BAYLOR COLLEGE OF MEDICINE

Lawrence Eichenfield, MD, discussed recent FDA approvals: tapinarof cream, 1% for AD in patients 2 years and older, and nemolizumab for moderate-to-severe AD in patients 12 years and up. Both treatments offer non-steroidal alternatives, with tapinarof providing a favorable safety profile and nemolizumab targeting IL-31, offering potential unique responses.

CAR-T therapy can benefit patients excluded from pivotal trials, according to Daniel Goyco Vera, MD. His study of 61 lymphoma patients found effective treatment despite exclusion criteria, highlighting the need for diverse trial recruitment to build trust and ensure generalizable results.

Aurion Biotech's Phase 1/2 CLARA trial showed AURN001, an allogeneic cell therapy for corneal edema, improved visual acuity and reduced corneal thickness. High-dose combination therapy was most effective, simplifying recovery compared to current treatments like DMEK. The therapy could treat up to 200 patients with one donor cornea, addressing global shortages. Phase 3 trials and FDA approval are next.

FDA approved tapinarof cream 1% for atopic dermatitis in ages 2 and older, representing a non-steroidal option with a new mechanism of action. It improves skin barrier and reduces inflammation, showing a remittive effect. Over 80% of participants aged 2-17 in ADORING 3 trial achieved clear or almost clear skin, with 52% reporting completely clear skin.

Researchers at Baylor College of Medicine and Texas Children’s Hospital found that loss of MeCP2 in adulthood causes immediate gene dysregulation, leading to Rett syndrome. They identified a molecular cascade driving disease independently of developmental contributions, with neuronal circuit-level deficits occurring after gene expression dysregulation.

Grace Liu from Sino Biological discusses Antibody-Drug Conjugates (ADCs), a cancer therapy combining precision targeting with potent cytotoxic drugs. ADCs consist of a monoclonal antibody, cytotoxic payload, and linker, balancing efficacy and safety. Sino Biological offers comprehensive ADC development solutions, aiding in the complex process from discovery to clinical studies.

Organon's tapinarof cream, 1% (VTAMA), an aryl hydrocarbon receptor agonist, has been FDA-approved for treating atopic dermatitis in patients aged 2 years and up. It offers a steroid-free option, improving skin integrity and reducing itch, with significant efficacy observed in phase 3 trials.

Researchers at Baylor College of Medicine developed FunMap, a machine learning-based approach integrating protein datasets and RNA sequencing data from 11 cancer types to assess cancer-associated mutations and understudied proteins, uncovering protein modules and predicting functions, with potential to inform therapeutic strategies.

Ally Schott's early endometriosis diagnosis at 12 led to limited treatment options and self-taught research. Despite its prevalence affecting 10% of women globally, endometriosis remains underfunded and poorly understood, with only two new drugs approved in the U.S. for pain management in the past decade. Women's health research generally suffers from insufficient funding and information deficits, hindering drug development. Recent federal efforts, including a $100 million investment by the Biden administration, aim to address these gaps, but future priorities under a new administration remain uncertain.

Brexucabtagene autoleucel (brexu-cel) showed similar efficacy in patients aged 60-69 with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) compared to those under 60, with complete remission (CR)/CR with incomplete count recovery (CRi) rates of 89% and minimal residual disease (MRD)-negativity rates of 59% in the older group. No significant differences in overall survival (OS) or progression-free survival (PFS) were observed across age groups. Safety findings, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), were largely consistent.