The disease Alzheimer’s is the most common cause of dementia, affecting more than 55 million people worldwide. Currently, the two main approaches for treatments to delay or slow disease progression target the accumulation of amyloid beta peptides and the tau protein. However, these strategies address only a small set of biological markers and mechanisms associated with Alzheimer’s disease.

Now, a scientific team led by researchers from Western, Stanford and California Universities (UCSF), tested a experimental drug which is designed to enhance the brain resilience in the changes caused by Alzheimer’s disease. The drug showed promising results in the first human clinical trial.

The drug, with the provisional name LM11A-31, targets the P75 neurotrophin receptor (P75NTR) found on cells in the brain. This receptor helps regulate various processes such as cell survival, growth and death. The drug enhances the passage of signals that promote cell survival and growth.

Although the primary purpose of the clinical trial was to assess the drug’s safety and tolerability in patients with mild to moderate Alzheimer’s disease, the researchers also collected multiple markers of brain pathology to assess whether it affected disease progression relative to a placebo. medicine.

Despite the relatively short duration of the clinical trial (26 weeks), it was found that the drug slowed development disease. The clinical trial also showed that it is safe and well tolerated.

“In a phase 2A clinical trial, the goal is to prove that the drug it does not cause side effects that are toxicexplained Taylor Schmitz, lead author of the study.

The researchers hope the drug will benefit patients even if given in later stages of the disease. Current treatments, such as monoclonal antibodies, which try to clear amyloid from the brain, are not as effective for patients in the later stages of the disease because significant damage has already been done to neurons.

“This drug is exciting because it directly affects the ability of neurons to survive. It promotes their overall integrity, their branching, and their synapses where they connect and communicate with each other,” said Haley Shanks, a neuroscientist and co-author of the study.

“In animal models, the drug was shown to preserve these neurons or reverse their damage, returning them to a healthy state,” he added.

At the clinic testheld in five European countriesinvolved 242 people with mild to moderate Alzheimer’s disease.

“We also observed changes in a biomarker of inflammation. The drug slowed the rise of this marker in the cerebrospinal fluid.” This is important because over the past five years, inflammation has become a key factor in understanding Alzheimer’s disease,” Schmitz added.

“An important part of the study was the collaboration of many independent experts in the fields of brain imaging and biomarkers. “It is of particular interest that this therapeutic approach appears to implicate key mechanisms of resilience and integrity of synaptic connections between brain cells,” said Longo.

The study findings were published in Nature Medicine.