World-first Alzheimer’s drug trial gets “very promising” results at UCL Queen Square Institute of Neurology

A world-first clinical trial at SRT grantee UCL Queen Square Institute of Neurology has shown “very promising” results for a new Alzheimer’s drug that “silences” the gene coded to produce tau – a protein which can form abnormal “tangles” thought to cause Alzheimer’s.

The trial involved 46 patients and looked at the safety of the drug – known as MAPTRx – and how well it targets the gene. MAPTRx was found to be safe, and biologically effective – reducing the amount of tau in the brain by over 50%.

Dr Catherine Mummery, a consultant neurologist who led the trial, said, “The results are a significant step forward in demonstrating that we can successfully target tau with a gene silencing drug to slow – or possibly even reverse – Alzheimer’s disease, and other diseases caused by tau accumulation in the future.”

The trial looked at three doses of the drug compared with a placebo and according to the study, the drug was well tolerated. Researchers also looked at levels of two forms of the tau protein in the central nervous system – a reliable indicator of Alzheimer’s – over the duration of the study. They found a greater than 50% reduction after 24 weeks in the treatment groups which received the highest dose of the drug.

This early clinical trial cannot prove that the drug improves the clinical condition of Alzheimer’s patients, but it does show biological signs of improvement. Dr Liz Coulthard, associate professor in dementia neurology at the University of Bristol, said; “As this is a phase 1 study, all it tells us is that the drug is good enough to take to full trials, i.e., the drug blocks the production of harmful protein without causing obvious dangerous side effects… Bigger trials are needed to test whether this effect actually helps individual patients. These trials are underway.”

Tara Spires-Jones, professor of neurodegeneration and deputy director of the Centre for Discovery Brain Sciences at the University of Edinburgh, also noted: “The data are very promising… I very much look forward to seeing results from the next stages of testing.”

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