MJFF Awards Grant to Cantabio to Develop Tau-focused Therapy

Cantabio Pharmaceuticals has been awarded a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to develop a small molecule that can reduce the aggregation of a protein called tau. The research may have applications in treating Parkinson’s and other brain diseases.

“We are proud to support Cantabio’s approach targeting this pathology and building potential to slow or prevent progression for millions of people,” Marco Baptista, PhD, vice president of research programs at MJFF, said in a press release.

The protein tau normally helps to stabilize microtubules, which are structures that help to give cells their shape. Under normal conditions, tau is present as a monomer (a single protein).

However, under disease conditions, tau protein can form fibrils, or clumps, that are thought to be toxic to cells. Tau aggregates are found commonly in the brains of people with Alzheimer’s disease, and emerging research suggests these abnormal protein clumps also are involved in Parkinson’s.

Toxic aggregates of another protein, alpha-synuclein, are characteristic of Parkinson’s. Alpha-synuclein aggregates may help to prompt the formation of tau fibrils, according to Cantabio.

“The importance of the aggregation of the tau protein in context of the onset and the progression of Parkinson’s disease (PD) has received limited research focus, although neurofibrillary tangles are a pathological hallmark of PD,” said Gergely Tóth, PhD, Cantabio’s CEO.

The overall goal of the newly funded project is to develop small molecules (pharmacological chaperones) that bind to tau in its monomer state, helping to stabilize the normal protein and prevent it from clumping up. These small molecules may stop or reverse the formation of tau fibrils, and could help to prevent tau clumping spurred by alpha-synuclein.

“A therapy to reduce tau aggregation would have application in Parkinson’s and across other brain diseases,” Baptista said.

The project, “Development of small-molecule inhibitors to reduce the aggregation of tau for the treatment of Parkinson’s disease,” will develop a novel small-molecule tau aggregation inhibitor through experiments done in vitro (in dishes) and in vivo (in living animals). The goal is to develop a molecule that has high therapeutic potential to treat, as well as help prevent, Parkinson’s and Alzheimer’s.

“We are pleased to have the support of The Michael J. Fox Foundation to explore the efficacy of Cantabio’s novel small-molecule tau aggregation inhibitors in PD models. These studies may enable the development of this therapeutic approach for the treatment of both Parkinson’s and Alzheimer’s diseases,” Tóth said.