Alzheimer's drugs - why an NHS ban may not be bad news

There has been news this week of what seemed at first sight like a major setback for Alzheimer’s researchers and patients seeking a disease-modifying drug. They thought they had got not one but two - lecanemab and donanemab. A cause then for huge celebrations with charities talking. of a major breakthrough. But the National Institute for Health and Care Excellence (NICE) has snuffed out that optimism by ruling that neither drug can be prescribed on the NHS because they do not provide enough benefit given their huge costs.

One cannot but sympathise with those who thought they had found a way of staving off dementia only to have it snatched away. But NICE’s decision makes sense and in the long run may prove beneficial in directing researchers to focus on more effective ways of slowing Alzheimer’s and other neurological diseases such as Parkinson’s.

The NICE briefing on its cost/benefit analysis of donanemab and lecanemab has very thin pickings in the benefits column contrasted with a whole list of negatives when it comes to costs. Patients given the drugs in phase 3 trials did appear to deteriorate more slowly than those given a placebo but this applied only to a relatively small subset of the Alzheimer’s population.

The committee studying lecanemab noted that “the treatment effect was small and that the duration of the treatment effect was uncertain.”

The cost of fortnightly infusions of the drug in hospital was high, and then there ere the costs of identifying suitable patients and monitoring them for side-effects with lumbar punctures and MRI scans.

One very serious potential side effect is brain swelling which has reportedly caused several deaths.

While the cost the NHS would have to pay for the drugs is confidential , patients in the US pay between £20,000 and £25,000 for them and it has been estimated that they could cost the NHS £1.5 billion a year.

The donanemab report shows that there is even a lack of clarity about whether the drugs work at all:

“The submission from the Association of British Neurologists stated it was unclear if the donanemab trial results were clinically meaningful and this is currently being debated in the clinical community.”

What this sentence only begins to hint at is that there is a bitter row ongoing in the Alzheimer’s research community over the causes of the disease and what that means for the path to a cure. The dominant camp for some years has put its weight behind the amyloid theory, the idea that the build up of a protein called amyloid in the brain is what triggers Alzheimer’s.

But a book called Doctored by the medical journalist Charles Pillar details a series of cases where data supporting the amyloid theory was falsified and alleges that the rush to invest billions in research into drugs combating amyloid build up has been generated by fraudulent scientific papers. I found the book both convincing and worrying although one review in the Lancet by John Hardy, who describes himself as part of what the author calls an “arrogant “cabal” of researchers who support the amyloid hypothesis” is dismissive.

Hardy, writing before the NICE ruling on lecanemab and donanemab, says “the risk–benefit relationships of these drugs are being appropriately discussed by regulators and in public fora. A book by a non-expert is not the correct place for these discussions.” Perhaps I am biased but over the years “non-expert” journalists sticking their noses in where they are not wanted have uncovered all sorts of important stories, from thalidomide to the Theranos blood testing fraud, that experts have been slow to spot.

The positive outcome of all this is that other areas of Alzheimer’s research, notably the role of another protein, Tau, which have struggled to get funding when it was mostly heading in the direction of the amyloid theory, may now get a better hearing.

The other thing that strikes me is the relative speed things happen in the Alzheimer’s world compared with the Parkinson’s research scene. I have written about the painful progress over 16 years of trials of exenatide, an existing diabetes drug that ultimately failed as a Parkinson’s disease modifier,. So I am amazed to learn that donanemab was only discovered in 2013 and by 2024 had been approved by America’s FDA. Even if it and lecanemab are eventually deemed failures, they will have failed fast by the standards of the pharmaceutical industry, if not Silicon Valley.

And that is why this week’s news of a partnership between Cure Parkinson’s and Alzheimer’s Research UK is so welcome. The two charities each have their own projects aimed at making clinical trials speedier - Cure Parkinson’s International Linked Clinical Trials and Alzheimer’s Research UK’s Clinical Accelerator. With researchers now finding similarities between what happens in the brain in Alzheimer’s and Parkinson’s the hope is that by working together they can deliver new drugs at a faster pace.

For a Parkinson’s research community which receives tiny amounts of government funding teaming up with their somewhat better resourced Alzheimer’s colleagues must make good sense. Which brings me, as ever, to the Movers and Shakers parliamentary petition calling for better care and more research funding for people with Parkinson’s.

The total on Thursday evening

We now have nearly 50,000 signatures on the petition so the target of 100,000 by September 10th, triggering a House of Commons debate, now looks realistic. But only if YOU sign up - yes, I’m looking at you.

Sign right here.