The first pill to slow down Alzheimer's disease is being analysed for potential use on the NHS, with regulators set to make a decision in April.
The daily medication, called hydromethylthionine mesylate (HMTM), was developed by British firm TauRX in Aberdeen.
If approved by both the Medicines and Healthcare products Regulatory Agency (MHRA) and the National Institute of Health and Care Excellence (Nice), it would mark the most significant advancement in NHS dementia treatment in decades.
The development follows the rejection of two other Alzheimer's drugs earlier this year on cost grounds.
If approved it would mark the most significant advancement in NHS dementia treatment in decades
,PA
HMTM works by preventing the build-up of tau protein clumps in the brain, which can damage neurons and lead to cognitive decline.
The oral medication could offer significant cost advantages over existing treatments like lecanemab and donanemab, which require regular hospital visits for intravenous administration.
NHS officials believe the pill, that can be taken at home, could reduce healthcare costs compared to treatments requiring two or four-weekly hospital sessions.
The drug was developed from methylene blue, a medicine previously used to treat malaria.
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The first pill to slow down Alzheimer's disease is being analysed for potential use on the NHS
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Professor Alistair Burns, former NHS clinical director for dementia, called the trial results "great news for people with Alzheimer's disease, their families and carers.
"We have reached an exciting time in the field of Alzheimer's disease treatment. After no new therapies for a generation, we are on the threshold of having a range of new treatments, including a tau-targeted oral therapy, which have the real potential to slow the disease process."
Trial results suggest HMTM has a better safety profile than other treatments, with fewer adverse side effects.
However, experts are divided on the drug's effectiveness, with concerns about unpublished and non-peer-reviewed clinical trial data.
A source close to the regulatory process claimed TauRX had provided limited evidence beyond the drug's effects of "making your urine turn green and your poo turn blue".
The source added that the company's case was "very difficult to understand" and appeared to be "trying to be complicated and confusing".
NHS officials believe a pill that can be taken at home could reduce healthcare costs
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The company has reportedly refused to accept previous trial results showing the drug performed no better than placebo.
This raises the possibility of HMTM becoming the third consecutive Alzheimer's treatment to face NHS rejection.
At the Alzheimer's Disease International conference in 2024, trial results showed no overall benefit over placebo on memory and cognitive decline, which the company attributed to unexpected placebo effects.
Dr Richard Oakley from Alzheimer's Society noted that in a small subset of participants with mild cognitive impairment, HMTM showed beneficial effects.
He highlighted that HMTM didn't cause the brain bleeding or swelling associated with drugs like donanemab or lecanemab.
However, Dr Oakley emphasised that without peer-reviewed publication of the phase 3 study results, it remains difficult to assess the drug's effectiveness.
The Nice committee is scheduled to make its decision no earlier than April.
