Tag: Medication

Posted on

Brain-Targeted Drug Discovery Barriers Drive Deep Science Ventures and Medicines Discovery Catapult Deal

Deep Science Ventures (DSV) and Medicine Discovery Catapult (MDC) agreed to collaborate to address challenges in delivering medicines into the brain.

One of medicine’s greatest challenges is ensuring that treatments reach the precise area of the body where they are needed. While recent scientific breakthroughs have identified numerous targets for neurological conditions, the difficulty of effectively transporting these treatments across the blood-brain barrier and into the central nervous system (CNS) remains a primary challenge for global health.

According to the World Health Organization’s Global Status Report on Neurology, over 40% of the global population is living with CNS diseases, making them a leading global cause of ill health and disability.

Directly addressing critical gaps in healthcare means these innovations have the potential to improve patient outcomes while creating clinical and commercial opportunities for biotech and pharma companies. Developing new solutions could unlock access for rare neurological disorders and expand treatment to large or underserved patient populations, including those with diseases such as Alzheimer’s, Parkinson’s, and various brain cancers.

The first phase of the partnership will see an in-depth review of the current medicines landscape conducted to identify opportunities for innovation. This information will then be used to identify systemic gaps in brain-entry technologies. The long-term ambition is for novel approaches that meet the investment criteria of the partners to be spun out into new ventures focused on high-impact solutions and to provide them with pre-seed funding.

Alzheimers research
Credit: Minerva Studio/Getty Images

A core part of DSV’s approach involves building future founding teams to form new companies that will address challenges across multiple sectors. Future founders will work on opportunities that have been pre-scoped by DSV, de-risking the standard founder proposition.

By combining DSV’s venture-building model with MDC’s drug discovery expertise and infrastructure, the partnership will aim to develop new approaches to ensure life-changing medicines reach the brain, according to Adam Tomassi-Russell, senior director, DSV.

“The blood-brain barrier remains one of the most complex issues in modern medicine and with over 40% of the world’s population facing neurological conditions, it’s imperative that we find an optimal solution to this problem,” said Tomassi-Russell. “By pooling our venture-creation expertise with MDC’s discovery capabilities, we can offer the right founders a frictionless environment in which to tackle the CNS delivery gap. If we can solve the ‘how’ of brain entry more effectively, we can unlock a new frontier of CNS therapeutics and address the huge unmet need in these diseases.”

“At MDC, we are committed to transforming bold ideas into better treatments,” added Nicola Heron, chief strategy officer, MDC. “This collaboration presents an opportunity to discover new technologies that could have a significant impact on patients and society. Through this partnership, we will strengthen the ecosystem for CNS innovation in the U.K. and beyond, enabling more medicines to reach patients faster.”

 

The post Brain-Targeted Drug Discovery Barriers Drive Deep Science Ventures and Medicines Discovery Catapult Deal appeared first on GEN – Genetic Engineering and Biotechnology News.

Posted on

Metabolic syndrome and perioperative neurocognitive disorders: epidemiology, mechanisms, and interventions

Perioperative neurocognitive disorders, an umbrella term encompassing preoperative cognitive impairment, acute postoperative delirium, and longer term postoperative neurocognitive disorders, represent significant complications for the growing population of older surgical patients. The rising prevalence of metabolic syndrome, defined by the clustering of abdominal obesity, insulin resistance, hypertension, and dyslipidemia, necessitates a deeper understanding of its impact on the perioperative brain. This comprehensive review elucidates the intricate epidemiological and mechanistic links between metabolic syndrome and the spectrum of cognitive decline. Epidemiologically, we disaggregate the risk profiles of individual components, demonstrating that distinct metabolic phenotypes serve as specific predictors for different phases of impairment. Mechanistically, we propose a sequential pathophysiological cascade where chronic systemic inflammation primes the brain for injury. Surgical stress triggers the failure of a compromised blood brain barrier, leading to the activation of the TLR4/NLRP3 inflammasome and the induction of central insulin resistance. These processes ultimately culminate in mitochondrial energy crises and synaptic degradation. To address these vulnerabilities, we evaluate an integrated perioperative strategy spanning preoperative metabolic optimization, intraoperative management, and emerging pharmacological interventions such as SGLT2 inhibitors and mitochondria targeted antioxidants. Critically, this review identifies a major knowledge gap regarding the absence of dedicated randomized controlled trials targeting the surgical metabolic syndrome population. Ultimately, our findings advocate for a clinical paradigm shift toward phenotype specific metabolic optimization to improve neurocognitive outcomes in these high risk patients.
Posted on

Autonomic nervous system dysfunction in irritable bowel syndrome: pathophysiology and therapeutic implications

This review synthesizes current evidence on autonomic nervous system (ANS) dysfunction in irritable bowel syndrome (IBS). Patients with IBS often exhibit sympathovagal imbalance–reduced vagal tone with relative sympathetic hyperactivity–which correlates with symptom severity and shows subtype specificity. The ANS orchestrates bidirectional brain–gut communication via interactions with psychosocial factors, low-grade neuroinflammation, and the gut microbiota. Key mechanisms include vagal afferent signaling by microbial metabolites, sympathetic regulation of mucosal immunity, stress-induced disruption of autonomic homeostasis, and neuroplastic changes in intestinal and central pain pathways. Emerging evidence supports therapeutic targeting of autonomic circuits through vagus nerve stimulation, pharmacological modulation of serotonin and adrenergic receptors, and microbiome-based interventions. Current challenges include methodological limitations in assessing neural dynamics and insufficient integration of multi-system interactions. Future research should employ multi-omics approaches to elucidate pathway-specific mechanisms and develop precision medicine strategies for this heterogeneous disorder.
Posted on

Post-Stroke Arm Immobility Improved with Spinal Cord Stimulation

Stroke is the leading cause of arm and hand hemiparesis, weakness or paralysis on one side of the body, with about 80% of stroke patients experiencing at least partial weakness. Many stroke survivors rank improving arm function as a high priority for recovery, but effective treatments are not currently available.

Researchers at the University of Pittsburgh have just published the results of a pilot clinical trial to address this unmet need. Their work, which uses cervical spinal cord stimulation (SCS) to enhance arm and hand function was published in Nature Medicine.

“From a clinical perspective, even modest improvements in arm strength or control can make a meaningful difference in daily life of stroke survivors,” said study co-author George Wittenberg, MD, PhD, professor at the University of Pittsburgh School of Medicine.

This small pilot study included seven patients with varying levels of muscle hemiparesis caused by stroke. All patients presented with profound motor defects, with Fugl-Meyer Assessment (FMA) scores ranging from 15 to 35 (for context, the higher the score, the less impairment, with scores below 50 indicating severe impairment and scores over 100 indicating minimal to no impairment). Each patient was implanted with two leads implanted unilaterally in the cervical spinal cord and underwent SCS stimulation and worked on motor activity under researcher guidance over a four-week period.

During SCS testing, all patients experienced improvement in motor function with an average increase of 32% in strength and increase of 5.6 FMA points on average. After approximately 8.6 hours of motor activity (5.5 hours of which were with the SCS on), the FMA scores improved on average by 6.6 FMA points.

Researchers adjust spinal cord stimulation settings in real time to test their effects on arm and hand movement during stroke rehabilitation. [UPMC and University of Pittsburgh Health Sciences]

“This approach is designed to rapidly help people move their arms better, even years after a stroke,” said co-senior author Marco Capogrosso, PhD, assistant professor at the University of Pittsburgh and director of the spinal cord stimulation laboratory at Rehab Neural Engineering Labs in the UPMC Rehabilitation Institute.

“The stimulation works mostly as an assistive technology—when it’s on, people can move better,” Capogrosso continued. “By stimulating the spinal cord, we can immediately allow residual connections between the brain and the spinal cord to work more efficiently, enabling better movement.”

“Some of the improvements we measure may look small from the outside, but many stroke survivors are just on the verge of being able to do something important,” said Wittenberg. “Even a small change in motor function can be very significant if it helps someone button a shirt, open their hand or return to an activity they care about.”

While the patients in the study experienced immediate improvements to motor function, maintained improvement depended on continued use of SCS. Additional assessments showed motor function declined without continued stimulation, suggesting that the future use of SCS may not be with short-term use in rehabilitation but rather an assistive neuroprosthetic technology.

“This study represents the conclusion of our initial feasibility phase and an important step toward real-world clinical application,” Capogrosso said.

Based on the positive results from this pilot program, the researchers plan to expand the patient poll for a larger clinical trial to better understand the consequences of long-term SCS use, with and without physical therapy.

“Our goal is to develop a technology that could eventually be used in everyday life, not just in the clinic,” Capograsso concluded. “These results give us confidence that spinal cord stimulation could become a practical, implantable option for helping stroke survivors use their arms when it matters most.”

The post Post-Stroke Arm Immobility Improved with Spinal Cord Stimulation appeared first on Inside Precision Medicine.

Posted on

Infant Brain Development Sheds Light on Parkinson’s Link with Autism

Changes in the brains of babies shortly after birth could help explain why people go on to develop Parkinson’s disease (PD) and why this is particularly common in people with autism spectrum disorder (ASD).

The findings, in Science Advances, suggest a reason why humans are vulnerable to neurodegenerative diseases in a way that is not seen among other primates.

Researchers were surprised to find increased expression in genes associated with PD among humans that was not found in chimps or rhesus monkeys.

Human changes in regulatory DNA operated across two cell lineages, one linked with autism and the other with PD.

“Patients with ASD are also more likely to develop PD, a link that is currently not understood,” commented the researchers, led by Menno Creyghton, PhD, from Erasmus University Medical Center in the Netherlands.

“Our analysis linking the two diseases through a common network could provide insight into common biology between the two diseases.”

Human brains have several differences to those of primates, including a larger volume, more projections between outermost areas, and greater complexity of neurons in the upper layer.

The researchers note that, soon after birth, babies experience protracted brain development that coincides with periods of extensive synaptic modeling.

To investigate differences with our close evolutionary relatives, they conducted a single-cell transcriptomic and epigenomic analysis of human, rhesus macaque, and chimpanzee brain tissue during early postnatal development.

Through this, the team identified human infant–specific programs and cell states that were not observed in adults and correlated these early life processes with susceptibility to PD in aged neurons.

Specifically, Creyghton and co-workers identified human gene expression changes in immature oligodendrocytes enriched in both ASD risk genes and gene expression losses in patients with ASD.

There were also gene expression gains in human babies enriched in PD risk genes for and among genes dysregulated in PD patients.

Both transcriptional programs were part of a core network that contained human-specific sequence changes in regulatory DNA. This lacked cell lineage specificity, operating in both the oligodendrocyte and neuronal lineages, with different downstream targets for each.

In the oligodendrocyte lineage, the downstream target genes were overrepresented in autism risk genes whereas in the neural lineage, PD risk genes were enriched.

“It is therefore possible that these pathologies could be maintained in the human lineage by complex antagonistic interactions across developmental stages,” the authors speculated.

They noted the “unexpected” correlation between human evolution and Parkinson’s disease.

“It has been suggested that humans are exceptionally vulnerable to neurodegenerative diseases including PD as similar disease pathology has not clearly been observed in other primate species,” the team pointed out.

“We show that the human infant–specific expression changes that are overrepresented in PD-deregulated genes are enriched in synaptic developmental genes.

“Imbalances in synaptic function can strongly affect neural vulnerabilities, especially in larger projection neurons. As such, pathological deregulation of such a program may play a role in PD susceptibility.”

The post Infant Brain Development Sheds Light on Parkinson’s Link with Autism appeared first on Inside Precision Medicine.

Posted on

STAT+: Supreme Court backs generic drugmaker in ‘skinny labeling’ case

The Supreme Court unanimously ruled that Hikma Pharmaceuticals did not infringe patents held by Amarin in a decision that makes generic drugmakers less vulnerable to lawsuits over so-called skinny labels.

The ruling overturned a lower court decision that sided with Amarin. Generic drugmakers had argued that, if the Supreme Court also ruled in favor of Amarin, they would be discouraged from making and selling lower-cost versions of brand-name medicines, which would maintain higher prices for prescription drugs.

At issue is skinny labeling, which refers to moves by generic companies that seek regulatory approval to market a medicine for a specific use, but not other patented uses for which a brand-name drug is prescribed. For instance, a generic drug could be marketed to treat one type of heart problem but not another. In doing so, the generic company seeks to avoid lawsuits claiming patent infringement.

Continue to STAT+ to read the full story…

Posted on

Cultural and intergenerational pathways between family alcohol use, trauma exposure, and probable PTSD in Taiwanese adolescents

BackgroundPosttraumatic stress disorder (PTSD) and alcohol use (AU) frequently co-occur, yet little is known about how family drinking patterns shape trauma exposure and PTSD risk in non-Western and Indigenous adolescent populations. Taiwan’s multi-ethnic eastern region—home to Amis and Atayal Indigenous groups—provides a unique context for examining culturally specific trauma pathways.ObjectiveTo examine how caregiver and adolescent alcohol use relate to exposure to traumatic events (TEs) and probable PTSD likelihood among Han Chinese and two Indigenous groups, Amis and Atayal, in eastern Taiwan.MethodsA cross-sectional survey was conducted among 751 junior-high students (Han, Amis, Atayal) in rural eastern Taiwan. Adolescents completed the Chinese UCLA PTSD Reaction Index and a trauma-exposure checklist. Caregiver and adolescent AU were assessed via self-report. Logistic regression tested associations among caregiver and adolescent AU, trauma exposure, and probable PTSD likelihood, adjusting for demographic factors.ResultsCaregiver AU was significantly associated with both trauma exposure and probable PTSD across all ethnic groups. While adolescent AU was associated with trauma exposure among Han and Amis youth, no such link was found for Atayal adolescents. Furthermore, exploratory testing rejected the self-medication hypothesis, lending support to the high-risk environmental framework. Trauma types most strongly associated with probable PTSD differed by ethnicity: sexual trauma and painful medical procedures among Han youth; bereavement among Amis adolescents; and witnessing community violence among Atayal adolescents. Indigenous participants showed consistently higher trauma exposure than Han peers.ConclusionsFamily drinking patterns exert strong intergenerational effects on adolescent trauma exposure and probable PTSD risk. The culturally distinct trauma profiles across Han, Amis, and Atayal adolescents highlight the need for trauma-informed, culturally grounded, and family-centered mental-health interventions. Reducing caregiver AU and addressing structural inequities affecting Indigenous communities may offer critical leverage points for preventing PTSD among youth in Taiwan and comparable multi-ethnic settings.
Posted on

Spectral EEG-guided adaptive neuromodulation for social anxiety disorder, performance-only subtype: a case report

Social anxiety disorder (SAD) is often complicated by anticipatory anxiety, hyperarousal, and variability in response to first-line treatments such as cognitive behavioral therapy (CBT) and pharmacotherapy. These limitations highlight the need for adjunctive modalities capable of enhancing the magnitude and durability of treatment response. This case report explores outcomes following spectral EEG-guided personalized repetitive transcranial magnetic stimulation (PrTMS) in a patient with SAD, performance-only subtype. Weekly psychometric assessments revealed reductions in social anxiety intensity, avoidance behaviors, and negative self-appraisal, along with improvements in mood and daily function. Symptom changes were quantified using the Liebowitz Social Anxiety Scale (LSAS), Social Phobia Inventory (SPIN), Patient Health Questionnaire-9 (PHQ-9), Generalized Anxiety Disorder 7-Item Scale (GAD-7), and Quality of Life Enjoyment and Satisfaction Questionnaire Short Form (Q-LES-Q-SF). Clinical improvements were accompanied by increased alpha band power, decreased delta band power, and an increased alpha-delta ratio on serial spectral resting-state EEGs. Our findings suggest that PrTMS may warrant further investigation as a potential adjunctive treatment for SAD. Further large-scale, blinded, and randomized studies are warranted to validate our observations on the feasibility of PrTMS for SAD.
Posted on

A socially assistive robot to support mental wellbeing in LGBTQ+ young people at risk of self-harm: a randomized controlled trial

Nature Medicine, Published online: 04 June 2026; doi:10.1038/s41591-026-04422-6

A randomized controlled trial integrating Purrble, a socially assistive tactile robot, into safety planning found that LGBTQ+ youth at risk of self-harm experienced improvements in Difficulties in Emotion Regulation Scale and fewer depressive symptoms compared to safety planning alone.