UNIVERSITY OF ARKANSAS SYSTEM

Breakthrough at UT Health San Antonio: Chemical Endocytic Strategy Could Transform IV Drugs into Oral Treatments

a month ago

• Researchers at UT Health San Antonio have developed "chemical endocytic medicinal chemistry," a novel approach that could enable large-molecule drugs to be taken orally rather than intravenously. • The discovery leverages CD36 protein receptors on cell surfaces to facilitate cellular uptake of large and water-soluble drugs, potentially overcoming the blood-brain barrier for treating conditions like brain cancer and Alzheimer's disease. • This paradigm shift in drug delivery could revolutionize pharmaceutical development, resurrect previously abandoned drug candidates, and enable more personalized medicine based on patients' varying CD36 expression levels.

Cannabidiol linked to better social, executive function in tuberous sclerosis complex

5 months ago

A study shows that cannabidiol (CBD) treatment in young people with tuberous sclerosis complex (TSC) leads to significant improvements in cognitive, behavioral, and executive domains, enhancing social engagement, mood, and communication.

Elevidys Gene Therapy Shows Sustained Benefits in Duchenne Muscular Dystrophy Patients

5 months ago

• Sarepta Therapeutics' Elevidys demonstrates sustained benefits and disease stabilization in ambulatory Duchenne muscular dystrophy (DMD) patients, according to Phase 3 EMBARK trial results. • Crossover-treated patients showed a 2.34-point improvement on the North Star Ambulatory Assessment (NSAA) compared to matched external controls after 52 weeks of Elevidys treatment. • Patients treated with Elevidys in Part 1 of EMBARK maintained clinically meaningful improvements in NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) at two years. • Muscle biopsies showed consistent micro-dystrophin expression, and MRI scans indicated minimal muscle pathology progression, reinforcing Elevidys's long-term efficacy and safety.

REGENXBIO's RGX-202 DMD Gene Therapy Enters Pivotal Phase 3 Trial

6 months ago

• REGENXBIO has dosed the first patient in the Phase 3 AFFINITY DUCHENNE trial evaluating RGX-202 for Duchenne muscular dystrophy (DMD). • RGX-202 demonstrated improved or stable function in treated patients compared to natural history controls, with significant improvements in NSAA scores. • The pivotal trial aims for accelerated approval in 2026, focusing on microdystrophin expression as the primary endpoint. • RGX-202 was well-tolerated in earlier phases, with no serious adverse events reported, offering promise for a broader DMD patient population.

Trump Administration Reshapes Health Agencies; FDA Approves Novel Cancer Therapies

7 months ago

• President-elect Trump nominates Robert F. Kennedy Jr. as HHS Secretary, Mehmet Oz to lead CMS, and Marty Makary to head the FDA, signaling a shift in healthcare priorities. • The FDA grants accelerated approval to asciminib for chronic myeloid leukemia and obecabtagene autoleucel for B-cell acute lymphoblastic leukemia, marking advancements in cancer treatment. • Revumenib receives approval for relapsed or refractory acute leukemia with KMT2A translocation, offering a targeted therapy for this specific genetic alteration. • A new Surgeon General's report highlights disparities in tobacco use, emphasizing the need for tailored interventions in vulnerable populations.

NIH Awards $2.9 Million to ACRI for Cystic Fibrosis Therapy Research

7 months ago

• The NIH has granted $2.9 million to ACRI to study how to improve the effectiveness of a key therapy for cystic fibrosis (CF). • The five-year study will investigate optimal dosing and potential genetic factors influencing response to triple combination therapy in CF patients. • Researchers aim to personalize CF treatment by understanding how individual patients process the drug and identifying the best dose for each person. • The study seeks to address the challenges of variable treatment response and side effects associated with triple combination therapy in CF patients.

Cilta-cel Demonstrates Sustained Survival Benefit in Pretreated Multiple Myeloma

8 months ago

• Updated data from the CARTITUDE-4 trial shows cilta-cel significantly improves overall survival in lenalidomide-refractory multiple myeloma patients compared to standard of care. • At 33.6 months follow-up, the 30-month overall survival rate with cilta-cel was 76.4% versus 63.8% with standard of care, reducing the risk of death by 45%. • The progression-free survival rate at 30 months was 59.4% with cilta-cel compared to 25.7% with standard of care, indicating a 71% reduction in disease progression or death. • Cilta-cel also demonstrated high overall response rates (84.6%) and minimal residual disease negativity, highlighting its potential to transform the myeloma treatment landscape.

UAMS Awarded $2.2 Million Grant to Investigate Efferocytosis in Retinopathy

8 months ago

• The University of Arkansas for Medical Sciences (UAMS) has received a $2.2 million grant from the National Eye Institute (NEI). • The grant will fund research into how modulating the immune response through efferocytosis can benefit patients with retinopathy. • Dr. Abdel Fouda's lab will lead the study, exploring methods to enhance myeloid cell-mediated efferocytosis in treating retinopathy. • The research aims to develop new therapies for ischemic and trauma-induced retinopathy by understanding efferocytosis' role.