Promise hits Parkinson’s disease therapy, as a Phase I/II clinical study has demonstrated the feasibility of transplanting stem-cell-derived dopamine progenitor cells into the brain. In preclinical research, a new platform uses the brain’s fluid transport pathways to effectively deliver AAVs to therapeutic targets in mice. We’ll also cover the accelerating infrastructure moment for AI-driven drug discovery, with billion-dollar investments flowing into end-to-end platforms driven by models and compute, rather than single drug assets. In business, Insilico’s revenue leaps as the company’s AI-developed lead candidate moves to Phase III, while Vertex acquires Crinetics for $10 billion.
Listed below are links to the GEN stories referenced in this episode of Touching Base:
A vaccine to prevent pancreatic cancer, targeting common KRAS mutations, was found to be safe and stimulated KRAS-specific T-cell responses in 90% of study participants who were at high risk of developing pancreatic ductal adenocarcinoma (PDAC), according to Phase I clinical trial results. After a median follow-up of 16.5 months, none of the participants developed pancreatic cancer, and some of the precancerous lesions shrank or stopped growing. The study represents the first proof of concept for the use of vaccines for interception of pancreatic cancer in human patients.
PDAC is an aggressive cancer that is often diagnosed at advanced stages and carries a low (five-year) survival rate. Approximately 10% of cases are associated with hereditary predisposition caused by pathogenic mutations in specific cancer susceptibility genes that are passed down from parent to child. The disease evolves over time from precursor lesions such as pancreatic intraepithelial neoplasia and intrapapillary mucinous neoplasms.
“Individuals at high risk due to hereditary predisposition or to the presence of a concerning pancreatic lesion detected on imaging usually undergo surveillance to monitor for changes over time,” said Neeha Zaidi, MD, associate professor of oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University. “If there is a high enough concern for transformation to cancer or if early cancer is detected, the current standard of care is surgical resection. However, the chances of recurrence are up to 80%, and many precursor lesions to pancreatic cancer are microscopic and thus undetectable by imaging.”
The researchers wanted to test if intercepting cancer development through noninvasive approaches could provide an effective strategy to prevent PDAC and improve survival in high-risk individuals. “Prevention and interception save lives and reduce the morbidity associated with cancer development and progression. This is especially important for cancers whose early-onset frequency is increasing and for which we do not have effective methods for early detection,” said Elizabeth Jaffee, MD, FAACR, deputy director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.
Mutations in the KRAS gene are the main oncogenic drivers in more than 90% of PDACs. The researchers previously developed mKRAS-VAX, an off-the-shelf synthetic long peptide vaccine targeting the six most common KRAS mutations found in PDAC and in most pancreatic precancer lesions.
In the new study, the team conducted a Phase I clinical trial to assess vaccination with mKRAS-VAX in 20 individuals at high risk of PDAC due to hereditary predisposition and radiographic evidence of a pancreatic lesion, typically in the form of a small cyst.
“The goal of this study was to test the safety of the vaccine and induction of durable immune responses,” said Jaffee. She added that the trial was based on preclinical data demonstrating the ability of a KRAS-targeted vaccine to prevent progression of early precancers in a genetically engineered mouse model of KRAS-driven pancreatic cancer.
Study participants received mKRAS-VAX via subcutaneous injections according to a prime-boost vaccination strategy, with priming doses on weeks one, three, and five and a boost dose on week 13. Blood was collected at different time points, and optional annual follow-up visits were offered for long-term immune monitoring.
Results showed that the vaccine stimulated mutant-KRAS-specific effector and central memory T-cell responses in 90% of participants. These responses remained detectable in the blood for up to two years after vaccination. “This long-lasting response is particularly noteworthy when assessing for possible interception of cancer, which requires long-lasting immunity,” said Zaidi. “In addition, the vaccine was safe and well tolerated, supporting its use in larger cancer interception studies.”
After a median follow-up of 16.5 months, none of the vaccinated individuals developed cancer. The researchers evaluated changes in cyst size as an exploratory clinical endpoint and found a higher rate of cyst reduction or resolution among the vaccinated individuals (37.5%) relative to an unvaccinated cohort with similar characteristics (6.8%).
“This research underscores the need for further funding to support the development of strategies that can intercept and prevent cancer development in high-risk individuals. More studies are needed to find the best vaccine approaches, the best targets, and the ideal timing for vaccination,” concluded Jaffee.
According to the authors, the study’s limitations include the small size and the fact that the trial was not designed to assess the clinical efficacy of the vaccine. “We observed evidence of stability or regression of the pancreatic cysts in association with the induction and durability of KRAS-specific T-cell responses,” said Michael G. Goggins, MD, professor of pathology, medicine, and oncology and the Sol Goldman Professor of Pancreatic Cancer Research at Johns Hopkins University School of Medicine. “However, larger studies are needed to demonstrate that this effect was in fact due to the vaccine.”
A decades-long mystery surrounding one of the nervous system’s most important molecular machines has been solved, providing a structural blueprint that could accelerate the development of targeted therapies for several inherited neurodegenerative diseases.
In a study published in Science Advances, researchers at the University of California, Davis report the first complete structure of kinesin-1—a motor protein that transports neurotransmitters, proteins, and other essential cargo throughout nerve cells—in its inactive state. The findings reveal how the protein is switched off until needed and identify structural features that could serve as targets for future drugs.
Although kinesin-1 was the first member of the kinesin superfamily to be discovered and has been studied for more than 40 years, scientists have never fully understood how cells regulate its activity. The new study provides what the authors call “a structural answer,” revealing how motor activity and cargo binding are coordinated.
Kinesin-1 is essential for healthy neurons. The protein walks along microtubules using energy from ATP, carrying cargo from the cell body to distant parts of nerve cells. When this transport system fails, neurons cannot deliver the materials needed to function and survive, contributing to diseases including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease type 2, and hereditary spastic paraplegia.
Using cryo-electron microscopy, the researchers captured the complete structure of kinesin-1 in its autoinhibited, or “off” state. They discovered that the protein folds into a compact configuration that simultaneously prevents the motor from moving and blocks cargo from attaching.
“This dual-inhibited kinesin architecture provides a comprehensive blueprint” for how the protein maintains its inactive state, the authors write. The structure also reveals distinct regulatory sites that can be unlocked to restore both movement and cargo transport.
The team also uncovered how the protein is activated. They propose that the microtubule-associated protein MAP7 binds kinesin-1 and triggers a series of structural changes that unfold the protein, releasing the motor domains and exposing the cargo-binding site. Once activated, kinesin can resume transporting essential cellular components through the neuron.
The findings have important implications for drug development because many disease-causing mutations disrupt kinesin-1’s ability to switch between its inactive and active states. Until now, researchers lacked the structural information needed to understand exactly how these mutations impair the protein or how they might be corrected.
With the complete structure now available, investigators can examine how specific mutations alter kinesin-1 and begin designing molecules that restore its normal function. Rather than replacing the defective protein, future therapies could stabilize its structure or correct the molecular interactions that prevent it from turning on.
The authors hope this discovery will aid in designing a molecule that would bind the mutant protein and correct its defect. They believe the study establishes “a clear foundation for future mutational studies” and provides “a powerful framework” for understanding how kinesin proteins are regulated across the broader superfamily.
While additional research is needed before therapies reach the clinic, the work provides the detailed structural roadmap that has long been missing. By revealing exactly how kinesin-1 is locked into its inactive state and how that lock can be released, the study identifies promising new targets for precision medicines aimed at restoring intracellular transport in neurodegenerative disease.
ATLANTA — Federal health officials have identified lettuce from Mexico served at Taco Bell locations across five U.S. states as a source of a widespread outbreak of diarrhea-causing parasite cyclospora.
The Centers for Disease Control and Prevention late Thursday warned consumers not to eat shredded iceberg lettuce from Taco Bell restaurants in Indiana, Kentucky, Michigan, Ohio and West Virginia. A record number of cyclospora cases have been reported in more than 30 states, and experts have said not every recent U.S. illness might be caused by a single source.
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Hiya. Today, we explore a gene therapy approach that’s drawing excitement but also skepticism, and see that Jennifer Doudna is moving into AI-designed gene editors that push beyond evolution.
Plus, our trusty podcasters unpack the buzzy prediction market Kalshi’s move into biotech.
By Mai El Shoush, Partnership Campaign Manager, SNF Global Center for Child and Adolescent Mental Health at the Child Mind Institute
Few events unite the world quite like the FIFA World Cup, demonstrating soccer’s unique ability to connect people across cultures and communities. As millions tune in, the moment offers a timely reminder on how sports can reach young people far beyond the field. This belief underpins the work of Grassroot Soccer, a valued partner of the SNF Global Center for Child and Adolescent Mental Health at the Child Mind Institute. They utilize the power of soccer to advance youth mental health — and have reached more than 25 million young people around the world through this approach.
The partnership incorporates three interconnected areas:
Building capacity for frontline workers
Generating evidence
Contributing to thought leadership on child and adolescent mental health in low-resource settings
The 2026 Men’s World Cup in North America and the upcoming 2027 Women’s World Cup in Brazil present an important opportunity for the organization to take its mental health programming from sub-Saharan Africa and adapt it to the U.S. context.
We spoke with Grassroot Soccer’s Mental Health Specialist Charmaine Nyakonda about the organization’s approach, the impact of their collaboration with SNF, and the growing role of sports in improving the mental health of young people and communities globally.
Grassroot Soccer’s Mental Health Specialist, Charmaine Nyakonda
As international attention increasingly turns towards soccer’s global reach with the 2026 FIFA Men’s World Cup, what role can sports play in advancing youth mental health care across diverse communities?
Soccer is the most popular and accessible sport in the world. From the largest global stages like the World Cup to the most remote rural villages, it’s played everywhere on the planet. As the World Cup shows, soccer has the power to captivate across geographies, cultures, and time zones.
Soccer (and sport in general) is a powerful tool that teaches youth important life skills and character-building lessons like resiliency, hard work, courage, trust, and teamwork. On top of this, soccer’s universal appeal across diverse communities means using the language of the game can also be an effective way to break through the stigma around sensitive but pressing topics like mental health. This is why Grassroot Soccer uses soccer as a teaching tool for mental health.
Grassroot Soccer has successfully touched the lives of millions of young people across settings worldwide. What has the team learned about the universal role that sports can play in supporting mental health, resilience, and social connection?
Across our programs, soccer serves as a universal entry point that hooks young people and meets them where they are. From there, our play-based activities use soccer games and metaphors to make learning about mental health fun and engaging. They break down stigma and create safe spaces where young people can feel comfortable opening up and being vulnerable. This drives exceptional participation and completion rates.
Importantly, our signature soccer-based mental health promotion and prevention program, called MindSKILLZ, is guided by core mental health design principles. This includes grassroots co-creation, trauma-informed practice, relationship-centered delivery, and strengths-based learning — which ensures that programs are ethical, culturally grounded, and safe for adolescents in both stable and crisis-affected settings.
“Soccer (and sport in general) is a powerful tool that teaches youth important life skills and character-building lessons like resiliency, hard work, courage, trust, and teamwork.”
What elements of Grassroot Soccer’s approach do you believe could serve as a transferable blueprint for other organizations seeking to improve outcomes for young people in different sectors and communities?
Our commitment to co-design with young people: We believe that young people are the experts on their lives and needs, so we intentionally apply the principle of “Nothing about us without us” to our work. We put this into practice by engaging young people at every point in the program life cycle to ensure our work meets their needs, from design to implementation to evaluation.
In 2023, Grassroot Soccer officially launched a Youth Advisory Committee (YAC) composed of young leaders that serves as an internal advisory and advocacy body for the organization. The Committee consists of SKILLZ Coaches, Master Coaches, and trainers between the ages of 18 to 30 from the communities we serve.
Mental health integration: Mental health promotion and prevention must be woven into every service that young people access. Mental health is both a driver and an outcome of young people’s overall well-being. Unaddressed mental health challenges undermine progress in HIV, sexual and reproductive health, education, and other youth development areas. And those same factors profoundly shape young people’s mental health in return.
What has made the partnership with the SNF Global Center particularly valuable, and what role do cross-sector and multidisciplinary partnerships play in expanding support and opportunities for young people?
The diverse cultural perspectives from Greece, Brazil, the United States, and South Africa — and especially youth voices — have enriched our understanding of the barriers young people face, the values that motivate them, and the creative solutions they come up with when it comes to mental health.
We’ve also benefited tremendously from the scientific and clinical expertise at the SNF Global Center. We were able to co-develop an open-access, near-peer emotional support online training that aims to enhance the capacity of frontline workers to identify, refer, and support common mental health problems among children and adolescents. This is especially important in low-resource settings.
Additionally, through thought leadership and advocacy collaboration, this partnership has been valuable in contributing to an important shift in the global conversation to framing low-resource settings (the Global South) as sources of innovation that can positively impact the Global North.
Over the years, what has most reinforced your belief in the power of soccer to support youth mental health, and what examples best bring that impact to life?
We’ve seen a few powerful stories of impact in humanitarian settings, where the need for mental health care is at its greatest but resources are at its most limited.
For example, in 2024, relentless downpours following a tropical cyclone caused rivers in Malawi’s Nkhotakota and Karonga districts to overflow, leading to widespread flooding impacting over 150,000 people and forcing many displaced residents to seek refuge in makeshift internally displaced person (IDP) camps.
In the camps, people had no access to mental health and psychological support services or safe spaces, and youth were not attending school. The Malawi Ministry of Health identified a major gap in mental health support for adolescents and young people and called on Grassroot Soccer to deliver MindSKILLZ to respond to this need.
Grassroot Soccer’s response team focused heavily on play, coping skills development, and trauma-informed support, creating critical psychological and physical safe spaces for youth within the camp environment.
Looking ahead, how would you like to see the conversation evolve at the intersection of sports, youth well-being, and mental health?
Firstly, we would like to see the conversation move beyond asking whether adolescent girls and young women like to play sports and instead asking, “What can we unlock when they do?”
For too long, sports (and soccer in particular) have been understood as primarily a significant part of adolescent boys’ and young men’s lives only. That assumption has shaped who gets access, who feels welcome, and whose well-being is centered. Grassroot Soccer’s own programming challenges this directly — we have reached more adolescent girls than boys, and in doing so have created space for girls to reconstruct gendered ideas about soccer. The conversation on sports, youth, and mental health needs to evolve to view sports games like soccer as universal tools that can transcend gender norms, age, and socio-economic background to support young people.
At the same time, we want to see the conversation take adolescent boys’ mental health more seriously, not less. Qualitative insights from Grassroot Soccer show that rigid masculine norms remain a profound barrier for boys.
More broadly, we ultimately want to see the conversation shift from individual resilience to multi-systemic access and inclusive enabling environments. Young people need culturally grounded, safe environments that work for them — and include them in design and delivery to help them develop into healthy adults who carry that capacity forward.
What do you see as the greatest opportunities for the next generation of young people, and what must we do collectively to help make that future possible?
With the largest generation of adolescents coming of age, their ability to live healthy and productive lives will have a profound impact on not just their own life trajectories, but also on the world. Tackling critical global issues from poverty to climate change will require a generation of young people who are strong, healthy, and empowered — and their mental health is central to all this.
At Grassroot Soccer, we are committed to investing in youth mental health to realize a world where mental health challenges have been normalized. We want young people to understand their own mental health, have practical coping skills to deal with frustration and aggression, and recognize the value in seeking support from others. This will help develop a population with reduced symptoms of depression and anxiety, as well as overall improved mental well-being. Then we can collectively realize our vision for a better future.
Through the SNF Global Center, the Child Mind Institute partners with organizations around the world to strengthen child and adolescent mental health systems by combining scientific expertise, local leadership, evidence-based innovation and youth voices. Grassroot Soccer is one example of how cross-sector partnerships can expand access to mental health support in communities where young people already learn, connect, and thrive.
<![CDATA[Learn how clinicians tackle mood and substance use disorders together, boost patient engagement, and expand evidence-based addiction medications for bipolar and alcohol use.]]>
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<![CDATA[Meta-analysis links traumatic brain injury to rising long-term psychosis risk; severity, age, and follow-up time guide smarter screening and care.]]>
Results from a Phase I clinical trial show early promise for a vaccine designed to prevent pancreatic cancer in people at high risk of developing the disease. Published in Cancer Discovery, the findings suggest it may be possible to intercept one of the most aggressive forms of cancer before it takes hold.
Pancreatic cancer is often diagnosed at advanced stages and grows rapidly, contributing to its poor survival rates. Yet the disease typically develops over many years from pancreatic cysts and other precursor lesions, creating a window of opportunity to intervene before those lesions turn into malignant tumors.
“Individuals at high risk due to hereditary predisposition or to the presence of a concerning pancreatic lesion detected on imaging usually undergo surveillance to monitor for changes over time,” said Neeha Zaidi, MD, associate professor of oncology at Johns Hopkins Medicine and co-senior author of the study. “If there is a high enough concern for transformation to cancer or if early cancer is detected, the current standard of care is surgical resection. However, the chances of recurrence are up to 80%, and many precursor lesions to pancreatic cancer are microscopic and thus undetectable by imaging.”
More than 90% of pancreatic cancers are driven by mutations in the KRAS gene, which also appear in many precancerous lesions. Zaidi’s team developed a vaccine targeting the six most common KRAS mutations, with the goal of training the immune system to recognize and attack cells carrying these mutations before they become cancerous.
The Phase I clinical trial evaluated the mKRAS-VAX vaccine in 20 participants with genetic predisposition to pancreatic cancer and a pancreatic abnormality identified through imaging. Patients received four doses of the vaccine over the course of 13 weeks and were monitored for side effects and immune responses for a median follow-up time of 16.5 months.
None of the patients developed pancreatic cancer or high-risk lesions during the study, and 90% of them developed a significant immune response to the vaccine. Memory cells against the six KRAS mutations were still detectable in the blood for as long as two years after vaccination.
“This long-lasting response is particularly noteworthy when assessing for possible interception of cancer, which requires long-lasting immunity,” said Zaidi. “In addition, the vaccine was safe and well tolerated, supporting its use in larger cancer interception studies.”
Although the main goal of the study was to prove the vaccine’s safety, an exploratory imaging analysis found that pancreatic cysts completely regressed in five participants and partially regressed in three, while the remaining remained stable.
The vaccine had previously been tested in pancreatic cancer patients who had undergone surgery and were at high risk of experiencing recurrence, where all participants remained disease free for at least five years. “We thought if we can see an immune response in patients with cancer, the vaccine should work even better in people who are at higher risk because of a family history, gene alteration or cyst on the pancreas,” said Zaidi.
Another trial is currently enrolling patients to study whether the immune response generated by the vaccine can infiltrate within precancerous lesions in addition to being detectable in the blood. If successful in larger clinical studies, the vaccine could offer the first interception strategy to reduce the number of pancreatic cancer cases.
“Prevention and interception save lives and reduce the morbidity associated with cancer development and progression. This is especially important for cancers whose early-onset frequency is increasing and for which we do not have effective methods for early detection,” says Elizabeth Jaffee, MD, deputy director of the Johns Hopkins Kimmel Cancer Center, co-director of the Skip Viragh Center for Pancreatic Cancer, and associate director of the Bloomberg~Kimmel Institute for Cancer Immunotherapy.
“This is just the beginning, but the findings suggest that the immune system is getting activated. We have more work to do, but this is a good start aimed at prevention, which no one had thought about doing before.”
