(Bloomberg) — After decades of researching a treatment for Alzheimer's, Claude Wischik is set to find out whether his lonely 30-year battle can lead to one of the first real treatments for the memory-destroying brain disorder that afflicts millions worldwide.
The co-founder of Singapore-based TauRx Pharmaceuticals Ltd. plans to present results from ongoing final human trials on its experimental Alzheimer's drug, LMTX, as early as July. An earlier study showed that patients given the company's treatment had better cognitive scores than those who didn't get it, according to research it published in the Journal of Alzheimer's Disease.
The stakes are huge, and researchers estimate that more than 40 million people worldwide are living with Alzheimer's and dementia. The world's largest pharmaceutical companies from Pfizer Inc. (NYSE:PFE) to Biogen Inc. (Nasdaq:BIIB) have poured resources into the field, but success has so far largely eluded scientists. There is still no cure for Alzheimer's and no medicines to slow the progression of the disease.
Existing drugs mainly offer symptomatic relief for patients, and advisory firm GBI Research estimates that the market for Alzheimer's therapies has the potential to expand from $5.2 billion in 2014 to $11.3 billion in 2021 in eight major countries including the United States and Japan.
Between 1998 and 2014, there were about 123 experimental Alzheimer's medicines under development that were halted or did not receive regulatory approval, according to a report by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA).
Successful trials would also be a boon for TauRx's backers, including its biggest shareholder, Malaysian investment holding company Genting Berhad, and Singapore's state investment company Temasek. Promising results might open the door for a possible initial public offering and tie-ups with large pharmaceutical companies, its executives say. If the trials are successful, the company would apply to both E.U. and U.S. regulators, and the timing of any approval would depend highly on the Phase 3 data, they said.
Wischik, a professor at the University of Aberdeen in Scotland, has spent his career looking for a drug to dissolve tangles of a protein called tau, one of the hallmarks of Alzheimer's brain abnormalities. The majority of Alzheimer's researchers have focused on another kind of protein clump known as beta-amyloid, which has had mixed results.
"Looking for ways to prevent tau aggregation is definitely a viable path for drug discovery," said Rosa Sancho, head of research at the charity Alzheimer's Research UK. If the drug turns out effective and safe, "for the field it would be incredible, there are no treatments at the moment and they are so desperately needed."
Success isn't guaranteed for TauRx. Sancho warns of pitfalls given the difficulties researchers have historically had with Alzheimer's. "I would be cautious because in the past few years we've had so many failures in trials," she said, referring to tests from many pharma companies that failed to produce a viable drug.
Other treatments
In 2013, as many as 5 million Americans were living with Alzheimer's disease, according to the U.S. Centers for Disease Control and Prevention (CDC).
Other experimental Alzheimer's treatments are also far along in clinical tests. Eli Lilly & Co. (NYSE:LLY) is conducting final stage trials on the beta-amyloid-directed drug solanezumab. Trial results announced in July last year showed that patients who had been taking its drug longer did better on a cognitive test than those who hadn't. This month the company said it had changed the primary goal of a test on the drug to focus solely on whether it can forestall changes that may occur early in the course of the disease.
