A new pill is being tested as a novel treatment that could potentially stop the progression of dementia and reverse the brain’s biological age. 

Retro Biosciences, the company behind the longevity tablet, is supported by Sam Altman, a key figure in modern artificial intelligence, and is preparing to initiate a clinical trial.

The pill, known as RTR242, functions by eliminating the accumulation of ‘gunk’ in the brain, which builds up over time and is linked to neurodegenerative conditions. 

In healthy brains, these toxic clumps of proteins that degrade the organ as it ages are cleared out.

In individuals affected by degenerative diseases like Lewy Body dementia, Alzheimer’s, and Parkinson’s, the brain’s cleaning systems become less effective, allowing harmful proteins to gather and spread, harming neurons and disrupting their operation.

This novel pill aims to enhance a natural cellular cleaning process called autophagy, the body’s internal recycling mechanism, which is naturally activated by activities such as fasting. It targets cellular waste, breaks it down, and removes it.

By eliminating this toxic accumulation, particularly in the brain, the pill is intended not only to reverse damage and restore function but also to go beyond merely slowing disease progression.

Retro Biosciences’ strategy, which aims to extend healthy life by a decade, differs from other Alzheimer’s treatments that concentrate on removing specific plaques, like amyloid, to slow down cognitive impairment.

The daily pill will enter phase one clinical trials in Australia with the first patient receiving the treatment by the end of 2025.

TV personality Wendy Williams was diagnosed with frontotemporal dementia in 2023

Bruce Willis announced in 2023 that he was also diagnosed with frontotemporal dementia

Rather than simply pushing life expectancy numbers higher, Retro Biosciences aims to develop treatments that preserve people’s health and vitality well into old age, compressing the period of decline.

Joe Betts-LaCroix, chief executive of Retro Biosciences and a scientist known for his discoveries in biophysics, as well as for creating the world’s smallest personal computer, told Business Insider: ‘Curing cancer would add about three years to life expectancy, and curing heart disease about four.

‘Adding ten years of healthy lifespan to the adult population will be an even greater impact — one of the greatest achievements in the history of healthcare.’

An estimated seven million Americans are living with Alzheimer’s disease, the most common type of dementia, and that figure is expected to rise to nearly 13 million by 2050.

Approximately a million Americans are currently living with Parkinson’s disease, with nearly 90,000 new diagnoses occurring each year. The number of people with PD is expected to increase to 1.2 million by 2030.

Retro Biosciences has not released preclinical data, but said the pill focuses on cells that are still alive but are sick and dysfunctional because they are clogged with toxic, misfolded proteins.

The pill is designed to kickstart the process of autophagy, which acts as the cell’s ‘housekeeper,’ removing damaged organelles and aggregated proteins that are harmful to the cell, such as tau and amyloid beta.

As people age, the genes that regulate and execute this process become less active, and the entire system becomes more sluggish, allowing cellular gunk to accumulate faster than it can be cleared.

Joe Betts-LaCroix, chief executive of Retro Biosciences [pictured], stated that while curing cancer or heart disease would add three to four years to life expectancy, adding 10 years of healthy lifespan to adults would be one of healthcare’s greatest achievements

By 2050, Alzheimer’s cases among Americans 65+ are projected to nearly double, from 6.7 million to 12.7 million, driven by an aging population and the absence of a cure

Specific toxic proteins that accumulate in diseases like Alzheimer’s, such as amyloid-beta and tau, are particularly harmful because they are notoriously difficult to break down.

They can clog the autophagy machinery itself, which eventually leads to even more accumulation, causing the system to fail completely.

When the autophagy process fails, harmful proteins like amyloid beta and tau can build up, which is a key mechanism in the development of Alzheimer’s disease.

By restarting autophagy, the pill helps the struggling cells clean up, which can potentially restore their function and prevent them from dying in the first place.

Other recently approved drugs for Alzheimer’s, including Leqembi and Kisunla, function by removing amyloid plaques, a sticky buildup in the brain, to slow the progression of symptoms. Retro’s strategy is fundamentally different.

Betts-LaCroix said: ‘I tend to be more interested in therapeutics that reverse aging than ones that slow it down because the “slow it down” thing just feels kind of like a weak sauce.’

He added that the company, whose other projects include an innovative stem cell treatment for leukemia and a stem cell-derived therapy for central nervous system diseases, aims to ‘reset some aspect of our biology back to essentially a younger age.’

The success of the imminent Australian trial is crucial, as the company needs positive data to secure its target of $1 billion in a Series A funding round for larger future studies, a sum that would instantly position Retro among the giants of the longevity field, such as Jeff Bezos’s Altos Labs, which has raised over $3 billion from top tech investors.