Clinical Trial News
Gene Therapy for Hemophilia A Shows Sustained Clotting Factor Expression and Reduced Bleeding
• A novel gene therapy, SPK-8011, demonstrates stable coagulation factor VIII expression in hemophilia A patients, offering a potential long-term treatment option.
• The phase 1/2 trial showed a 91.5% reduction in bleeding episodes among participants, with most maintaining FVIII expression for the duration of the study.
• The therapy involves a recombinant AAV vector engineered to produce FVIII in liver cells, providing a one-time, disease-altering approach.
• While most participants responded well, two experienced a loss of expression due to an immune response, highlighting the need for further research.
Fasting-Mimicking Diet Shows Promise in Boosting Anti-Tumor Immunity in Cancer Patients
• A clinical trial reveals that a short-term, severe calorie restriction diet is safe and feasible for cancer patients undergoing standard treatments.
• The fasting-mimicking diet (FMD) leads to metabolic changes, including reduced blood glucose, insulin, and growth factor levels, potentially hindering cancer cell growth.
• FMD enhances anti-tumor immunity by decreasing immunosuppressive cells and increasing activated T cells, both systemically and within the tumor microenvironment.
• Researchers emphasize that while promising, further trials are needed to confirm the anti-tumor efficacy of FMD in improving cancer therapy outcomes.
Singapore Launches Project Golden Years: Groundbreaking Qualitative Study on Dementia Prevention Attitudes
• The National University of Singapore and Cerner Enviza have collaborated on Project Golden Years, investigating evolving attitudes towards dementia prevention in Singapore through real-world qualitative research.
• The study represents a significant advancement in understanding behavioral patterns and prevention strategies for dementia, combining academic expertise with real-world evidence methodology.
• The research findings will be featured in the ELICIT podcast series, highlighting the growing importance of qualitative real-world evidence in pharmaceutical research and patient care.
Shorter Course of Radiation Therapy Proves Safe and Effective for Post-Surgery Prostate Cancer Patients
• A large clinical trial found that hypofractionated post-operative prostate bed radiotherapy (HYPORT) delivered over 5 weeks is as safe as conventional 7-week treatment, with equivalent quality of life outcomes after 6 months.
• Patients receiving the shorter HYPORT treatment reported more bowel side effects immediately following therapy, but these differences disappeared by the 6-month follow-up with no increase in urinary complications.
• The study results could significantly improve treatment accessibility and reduce financial burden for prostate cancer patients, as the shorter course offers equivalent cancer control with fewer hospital visits.
EMA Grants Orphan Drug Designation to Devimistat for Refractory Burkitt's Lymphoma
• The European Medicines Agency (EMA) has granted orphan drug designation to Rafael Pharmaceuticals' devimistat for treating refractory or relapsed Burkitt's lymphoma.
• Burkitt's lymphoma, a rare and aggressive form of non-Hodgkin's lymphoma, disproportionately affects children, representing up to 30% of pediatric NHL cases.
• Devimistat targets the mitochondrial tricarboxylic acid cycle, crucial for tumor cell proliferation, and has received multiple orphan drug designations from both EMA and FDA.
• A Phase 2 trial is underway to investigate devimistat's efficacy in relapsed or refractory Burkitt's lymphoma, offering hope for patients with limited treatment options.
Arbor Biotechnologies Secures $215M Series B to Advance CRISPR Gene Editing Platform
• Arbor Biotechnologies, co-founded by CRISPR pioneer Feng Zhang, has raised $215 million in Series B funding, bringing total capital raised to over $300 million for advancing liver and CNS disease programs.
• The company leverages AI and machine learning to develop the industry's largest proprietary CRISPR genomic editors toolbox, enabling targeted treatment of genetic diseases at their root cause.
• Arbor is expanding beyond CRISPR nucleases to explore precision editing innovations like CRISPR transposases, while maintaining collaborations including a $1.2 billion partnership with Vertex Pharmaceuticals.
DeepMind Launches Isomorphic Labs to Transform Drug Discovery Through AI Innovation
• Alphabet's DeepMind has established Isomorphic Labs, a standalone AI-driven drug discovery company that will leverage the breakthrough AlphaFold protein structure prediction technology.
• The new venture will be led by DeepMind founder Demis Hassabis and aims to revolutionize drug discovery by implementing an AI-first approach while building partnerships with pharmaceutical companies.
• Isomorphic Labs will utilize DeepMind's AlphaFold2 database, which has successfully mapped nearly all human proteins and achieved sufficient precision for drug design in one-third of its protein models.
HPV Vaccine Shows 87% Reduction in Cervical Cancer Cases in Landmark English Study
• A groundbreaking study by King's College London demonstrates that routine HPV vaccination has led to an 87% reduction in cervical cancer cases in England, with over 10 million doses administered since 2008.
• The research provides the first direct evidence of HPV vaccine effectiveness, showing nearly complete prevention in women vaccinated at ages 12-13, with cases dropping from 500 per year to just five over an 11-year period.
• The vaccination program has prevented approximately 450 cervical cancer cases and 17,200 pre-malignant lesions, with effectiveness varying by age at vaccination: 62% reduction for ages 14-16 and 34% for ages 16-18.
WHO Solidarity Trial Finds Remdesivir, Hydroxychloroquine, Lopinavir, and Interferon Ineffective Against COVID-19
• The WHO's Solidarity Trial evaluated the efficacy of remdesivir, hydroxychloroquine, lopinavir, and interferon beta-1a in hospitalized COVID-19 patients, comparing them to standard care.
• The trial, conducted across 405 hospitals in 30 countries with over 11,000 participants, found that none of the drugs significantly reduced mortality.
• Results indicated that these antiviral regimens had little to no impact on overall mortality, initiation of ventilation, or duration of hospital stay for COVID-19 patients.
• The study's findings suggest that resources should be directed towards other therapeutic strategies for managing hospitalized COVID-19 patients.
Highlighted Clinical Trials:
Institut National de la Santé Et de la Recherche Médicale, France
Posted 3/22/2020
FDA Approves Novartis' Scemblix for Resistant Philadelphia Chromosome-Positive CML
• The FDA has approved Scemblix (asciminib) for Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) in adults after two or more tyrosine kinase inhibitors (TKIs).
• Scemblix, a first-in-class ABL myristoyl pocket binder, offers a novel approach for patients with resistance or intolerance to existing TKI therapies.
• Clinical trials demonstrated Scemblix nearly doubled the molecular response rate compared to bosutinib, with a lower rate of treatment discontinuation due to adverse reactions.
• The approval also includes patients with the T315I mutation, who typically have limited treatment options and face significantly worse outcomes.