STAT+: 931 days. The drug approval scandal hiding in plain sight

This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox.

Northwest Biotherapeutics, a public biotech company developing a treatment for brain cancer, submitted a marketing application to U.K. regulators in late December 2023. The review was supposed to take 150 days, under an expedited regulatory pathway for drugs that address serious unmet medical needs.

As you’re reading this newsletter, two years, six months, and 18 days have elapsed without an approval decision. Why the extra-long delay? Regulators at the U.K’s Medicines and Healthcare products Regulatory Agency, or MHRA, won’t comment, telling anyone who inquires, including me, that it’s up to Northwest Bio to provide an update on its brain cancer treatment, called DCVax.

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STAT+: AstraZeneca, Ionis report major trial failure with heart disease drug

LONDON — In a blow to its cardiovascular aspirations, AstraZeneca said Thursday that its drug for a heart disease — one that has become an increasingly competitive target for biopharma companies — failed in a pivotal trial.

The drug, called Wainua, on which AstraZeneca is partnered with Ionis Pharmaceuticals, did not outperform placebo in reducing cardiovascular death and clinical events for patients with a condition known as ATTR-CM, or transthyretin-mediated amyloid cardiomyopathy. 

AstraZeneca’s U.S. shares were down some 8% in premarket trading, while its London-listed shares were down 9% early Thursday. Ionis shares, meanwhile, were down 12% in premarket hours.

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STAT+: The quest to save Grace — and clear the way for rare disease patients everywhere

SAN FRANCISCO — Matt Wilsey adjusted the plastic tube coming out of his 15-year-old daughter’s stomach and tried, again, not to think beyond the next 15 minutes. His job was to be there with Grace and let his wife, Kristen, rest. He could not think about the future. He could not wonder, again, if he had made the right choice or if his daughter would survive.

Three weeks before, Grace received a gene therapy meant to save her life and spare her further harm from NGLY1 deficiency, an ultra-rare genetic condition that came with a cascade of profound developmental challenges, preventing her from ever speaking or walking with ease. 

Wilsey fashioned the therapy himself.

He did not sit in the lab. He did not inject the rats or slice open their brains. But he had hired the scientists who did. He recruited advisers, including Nobel Prize winners, brought together the families of other children diagnosed with the condition, and pulled together an A-team of investors and donors. Through it all, he was sustained by his devout Catholic faith.

He came to accept that Grace would never live an independent life. But he hoped the drug would allow her to live longer, maybe even say a few words. And he believed the game plan he wrote might serve as a guide to curing hundreds of other rare diseases. 

“We carry the hopes of many,” he wrote to his staff once. “I’m not just talking about NGLY1 families. I receive emails, calls, and texts from professionals and other advocates. They are blown away by what we have accomplished and hope we are an ice breaker for them. Our trial has the potential to really boost / save a decimated field.”

Then the drug meant to save Grace’s life landed her back in the hospital, feebler than she had ever been. He sat beside his daughter, with her soft eyes and long braided hair, her face all puffed up, and prayed her condition would improve.

For a father and his sick child, it was a matter of life and death. But the entire pharmaceutical industry was watching, too.

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What happens on ‘MAHA Monday’?

The Great American State Fair in Washington wraps up this week. During its two-week run, the programming included two “MAHA Mondays,” which featured speakers and administration officials representing the Make America Healthy Again movement. 

STAT Washington correspondent Chelsea Cirruzzo and I stopped by the second MAHA Monday earlier this week. We spoke with Health and Human Services senior adviser Calley Means about the event and how health and wellness fits into the American identity. We also spoke with acting Surgeon General Stephanie Haridopolos about a recent surgeon general’s advisory on the harms of screen use.

Four nuclear reactors hit a big milestone in the US

I was really looking forward to July 4, and not just because I love a poolside barbecue. This year the American holiday also marked a big symbolic deadline for US nuclear power.

Last year the Trump administration set a goal to see three new microreactors achieve criticality, a technical milestone establishing that a reactor can sustain a chain reaction, by the nation’s 250th birthday. And just in time, four reactors did so.

It was a lofty goal, and seeing not just three but four companies meet it is certainly a positive sign for emerging nuclear technologies at a time when the world is facing increased need to increase electricity supply and address climate change with emissions-free technologies.

But achieving criticality doesn’t mean a reactor is ready to provide electricity for the grid (or at all, for that matter). Let’s untangle what this program’s success could mean for nuclear power in the US, and where these companies might go from here.

The Reactor Pilot Program essentially opened a special door for prototype reactors to fast-track development. In August, the US Department of Energy selected 11 reactor projects for the program and offered them land and support from the national labs system. These are all microreactors; the large light-water reactors that dominate the grid today are tens or even hundreds of times their size. 

Antares Nuclear was the first to achieve criticality, reaching the milestone in June in its Mark-0 test reactor. Reactors from Valar Atomics, Deployable Energy, and Aalo Atomics followed. (Aalo hit the mark in the early hours of July 4—an inspiring example of just barely meeting a deadline.)

The speed with which these companies hit this milestone is impressive, especially in an industry known for massive projects that frequently blow past deadlines and stated budgets. (Valar, Antares, and Aalo were all founded in 2023, and Deployable started in 2025.) But reaching criticality and running a reactor that can produce electricity are two totally different things.

All these reactors reached what’s called zero-power criticality. Basically, it’s a test of whether you can start a nuclear chain reaction, with no meaningful power coming from the reactor. “A zero-power-criticality test can be achieved without making real engineering progress on fuel or design,” Kathryn Huff, a former assistant secretary for nuclear energy and chair of the Department of Nuclear Engineering and Engineering Physics of the University of Wisconsin–Madison, said on an episode of the Catalyst podcast earlier this year.

Now, with the completion of this program, the companies will need to continue their work to make power, which could involve some big technical challenges. In some cases they’ll need to add significant equipment, like the cooling systems to transfer the heat out of the reactor core.

The companies are projecting aggressive timelines moving forward. Aalo says it’s already begun work on the second reactor and plans to produce 10 megawatts of electricity to power an on-site data center in 2027. Deployable Energy says it plans to deploy commercial reactors by 2028

I tend to take timelines from startups, especially in nuclear, with a grain of salt. Not only are these remarkably complex technical machines, but companies often run into problems outside their own control, like regulatory challenges—which these new projects could soon face. 

The Nuclear Regulatory Commission is in charge of civilian and commercial nuclear use in the US, and historically, the process to get nuclear reactors approved has been quite slow.

The agency did propose a new framework for microreactor approvals earlier this year, which is designed to speed up the process—but it’s yet to be seen how quickly things will move. (And it’s worth noting here that some nuclear experts have questioned whether the agency under the Trump administration is loosening nuclear rules too much.)

Some nuclear supporters aren’t applauding the microreactor milestone. Federal focus on the program is an “unhelpful diversion” from goals to meaningfully increase nuclear capacity, according to one analysis by Third Way, a public policy think tank. “Artificially accelerating project timelines is a short-term solution, not a long-term fix,” the memo reads. 

Criticality is a big first step, but a lot will still have to happen for any of these microreactors to come online, much less for these small reactors to be a significant source of electricity for the grid. 

This article is from The Spark, MIT Technology Review’s weekly climate newsletter. To receive it in your inbox every Wednesday, sign up here

Biasing Food Value Construction

Conditions: Anorexia Nervosa

Interventions: Behavioral: Training food attributes

Sponsors: University of Chicago; National Institute of Mental Health (NIMH)

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Implementing DBT in Short-Term SUD-treatment

Conditions: Substance Abuse Disorder; Dialectical Behavior Therapy; Emotion Management Skills

Interventions: Behavioral: DBT-informed milieu therapy; Behavioral: Treatment as usual

Sponsors: Helse Møre og Romsdal HF

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