Research on treating disease with medication. Covers psychedelics, combination cancer therapy, opioid treatment in pregnancy, and personalized guidelines.
A pilot study proposes a promising global genomic assay for diagnosing molecular subtypes in pediatric B-ALL, leading to more accurate diagnosis and targeted treatment options. RNA sequencing analysis accurately identified subtypes in all known cases and determined genetic subtype in 79% of previously unknown cases.
Researchers found that high levels of FSP1 are associated with relapse after cisplatin treatment in head and neck squamous cell carcinoma. Targeting FSP1 can attenuate tumor stemness and downregulate invasion and metastatic rates, suggesting a potential new approach for treating drug-tolerant persister cancer cells.
Researchers develop technology that alleviates retinal pathologies by targeting mitochondrial chaperone TRAP1, which is implicated in the breakdown of blood-retinal barrier and pathological neovascularization. This treatment approach holds great promise for revolutionizing the treatment landscape for ischemic retinopathy.
Researchers developed an mRNA therapeutic that combats ovarian cancer by producing functional p53 protein, shrinking and killing tumors. The treatment is effective against metastases and has shown promise in preclinical studies.
A new study published in Neurology found that young Black and Hispanic women with multiple sclerosis face greater challenges in pregnancy, have more advanced disease, and experience higher inflammation levels, which may signal MS progression. The researchers also noted disparities in healthcare access and treatment.
Researchers developed computational algorithms to analyze patient data and discovered a protein, NME2, that works closely with MYC in advanced prostate cancer cells resistant to enzalutamide. Reducing NME2 levels restored the drug's efficacy, offering new hope for patients who initially respond poorly.
Researchers have effectively targeted three distinct groups of cholangiocarcinoma, a lethal disease caused by liver fluke parasite or herbal carcinogen exposure. Experimental models show that specific treatments targeting these pathways can slow cancer growth and improve prognosis for CCA patients
Researchers found that 94% of patients with posterior cortical atrophy had Alzheimer's pathology, while only 70% of those with memory loss did. Early identification of PCA may lead to better treatment options for Alzheimer's patients.
Stacyann Bailey studies how cancer treatment affects bones, hoping to prevent fractures and improve patient outcomes. Up to 50% of patients with metastatic bone disease develop fractures due to treatment, making her work crucial for their quality of life.
A team of researchers from UMass Amherst and Seattle Children's Research Institute found that prior exposure to certain bacteria changes the lung's innate immune response, making it more vulnerable to tuberculosis. The study suggests that remodeling the innate immune system could be a more effective strategy in fighting TB.
Researchers at Nagoya University have discovered a relationship between ALS progression and the disruption of mitochondria-associated membranes (MAM) and TBK1 activity. Decreased activation of TBK1 is linked to motor neuron death in ALS patients and mice with disrupted MAM.
Researchers at Cleveland Clinic have developed a peptide therapeutic that blocks aggressive cancer cells from multiplying rapidly. The treatment disrupts the molecular processes behind cancer growth and induces tumor cell death, making it a promising new strategy for treating triple-negative breast cancer.
Researchers discuss reductive carboxylation of glutamine as a potential target in acute myeloid leukemia (AML), an aggressive cancer with poor patient outcomes. The approach aims to weaken tumor cell survival mechanisms, potentially leading to novel therapies and improved patient outcomes.
Researchers at St. Jude Children's Research Hospital have developed a molecular glue that sticks to the cancer-related protein casein kinase 1 alpha (CK1α), leading to its destruction. The compound, SJ3149, displays broad anti-cancer activity and may have clinical utility as an alternative to conventional small molecule inhibitors.
A systematic analysis of cancer cells identifies 370 candidate priority drug targets across 27 cancer types. Researchers used machine learning methods to find promising targets and linked them to specific biological markers and genetic features.
A new task force launched by C-Path aims to accelerate drug development for mitochondrial and inherited metabolic diseases. The task force will leverage C-Path's expertise in data management standards, biomarkers, and regulatory science to generate solutions and contribute to regulatory decision-making.
Researchers found a shared genetic trait that predicts a higher risk of small lung vessel disease and its severe form, pulmonary arterial hypertension. This discovery could lead to personalized treatments for patients with limited oxygen response.
Researchers unveiled a previously unknown effect of PG545 in ovarian cancer cells, inducing DNA damage and promoting autophagic degradation of RAD51. This breakthrough could aid in selecting the most appropriate treatments for ovarian cancer patients with PARPi resistance.
Researchers have successfully used mRNA technology to correct a rare genetic disease in mice, demonstrating its potential therapeutic use. The treatment corrected the lethal consequences of the disease and restored glutathione metabolism.
Researchers from Zhejiang University School of Medicine have revealed key connections between clinical characteristics, neuroimaging, and treatment in obsessive-compulsive disorder (OCD). The study found that about 13-36% of patients show poor insight, linked to more severe symptoms and poorer treatment outcomes.
A large-scale German-wide randomized controlled trial found that high-dose therapy with glucocorticoids is not more effective than standard treatment for sudden hearing loss. However, it was associated with a higher risk of side effects. Several hundred thousand people worldwide suffer from sudden hearing loss each year.
A new study from Sylvester Comprehensive Cancer Center suggests that prophylactic treatment can significantly reduce the rate of cytokine release syndrome (CRS) in multiple myeloma patients. This approach could eliminate hospital stays and broaden access to immunotherapy treatments for more cancer patients.
GFH009 inhibits tumor growth and induces apoptosis in various HHM-derived cell lines. The compound's mechanism of action involves rapid 'on-off' inhibition of CDK9, which exerts a proapoptotic effect on cancer cells.
Researchers have developed a new method to generate cyclic peptides that can target diseases and be administered orally, overcoming challenges in protein binding. The approach enables high-throughput screening and has shown substantial bioavailability in rats, opening possibilities for treating various diseases.
The number of new dermatologic drug approvals by the FDA increased significantly between 2012 and 2022, with nearly half considered first in class or indication. This surge in approvals was deemed clinically useful or to have high added therapeutic benefit by health technology assessment organizations.
The use of 'trip-killers' to shorten psychedelic drug trips is a growing concern among doctors, with benzodiazepines and antipsychotics being the most frequently recommended options. However, warnings about their potential side effects are rarely included, highlighting a need for greater awareness.
A clinical trial found that an experimental drug, BI 690517, reduced albuminuria in 70% of patients when paired with a standard-care medication. The study also showed that the combination of the two drugs mitigated the risk of hyperkalemia.
Patients with lower socioeconomic status in their neighborhoods have a higher risk of nonadherence to guideline-directed medical therapy for heart failure. Neighborhood-level socioeconomic factors play a significant role in shaping healthcare outcomes.
A comprehensive review of targeted therapies for lupus nephritis discusses the challenges of current treatments and proposes strategies to overcome obstacles. Recent advancements in B-cell targeting and alternative approaches such as CAR-T cells are highlighted.
Researchers explore the properties of cytostatic persisters in cancer treatment, highlighting their therapeutic potential and challenges. The study suggests that targeting these persisters before resistance emerges can reduce cancer recurrence.
A novel synthesis method enables easy linkage of therapeutic oligonucleotides to peptide markers, streamlining the process and making it more accessible and cost-effective. This breakthrough has the potential to produce more effective and targeted RNA-based drugs.
Using metformin plus insulin to treat preexisting type 2 or gestational diabetes diagnosed early in pregnancy did not show a reduction in composite neonatal adverse outcomes. The study suggests that the effect of reducing large-for-gestational-age infant risk after adding metformin to insulin warrants further investigation.
A Northwestern University study found that nearly two-thirds of patients discontinue or change their medication within a year, highlighting the need for better understanding of barriers to treatment adherence. The high discontinuation rate for GLP-1 RAs may be due to gastrointestinal side effects.
A recent study published in JNCCN found that patients from underrepresented groups treated with first-line immunotherapy for advanced Non-Small Cell Lung Cancer (NSCLC) showed similar survival outcomes as their White counterparts. The study analyzed data from 248 patients and highlighted the need for more diverse patient representation...
A landmark study found that 92% of patients with severe asthma can safely reduce inhaled steroid dose and more than 60% can stop all use, minimizing side effects like osteoporosis, diabetes, and cataracts. The SHAMAL study used biologic therapy benralizumab to control severe asthma without steroids.
Researchers leverage AI to analyze healthcare data and identify new targets for effective therapies and accelerate drug development in aging research. AI can tailor cancer treatment more precisely to individual patients' unique aging profiles, optimizing treatment outcomes and minimizing risks.
Researchers found that a person-centered approach, trusting relationship, and proper training are crucial for effective peer support. Trained peer support can help plug gaps in medication advice and improve patient adherence to treatment.
Researchers at the University of Turku found that bexmarilimab therapy alters macrophage behavior to promote anti-tumor immune defense. The therapy was well-tolerated and stabilized disease progression in patients with advanced-stage cancer, inducing tumor-associated macrophage and lymphocyte activation.
Researchers found that a breakthrough infection builds upon existing memory B cell pool and introduces random mutations that prepare antibodies for new variants. Broad neutralizing antibodies can neutralize all tested Omicron variants.
Researchers found that BCMA-positive extracellular vesicles (EVs) in plasma levels correlated with myeloma patient responses to belantamab-mafodotin therapy. High EV levels preceded FLC progression and were associated with mafodotin-induced eryptosis.
A team of Kyoto University researchers found that macrophages produce granulomas through a hyperactive metabolic pathway called the pentose phosphate pathway. Inhibition of this pathway showed therapeutic efficacy in reducing granuloma formation in vitro and in mouse tissue models.
Imetelstat, a telomerase inhibitor, successfully treated anaemia in MDS patients for up to one year, reducing the need for red blood cell transfusions. The drug offers a novel mechanism of action for patients with lower-risk MDS who do not respond to standard treatment.
A study by the University of the Basque Country uses game theory to establish that tumours with less cellular heterogeneity are more aggressive. The work suggests a fresh theoretical approach for new therapeutic strategies, focusing on preserving high intratumour heterogeneity.
A team of researchers at UMass Amherst has discovered a crucial role played by the enzyme UGGT in protein folding. The study reveals how UGGT 'tags' misfolded proteins with specific sugars, enabling chaperones to identify and correct errors.
Researchers have discovered a new approach to targeting transcription factors in prostate cancer by exploiting the formation of protein droplets. This process can be used to inhibit androgen receptor activity, which is essential for the growth and survival of cancer cells.
Researchers have identified macrocyclic compounds as a potential solution for targeting proteins critical to tumorigenesis, blocking nearly 80% of cancer's signature characteristic events. Additionally, these small compact molecules can effectively overcome drug resistance by binding to mutant proteins and inhibiting their activities.
Researchers developed a flexible new platform that solves part of the daunting delivery problem in cell and gene therapies. The workhorses behind this platform are extracellular vesicles, which bind to target cells and effectively transfer drugs inside.
Researchers found that 1,8-cineole inhibits inflammatory pathways in the gut, while ginsenoside Rk2 alleviates liver inflammation and restores intestinal barrier function. Another compound, specnuezhenide, modifies gut microbiota and has potential to inhibit colorectal tumor growth.
Researchers at NTNU are developing a new therapy for rare hereditary diseases like DOOR syndrome using mini-brains grown in the lab. The treatment involves injecting a virus with a healthy OXR1 gene to produce the missing protein, which may help stop or reverse the disease.
A study of 278,000 individuals in Sweden found that longer cumulative duration of ADHD medication use was associated with an increased risk of hypertension and arterial disease. Clinicians should regularly monitor cardiovascular signs and symptoms during long-term treatment.
A new study found that vosoritide treatment increases height, facial volume, and foramen magnum size in young children with achondroplasia, reducing sudden infant death syndrome, sleep apnea, and surgery needs. Vosoritide may improve quality of life and potentially save lives by addressing the root cause of the condition.
A Phase II clinical trial has shown a clear clinical benefit of combining Dabrafenib and Trametinib in treating BRAF mutated low-grade paediatric gliomas. The combination therapy improved overall response rate by over four-fold and increased median progression-free survival.
Researchers at Hokkaido University found that cancer stem cells cause macrophages to age, suppressing their antitumor activity. Supplementing mice with nicotinamide mononucleotide restored macrophage function and prevented tumor growth.
Researchers have summarized recent advances in enzyme-activated near-infrared fluorescent probes for non-invasive visualization of enzyme dynamics and disease status. These probes offer high specificity, sensitivity, and biocompatibility, making them valuable assets in biomedical research and healthcare.
Researchers at Nagoya University identified the link between iron and blindness caused by ocular toxoplasmosis, a parasite affecting one-third of the world's population. Controlling iron levels has shown promise as a potential cure.
A randomized clinical trial of 2,858 patients with new-onset and worsening chronic heart failure shows that torsemide and furosemide have no significant difference in clinical or patient-reported outcomes at 12 months. Torsemide is associated with better outcomes for patients with de novo heart failure.
The Max Foundation has started treating three patients with advanced breast cancer, including a CDK4/6 inhibitor therapy, in Mozambique. This marks the first time this treatment is available in low-income countries and aims to improve outcomes for women living with advanced breast cancer.
Lepodisiran, a short interfering RNA, targets hepatic synthesis of apolipoprotein(a), reducing serum lipoprotein(a) concentrations. Phase 1 study results support further investigation of lepodisiran's efficacy in treating elevated lipoprotein(a) levels.
A survey found that 42% of primary care physicians are unaware of biologic treatments for asthma, and 82% do not get necessary labs to determine treatment options. This lack of knowledge can lead to delayed referrals to asthma specialists and reduced effectiveness in managing symptoms.
The C-Path Neuroscience Annual Workshop brought together stakeholders to chart a transformative course for neurology research and drug development, focusing on chronic progressive diseases such as Alzheimer's and Parkinson's. Key highlights included recommendations for innovative therapies and tools to address complex disorders.