A leading Scots scientist has defended controversial claims that an experimental drug could slow progression for Alzheimer’s disease, saying it has been backed in an upcoming study.
Researchers told a major Alzheimer’s conference in Toronto that the twice-daily LMTX pill was the first ever to slow brain shrinkage in dementia patients, which is a way of measuring degeneration of brain cells.
Professor Claude Wischik, of Aberdeen University, has been working on the potential treatment for 30 years, which focuses on dissolving proteins – known as tau – that form into toxic tangles in the brain. The 15-month trial examined 891 people with mild to moderate Alzheimer’s who were given either LMTX or a low dose of the drug to act as a placebo.
Overall the trial failed in its aim to slow progression of the disease, as the majority did not see any benefit.
However a secondary analysis of 15 per cent of patients, who were not already taking other drugs, saw improvements to thinking power and a reduction in the death of brain cells.
The claims have sparked concern among scientists as only a small number of patients were involved, but Prof Wischik told The Scotsman the findings have been backed up in a further study of 800 patients due to be published in the autumn.
Prof Wischik, professor of psychiatric geratology at Aberdeen University and co-founder of the spin out company TauRx, said: “It could have been a chance finding but the fact that it has been replicated in a second study gives me confidence.
“These are result that are difficult to set aside as being meaningless.”
The team aims to apply for a licence for the drug after publication of the further study later this year.
Dr Serge Gauthier, of McGill University, Canada, who presented the findings to the conference, said: “In a field that has been plagued by consistent failures of novel drug candidates in late-stage clinical trials and where there has been no practical therapeutic advance for over a decade, I am excited about the promise of LMTX as a potential new treatment option for these patients.”
The results of this trial were hotly anticipated as there are no meaningful treatments for Alzheimer’s disease, which affects around 90,000 Scots.
A number of experts were unconvinced by the findings, and called for more research to be done.
One of the major issues with LMTX – sometimes known as LMTM – was there was no explanation why it does not work when patients were taking other drugs.
Dr David Reynolds, chief scientific officer at Alzheimer’s Research UK, said: “A silver lining appears to be some encouraging signals from people who took LMTM as a monotherapy, but it’s hard to draw firm conclusions from the small numbers of people in this group.
“In the 15 per cent of people who were taking LMTM alone, there were marked benefits on day-to-day function as well as memory and thinking which must be explored further.
“Despite a lack of clarity around exactly how LMTM targets tau, the promising observation that those treated with the drug as a monotherapy showed reduced brain shrinkage provides evidence that the drug may act on the disease process. While today’s announcement marks an important step in the evolution of Alzheimer’s clinical trials, we must be cautious in our interpretation.”