Tag: Medication

Posted on

STAT+: Pharma’s drug spending deflection

This is the online version of STAT’s weekly email newsletter Health Care Inc. Sign up here.

Hello! Wanna drop some cars in an open pasture? The internet does it again. Drop some tips and feedback here: bob.herman@statnews.com.

PhRMA doesn’t want you to look at drug spending

Lobbying groups frequently send news releases and talking points to journalists as a way to influence reporting. Right now, the pharmaceutical industry’s primary lobbying shop, PhRMA, wants everyone to ignore the clear-cut rise in prescription drug spending. 

Continue to STAT+ to read the full story…

Posted on

STAT+: Pharmalittle: We’re reading about a U.S. peptide panel’s votes, Amgen scuffling with the FDA, and more

Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating because that oh-too-familiar routine of meetings, deadlines, and the like has returned with a vengeance. You knew this would happen, yes? To cope, we are relying, as always, on a cuppa stimulation. Our choice today is Earl Grey, an old standby. Feel free to join us. Remember, no prescription is required. Meanwhile, here are some items of interest to help you on your journey today, which we hope will be productive and meaningful. Best of luck and, of course, do keep in touch. …

A U.S. Food and Drug Administration advisory panel on Friday recommended that compounding pharmacies be allowed to manufacture the peptides epitalon and semax, but narrowly voted to recommend against manufacturing emideltide, STAT writes. The votes, which followed the panel’s decision on Thursday to recommend allowing pharmacies to make four other peptides, bring U.S. Health and Human Services secretary Robert F. Kennedy Jr. one step closer to his mission of making these unapproved compounds more available for Americans. Peptides have become increasingly popular in the U.S., driven by endorsements from social media influencers.

Amgen submitted new evidence to the FDA ​as it seeks a hearing to challenge the proposed ‌withdrawal of its rare disease drug Tavneos from the U.S. market, Reuters says. In April, the agency proposed withdrawing the drug, which treats a rare autoimmune ​disease that damages blood vessels, citing a lack of proven effectiveness ​and false statements in its original marketing application. Amgen strongly disagrees with the FDA and noted its submission includes more than 70 real-world studies involving over ​2,200 patients supporting the drug’s effectiveness and safety.

Continue to STAT+ to read the full story…

Posted on

Resting-state functional connectivity of the sensorimotor network in medication-naïve Chinese children with ADHD: cross-sectional associations with hyperactivity/impulsivity and executive function

BackgroundAttention-deficit/hyperactivity disorder (ADHD) is a prevalent neurodevelopmental disorder, often—but not always—characterized by inattention, hyperactivity/impulsivity, and deficits in executive functions (EFs); some individuals may also exhibit sensorimotor dysfunction. While SMN dysfunction has been implicated in ADHD, the patterns of SMN functional connectivity (FC) and their relationships with clinical symptoms and EFs remain inconsistent, partly due to methodological heterogeneity.MethodsThe study included 62 medication-naïve patients with ADHD (aged 6–15 years) and 46 healthy controls matched for age, sex, and IQ. Clinical symptoms and EFs were assessed using CPRS, IVA-CPT, and Stroop tests. Resting-state fMRI data were acquired, and ROIs within SMN, SN, DMN, and FPN were selected from the Dosenbach atlas. FC was analyzed using Network-Based Statistics (NBS). Partial correlations explored FC–behavior relationships in the ADHD group, controlling for age, sex, and IQ.ResultsThe ADHD group showed significantly higher CPRS hyperactivity/impulsivity scores and poorer IVA-CPT and Stroop performance. Compared to controls, ADHD showed increased FC within SMN (right posterior insula to left precentral gyrus/parietal lobe), between SMN–DMN (precentral gyrus, dorsal frontal cortex, temporal lobe, angular gyrus, posterior cingulate cortex, and precuneus), and SMN–SN (middle insula, superior parietal lobule, superior temporal gyrus, basal ganglia, and fusiform gyrus). Enhanced intra-SMN FC was negatively correlated with impulsivity/hyperactivity and hyperactivity index scores. Enhanced SMN–DMN FC was negatively correlated with hyperactivity symptoms and positively correlated with control quotients in IVA-CPT. Enhanced SMN–SN FC was negatively correlated with Stroop correct responses and positively correlated with omission errors.ConclusionsIn this medication-naïve pediatric sample, distinct SMN connectivity patterns differentially related to ADHD symptoms and executive function. However, given the cross-sectional design and modest sample size, these associations cannot establish causality; they require replication in longitudinal studies and larger cohorts to clarify whether they reflect developmental variation, neurobiological subtypes, or epiphenomena.
Posted on

Autonomic dysregulation and nonsuicidal self-injury: findings from a cross-sectional online survey

IntroductionDysregulation of the autonomic nervous system has been implicated in nonsuicidal self-injury (NSSI) in laboratory studies of physiological reactivity, but it remains unclear whether self-reported everyday autonomic symptom burden is associated with NSSI severity.MethodsWe conducted a preregistered cross-sectional online survey of N = 1,002 German-speaking adolescents and young adults aged 14–30 years (M = 23.43; 73% women). Autonomic symptoms were assessed with the COMPASS 31; NSSI presence and frequency were measured with the SITBI-R across four reference periods (past week, month, year, lifetime). Data were analyzed using zero-inflated negative binomial models.ResultsThe 12-month NSSI prevalence was 22.6%. Higher COMPASS 31 scores were associated with lower odds of reporting zero NSSI episodes across all reference periods and with higher NSSI episode counts for the past month, past year, and lifetime; the past-week count association was not statistically significant. These associations held after controlling for childhood adversity, perceived stress, medication, and hormonal contraception.DiscussionThese results indicate that self-reported autonomic symptom burden was robustly associated with NSSI involvement, particularly with the likelihood of any NSSI and with episode counts over longer reference periods. These findings suggest that self-reported autonomic symptom burden may represent a clinically relevant somatic correlate of NSSI severity.
Posted on

STAT+: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease

RA Capital and a fleet of other investors are putting $175 million behind Oak Hill Bio, a startup trying to revive an experimental Angelman syndrome drug that Roche shelved. 

Oak Hill is competing with Ultragenyx Pharmaceutical and Ionis Pharmaceuticals, both of which are already running Phase 3 trials of similar drugs for the same disease. Ultragenyx will have results this year.

Oak Hill is betting it has the best molecule.

Continue to STAT+ to read the full story…

Posted on

STAT+: As some medical schools embrace RFK Jr.’s nutrition push, they deftly sidestep MAHA priorities

When students return to McGovern Medical School at UT Health Houston this year, they will get to participate in hands-on nutrition experiences, including cooking demonstrations in the teaching kitchen, tours of the campus “holistic garden,” and the opportunity to take a culinary-medicine-focused elective. 

These immersive programs are a response to health secretary Robert F. Kennedy’s call for medical schools to teach more nutrition — part of the Trump administration’s push to address the rising rates of chronic diseases in the United States. Kennedy has blamed the problem, in part, on doctors not routinely discussing diet and nutrition with their patients.

Lessons in culinary medicine are part of the 71 nutrition-related topics that the Department of Health and Human Services wants future physicians to be well versed in. Other topics in the department’s Advancing Nutrition Education push include enhancing nutrient bioavailability through soaking, sprouting, and fermenting foods as well as prioritizing food-based medicine as the primary approach to manage chronic diseases driven by metabolic dysfunction.

The vast majority of 163 M.D.-granting and 48 D.O.-granting schools aren’t yet part of Kennedy’s new initiative, including several leading institutions. So far, some 73 medical schools — 54 M.D. programs, 19 D.O. programs — across 36 states have signed on.

Even as some medical schools heed Kennedy’s call for more nutrition education, several told STAT that they plan to steer clear of topics that aren’t backed by evidence, including some that align with the priorities of the Make America Healthy Again political movement championed by the health secretary. Nutrition experts cautioned that some of those topics could lead to “wellness creep” in medical education.

Continue to STAT+ to read the full story…

Posted on

Genetic Variants Linked to CAR T-Cell Efficacy and Safety

Scientists have identified variants in three genes that influence both the efficacy of CAR T-cell therapy and the likelihood of severe side effects. Published today in Science Immunology, these findings represent a first step toward adopting a precision medicine approach in an increasingly used form of cancer immunotherapy. 

CAR T-cell therapies have undeniably transformed the treatment of blood cancers, with a rapidly growing number of approvals over the past decade and progressively expanding toward solid tumors. While genetic variants associated with immune-related side effects have been identified for other forms of cancer immunotherapy, such as checkpoint inhibitors, the role of human genetics in CAR T-cell therapy has remained largely unexplored. However, this is particularly important given that most CAR T cells are manufactured from a patient’s own immune cells, meaning the therapy itself carries the patient’s unique genetic makeup. 

“Unlike traditional therapeutics that are essentially identical across all patients, CAR T cells are bespoke and harbor all of the ancestral genetic polymorphisms of their parental T cells,” write the study authors, led by Marcela V. Maus, MD, PhD, professor of medicine at Harvard Medical School and director of the Cellular Immunotherapy Program at Massachusetts General Hospital. 

Maus and colleagues analyzed data from 236 patients with aggressive lymphoma enrolled across two clinical trials, who had been treated with axicabtagene ciloleucel, a CAR T-cell therapy commercialized by Gilead under the name Yescarta. The analysis combined whole-genome sequencing with detailed biomarker and functional analysis to identify variants affecting the treatment’s efficacy and toxicity.

Among patients who developed treatment-induced toxicity, many carried variants of the STXBP2 gene. Follow-up experiments in human T cells showed that these variants increased the production of inflammatory cytokines and activated macrophages. Consistent with these findings, mutations in the STXBP2 gene have previously been linked to inflammatory bowel disease and a rare immune disease marked by excessive T cell activation. 

In contrast, variants in the ADMTSL3 gene were associated with protection against treatment-related toxicity, while variants in the PTPN22 gene were strongly linked to enhanced CAR T-cell expansion—a critical factor that determines treatment efficacy.  

“These findings demonstrate that germline genetics shape the safety and activity of engineered immune cell therapies, affecting future design and patient management,” write the scientists. Going forward, they plan to expand this research into larger patient cohorts and a wider range of CAR T-cell therapies to investigate how different genetic variants influence treatment efficacy and the risk of severe side effects across broader clinical settings. 

Beyond opening new avenues for precision medicine in CAR T-cell therapy, the researchers noted that these findings could also inform the development of off-the-shelf CAR T-cell therapies where donor-derived T cells are used instead of a patient’s own cells. This approach could enable a more precise selection of donor T cells optimized to maximize therapeutic benefit while minimizing toxicity risks. 

The post Genetic Variants Linked to CAR T-Cell Efficacy and Safety appeared first on Inside Precision Medicine.