NATIONAL INSTITUTES OF HEALTH

IU researcher Paul Macklin co-leads $4M NIH grant to integrate PhysiCell and CoGAPS into a cloud-based platform for cancer research, aiming to improve simulations and data analysis.

Antidepressants like escitalopram may boost cognitive function via the 5HT4 receptor, improving memory and thinking skills.

Zevra Therapeutics announces FDA approval for Miplyffa (arimoclomol) to treat Niemann-Pick disease type C (NPC) in patients aged two years and older, in conjunction with miglustat. The approval is based on comprehensive data from a 12-month trial demonstrating Miplyffa's safety and effectiveness, halting disease progression over 12 months. A 48-month open-label extension study further confirmed improved outcomes. Miplyffa is set to launch in the US market by autumn/winter 2024.

WashU's annual research funding surpasses $1 billion, supporting diverse projects from Alzheimer’s to air pollution, driving local economic growth. Funding from federal agencies and others nearly doubled in a decade, attributed to increased faculty, research quality, and infrastructure. WashU Medicine, ranked No. 2 in NIH funding, leads in innovative research and faculty growth. New facilities like the Jeffrey T. Fort Neuroscience Research Building foster collaboration and discoveries, while regional research addresses local issues. WashU's partnerships and startups contribute to the local economy, with expectations of continued funding growth in neuroscience, public health, and more.

Dr. Rozalina McCoy discusses CGM innovations from the ATTD 2024 Conference, focusing on CGM diabetes quality measures, hypoglycemia progress, and CGM-guided precision diabetes management. She highlights the utility of CGM data, the importance of detecting hypoglycemia, and the role of CGM in guiding nutrition and exercise for diabetes management, emphasizing the complementary use of A1c and CGM data for patient care.

Kratom, an herbal substance from Southeast Asia, produces opioid- and stimulant-like effects and is used for managing withdrawal symptoms, pain, and mental health issues. Despite FDA warnings about potential adverse events like liver toxicity and seizures, recent FDA research suggests kratom capsules may be safe in high doses. Kratom is not a controlled substance in the U.S., but several states have banned its active alkaloids. Kratom products are marketed for mood, focus, and energy enhancement, with companies advising caution and responsible use.

Tiziana Life Sciences receives $4 million NIH grant for intranasal anti-CD3 research in Alzheimer’s disease, aiming to modulate immune activity and slow progression. The grant supports preclinical and clinical studies, with plans to initiate first-in-human trials in the coming months. Foralumab, a fully human anti-CD3 monoclonal antibody, has shown potential in treating neurodegenerative conditions.

Black patients with TNBC received immunotherapy less often than white patients, but outcomes were similar when treated. Socioeconomic factors were main barriers for Black patients, while Medicare enrollees and those in community programs had lower immunotherapy rates. Immunotherapy mitigated racial survival differences in metastatic TNBC.

Symptoms related to sexual health in early-stage breast cancer patients on adjuvant endocrine therapy (AET) were linked to lower adherence in Black women, according to a study presented at the 17th AACR Conference. Both Black and white patients reported lower mental quality of life due to vaginal itchiness, painful intercourse, and loss of interest in sex. Black patients also experienced lower physical quality of life from loss of interest in sex. Vaginal dryness and decreased libido were linked to lower AET adherence in Black women only. The study highlights the need for addressing sexual health symptoms to improve treatment adherence.

The FDA has approved arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC) in patients aged 2 years and older, in combination with miglustat. This approval is based on a phase 2/3 trial showing arimoclomol met its primary outcome measure of improving the NPC Clinical Severity Scale score. Arimoclomol, an orally delivered medication, increases the activation of transcription factors EB and E3, leading to upregulation of lysosomal genes. Zevra Therapeutics plans to launch the therapy in the U.S. within 8 to 12 weeks.