Radically different
Nature Medicine, Published online: 10 July 2026; doi:10.1038/s41591-026-04497-1
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Jeremy Faust cuts through a hallway of Boston’s Brigham and Women’s Hospital on his way to see a patient who is struggling to breathe. It’s the start of his evening shift, and the emergency department hums with ambient sound: bleeping monitors, the rumbling wheels of medical carts, the squeaky soles of hustling staff. People on gurneys line the corridor, some wincing in pain, others chatting with relatives.
On this Wednesday evening in May, Faust is working what is typically a quieter shift, as far as emergency departments go. Still, he is overseeing a team of doctors, students, and physician assistants, and will tend to more than two dozen patients before signing off for the night.
It’s an understatement to say Faust likes to keep busy. Minutes earlier, he’d posted an article on his influential Substack newsletter, Inside Medicine, providing an update on a major international news story. An alert sent to the newsletter’s nearly 85,000 subscribers announced his “scoop”: Twenty-six passengers aboard the MV Hondius, the hantavirus-hit cruise ship docked at the time off Cape Verde, had disembarked much earlier than previously known — raising the possibility they could spread the rare virus in the United States.
A landmark Phase I/II clinical study led by researchers at Skåne University Hospital and Lund University has shown that transplanting stem-cell-derived dopamine progenitor cells into the brain is feasible. Eight patients with Parkinson’s disease (PD) received transplants of STEM-PD, a cryopreserved, off-the-shelf dopaminergic progenitor product derived from human pluripotent stem cells. The three-year Phase I/II, open-label, multicenter, single-arm, dose-escalation study identified no serious side effects linked to the transplanted cells during the first year of follow‑up.
“The possibility of replacing dopamine neurons that are lost in Parkinson’s disease has been a long-standing goal in the field,” said Malin Parmar, professor of cellular neuroscience at Lund University, and lead of the STEM-PD program. “The findings represent an important milestone for regenerative medicine approaches in Parkinson’s disease and support continued clinical development of stem cell-based therapies.”
Results from the study were reported by Parmar and colleagues in Nature Medicine. In their paper, titled “Human embryonic stem cell-derived dopaminergic cells for Parkinson’s disease: a Phase I/II open-label trial,” the team wrote, “In conclusion, particularly in the context of other recently published trials using human PS cell-derived dopaminergic cell therapies for PD, these findings further support the continued development of this therapeutic approach, including evaluation of the STEM-PD product in larger patient cohorts.”
Parkinson’s disease is the second most common neurodegenerative disorder after Alzheimer’s disease, the authors wrote. In Parkinson’s disease, patients lose nerve cells in the brain that produce dopamine, which leads to symptoms such as slowness of movement, stiffness, gait disturbance, and tremor. “The hallmark pathology of PD involves progressive degeneration of dopaminergic neurons in the substantia nigra pars compacta (SNpc) and the subsequent loss of their projections to the striatum,” the team explained. Current treatments are medications that replace the lost dopamine, but over time these medications often become less effective and cause side effects.
“Intracerebral transplantation of stem cell-derived dopaminergic progenitors to replace lost endogenous dopaminergic neurons offers a new potentially restorative therapeutic approach for PD,” the investigators continued. Scientists have been working to develop standardized and scalable dopamine cell therapy products derived from pluripotent stem (PS) cells, including human embryonic stem (ES) cells and induced PS (iPS) cells, they noted. “Several such products are now in clinical development … with early safety and feasibility data emerging.”
The transplanted stem cell-based dopamine nerve cell product tested in the newly reported trial is designed to replace the cells that produce dopamine, and the goal is that after being transplanted, the transplanted cells will mature into new dopamine-producing nerve cells in the brain. The STEM-PD trial aimed to evaluate the safety, tolerability, and feasibility of intraputaminal transplantation of STEM-PD in patients with moderately advanced PD.
Eight individuals with Parkinson’s disease received the transplanted cell product at two different doses, followed by 12 months of immunosuppression to prevent graft rejection. All patients were treated at Skåne University Hospital. Seven participants completed 12-month follow-up, and one participant died from a pulmonary infection that was not directly related to the cell product.
The surgical procedure was generally well tolerated, and no graft-induced involuntary movements were observed in the transplanted participants. Clinically, patients remained stable. Imaging using dopamine PET scans provided early indications of graft survival at both 6 and 12 months post-transplantation. Six of the seven participants substantially reduced their dopaminergic medication, a result that will be evaluated over time. In their paper, the team wrote in summary, “This Phase I/II clinical trial involving the bilateral intraputaminal transplantation of the STEM-PD dopaminergic progenitor cell product demonstrates its feasibility with no unexpected safety concerns from the cell product.”
Roger Barker, MD, professor of clinical neuroscience at the University of Cambridge, clinical lead of STEM-PD and clinical PI at the U.K. site, said: “This represents an exciting new departure on repairing the brain of individuals with Parkinson’s using dopamine cells- an approach pioneered in Lund some 40 years ago using fetal dopamine cells. The STEM-PD trial harnessing the expertise of scientists and clinicians from Lund and Cambridge has enabled us to undertake and deliver on one of the first ever stem cell-derived dopamine cell therapies for patients with Parkinson’s, and we hope this will be the beginning of an exciting new programme that may ultimately benefit the wider Parkinson’s community.”
Gesine Paul-Visse, MD, professor in neuropsychiatric research and lead PI at Skåne University Hospital, said, “Reaching this primary endpoint and being able to show that the cell product is safe is a great achievement for this trial, our team, the participating patients, but also for all patients suffering from Parkinson’s disease. We are hopeful that the early signs of cell survival and clinical improvement we observe will continue to increase over time and are excited to continue the development of this cell therapy.”
The STEM-PD research team will now continue the long-term follow-up of the participants to further evaluate safety, graft function, and clinical benefit. “Secondary and exploratory outcomes will evaluate the course and efficacy of clinical features, the survival of grafted dopaminergic cells at 36 months as well as additional safety signals occurring between 12 and 36 months and any dose–response effects,” the investigators stated. “Further evaluation of the grafts up to 36 months will determine whether the implanted cells continue to grow, mature and reinnervate the putamen after 12 months.”
STEM-PD builds on decades of research in dopamine cell replacement therapy for PD at Lund University and pioneering work in translation of pluripotent stem cell technology from experimental studies into clinical evaluation. The STEM-PD trial is the first pluripotent stem cell trial approved in Sweden and the first for PD in Europe. “The initiation and execution of this clinical trial have only been possible through close collaboration between scientists, clinicians, GMP manufacturing teams, regulatory experts and, most importantly, the participating patients,” concluded Parmar. In their report, the authors stated, “The STEM-PD trial adds further important confirmatory and complementary evidence to the recently reported feasibility and short-term safety of PS cell-derived dopaminergic progenitor transplantation in PD.”
The academic Phase I/IIa trial was conducted in collaboration with Novo Nordisk. Cellular Intelligence, a Boston-based company, recently acquired the STEM-PD program and will lead its next phase of clinical development, including a planned Phase II trial. The STEM-PD cells and their continued development hold IND clearance with FDA Fast Track Designation, and Cellular Intelligence aims to advance the program through Phase III to market approval.
STEM-PD is an academic European clinical translation initiative, focused on developing stem cell-based therapies for Parkinson’s disease. The program is led from Lund University with partners from Skåne University Hospital, Cambridge University Hospital, and University College London and combines expertise in stem cell biology, GMP manufacturing, neurosurgery, clinical neurology, and regenerative medicine to advance pluripotent stem cell-derived dopamine neuron therapies toward clinical application.
The post Stem Cell Therapy Shows Promise in First Human Parkinson’s Disease Trial appeared first on GEN – Genetic Engineering and Biotechnology News.
By taking advantage of the microbe-targeting capabilities of bacteriophages (phages), researchers at McMaster university have devised a supportive therapy for Crohn’s disease that disarms a type of E. coli bacteria that drives inflammation in the gut without disrupting the broader gut microbiome. The team showed that the phage therapy also improved responses to low doses of a conventional corticosteroid in a mouse model of Crohn’s disease, suggesting treatment could both improve outcomes and lower the risk of side effects from standard therapies.
Senior and co-corresponding author Elena F. Verdu, PhD, professor in the Department of Medicine and director of the Farncombe Family Digestive Health Research Institute, and colleagues reported on their study in Science Translational Medicine, in a paper titled “Phage intervention improves colitis and response to corticosteroids by attenuating virulence of Crohn’s disease–associated bacteria,” in which they noted “By reducing bacterial virulence mechanisms without substantially disrupting microbial balance, phage-based treatments align with personalized microbial therapeutics in IBD.”
Inflammatory bowel diseases (IBDs) such as Crohn’s disease and ulcerative colitis (UC) are chronic inflammatory conditions of the gastrointestinal (GI) tract that have strong microbial components, with patients often displaying disruptions in the delicate microbiome of the gut. “Recent studies have demonstrated that changes in the composition and function of the gut microbiota precede CD onset by up to five years,” the author wrote. However, they continued, “Current medications focus on suppressing symptoms and inflammation and can fail, in part, because they do not address underlying microbial drivers.”
Patients with Crohn’s disease tend to harbor strains of adherent-invasive E. coli (AIEC) that adhere to and invade epithelial cells in the intestines. “AIEC is distinct from other IBD-associated taxa because of its ability to adhere, invade, and survive in intestinal epithelial cells,” the team continued. Clinicians will sometimes prescribe antibiotics during symptom flares to try to target these bacteria, but antibiotics aren’t a long-term solution. They are non-specific and can perturb the microbiome even further, and with frequent use lead to side effects.
AIEC bacteria can be difficult to identify and selectively target, making them an important test case for more precise microbiome-based therapies. “One challenge is that AIEC are defined by what they do, not simply by how they appear in a microbiome analysis,” said Verdu. “To identify them, we need to test their behavior, such as their ability to adhere to and invade intestinal cells and persist in immune cells.”
Verdu, together with first author Kyle Jackson, PhD, at Farncombe Family Digestive Health Research Institute, and colleagues theorized they could navigate these roadblocks and neutralize virulent E. coli using bacteriophages. “Phages work like a lock-and-key system—each phage targets only certain bacteria. That precision gives us a way to intervene without wiping out the entire microbiome,” explained co-author Zeinab Hosseinidoust, PhD, associate professor in the Department of Chemical Engineering and the School of Biomedical Engineering.
The concept of using phages to target inflammatory bacteria is already being trialed, the authors noted in their paper. “Commercial phage ‘cocktails’ are presently being evaluated in clinical trials (NCT04737876 and NCT03808103) against proinflammatory bacterial taxa associated with IBD. These trials seek to target adherent-invasive Escherichia coli (AIEC), a group of bacteria enriched in patients with active CD compared with those in remission or healthy individuals.”
For their newly reported study the team developed a Crohn’s disease mouse model with a defined microbiome, and then screened a collection of phages, looking for those that could infect one strain of E.coli, NRG857c, isolated from patients with Crohn’s disease.
The results showed that their approach significantly reduced gut inflammation. The phages did not eliminate the bacteria entirely. Instead, they altered their behaviour by supressing a molecular “grappling hook” that helps AIEC attach to the gut lining and trigger immune responses. When that virulence mechanism was turned off, inflammation subsided.
One phage, designated HER259, countered the bacteria’s virulence by infecting it and switching off a genetic region named fimS. This region promotes the expression of FimH, which supports the bacteria’s ability to adhere to cells and trigger inflammation.
“HER259 ameliorated colitis in gnotobiotic models and attenuated the virulence of AIEC strain NRG857c, including suppression of the FimH adhesin through inversion of the fimS promoter to its ‘off’ orientation,” the authors wrote. “Withdrawal of HER259 treatment led to reversion of the fimS promoter and reactivated colitis.”
Hosseinidoust noted, “The bacteria were still there but they lost the traits that drive inflammation. We like to think of it as knocking out a few teeth. The bacteria can’t do as much damage anymore.” Withdrawing HER259 treatment led to reversion of the fimS promotor, and reactivated colitis, the authors noted.
The researchers also found that phage therapy enhanced the effectiveness of a commonly used steroid treatment for IBD, budesonide. When combined with the phage, a lower-than-standard dose of the drug produced benefits comparable to higher doses of the drug alone.
The HER259 phage in addition enhanced the therapeutic effect of subtherapeutic budesonide independent of microbial drug metabolism, the team wrote. While phages have previously been shown to increase the effectiveness of antibiotics, this is the first time a positive collaboration between phage and a non-antibiotic drug has been reported.
“In summary, using phage HER259 and NRG857c as a model phage–proinflammatory bacterium pair, we reveal a previously unknown mechanism by which targeted phage therapy disrupted key virulence markers in the CD-associated E. coli strain, reducing acute and chronic colitis, preventing reactivation, and enhancing therapeutic responses to budesonide.”
The findings point to a precision‑medicine approach for IBD. The bacterial function targeted by the phage can be measured in stool samples and was found to be higher in a subset of patients with Crohn’s disease, suggesting a potential way to identify those who could benefit most from this therapy.
“If we can identify which patients carry the harmful bacterial function, we could, in the future, intervene with a targeted therapy designed specifically to turn down that activity,” says Verdu. “This is what personalized medicine should look like: matching the right biological tool to the right patient,” added Hosseinidoust.
Next steps for the team include evaluating broader collections of bacterial strains from IBD patients and developing combinations of phages—work that brings the approach closer to human trials.
The post Crohn’s Disease Phage Therapy Neutralizes Inflammatory <i>E. coli</i> in Mouse Model appeared first on GEN – Genetic Engineering and Biotechnology News.
A multi-skilled biomedical AI agent can automate biomedical research tasks, driving forward basic research into practical application.
The AI-powered “co-scientist” Biomni autonomously executes diverse research tasks, working across complex tasks across fields as diverse as genomics, immunology, pharmacology, and clinical medicine.
The large-language model (LLM), outlined in Science, mines through biomedical literature interpreting requests, both composing and executing multi-step workflows.
It can formulate hypotheses, performs complex bioinformatics analyses, and design rigorous experimental protocols, with tests showing comparable accuracy to experts while taking a fraction of the speed.
“Biomni is able to understand a simple question like, ‘Why are these patients responding differently to the drug?’” explained research Kexin Huang, who was studying for his PhD at Stanford University at the time of the research.
“Then it digs in, doing a lot of the scientific legwork.”
Initially, the researchers constructed a unified and comprehensive biomedical action space by systematically analyzing 2500 biomedical research papers spanning 25 distinct subfields, curated from literature repositories.
From this foundation, they developed an LLM-powered action discovery agent capable of reading papers and extracting key tasks, tools, and databases essential to driving biomedical discoveries.
These elements are then chosen and developed into Biomni-E1, the foundational environment that defines the biomedical action space for agentic interaction and includes 150 specialized biomedical tools, 105 software packages, and 59 databases.
The team then designed Biomni-A1, a general-purpose agent architecture capable of flexibly executing a broad spectrum of biomedical tasks by using tools and datasets provided by Biomni E1.
After a user query is entered, the agent uses a retrieval system to identify the most relevant tools, databases, and software needed.
It then applies LLM-based reasoning and domain expertise to generate a detailed, step-by-step plan. Each step is expressed through executable code, enabling precise and flexible compositions of biomedical actions—an essential 10 feature given the domain’s reliance on highly specialized tools and data resources.
This integrated system allows Biomni to efficiently generate solutions for challenging, large-scale biomedical problems, but also to generalize to tasks across previously unseen areas of biomedical research.
In this way it removes the laborious work from biomedical science, allowing researchers to focus on creating hypotheses and innovative experiments and collaborating across disciplines.
“The hurdle in biomedical science is not intelligence or ideas; it is mechanics,” said researcher Jure Leskovec, PhD, also at Stanford.
“It’s this laborious stuff that slows innovation. Biomni can do this work in minutes.”
The team tested Biomni’s practical capabilities through five case studies: analyzing wearable sensor data; performing comprehensive bioinformatics analyses on massive raw datasets such as single-cell RNA-seq and ATAC-seq data; designing laboratory protocols to assist wet-lab researchers; optimizing a protein sequence for better thermostability; and orchestrating robotics wet-lab instruments.
In one test, more than 450 files of real-world continuous glucose monitoring, food intake, and physical activity data from a single person was uploaded and Biomni asked to find interesting and plausible hypotheses.
The researchers asked a simple question: “Analyze this data, find interesting and plausible hypotheses.” In just 40 minutes, the AI-agent identified patterns relating food intake and body temperature that would have taken an estimated 60 or more hours for a human to complete.
“With Biomni, we introduce a scalable, general-purpose biomedical AI agent, pointing toward a future in which AI agents work alongside human researchers to accelerate biomedical discovery from basic research to translation,” the authors concluded.
A prototype of the AI agent is already being used by more than 10,000 labs in academia and industry, making it the most widely used AI co-scientist system in biomedicine.
The post AI Tool Speeds Biomedical Research Progress appeared first on Inside Precision Medicine.
Reliance of artificial intelligence (AI) has been increasing across all fields, including in the clinic. How to ensure AI is integrated practically, effectively, and ethically has been the focus of many discussions and debates, however final consensus almost always lands on the idea that however AI is integrated into clinical use, conscientious human oversight is necessary.
To test the veracity of this goal, a team of researchers in Spain put over 200 medical doctors to the test to determine if the clinicians would trust their experience and training over AI recommendations.
“It is important to investigate the errors that humans (including doctors) make when working with algorithms, in order to learn how to minimize the problems that arise from them,” said co-author of the study, Fernando Blanco, PhD, researcher at the Mind, Brain and Behavior Research Center (CIMCYC) in Granada, Spain.
“We wanted to see if professional physicians would act differently so that they would notice and correct these errors,” the authors wrote in their paper published in PLOS Digital Health.
The researchers created treatment plan options for a series of fictitious patients with a rare disease. They asked 223 physician participants whether or not to provide a treatment to a patient based on whether the patient was classified by AI as being highly or lowly sensitive to the treatment plan. The physicians were then presented with patient recovery data and asked to rate their perception on how reliable the AI classification was.
In these experiments, both groups of patients responded to treatment with similar sensitivity, resulting in ineffective AI recommendations that could be identified using the patient recovery data.
“In the first experiment, the treatment worked moderately (and equally) well for both groups. In the second, the treatment did not work at all for either group,” the authors wrote. They expected that “in both experiments, participants would administer the treatment less often to the fictitious patients classified as lowly sensitive to the treatment, consistently with the AI classification.” However, this was not the case.
“In both experiments, physicians mostly trusted the AI’s classifications and had trouble learning from the feedback,” said lead author Aranzazu Vinas, PhD, University of the Basque Country, Spain. “Furthermore, in the second experiment, professionals did not notice that the treatment was completely ineffective.”
These results present a major concern for the medical community in its use of AI in the clinic. While AI can be highly effective and useful for data collection and summary, supervision and critical thinking are still required for effective and safe patient care. This study highlights the need for physicians to take the time to critically consider all available data, regardless of recommendations by AI in their diagnostic and treatment decisions.
“People tend to say that there is always a human controlling the algorithm,” opined Helena Matute, PhD, professor, University of Deusto, Spain on the team’s findings “but our experiments show that doctors (as well as anyone else) have problems in learning from the available evidence when it contradicts the suggestions of an algorithm.”
The post Clinicians Trust Faulty AI Recommendations Over Experience appeared first on Inside Precision Medicine.
A novel sequencing strategy that combines low-pass whole-genome sequencing with deep whole-exome sequencing in a single assay could significantly lower the cost of large-scale genomic studies without sacrificing analytical performance, according to a study published in Nature Genetics. The approach, known as blended genome–exome (BGE) sequencing, may help accelerate precision medicine initiatives by making comprehensive genomic profiling more accessible across diverse populations.
Researchers from Massachusetts General Hospital and the Broad Institute of MIT and Harvard developed BGE to overcome a persistent tradeoff in human genomics. High-coverage whole-genome sequencing offers the most comprehensive view of genetic variation but remains prohibitively expensive for many population studies. Genotyping arrays and exome sequencing are more affordable but either miss large portions of the genome or introduce bias by relying on variants selected primarily from European ancestry populations.
The BGE workflow integrates low-pass whole-genome sequencing at 1–4x coverage with deep exome sequencing at 30–40x coverage within a single library preparation and sequencing run on Illumina’s NovaSeqS4. The result is a unified dataset capable of supporting genome-wide association studies, rare variant discovery, copy number variant (CNV) detection, and polygenic analyses at approximately 28% of the cost of conventional 30x whole-genome sequencing.
The investigators validated the approach in more than 53,000 participants enrolled in the Populations Underrepresented in Mental Illness Associations Studies (PUMAS) Project, which includes African, African American, Hispanic/Latino, and Colombian cohorts. The scale and diversity of the study allowed the researchers to assess performance in populations that have historically been underrepresented in genomic research.
Imputed genotypes generated from BGE showed excellent agreement with Illumina Global Screening Array data, achieving concordance exceeding 95% for variants with minor allele frequencies above 1%. Importantly, performance remained consistent across multiple ancestry groups and local ancestry backgrounds, addressing one of the major limitations of conventional array-based genotyping.
The platform also demonstrated strong performance for clinically relevant structural variation. Using established computational pipelines, investigators achieved approximately 90% positive predictive value for protein-coding CNVs spanning three or more exons compared with deep whole-genome sequencing. In benchmarking studies, the method successfully detected all validated de novo coding CNVs in a reference autism cohort while maintaining low false-positive rates.
Beyond analytical performance, the study highlights potential operational advantages. By combining genome and exome sequencing into a single workflow, BGE simplifies laboratory processing, reduces the need for multiple assays, and minimizes sample attrition between sequencing platforms. These efficiencies could prove valuable for national biobanks, health system sequencing programs, and pharmaceutical research efforts that increasingly require genomic datasets from hundreds of thousands of participants.
The technology may also advance equity in precision medicine. Because low-pass genome sequencing does not depend on predefined variant content, it avoids many of the ascertainment biases associated with traditional genotyping arrays. The authors found that BGE captured substantially more coding and noncoding variants than array-based approaches while maintaining high-quality rare variant detection through deep exome coverage.
The researchers acknowledge that imputation performance remains influenced by the diversity of available reference panels, particularly for Indigenous American ancestry. However, as more globally representative reference datasets become available, they expect the accuracy of low-pass genome imputation to improve further.
As precision medicine increasingly depends on large, ancestrally diverse genomic datasets, technologies that balance cost, scalability, and comprehensive variant detection will be essential. BGE sequencing offers a practical alternative to deep whole-genome sequencing, enabling broader participation in genomic discovery while preserving much of the analytical power needed to identify clinically meaningful genetic variation.
The post Blended Genome–Exome Sequencing Slashes Costs Without Quality Loss appeared first on Inside Precision Medicine.