The difference between the origin of Alzheimer's disease (AD) and its symptoms is an obstacle to finding effective treatments. Scientists focused on amyloid-β (Aβ) plaques and tau aggregates to slow neurodegeneration and cognitive decline. Without identifying what causes AD, approved treatments do not provide much benefit. However, new findings suggest that restoring lithium levels in the brain could prevent and treat AD. Not just any lithium would work, just the forms that do not bind to Aβ.

Abnormal tau aggregation is a hallmark of Alzheimer’s disease (AD) and a major contributor to neurodegeneration, synaptic dysfunction, and progressive functional decline. Antibodies against extracellular tau represent a potential therapeutic approach aimed at reducing pathological spread and delaying the clinical progression of AD. Researchers from Merck & Co., Inc. presented the preclinical development of MK-2214, a murine IgG2a monoclonal antibody that selectively targets the pathological phospho-epitope pSer413 of the tau protein.

Lysoway Therapeutics Inc. announced that it has received a research grant of $2.93 million from The Michael J. Fox Foundation for Parkinson's Research. The funding will support the preclinical development of Lysoway’s highly brain-penetrant small molecule mucolipin TRP Cation channel 1 (MCOLN1/TRPML1) agonist.

In the tumor microenvironment, cancer cells activate various signaling pathways to promote their growth. This includes the formation of blood vessels. However, the circulatory system is not the only one attracted to the tumor. Researchers at Sanford Research have uncovered a mechanism to circumvent the immune response that would destroy them.

Alterity Therapeutics Ltd. helped develop a new neuroimaging biomarker called the multiple system atrophy index (MSA-AI), which looks to be a more reliable biomarker for tracking disease progression of MSA.

Vertex Pharmaceuticals Inc.’s bad news from a phase II pain study and separately on the regulatory front caused shares (NASDAQ:VRTX) to close Aug. 5 at $393.83, down $78.44, or 16%. Vertex rolled out top-line results from the phase II dose-ranging study to test the safety and efficacy of its NaV1.8 pain signal inhibitor, VX-993, in tackling acute pain after bunionectomy.  

Subtyping is what made precision medicine in cancer a reality. And for successful drug discovery in all its stages, finding subtypes in Alzheimer’s disease is all but imperative. Prior to the approval of the modestly effective Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd.), Kisunla (donanemab, Eli Lilly and Co.), and the since-withdrawn Aduhelm (aducanumab, Biogen Inc./Eisai Co. Ltd.), more than a dozen failed phase III clinical trials were all that amyloid-targeting drugs had to show for themselves for decades of effort.

Deep learning tools for protein design can also be used to create molecules that bind to them. Certain peptides, such as intrinsically disordered proteins (IDPs), are challenging to target due to their variable nature. However, scientists from the lab of Nobel laureate David Baker have developed a method to generate binders for IDPs by searching the world’s largest protein database with their AI-powered tool RFdiffusion.

At the Alzheimer's Association International Conference (AAIC) 2025, researchers from Acadia Pharmaceuticals Inc. have presented novel results on the assessment of the pharmacologic and pharmacokinetic (PK) properties and nonclinical safety of ACP-204.