo writes. “The discoveries that will change medicine a generation from now are probably being chased today by people asking questions no one else sees the value of yet.”

Now, amid growing pressure to prioritize research with immediate clinical applications, Gallo argues that science's most consequential discoveries often emerge from questions whose practical value isn't yet apparent. As he puts it, you can't just fund the obvious.