Clinical Trial News

Azithromycin distributions to children 1-59 months in sub-Saharan Africa reduced all-cause mortality, challenging WHO's recommendation to limit to infants in high-mortality areas. The AVENIR trial showed broader use saves lives, despite AMR concerns.

A bowel cancer treatment for the aggressive BRAF V600E mutation, similar to Dame Deborah James's case, has been approved for NHS use. The drug combination, trifluridine-tipiracil hydrochloride and bevacizumab, extends life by nearly a year for patients with no other treatment options, with some rare cases gaining up to seven more years. This approval is a significant step in fighting the incurable genetic form of bowel cancer, which often affects the young.

C. Michael Gibson discovered high lipoprotein(a) levels, inherited from his grandfather, despite decent cholesterol. Research is ongoing for medications to lower lipoprotein(a) and other approaches to reduce cardiovascular disease risk. Statins are effective but not sufficient, prompting exploration of new treatments targeting various cholesterol types, including HDL and remnant cholesterol. Clinical trials are underway, with mixed results so far, but optimism remains for potential breakthroughs.

Leqembi, an Alzheimer's drug from Biogen and Eisai, offers early-stage patients more independent living time but faces hurdles like reimbursement issues, diagnostic tests, and neurologist availability. Missie Meeks, an early recipient, reports improved daily functioning. Despite risks like brain swelling and bleeding, some patients find the process worth it for extended normal life.

Longboard Pharmaceuticals announced positive interim results from the PACIFIC Study OLE, showing a 57.7% median reduction in countable motor seizures over ~9 months for bexicaserin in DEEs, with sustained efficacy and a favorable safety profile. The company plans to initiate a global Phase 3 program later this year.

Researchers from NUS Medicine's WisDM used CURATE.AI to treat a patient with Waldenström macroglobulinemia, achieving improved red blood cell levels and cost savings. The AI platform, which adjusts treatment based on individual responses, demonstrates potential for personalized rare disease treatments despite small patient populations.

Savara presents Phase 3 IMPALA-2 trial data at ERS 2024 showing significant improvements in disease severity, DLCO%, SGRQ Total Score, and GGO with molgramostim in aPAP patients.

Dupilumab therapy over 3 years significantly improves chronic rhinosinusitis with nasal polyps (CRSwNP) symptoms, disease-related impairment, nasal polyp burden, and olfactory function, according to a multicenter, retrospective analysis involving 150 adult patients. The study, led by Patrick Huber, found sustained effectiveness by the 36-month mark, with 76.5% of subjects showing an 'excellent' treatment response. Adverse events were mostly self-limiting.

Nanorobots guided by magnetic fields could revolutionize stroke prevention by sealing off brain aneurysms more safely and precisely than traditional methods. These bots, which release a clotting agent when heated, have successfully blocked aneurysms in rabbit trials without obstructing overall blood supply. Future research aims to refine this technology for larger animals and deeper brain aneurysms.

FDA advisory panel rejected MDMA-assisted therapy for PTSD due to concerns over clinical trial design, potential abuse, and misconduct allegations. April Pride, CEO of SetSet, views this as a setback for psychedelic medicine, advocating for regulatory reforms and harm-reduction education. Despite the rejection, Pride remains optimistic about the future of psychedelic medicine, expecting innovation in safer compounds and protocols.