Tag: News Approved

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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.

Continue to STAT+ to read the full story…

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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.

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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. 

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Cognitive Training for Functioning in PTSD

Conditions: Posttraumatic Stress Disorder

Interventions: Behavioral: COGENT (Cognitive ENhancement Training); Behavioral: ST (Sham Training)

Sponsors: VA Office of Research and Development

Not yet recruiting
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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

Not yet recruiting
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Dissociation and PTSD in women with a history of childhood sexual abuse: a pilot examination of a specialized inpatient unit

BackgroundWorldwide, there are relatively few specialized inpatient units dedicated to women with histories of childhood sexual abuse (CSA) and comorbid psychiatric disorders. This pilot study examined dissociation and PTSD among women admitted to such a specialized integrative inpatient unit in Israel. We conduct an in-depth analysis of the role of dissociation in these women’s clinical picture, as well as in their treatment response.MethodsThe study included two phases. Phase 1 used a cross-sectional design to assess the complex inter-relationships between PTSD and various facets of dissociation in women with CSA histories admitted to a specialized inpatient unit (N = 108). Phase 2 focused on a sub-sample of participants (N = 28) who completed the inpatient program and completed admission and discharge assessments. Measures included the PTSD Checklist for DSM-5 (PCL-5) and the Dissociative Experiences Scale (DES-II).ResultsIn Phase 1, dissociative symptoms were positively correlated with overall PTSD severity and all PTSD symptom clusters, with the dissociative sub-measure of Absorption showing the strongest associations. In Phase 2, PTSD symptoms significantly decreased following treatment. In line with phase 1, reductions in Absorption were associated with improvements in overall PTSD severity and specifically in Hyperarousal symptoms.ConclusionsFindings from this pilot study indicate the therapeutic potential of a specialized integrated inpatient unit for women with CSA histories. Importantly, our results indicate that dissociation should be regarded as a major therapeutic target, most notably patients’ tendency for maladaptive absorption. These preliminary results should be expanded upon in larger, controlled clinical trials, further elucidating the role of dissociation and other mechanisms of change in similar units.
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STAT+: RFK Jr. plans to create list of injuries caused by Covid-19 vaccines

WASHINGTON — Health secretary Robert F. Kennedy Jr. is preparing to make it easier for people to claim that they were injured by a Covid-19 vaccine and receive compensation. 

Kennedy is set to start the process of compiling a list of injuries that are presumed to be caused by Covid shots. People with those conditions could then ask for compensation from the government. It’s not clear what conditions may make the list — and that’s something that outside experts are keeping a close eye on.

Kennedy has long been critical of vaccines, saying none have been adequately safety tested. He’s said that he plans to overhaul a similar but separate program that provides compensation for individuals who claim injury by a vaccine recommended by the federal government. 

Continue to STAT+ to read the full story…

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Virtual Patients Will Train Future Mental Health Clinicians

Researchers from the University of Pennsylvania and New York University have received a $4 million grant from the Wellcome Trust to develop an AI-driven platform to train mental health clinicians using simulations of real patients. 

Within the next two years, the partners will work on the development of the STELLAR platform, which stands for Steering-Vector Enhanced LLM Agents for Realistic Digital Twins in Mental Health. The platform will create digital twins of patients that trainees can use to practice conducting clinical interviews and evaluating psychiatric symptoms. 

“STELLAR brings together behavioral data, clinical expertise, and AI to ask a very practical question,” said Sharath Chandra Guntuku, PhD, associate professor of computer and information science at Penn Engineering. “Can we build training tools that better prepare clinicians for how varied and complex patients are?”

Preparing future mental health clinicians for clinical interviews can be challenging as patients will often report overlapping symptoms that shift over time and subjective experiences that can be expressed differently by each individual. STELLAR will give trainees an ethical option for trainees to simulate interviewing patients with a broad range of symptoms, backgrounds, and clinical scenarios.

“In psychiatry, the details of symptom experience matter: how someone describes distress, how symptoms overlap, how severity changes over time, and how context shapes the clinical interaction,” said Raquel E. Gur, MD, PhD, professor of psychiatry, neurology, and radiology at Penn’s Perelman School of Medicine.

Patient simulations will be created drawing from clinical data from the Philadelphia Neurodevelopmental Cohort, a repository including psychiatric assessments and clinical interviews from thousands of young people created by Penn Medicine and the Children’s Hospital of Philadelphia. Rather than copying individual patients, the simulations will create composites based on real-world data for clinicians to practice realistic conversations in the context of a clinical interview. 

This will allow trainers to precisely control the symptoms students encounter, their intensity, and how they interact with each other. For instance, a trainee may practice interviewing a patient with mild anxiety and another whose anxiety overlaps with depression or psychosis to learn how to distinguish the differences in presentation between both.

Because many mental health symptoms manifest beyond formal clinical settings, the platform will also be trained using data from social media platforms, where people discuss mental health symptoms in everyday language.

“Patient simulations will only be useful for clinician training if they are grounded in real clinical speech and evaluated as clinical interactions, not just plausible AI dialogue,” said Neville Ryant, PhD, researcher at Penn’s Linguistic Data Consortium. “[Our] role is to bring speech and language science into the core of the project: adapting speech-recognition tools to clinical interviews, creating high-quality transcripts and annotations, and helping evaluate both what the simulations say and how they say it. That includes assessing the language generated by the models, the naturalness of synthetic voices, how well those voices reflect target speech patterns, and the behavior of the avatar during real trainee interactions.”

To ensure the conversations are realistic, respectful, and useful to trainees, the team will involve people with lived experience of mental health conditions as well as family members and caregivers to provide their perspective into the evaluation process. Their feedback will help researchers assess the accuracy of simulations, avoid stereotyping patients, and prepare trainees for complex and nuanced clinical conversations with real patients. 

“The promise of this approach is that we can move beyond stylized and potentially biased simulations,” said João Sedoc, PhD, assistant professor of technology, operations and statistics at NYU’s Stern School of Business. “If we can create digital patients that simulate controllable plausible symptom expression and responsibly evaluate, we can augment current clinician training practices with the kinds of conversations that are essential to better mental health care.”

The post Virtual Patients Will Train Future Mental Health Clinicians appeared first on Inside Precision Medicine.