After decades of trying and dozens of failed trials, amyloid targeting has paid off with the first disease-modifying agents reaching the market. But success does not mean slam dunk. Aduhelm (aducanumab, Biogen Inc.) was dogged by controversy throughout its brief tenure, and Biogen pulled the plug on it in early 2024. Leqembi (lecanemab, Biogen Inc.) has received full approval. In this second installment of a three-part series on Alzheimer’s, BioWorld looks at the nuanced view of amyloid’s role in the disease.

At a recent meeting on “Research priorities for preventing and treating Alzheimer’s disease and related dementias” (ADRD), convened by the National Academies, one consensus priority on ADRD research was that there needs to be more of it at every stage. Several speakers presented stark numbers on the relative volume of research in cancer and neurodegeneration. Research output, measured in peer-reviewed papers, for dementia is estimated to be around 10,000 papers annually, compared to 150,000 for cancer, while AD clinical trials are also few and far between compared to cancer trials. This final installment of BioWorld’s series on Alzheimer’s explores some of the reasons for this discrepancy along with the latest advances and ongoing efforts to accelerate research and drug development in the field.

Ironically, the first person to be diagnosed with what is now Alzheimer’s disease was missing its major risk factor. When she first began showing symptoms of dementia in 1901, Auguste Deter was not particularly old. Despite Deter’s case, aging is the largest risk factor for developing Alzheimer’s, by a large margin. But “geroscience has not been translated into drugs for Alzheimer’s disease,” Howard Fillit, Alzheimer’s Drug Discovery Foundation co-founder and chief scientific officer, told BioWorld. “We’re just starting to see that cross-fertilization now.”

Alzheimer’s disease (AD) is recognized worldwide for its debilitating symptoms of declining cognitive function and gradual memory loss. What remains less clear is exactly what causes the neurodegenerative disease, and how to treat it. “Alzheimer’s disease is characterized by two key pathologies – beta-amyloid plaques and tau neurofibrillary tangles.” Seung-Yong Yoon, CEO of Adel Inc., told BioWorld. “Adel is looking to develop a tau-targeting drug, considering tau has been more correlated with AD symptom progression, and the industry’s need for tau pipelines.”

Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later.

In what represents its first patenting, Sonura LLC has been issued with a patent for an aural device designed to protect the hearing of infants in a neonatal intensive care unit (NICU) by active filtering, while, at the same time, providing aural stimulation for neurological development.

The U.S. FDA granted Neuronetics Inc. clearance for use of its Neurostar advanced therapy as an adjunctive treatment of major depressive disorder in patients aged 15 to 21, making it the first transcranial magnetic stimulation treatment (TMS) cleared for this age group, the company said.

The U.S. FDA has posted another proposed ban of the use of electrical stimulation devices (ESDs) for the treatment of self-injurious behavior, which revisits the controversy over their use by the Judge Rotenberg Center.

Graymatters Health Ltd. seeks patent protection for its non-invasive, self-neuromodulation approach for treating post-traumatic stress disorder. In what represents the company’s second PCT filing, GMH’s co-founders, Oded Kraft, Rani Cohen and Shai Attia, describe a method for achieving a reduction of at least six points in a Clinician-Administered PTSD Scale for DSM-5, following neurofeedback treatment delivered for eight to 12 consecutive weeks.

Laboratory Corporation of America Holdings expanded its blood biomarker test portfolio for Alzheimer’s disease, making its pTau217 test available by prescription in the U.S. as well as for research globally. The standalone test can also be used in monitoring patients undergoing treatment for the neurodegenerative disease.