Scientists develop new molecule that could halt, cure Alzheimer’s

A small molecule that disrupts the mechanism through which neurons become dysfunctional with the onset of Alzheimer’s disease (AD) has been developed by a team of scientists led by Prof T. Govindaraju from Jawaharlal Nehru Centre for Advanced Scientific Research (JNCASR). The molecule could be a potential drug candidate to halt or cure the leading cause of dementia (70%-80%) worldwide, according to the Ministry of Science & Technology.

Abnormal levels of naturally-forming protein in brains affected by Alzheimer’s disease clump together to form plaques that collect between neurons and disrupt cell function. This is caused by production and deposition of the amyloid peptide (Aβ) that accumulates in the central nervous system. The multifactorial nature of Alzheimer’s disease, attributed to multifaceted amyloid toxicity, has kept researchers from developing effective treatment, a statement from the Ministry of Science & Technology said on Wednesday.

The JNCASR team designed and synthesized a set of novel small molecules and identified a lead candidate which they found could reduce the toxicity of Amyloid Beta (Aβ) toxicity, the statement explained.

Jawaharlal Nehru Centre for Advanced Scientific Research (JNCASR) is an autonomous institute of the Department of Science & Technology.

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Detailed studies established the molecule called TGR63 as the lead candidate to rescue neuronal cells from amyloid toxicity, the statement said. Remarkably, the molecule was also found to reduce amyloid burden in the cortex and hippocampus, or a complex part embedded deep into the temporal lobe, thereby reversing cognitive decline. This research has been published recently in the journal Advanced Therapeutics.

Common treatments available now provide only temporary relief to those affected by Alzheimer’s. There are no approved drugs that directly act on the disease mechanisms of Alzheimer’s disease. There is an unmet need to develop drug candidates to halt or cure Alzheimer’s disease, the statement said.

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Mice brain affected with Alzheimer’s disease when treated with TGR63 showed a significant reduction of amyloid deposits, validating its therapeutic efficacy. The mice also showed reduction of learning deficiency, memory impairment, and cognitive decline as revealed by distinct behavioural tests. These key attributes have validated the potential of TGR63 as a promising drug candidate for the treatment of Alzheimer’s disease.

Alzheimer’s disease severely affects patients, families, caregivers and, hence, is a major societal and economic challenge globally. The novel drug candidate TGR63 developed by the JNCASR team has potential as a promising drug candidate for AD treatment.