Translational Bytes

Jul21 Model-Informed Dosing for DNDI-6148 in Cutaneous Leishmaniasis

For the neglected tropical skin infection cutaneous leishmaniasis, there is only one approved oral treatment, which is limited by safety and efficacy concerns, underscoring the urgent need for better oral therapies. Drugs for Neglected Diseases initiative (DNDi) has identified DNDI-6148 as a precli...

For decades, researchers have sought ways to control the placebo effect, mostly by educating patients about placebo mechanisms. But what about the study team itself? After all, in early-phase inpatient trials, clinicians, nurses, and coordinators spend days in close, repeated contact with parti...

Pregnant or lactating women with COVID-19 are at increased risk of developing severe disease or complications, such as hypertension, preeclampsia, or a need for intensive care admission. 1,2 However, because of perceived safety concerns regarding infant exposure, breastfeeding women were excluded ...

Neuroblastoma, the deadliest solid childhood cancer outside the brain, is thought to contain two cell states: aggressive, stem-like mesenchymal cells and committed, neuron-like adrenergic cells. Adrenergic cells comprise the bulk of tumors and are generally more sensitive to traditional therapy, wh...

Real-world data (RWD) and real-world evidence (RWE) have the potential to transform how we study the safety, effectiveness, and value of medicines and medical technologies. As researchers, we have opportunities to generate insights from routine clinical care that can complement clinical trials and ...

In the recent Clinical and Translational Science perspective piece authored by Drs. Linda Jeng and Jeffrey Siegel, they illuminate a critical pathway for bringing treatments to patients faster: the use of surrogate endpoints in drug approval decisions. The authors conclude that when rigorously ...

Pharmacogenetics (PGx) is a form of precision medicine that applies knowledge of drug-gene interactions to maximize effectiveness and reduce toxicity of medications. Given the elevated risk of severe, and sometimes life-threating, chemotherapy-associated toxicities in genetically susceptible pati...

Interleukin‑6 (IL‑6) plays a central role in ulcerative colitis (UC) by activating two distinct pathways: classic signaling, mediated by the membrane‑bound IL‑6 receptor and supporting homeostasis and tissue repair, and trans‑signaling, mediated by the soluble IL‑6 receptor and driving pro‑inflamm...

Chimeric antigen receptor (CAR)-T cell therapy has achieved remarkable success in the treatment of hematological malignancies and demonstrated promising antitumor activity in solid tumors. Accurate characterization of CAR-T cell kinetics is essential for understanding the efficacy and safety of the...

A randomized, double-blind, placebo-controlled Phase I study in 37 healthy adults of Black African origin assessed the cardiac safety of combining pyronaridine (PYR) and piperaquine (PQP), two oral long-acting antimalarial agents with potential for monthly chemoprevention. PQP is known to produce a...

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