Antibody Promotes Nerve Regeneration in Spinal Cord Injury Patients

Swiss research shows treatment with an antibody known as NG101 promotes regeneration of damaged spinal cord tissue in some people with spinal cord injury by blocking a protein called Nogo-A that normally suppresses nerve regrowth in the central nervous system.

Compared to placebo, participants treated with the antibody showed faster lesion volume shrinkage and a significantly slower loss of spinal cord tissue, both in terms of size and myelin content, above the injury site.

This study, published in Nature Communications, is an additional analysis of a subset of 106 patients of a Phase IIb study published last year in The Lancet Neurology and carried out by Swiss and German academic researchers as well as the Swiss biotech NovaGo Therapeutics, which is developing the antibody. The original trial population included 126 people.  It did not robustly meet its primary endpoint across the full patient population but showed promising signals in some motor-incomplete patients.

The current study specifically looked at people for whom magnetic resonance imaging (MRI) data were available. It investigated whether NG101 produced measurable structural changes in the spinal cord over time, whether MRI could detect those changes even in patients without obvious clinical improvement, also whether combining imaging with electrophysiology could better identify likely responders to inform more efficient future trial designs.

In the Phase IIb study the antibody was delivered directly into the spinal canal to 106 participants with acute cervical spinal cord injury within weeks of injury. Over a six month follow up period, the team measured lesion volume, spinal cord cross-sectional area, and myelin integrity.

The patients treated with NG101 showed a slowing of damage and faster injury improvement than those in the placebo group. The researchers believe this suggests the drug either slows post-injury neurodegeneration or actively promotes nerve fiber sprouting.

“Our data suggest that NG101 antibody treatment demonstrates both focal and remote structural preservation following cervical spinal cord injury, consistent with regenerative mechanisms seen in preclinical models,” write lead author Patrick Freund, MD, PhD, a professor and researcher at the University of Zurich and Balgrist University Hospital, and colleagues.

“These changes are detectable even in participants without overt functional improvement, underscoring the value of quantitative MRI in revealing treatment effects that may be missed by clinical scores alone.”

The post Antibody Promotes Nerve Regeneration in Spinal Cord Injury Patients appeared first on Inside Precision Medicine.

Single Procedure Reduces Colorectal Cancer Risk Decades Later

A single screening procedure could reduce the risk of colorectal cancer for decades, Norwegian trial findings indicate.

Once-only flexible sigmoidoscopy to screen for CRC and remove precancerous lesions provided sustained benefits, according to 23-year follow up of the Norwegian Colorectal Cancer Prevention (NORCCAP) trial.

Men experienced a far greater reduction in CRC incidence than women with the procedure, which involves using a thin, flexible tube to view the lower part of the large bowel.

Their risk of death relating to the cancer also decreased by a third, with no significant survival benefits for women.

“Our findings suggest that a single flexible sigmoidoscopy can have a long-lasting effect on CRC incidence and death,” reported Edoardo Botteri, PhD, from the Norwegian Institute of Public Health in Oslo in the Annals of Internal Medicine.

CRC is the third most common cancer worldwide and, while several screening tests are available, there is not enough evidence to recommend one over another.

As a result, many countries use fecal immunochemical testing (FIT) for CRC screening, whereas others use endoscopic screening with colonoscopy or sigmoidoscopy.

To investigate the potential benefits of sigmoidoscopy further, the team randomly assigned Norwegians aged between 50 and 64 year and living in the city of Oslo or Telemark county to either a once-only sigmoidoscopy examination or usual care without screening in a 1:3 ratio.

Participants invited for flexible sigmoidoscopy were further randomly assigned to either bring or not to bring three successive stool samples for fecal blood testing on attending their sigmoidoscopy procedure.

Those with positive screening results—involving any polyp at least 10 cm, any adenoma, CRC, or positive FIT result—were referred for colonoscopy within six weeks. Adherence to colonoscopy after a positive sigmoidoscopy result was 96%.

Overall, 100,210 persons were randomly assigned to a group, and 98,654 were included in intention-to-screen analyses.

This included 20,552 individuals in the screening group and 78,102 in the no-screening group. Participation with screening was 61.4% in men and 64.7% in women.

In men, the 23-year cumulative risk for CRC was 4.3% with screening and 6.0% without, while in women the corresponding risks were 4.2% and 4.7%.

In men, the 23-year cumulative risk for CRC death was 1.4% in the screening group and 2.2% in the no-screening group, while for women this was 1.3% and 1.4%, respectively.

The effect was strongest for rectosigmoid cancer. Adding fecal blood testing to sigmoidoscopy did not change the benefits of screening.

“Our findings indicate that among men screening was associated with an approximately 28% reduction in CRC incidence and a 37% reduction in CRC death over long-term follow-up, corresponding to absolute risk reductions of 1.7 and 0.8 percentage points, respectively,” the researchers reported.

“In women, screening was associated with a more modest relative risk reduction in CRC incidence (11%; absolute reduction, 0.5 percentage points), with no corresponding reduction in CRC death.”

The post Single Procedure Reduces Colorectal Cancer Risk Decades Later appeared first on Inside Precision Medicine.

First Clinical Trial for a GLP-1 Gene Therapy Greenlit in Europe

Fractyl Health has received regulatory approval in the Netherlands to start the first-ever clinical trial for a gene therapy to treat type 2 diabetes. The upcoming Phase I/II clinical trial will test the safety and preliminary efficacy of RJVA-001, a gene therapy designed to locally deliver GLP-1 receptor agonist drugs with the goal of reducing the side effects associated with oral administration. 

“GLP-1 medicines have changed what is possible in obesity and type 2 diabetes, but they require chronic, high-dose systemic exposure that many patients cannot or do not sustain,” said Harith Rajagopalan, MD, PhD, co-founder and CEO of Fractyl Health. “RJVA-001 takes a different path: a potential one-time, pancreas-targeted gene therapy designed to enable the body to produce GLP-1 in response to meals: physiology, not pharmacology.” 

Based in Burlington, Massachusetts, Fractyl Health develops novel approaches to treating obesity and type 2 diabetes. The company’s lead program, an endoscopic procedure to maintain weight loss after discontinuing GLP-1 treatment for obesity, is currently being evaluated in a pivotal clinical trial. 

RJVA-001 leverages an engineered version of the human insulin promoter to trigger the production of GLP-1 in pancreatic beta cells when glucose levels rise after eating. The gene therapy is delivered using a minimally invasive endoscopic infusion directly into the pancreas, which is guided by ultrasound. 

The Phase I/II study will evaluate the safety, tolerability, and preliminary efficacy of three escalating doses of the gene therapy. Participants will include adults with type 2 diabetes who have previously responded well to oral GLP-1 treatment yet continue to experience difficulties controlling their blood sugar levels. 

“With this authorization, RJVA-001 becomes the first AAV gene therapy candidate to enter clinical development for type 2 diabetes,” said Rajagopalan. “We expect to dose the first patient and report initial data in the second half of 2026.”

Later this year, Fractyl expects to expand the study to additional sites in Australia, where the company has already submitted a clinical trial application and is currently awaiting a response from regulators. 

RJVA-001 will be the first candidate from Fractyl’s Rejuva platform to enter clinical development. This smart GLP-1 gene therapy platform focuses on the development of next-generation adeno-associated virus (AAV)-based gene therapies that are locally delivered to the pancreas. Another candidate developed through this platform includes a dual GIP/GLP-1 gene therapy to treat obesity, currently in preclinical development.  

“For decades, we have managed [type 2 diabetes] as a chronic, progressive disease that inevitably worsens over time. With this authorization, we are preparing to test, for the first time in humans, whether a one-time, pancreas-targeted gene therapy delivered via a routine endoscopic procedure could provide durable metabolic control by enabling physiologic, nutrient-responsive GLP-1 expression at the source of disease,” said Jacques Bergman, MD, PhD, professor of gastrointestinal endoscopy and deputy chair of the department of gastroenterology and hepatology at Amsterdam UMC, and a principal investigator of the upcoming clinical trial. 

“Patients who remain inadequately controlled despite maximally tolerated GLP-1 receptor agonists and multiple oral agents represent a population with significant unmet need. If successful, RJVA-001 could transform how we think about [type 2 diabetes], from a chronic disease you manage every day to one that could potentially be treated once.”

The post First Clinical Trial for a GLP-1 Gene Therapy Greenlit in Europe appeared first on Inside Precision Medicine.

STAT+: European Union inks a draft deal to boost local production of medicines and avoid shortages

The European Union reached a provisional deal to strengthen the supply of essential medicines — such as antibiotics, insulin, vaccines, and painkillers — and avoid shortages by ‌boosting domestic production and reducing reliance on imports.

Known as the Critical Medicines Act, the initiative seeks to avoid the kinds of withering drug shortages that were seen during the Covid-19 pandemic. Specifically, the proposal targets supply-chain weaknesses for more than 200 medicines that are considered critical ​to the region’s health security, including treatments for rare diseases.

“With today’s agreement, we are taking practical action to reduce our vulnerabilities, diversify supply chains and strengthen Europe’s capacity to produce critical medicines and their ingredients closer to home,” said Neophytos Charalambides, the health minister for Cyprus, in a statement.

Continue to STAT+ to read the full story…

Alexa Grasso on Mental Health Fitness 

Alexa Grasso shares her routine for staying mentally strong in and out of the Octagon

In recognition of Mental Health Awareness Month, the Child Mind Institute has launched the Mental Health Fitness campaign — a national call to action highlighting the importance that caring for one’s mental health is just as important as physical health. 

Alexa Grasso knows the importance of prioritizing her mental well-being alongside her physical training. She uses breathwork to stay grounded, acknowledges emotions instead of suppressing them, and practices mindfulness — highlighting that true strength comes from training the mind with the same discipline as the body.

“What I do when I feel stressed or overwhelmed by life, is breathing. It’s super important to breathe, to think clearly and give time to each emotion. To live it.”


About Anna Sitar

Alexa Grasso is a Mexican mixed martial artist and one of the top competitors in women’s flyweight diving of the UFC. Known for her sharp boxing and calm presence in the Octagon, she made history in 2023 by becoming the first Mexican-born woman to win a UFC championship. Grasso’s rise through the sport has been marked by discipline and resilience, earning her recognition as a trailblazer for Mexican athletes in MMA.

About Mental Health Fitness

For decades, we’ve understood that physical fitness doesn’t just happen — it takes skills, regular practice, and a supportive environment. The same is true for mental health. Developed by experts at the Child Mind Institute for three different age groups, our Mental Health Fitness guides have been used by more than 1.8 million students, caregivers, and educators to build emotion regulation skills and resilience. Whether your child is 5 or 15, struggling or thriving, they can learn these skills. And you can practice alongside them. Learn more at Mental Health Fitness.

Related Resources

The post Alexa Grasso on Mental Health Fitness  appeared first on Child Mind Institute.

STAT+: Makary departs FDA amid turmoil as Diamantas, agency’s top food official, steps in

WASHINGTON — Food and Drug Administration Commissioner Marty Makary is resigning from his role, and Kyle Diamantas, the top food regulator at the agency, will step in as acting commissioner.

President Donald Trump confirmed Makary’s resignation while taking questions from reporters ahead of his trip to China.

“He’s going to go on and he’s going to lead a good life,” Trump said of Makary. “He was having some difficulty. He’s a great doctor. He’s going to go on and do well. Everybody wants that job.”

Continue to STAT+ to read the full story…

Finding Answers When Your Child Is Struggling: Introducing Ask Kai 

Every day, parents reach out to the Child Mind Institute with questions that keep them up at night: Why does my daughter have such strong emotions? Why can’t my son sit still in class? Is this normal anxiety or something more?.

These questions are urgent, but finding answers isn’t easy. The wait time to see a children’s mental health professional can be months or even years. Many families don’t know where to start, what’s normal for their child’s age, or whether their concerns warrant professional help. And with nearly 1 in 5 children experiencing a mental health challenge within the U.S. alone, too many families are navigating this uncertainty on their own.

That’s why we built Ask Kai — a free, evidence-based symptom checker designed specifically for children’s mental health, available in both English and Spanish.

Meet Ask Kai

Ask Kai is a conversational symptom checker that helps parents and caregivers understand their child’s behavior and points them to appropriate resources. Through a streamlined series of questions and prompts, Ask Kai gathers information about your child’s challenges and provides personalized recommendations in minutes.

Ask Kai doesn’t diagnose your child. Instead, it helps you:

  • Understand whether your concerns align with common mental health challenges
  • Learn what to look for and what questions to ask
  • Find evidence-based resources specific to your child’s needs
  • Know how to find additional support if needed

Think of Ask Kai as a knowledgeable guide who helps you make sense of what you’re seeing and points you toward your next best step.

How Ask Kai works

If you’ve ever used a symptom checker for physical health, you know the challenge: enter “headache,” and you might walk away convinced you have everything from a sinus infection to a brain tumor. Mental health symptom checkers face even greater challenges. Because every child is different, what looks like defiance in one child could look like anxiety in another, and a behavior that’s appropriate at age five might be cause for concern at age ten. On top of that, mental health conditions often overlap.

So how did we build a tool that captures this complexity without overwhelming families? We focused on expertise and evidence:

  • Clinicians and data lead the way: Ask Kai was built using thousands of child mental health evaluations — open datasets spanning different ages, backgrounds, and conditions — and developed in close collaboration with child psychologists and psychiatrists.
  • Evidence-based question selection: Rather than asking hundreds of questions, we used machine learning to identify which combinations of questions provide the most meaningful information without burdening families.
  • Comprehensive resource library: Every recommendation Ask Kai makes is matched to our extensive collection of guides, articles, expert perspectives, and pathways to professional help.

Here’s what happens when you use Ask Kai

Step 1: Initial Screening

You’ll answer a brief set of questions about your child’s behavior, emotions, and how these challenges affect daily life. You’ll also have the chance to describe in your own words what brought you to Ask Kai. These questions cover the areas where we see the most common concerns.

Step 2: Personalized Deep Dive

Based on your responses, Ask Kai selects targeted follow-up questions that dig deeper into the areas you flagged, whether that’s attention and focus, social anxiety, learning, or other behavioral challenges.

Step 3: Matching You to Resources

Ask Kai analyzes your complete response pattern, including the severity and impact of the behaviors you described, and provides a report with recommendations relevant to your child’s age, challenges, and needs.

What Ask Kai can assess

We designed Ask Kai to explore the areas where we can provide the most help to the most families. Ask Kai offers comprehensive screening and resources for:

ADHD (Attention-Deficit/Hyperactivity Disorder)

Attention-deficit/hyperactivity disorder (ADHD) is a neurodevelopmental disorder that is characterized by difficulties with attention, organization, and impulsive behaviors. Symptoms are usually divided into inattentive behaviors or hyperactive and impulsive behaviors. Inattentive symptoms may include making careless mistakes, being easily distracted, difficulty listening to instructions, trouble with organization, and forgetfulness. Hyperactive/impulsive symptoms may include fidgeting or squirming, trouble playing quietly, extreme impatience, as well as constant talking and interrupting.

Autism Spectrum Disorder

Autism spectrum disorder (ASD) is a neurodevelopmental disorder that begins in utero, but children may not get diagnosed until they’re in preschool or even older, when symptoms become more apparent. The disorder is characterized by deficits in social communication skills as well as restrictive or repetitive behaviors. Symptoms include a wide range of impaired cognitive abilities, language skills, and behaviors. These symptoms have been thought of as a set of disorders but are now being considered one disorder that presents along a spectrum.

Depression

Depression is a mood disorder that can cause children and teenagers to feel very sad and hopeless. Kids with depression have trouble enjoying things they used to love. They may also seem listless and easily annoyed.

Generalized Anxiety Disorder

Generalized anxiety disorder is characterized by excessive, persistent, and unreasonable worries about everyday things, like doing well in school or sports. In general, kids with this disorder worry a lot about being perfect.

Oppositional Defiance Disorder

Oppositional defiance disorder is a disruptive behavior disorder characterized by ongoing persistent, age-inappropriate disobedience and resistance to authority. To be diagnosed with this disorder, children would have had to display extreme behavior issues for at least six months. Diagnosis occurs around early elementary school ages and stops around adolescence.

Social Anxiety Disorder

Social anxiety disorder is a type of anxiety characterized by such intense self-consciousness and fear of embarrassment in social situations that the individual avoids social events; also known as social phobia. While some kids with this disorder are specifically afraid of performance engagements like public speaking or sporting events, others are scared of general social situations.

Specific Phobia

Specific phobia is an anxiety disorder characterized by an excessive and irrational fear of an object, situation, or place. Common specific phobias include dogs, clowns, bugs, the dark, and loud noises.

Elimination Disorders (Enuresis & Encopresis)

For young children, bathroom troubles are often a normal part of growing up. But once kids pass potty-training age, peeing or pooping in places other than the toilet might be a sign of an underlying issue. If it involves urine, it’s called “enuresis.” If it involves feces, it’s called “encopresis.”

Nonverbal Learning Disorder (NVLD)

Nonverbal learning disorder (NVLD) is a condition characterized by difficulty processing visual-spatial information — which involves the brain’s ability to interpret and respond to visual input, including where things are in space. These skills are used to do things like putting together a puzzle or reading a diagram.

Specific Learning Disorder (Dyslexia, Dyscalculia, & Dysgraphia)

Specific  learning disorder is a condition that causes children to have difficulty with reading, writing, and/or math. If they have trouble with reading, the disorder is called dyslexia. If they have trouble with writing, it’s called dysgraphia. If they have trouble with math, it’s called dyscalculia. Symptoms are typically first noticed when the child is in preschool or early elementary school.

These ten areas represent some of the most common mental health concerns in childhood. They’re also areas where we have robust data, validated assessments, and comprehensive resources.

What if your concerns aren’t on this list?

Ask Kai can still help. When you describe your situation in your own words, Ask Kai analyzes your response to identify additional concerns and match you to appropriate resources. Your child’s challenges don’t need to fit neatly into one of these categories for Ask Kai to provide value.

However, we will only make recommendations when we’re confident in the evidence behind them. If a particular concern isn’t well represented in our data, we won’t try to provide guidance in that area. Regardless of the results, everyone receives a core set of resources that we believe are helpful for all families navigating children’s mental health.

Your privacy matters

We take data privacy seriously:

  • Your responses are confidential and secure
  • Free-text responses are analyzed using secure AI systems
  • We don’t share your individual information with third parties
  • In addition to providing you with resources, your data will only be used to improve Ask Kai

Try Ask Kai Today

If you’re wondering whether your child’s behavior is typical or cause for concern, Ask Kai can help. In just 10 to 15 minutes, you’ll get personalized insights and resources to guide your next steps.

Start Your Assessment

You don’t have to figure this out alone. We’re here to help.

If your child is in crisis, expressing thoughts of self-harm, experiencing severe symptoms, or in immediate danger, please seek emergency help right away. Call 988 (Suicide and Crisis Lifeline), text “HELLO” to 741741 (Crisis Text Line), or go to your nearest emergency room.

Frequently Asked Questions

How long does it take?

Most families complete Ask Kai in 10–15 minutes.

Who should use this tool?

Parents, caregivers, and professionals working with children ages 4–18.

Will I get a diagnosis?

No. Ask Kai provides screening information and resources, but only a qualified clinician can provide a diagnosis.

What if I need immediate help?

If your child is in crisis, please call 988, text “HELLO” to 741741, or visit your nearest emergency room.

What makes this different from other symptom checkers?

Ask Kai was built specifically for children’s mental health, uses evidence-based assessments, provides personalized follow-up questions, and was developed in close collaboration with child mental health professionals.

The post Finding Answers When Your Child Is Struggling: Introducing Ask Kai  appeared first on Child Mind Institute.

Supreme Court extends mifepristone deadline

Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here.

Good morning. My co-workers and pals Isabella Cueto and Lev Facher have been talking about alcohol for years. As STAT’s reporters on chronic disease and addiction, respectively, it’s right at the intersection of their beats, yet rarely covered as a public health issue. I’m happy to share that all their talking turned to reporting, and now an incredible series. The first parts are up now. Scroll down or skip ahead to start reading

Read the rest…

World Models: 10 Things That Matter in AI Right Now

World models recently made our list of 10 Things That Matter in AI Right Now. Watch executive editor Niall Firth explain why this emerging area of AI is gaining so much attention.

Join MIT Technology Review editors and reporters for a subscriber-only Roundtables discussion, “Can AI Learn to Understand the World?” exploring how AI may evolve to better reason about the real world and what this could mean for the future of AI systems.

Related Stories:

Speakers:

Mat Honan
Editor in Chief
Will Douglas Heaven
Senior Editor, AI
Grace Huckins
AI Reporter
<![CDATA[Joseph F. Goldberg, MD, urges evidence-based psychiatric deprescribing: continual med reviews, shared decisions, and smarter polypharmacy reduction.]]>