Clinical Trial News

A recent Phase 2a study on BRAVYL (oral fasudil) has shown promising results in slowing the progression of ALS, with significant reductions in neurodegeneration biomarkers and improvements in clinical outcomes. The study also highlights BRAVYL's potential in reducing TDP-43 pathology, a key factor in ALS progression.

EMA's CHMP has recommended not renewing Translarna's EU marketing authorisation, citing unconfirmed effectiveness for Duchenne muscular dystrophy patients with a nonsense mutation. Despite re-examination, including study 041 and patient registry data, Translarna's benefits were not statistically significant, leading to a negative benefit-risk balance conclusion.

Mayo Clinic researchers have made significant progress in the search for effective glioblastoma therapies, developing a treatment with a nearly 50% cure rate in laboratory models. This innovative approach combines a small molecule inhibitor with an FDA-approved drug, showing promising results without adverse effects. The research focuses on targeting molecular motors and overcoming drug resistance, offering new hope for treating this lethal brain tumor.

A Duncan family's ER visit revealed their infant son Bennett had leukemia, leading to months of treatment, including chemotherapy and CAR-T therapy, a clinical trial. The family, supported by their community and a GoFundMe, hopes for remission and a bone marrow transplant, aiming for a homecoming by summer.

The CheckMate 8HW trial showed nivolumab plus ipilimumab significantly reduced disease progression or death risk by 79% in MSI-H/dMMR metastatic colorectal cancer patients, compared to chemotherapy. With a 24-month progression-free survival rate of 72% vs 14%, and fewer severe adverse events, this immunotherapy combination may offer a new first-line treatment option.

FDA approved eflornithine (Iwilfin) to reduce relapse risk in high-risk neuroblastoma patients post-therapy. Based on Study 3b vs. ANBL0032 trial, it showed improved event-free and overall survival. Common side effects include otitis media and diarrhea. Approved after FDA committee's positive vote.

Jacobio Pharmaceuticals presented glecirasib's efficacy in treating KRAS G12C mutated pancreatic and other solid tumors at ASCO GI 2024. With a 48% cORR and 90% DCR in evaluable patients, glecirasib shows promise over standard treatments, supported by safety and tolerability data. It received BTD for pancreatic cancer, aiming to expedite its development and patient access.

PeproMene Bio, Inc. announced a patient in their Phase 1 trial for PMB-CT01 (BAFFR-CAR T Cells) achieved complete remission post-treatment for relapsed/refractory B-cell acute lymphoblastic leukemia. The trial, conducted at City of Hope, showed minimal toxicity and durable remissions, highlighting PMB-CT01's potential in treating B-cell malignancies.

Early HIV treatment within four weeks of infection may enable long-term virus control without medication, a study shows. Starting ART early optimizes immune cells' development, enhancing natural virus elimination. Delayed treatment reduces these benefits. Despite decreasing HIV infections and AIDS mortality in Europe, scaling up testing and treatment is crucial to meet 2030 AIDS epidemic targets.

Faricimab demonstrated good visual stability and allowed for extended treatment intervals in patients with neovascular age-related macular degeneration, as per real-world data from the FARETINA-AMD study presented at Retina 2024.