New Study Finds Neuroprotective Drug Effective in Mild to Moderate Alzheimer's

A groundbreaking study published in Nature Medicine has revealed promising results for a novel neuroprotective drug candidate, LM11A-31, in treating Alzheimer's disease. Over a span of 26 weeks, patients with mild to moderate Alzheimer's disease tolerated the drug well, and it demonstrated safety across various spinal fluid and imaging biomarkers. These positive outcomes suggest that LM11A-31 merits further investigation in more extensive trials to assess its full potential in terms of safety and efficacy.

The research was funded by the Alzheimer's Drug Discovery Foundation (ADDF), highlighting the progress being made towards innovative treatments that focus on the biology of aging. This approach aims to complement existing anti-amyloid therapies and expand the arsenal of Alzheimer's treatments. According to Howard Fillit, MD, Co-Founder and Chief Science Officer of the ADDF, the advancement of such a novel drug would have been unimaginable a decade ago. The rapid evolution in the field signifies a shift towards therapies targeting the underlying biology of Alzheimer's, much like the precision medicine approach seen in cancer care.

Phase 2a exploratory studies like this one are crucial for generating initial safety and tolerability data in the target population, alongside providing essential clinical and biomarker data. These data inform the design of more rigorous phase 2b and 3 studies.

Frank Longo, MD, PhD, Founder of PharmatrophiX and a prominent figure at Stanford University, expressed optimism about the study's findings. He emphasized the importance of ADDF's funding in reaching this milestone, reflecting on his 25-year-long relationship with the foundation, which began when LM11A-31 was first discovered. This partnership has been vital in advancing the drug from basic research through preclinical studies to the clinical phase.

LM11A-31 functions by activating nerve cell survival pathways while inhibiting degenerative ones, addressing various Alzheimer's-related pathologies. These include resilience against amyloid, reduction of tau pathology, and mitigation of microglial pathology. Developed by PharmatrophiX, a biopharmaceutical company, LM11A-31 targets neurodegenerative disorders with the potential to modify the disease.

The study's findings are part of a broader trend in Alzheimer's research, which is increasingly focusing on diverse causes like inflammation, metabolic disturbances, and vascular dysfunction. Nearly 75% of drugs in the pipeline are exploring these novel targets. The next phase involves combination therapy trials that aim to combine multiple drugs into one treatment regimen.

Dr. Fillit noted that the advancement of novel, multifaceted drugs like LM11A-31 marks a significant step forward in Alzheimer's research. These therapies have the potential to target numerous underlying disease mechanisms, demonstrating the impact of ADDF's investments in transforming the treatment landscape through cutting-edge science.

Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer's Drug Discovery Foundation is dedicated to accelerating the discovery of drugs to prevent, treat, and cure Alzheimer's disease. The foundation is the only public charity solely focused on funding Alzheimer's drug development, employing a venture philanthropy model to support research in both academia and the biotech industry. The ADDF's contributions have been pivotal in bringing significant innovations like the first Alzheimer's PET scan and blood test to market, and continue to fuel a robust and diverse drug pipeline. Through the support of its donors, the ADDF has awarded over $290 million to fund more than 750 drug discovery programs, biomarker programs, and clinical trials across 20 countries.