Background: Dietary counseling is an essential complement to the growing use of glucagon-like peptide-1 receptor agonists (GLP-1 RAs) to achieve sustainable weight loss. Diverse patient needs, the demand for ongoing support, and limited resources underscore the potential of using digital support tools. Objective: This study aimed to evaluate the usability, treatment satisfaction, and weight effects of a personalized nutrition advisor (PNA), a prototype decision-support tool designed to facilitate personalized weight management counseling. Methods: We assessed usability, treatment satisfaction, and weight effects of a web-based PNA prototype dashboard designed to help dietitians deliver personalized remote dietary counseling in overweight adults (BMI ≥28 kg/m² at pharmacotherapy initiation) who had achieved ≥5% weight loss with GLP-1 RA therapy over 12-weeks. The PNA dashboard generated tailored nutritional recommendations to help participants achieve personalized weight loss goals based on an energy balance model that estimated daily caloric intake and nutritional quality using frequent body weight measurements, dietary logs, and physical activity data from a lifestyle-tracking app. To evaluate the PNA’s usability, we used a convergent parallel mixed-methods approach, combining a validated quantitative usability survey and qualitative focus-group interviews and feedback questionnaires with both dietitians and patients, alongside patient-reported satisfaction metrics for PNA-guided consultations (treatment satisfaction, general self-efficacy, and perceived health impact). Thematic analysis with a hybrid deductive-inductive methodology was applied to qualitative data, using 4 a priori themes derived from this study’s conceptual framework regarding prior expectations, perceived utility and satisfaction, usability challenges, and suggestions for clinical integration. Percent weight loss during the intervention was assessed as an exploratory outcome. Results: Among 78 participants (58/78, 74% women), on GLP-1 RA (mean weight loss of −13.3%, SD 6.7%, after 10, SD 5, months of treatment), between 3 and 5 remote nutrition consultations were delivered with PNA support. After the 3-month intervention period, weight loss was sustained in all participants (−0.2%, SD 2.5%), with 21% (16/78) suspending the GLP-1 RA due to drug shortages. Dietitians rated the PNA’s usability as moderate (62%) on the Healthcare Systems Usability Scale. Patients evaluated the perceived health impact of the PNA-assisted consultations at 19.1 (SD 3.5) of 25 on the user version of the Mobile Application Rating Scale. No significant differences were observed in patient-reported treatment satisfaction (=.78) or self-efficacy (=.57) before and after PNA-assisted care. Qualitative analysis identified convergence with quantitative findings across themes of usability, treatment satisfaction, and clinical integration, with divergence observed for self-efficacy and tracking motivation. Both patients and dietitians found the PNA valuable for generating actionable nutritional insights. Conclusions: While effective implementation of tools such as the PNA prototype demands dietitian training and workflow integration, they offer scalable, personalized nutrition or lifestyle guidance—as a therapeutic decision-support tool within weight loss interventions, with or without adjunct GLP-1 RA pharmacotherapy. Trial Registration: ClinicalTrials.gov NCT05997771; https://clinicaltrials.gov/study/NCT05997771
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Inside the first-of-its-kind Enable Injections enFuse drug delivery wearable
When Sanofi won FDA approval in July 2026 for the first anticancer treatment administered with an on-body injector (OBI), the Enable Injections enFuse platform made it possible. The enFuse OBI secured its initial combination product FDA approval in 2023 for adults with paroxysmal nocturnal hemoglobinuria, a rare blood disorder. That milestone made enFuse the “first…
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Spinal Cord Injury Stem Cell Transplant Clears Phase I, Improves Motor Scores
Researchers from Keio University School of Medicine and Keio University Regenerative Medicine Research Center in Japan transplanted neural stem/progenitor cells derived from induced pluripotent stem cells (iPSCs) into the injured spinal cords of four men with recent, complete cervical spinal cord injuries. The first-in-human Phase I study, published in Nature Medicine, primarily evaluated safety, following participants for up to four years after treatment.
Spinal cord injury affects more than 20 million people worldwide and often results in permanent paralysis because the adult spinal cord has only limited capacity to regenerate. Current treatments—including surgery and rehabilitation—can stabilize the injury and maximize remaining function but cannot rebuild the damaged neural circuits responsible for movement and sensation.
The transplanted cells were manufactured from clinical-grade iPSCs under strict quality controls before being differentiated into neural stem/progenitor cells. Approximately two million cells were injected directly into each patient’s spinal cord injury site two to four weeks after injury while patients received temporary immunosuppressive therapy to reduce the risk of rejection.
The trial met its primary objective. Researchers observed no tumor formation, abnormal cell growth, or other serious complications attributable to the transplanted cells during the initial 52-week study period or the subsequent long-term follow-up. Imaging studies likewise revealed no evidence of graft-related abnormalities, addressing one of the field’s greatest concerns regarding therapies derived from pluripotent stem cells.
Although safety was the principal endpoint, investigators also tracked neurological recovery. All four participants showed improvements in motor function, and two improved enough to advance from complete paralysis (American Spinal Injury Association Impairment Scale grade A) to grades C or D, indicating recovery of some voluntary movement below the injury level. Median motor scores improved by 13 points after one year, exceeding the recovery typically observed in a comparable historical patient registry, although the researchers caution that the small, uncontrolled study cannot establish that the stem cell treatment caused these gains.
The encouraging results build on years of preclinical research showing that transplanted neural stem cells can differentiate into neurons and supporting cells, promote remyelination, stimulate regrowth of damaged nerve fibers, and release molecules that support tissue repair. Animal studies have also suggested that the transplanted neurons can integrate into existing spinal cord circuits, although such integration cannot yet be directly confirmed in patients.
The investigators emphasize that many questions remain before the therapy could become a standard treatment. The study enrolled only four participants, lacked a placebo control, and included only men with recent cervical spinal cord injuries. Larger randomized clinical trials will be needed to determine whether the treatment consistently improves neurological recovery and to identify which patients are most likely to benefit. Researchers also plan to continue monitoring participants to assess the long-term safety of the transplanted cells.
Even with those caveats, the findings represent a significant advance for regenerative medicine. Rather than demonstrating a cure for paralysis, the study establishes that carefully manufactured iPSC-derived neural stem cells can be transplanted into the human spinal cord without the serious safety issues that have long challenged the field. That achievement provides a critical foundation for the next generation of clinical trials aimed at determining whether stem cell therapy can ultimately restore function after devastating spinal cord injuries.
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Pregnancy Complications Increase the Risk of Peripheral Artery Disease
Having a common birth complication such as preterm birth, preeclampsia, high blood pressure and gestational diabetes puts women at increased risk of developing peripheral artery disease in later life.
As reported in PLOS Medicine, all five of the common complications listed were associated with an increased risk for peripheral artery disease, a form of atherosclerosis in the legs, up to 46 years after giving birth in a large Swedish study.
Overall, women with gestational diabetes had almost four-times higher risk of developing peripheral artery disease than women who had a healthy pregnancy with preeclampsia, high maternal blood pressure, preterm birth or small for gestational age babies increasing risk 1.3–1.7-fold.
“Peripheral artery disease affects more than 230 million people worldwide and is a strong predictor of future stroke, ischemic heart disease, and premature mortality,” write Casey Crump, MD, PhD, an epidemiologist and family physician at McGovern Medical School, and colleagues.
“Despite its high prevalence and clinical importance, it is understudied compared with other cardiovascular diseases.”
Pregnancy complications such as gestational diabetes and preeclampsia are known to increase the risk of other types of cardiovascular disease so Crump and team carried out a study to assess if this was also the case for peripheral artery disease.
The team drew on Swedish national birth and health registries to follow more than 2.2 million women who had single‑baby pregnancies between 1973 and 2015. They focused on the five common birth complications and tracked new diagnoses of peripheral artery disease from hospital, specialist clinic, and primary care records for up to 46 years after childbirth.
The researchers compared women with and without these complications while accounting for age, education, income, smoking, weight, and existing conditions like hypertension or diabetes. The also compared sisters to check whether shared genes or family lifestyle could fully explain the patterns they saw.
Overall, 13,211 women developed peripheral artery disease, mostly in their early 60s. All the complications were linked to higher risk of arterial disease, with the association becoming stronger with age. Experiencing more than one complication also increased risk further and women with three or more had around three times higher risk of peripheral artery disease in later life than women with none. The research also showed links were not likely to be genetic or related to a shared upbringing.
“Women who experience an adverse pregnancy outcome need early preventive actions and long-term clinical follow-up to reduce their risk for peripheral artery disease and other associated cardiovascular diseases,” write the authors.
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Click Chemistry Boosts Antibody–Drug Conjugates Against Heterogeneous Tumors
Antibody–drug conjugates (ADCs) have transformed cancer treatment by delivering potent cytotoxic drugs directly to tumor cells. But because each ADC recognizes only a single molecular target, many cancers become difficult to treat as tumors evolve into a patchwork of cells with different biological characteristics. A preclinical study published in Nature describes a modular strategy that enables existing antibodies and ADCs to assemble inside the body, allowing them to engage multiple tumor targets without creating entirely new drugs.
Researchers at Washington University School of Medicine modified FDA-approved antibodies and HER2-directed ADCs with complementary chemical groups that bond together through bioorthogonal “click chemistry” after sequential administration. The resulting complexes produced stronger antitumor activity than conventional ADC therapy in mouse models of pancreatic, gastric, and breast cancer.
The work addresses a longstanding obstacle for ADCs: they are most effective when nearly every tumor cell displays the same molecular target.
“Strategies that enhance ADC delivery to the tumor without requiring uniformly high antigen expression are needed to address heterogeneous and treatment-resistant tumors,” the authors write.
To test that concept, the researchers paired HER2-directed ADCs with antibodies targeting either HER2 or EGFR, a receptor frequently associated with treatment resistance. Because the molecules assemble after entering the circulation, the approach offers a modular alternative to engineering entirely new bispecific antibodies.
The strategy proved particularly effective in cancers with low, ultralow, negative, or heterogeneous HER2 expression, expanding activity into tumors that often respond poorly to existing HER2-targeted therapies.
“This strategy enabled targeted delivery of HER2-directed ADCs… to EGFR-high or EGFR-low cancer cells across the spectrum of HER2 expression,” the authors conclude.
The improved targeting translated into substantially better outcomes in the pancreatic cancer model. About 90% of treated mice survived for 120 days, whereas animals receiving conventional ADC therapy survived less than 80 days on average. The researchers also optimized the platform to reduce off-target drug accumulation in the liver, an important consideration for limiting toxicity.
Rather than relying exclusively on one receptor to deliver therapy, the system can exploit interactions between multiple tumor-associated receptors to improve drug uptake.
“The therapeutic benefit probably reflects a combination of bioorthogonal chemistry-driven ligation and tumor receptor biology rather than exclusively receptor colocalized assembly,” the authors write.
The click chemistry platform also may accelerate development of precision therapies. The linking molecules can be manufactured in as little as one to three days, making it possible to rapidly pair different clinically approved antibodies and ADCs for individual tumor profiles. Although demonstrated using HER2-targeted therapies, the researchers say the modular approach could be adapted to many receptor combinations and cancer types. They are particularly interested in applying the technology to difficult-to-treat tumors such as brain cancers, where improving drug delivery remains a major challenge.
“Beyond direct cytotoxic delivery, the modular customization of this approach offers potential for immune modulation, diagnostic imaging and combinatorial payload strategies,” the authors conclude. “As such, this approach provides a flexible foundation for the optimization of antibody-based therapeutics in oncology and other diseases.”
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STAT+: Rare disease drugmakers try to avoid Trump price cuts
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Messaging wars: affordability vs. fraud
Rising out-of-pocket health care costs remain voters’ top health priority overall, but Republicans are more worried about fraud in government health programs, according to a KFF poll.
Exploring the Relationship Between Parental Trauma, Parenting Style, and Children’s Medical Treatment Adherence: Cross-Sectional Study
Background: Medication adherence is defined as the degree to which the medications taken reflect the prescribed intention and is influenced by various factors. Different factors, such as parental trauma or parenting style, may influence their child or children’s medical treatment adherence. Authoritative, or “flexible,” parenting is known to develop the most nurturing relationship between parent and child or children. The relationship between parental factors, such as parenting style and trauma level, and their influence on child medication adherence is being explored. Objective: This study examines the relationship between parental trauma, parenting style, and their child or children’s adherence to medical recommendations. Methods: Participants were recruited through Amazon’s Mechanical Turk, an online crowdsourcing platform. Participants were aged 18 years or older, a caregiver for at least 1 child younger than the age of 18 years, and had at least 1 child who was prescribed medication within the past 3 months. Parental trauma level was measured via the Adverse Childhood Experiences Questionnaire, and parenting style was measured via the Parental Authority Questionnaire–Revised. Descriptive statistics, including chi-square tests, 2-tailed tests, and adjusted logistic regressions, were used to determine the association between parental trauma, parenting style, and their child or children’s medical adherence. Results: A total of 720 participant responses were analyzed. Children who were medically adherent were younger (=.02). Caregivers’ mean age was 36.8 (SD 7.97) years, and 35.4% (n=255) identified as male. An increase in caregivers’ adverse childhood experiences score was marginally associated with an increased risk of medication nonadherence among their children (adjusted odds ratio 0.94; =.07). Conclusions: Similar to other studies, this study showed that parents of children who adhere to medical treatment practice an authoritative parenting style. Uniquely, it also showed that an increased adverse childhood experiences score was only marginally associated with an increased risk of medical nonadherence.
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Blood Cancer Therapies Informed by Spatial Remodeling of Bone Marrow
In a new study published in Leukemia titled, “Spatial remodeling of bone marrow architecture defines tissue-state signatures of disease activity and therapeutic response in myelodysplastic neoplasm,“ researchers from Weill Cornell Medicine have developed a new AI-based method to assess patients with a form of blood cancer called myelodysplastic neoplasms (MDS). The approach compares the size and shape characteristics of hematopoietic cells in the patient’s bone marrow sample with those in healthy bone marrow to generate a score that reflects disease severity.
MDS is a form of blood cancer that usually affects older adults. About one-third of patients progress to a more aggressive cancer called acute myeloid leukemia. MDS patients must undergo frequent biopsies to identify signs of remission or worsening disease.
“It’s fundamentally a chronic and progressive disease,” said Sanjay Patel, MD, clinical chief of hematopathology, and an associate professor of pathology and laboratory medicine at Weill Cornell Medicine. “With the current methods pathologists use to assess MDS samples, there are some clear-cut cases and a lot of gray area.”
The researchers developed a new method using fully deidentified patient samples that may provide more clarity for patients with MDS and their physicians.
“Using AI to assist us in looking at the spatial architecture of the bone marrow using widely available laboratory assays allows us to improve our capability to assess patients’ prognosis and perhaps triage them for precision therapies,” said David Redmond, PhD, assistant professor of computational biology research in medicine at Weill Cornell Medicine.
The MDS-Microarchitectural Perturbation Score (MDS-MAPS) score ranks patient samples based on 82 features associated with normal tissue and different genetic subtypes of MDS. The tool uses routinely collected samples, tissue staining protocols, and imaging technologies that could be implemented in most hospital pathology departments and laboratories.
“We can generate an MDS-MAPS value for a patient at diagnosis and track how it changes over time,” said Patel.
The next step will be to validate the method in larger cohorts of patient samples. The researchers will collaborate with Pinkal Desai, MD, associate professor of medicine at Weill Cornell Medicine and a hematologist/oncologist at NewYork-Presbyterian/Weill Cornell Medical Center, to study how the tool performs on other forms of MDS, and increasingly recognized precursor conditions.
“Patients with MDS and related precursor conditions have many mutations as part of the disease biology and each patient’s molecular signature is different,” said Desai. “We have always wondered if these mutations signal a different spatial pattern and whether these patterns have an impact in predicting how patients progress and respond to treatments. Together we are poised to harness technology to answer real-world clinical questions.”
The post Blood Cancer Therapies Informed by Spatial Remodeling of Bone Marrow appeared first on GEN – Genetic Engineering and Biotechnology News.
New Center for Therapeutic Genetics to Develop Rare Disease Genetic Medicines
It’s estimated that nearly 400 million people across the globe live with a rare disease. Though many of these diseases are progressive, debilitating, and even life-threatening, less than five percent of them have an effective and approved treatment.
Nearly half of the rare disease patients are children and it’s estimated that 30% of children with a rare disease will not survive past their fifth birthday. Though there have been some major successes in developing and providing treatment for some children with rare disease, including Baby KJ Muldoon—who was the first patient to receive a personalized CRISPR gene editing therapy—N = 1 therapies are challenging on many levels, and these stories cannot be the functional standard practice for treating patients with rare disease.
“What if treating genetic disease could be as routine as life-saving surgery?” ask David Liu, PhD, core institute member at the Broad Institute, and Winston Yan, MD, PhD, physician-scientist at the Broad institute and director of the new Center for Therapeutic Genetics (CTG) which is working to answer this very question.
“Scaling and sustaining treatment for ultra-rare disease is a hard problem that many scientists, clinicians, patients, and drug developers are working on,” says Yan.
Along with Yan and Lui, the CTG is founded by leaders in genetic medicine, including Cat Lutz, PhD, vice president, of The Jackson Laboratory’s rare disease translational center, Timothy Yu, MD, PhD, physician and researcher at Boston Children’s Hospital, and Wendy Chung, MD, PhD, chief of pediatrics at Boston Children’s Hospital. Their aim is simple: to develop genetic medicines that can treat patients with rare diseases in a scalable and repeatable way, while sharing the methods, data, and training learned through this practice.
“What we have here is a group of leaders who believe that by approaching genetic medicines not as products, but as a standardized clinical procedure, and by sharing what we learn openly across institutions, we can make precision genetic medicine faster, safer, less expensive, and more accessible to patients and families in need,” says Yan.
The impetus behind developing the CTG is centered on patient care and utilizing ever growing biotechnology.
“We receive messages every week from parents asking for help: Can we do for their child what has been done for other children who have received treatments?” shares Liu. “The honest answer today is usually ‘not yet,’ often not because the science doesn’t exist, but because we don’t yet have the infrastructure to bring these treatments to many patients. CTG is our commitment to closing that gap, so that eventually every family who needs this kind of treatment has a path forward.”
Though the center is in its founding phase and not yet working with patients, the programs are anticipated to include the development of precision gene-editing treatments for multiple diseases, including rare forms of genetic epilepsies, supported by a recent $34.5 million award from the ARPA-H THRIVE program.
“What we are building together is scalable treatment, something our institutions will learn to do well and repeat again and again. At Boston Children’s, we see children every day for whom a diagnosis is only the beginning of a much longer journey,” says Chung.
By utilizing technology, like CRISPR or base editing or prime editing, the goal is to develop a scalable system, not just one-off treatments. The founders of CTG aim to develop programmable and modular medicines that can be customized to the patient needs, not unlike the tailored pre-operational plans that are developed for each patient undergoing a standard practice surgery.
“We used to see precisely tailored therapies for children with genetic diseases as remarkable exceptions. CTG is built on the conviction that they don’t have to be—that the methods we develop for one rare disease will carry to the next, and eventually to the many,” says Yu.
Lutz concurs, adding, “We are at a moment in genomic medicine where, for many rare diseases, the question is no longer whether we can treat them, but whether we will build the systems to do it.”
Standardizing precision medical care using programmable genetic medicines for treating rare diseases is only the beginning. Best practices and processes for addressing rare disease can translate to treating more widespread diseases as well.
“CTG exists to close a major gap in care and ensure that what we learn from one disease accelerates treatment for the next,” concludes Chung.
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Curcumin attenuates α-synuclein pathology in Parkinson’s disease model mice through modulation of UBC9-associated SUMOylation signaling
BackgroundPost-translational modifications, particularly SUMOylation, plays a crucial role in α-synuclein (α-syn) aggregation, a key pathological feature of Parkinson’s disease (PD). Curcumin, a natural polyphenol, has shown neuroprotective potential, but its effects on SUMOylation-related signaling in PD remain unclear.ObjectiveThis study aimed to investigate whether curcumin modulates α-syn SUMOylation and to elucidate the underlying molecular mechanisms in PD model mice.MethodsA PD model was established in male C57BL/6 mice via unilateral intrastriatal injection of α-syn preformed fibrils (PFFs). Six months after α-syn PFFs injection, mice were treated intravenously with curcumin (25 mg/kg/day) or vehicle for 1 month. Behavioral tests (open field, rotarod) assessed motor function. Neuropathology was evaluated by immunohistochemistry and western blotting for tyrosine hydroxylase (TH), phosphorylated α-syn (p-syn), SUMOylation pathway components (SUMO1, SAE2, UBC9, PIAS1/2), and ubiquitin. Striatal dopamine levels were measured by HPLC.ResultsCurcumin treatment ameliorated motor deficits and anxiety-like behaviors in PD mice. It partially preserved dopaminergic neurons and reduced p-syn aggregation in the substantia nigra, accompanied by increased striatal dopamine levels. Mechanistically, curcumin was associated with reduced SUMO1 and increased ubiquitin levels, suggesting modulation of SUMOylation-related signaling. Among SUMOylation enzymes, UBC9 expression was decreased, whereas E1 (SAE2) and E3 (PIAS1/2) components were not substantially affected.ConclusionOur findings demonstrated that curcumin exerted neuroprotective effects in a PD model by attenuating α-syn pathology. The protective mechanism involves the inhibition of α-syn SUMOylation, primarily through the downregulation of the UBC9 enzyme. This study identifies UBC9-mediated SUMOylation as a potential target for curcumin and highlight a promising strategy for modifying α-syn-associated pathology in PD.