Regulators urged to approve game-changing Alzheimer's drug donanemab

Regulators urged to approve game-changing new Alzheimer’s drug that slows decline by up to 60% amid hope donanemab could be on the NHS in two years

  • Experts say Brits with condition deserve not to left in limbo over the treatment
  • READ MORE: Incredible transformation of  Alzheimer’s patient on new drug

Britain’s regulators are being urged to rapidly approve new Alzheimer’s drugs that have been hailed as a ‘turning point’ in the fight against the disease.

Donanemab was found to significantly delay the worsening of symptoms in people with early stage Alzheimer’s in a landmark trial. 

The drug also slowed the progress of the disease by up to 60 per cent. 

It follows lecanemab to be the second drug to offer hope to patients and raises the prospect of the disease one day being treated like asthma or diabetes. 

The pharmaceutical firm behind donanemab, Eli Lilly, expects to apply for approval to sell the drug in the UK within the next six months.

Donanemab is given to Alzheimer’s patients through an IV infusion once a month. The monoclonal antibody — a man-made version of proteins produced by the body to fight-off harmful substances — travels to the brain . Once inside the organ, donanemab binds to toxic build-ups of amyloid plaque — a hallmark sign of the memory-robbing disease. This prompts immune cells, known as microglia, to clear them

Mike Colley started having problems with his memory and decision making a couple of years ago. Pictured with his son Mark

Experts hope this means that it will become available for use on the NHS as soon as 2025.

Dr Susan Kohlhaas, executive director of research and partnerships at Alzheimer’s Research UK, told the Guardian: ‘We now have two potentially life-changing Alzheimer’s treatments on the horizon.

‘We need to see rapid regulatory decisions so people who could benefit from these treatments aren’t left in limbo. 

‘After 20 years without new Alzheimer’s medicines, people affected by this disease deserve to have answers about new treatments as quickly as possible.’

Unveiling the full trial results of donanemab at the Alzheimer’s Association International Conference in Amsterdam yesterday, American pharmaceutical giant Eli Lilly and Company said it had already sought approval from US regulators the Food and Drug Administration (FDA).

READ MORE: ‘My dad is so full of life again – now we have hope’: Incredible transformation of 80-year-old with Alzheimer’s taking game-changing new drug donanemab 

The company said it expected to apply to UK regulators within six months.

The Medicines and Healthcare Products Regulatory Agency (MHRA) will determine if both drugs are safe for UK use, under the first step of approval.

Drugs watchdog NICE will then assess whether they are cost effective for the NHS.

These processes are likely to take at least 18 months, giving the health service time to gear up for any mass roll-out.

The first-generation drugs are hoped to pave the way for even more effective future therapies.

Charities also called on health officials to ensure the UK is ready to roll out this initial wave of treatments, which have the potential to help 720,000 people.

While welcoming the ‘new era’ in treatment, they warned only 2 per cent of patients could receive the drug at present because diagnosis in Britain is inadequate.

Richard Oakley, research chief at the Alzheimer’s Society, said: ‘This is truly a turning point in the fight against Alzheimer’s and science is proving that it is possible to slow down the disease.

‘Treatments like donanemab are the first steps toward a future where Alzheimer’s could be considered a long-term condition alongside diabetes or asthma. 

‘People may have to live with it, but they could have treatments that allow them to effectively manage their symptoms and continue to live fulfilled lives.’

But he said having the resources to diagnose Alzheimer’s would be key to making treatments such as donanemab and lecanemab available.

He added: ‘We can’t have a situation where treatments are approved for use in the UK but people aren’t diagnosed early or accurately enough to be eligible.

Mr Colley surprised his family at his own 80th birthday party by belting out Frank Sinatra’s My Way. He told BBC News: ‘That’s the confidence I have now. I’d never have done that even 12 months ago.’

‘We need early, and accurate, diagnoses available for everyone and the NHS ready to roll out treatments such as donanemab and lecanemab if and when they are approved in the UK.’

Given as a monthly infusion in hospital, donanemab was found to halt mental decline for more than a year in around half of patients. It was most effective in under-75s in the earliest stages of the disease.

Researchers examined almost 1,800 people with early-stage Alzheimer’s, with patients given either donanemab or a dummy drug for 18 months.

Those at the very earliest stage of disease, known as mild cognitive impairment, had the greatest benefit, with a 60 per cent slowing of decline.

Among patients with early Alzheimer’s, whose brain scans showed low or medium levels of a protein called tau, the drug was found to slow clinical decline by 35 per cent.

READ MORE: Are we REALLY at a ‘turning point in the fight against Alzheimer’s’? Breakthrough new drugs halt cruel disease’s decline… but experts warn crippling side effects (and cost) may outweigh any of the benefits

Levels of tau are a marker of how far the disease has progressed. When the results were combined to include people who had higher levels of that protein, there was a 22 per cent slowing in disease progression.

The drug works by using the immune system to remove a protein called amyloid, which builds up in toxic plaques in the brain and stops the cells communicating.

Consequently, donanemab is effective only in early-stage Alzheimer’s and not other forms of the disease, such as vascular or frontotemporal dementia.

Researchers said some patients were taken off the drugs after as little as six months because scans showed their amyloid had completely disappeared. 

Early indications suggest the plaques, which were decades in the making, would take several years to return.

Lecanemab, made by drug companies Eisai and Biogen was shown to slow decline by 27 per cent, leading to its US approval this month.

Scientists said the successes of both donanemab and lecanemab ended the decades-long debate over whether the accumulation of sticky amyloid plaques was at least partly responsible for dementia.

Dr Maria Carrillo, Alzheimer’s Association chief science officer, said: ‘We used to even just a short year ago still argue about the fact that amyloid and perhaps tau were not connected, and that amyloid might not have anything to do with this disease.

‘And today we are seeing evidence upon evidence that that is not the case, in that they are very closely linked. 

‘And in fact, removing one does impact the other and improves outcomes for people. So that is really a big message for the field.’

As well as delaying the worsening of symptoms for between 4.5 to 7.5 months on average, the drug also meant patients could continue to perform daily activities for longer.

Researchers have unveiled that donanemab slowed cognitive decline in Alzheimer’s by 35 per cent by removing toxic plaques in the brain 

Dr Mark Mintun, of Eli Lilly, said at the Amsterdam conference this week that it could help people with early, symptomatic Alzheimer’s to ‘keep working, enjoying trips, sharing quality time with family’ and ‘feel like themselves, for longer’.

However, the treatment was not without some serious side effects such as brain swelling, which occurred in up to a third of patients. 

Three deaths were linked to the medication.

Professor Paresh Malhotra, head of neurology at Imperial College London, said: ‘If the drug is approved in the UK, then NHS services will need to adapt considerably to provide it and there will need to be honest discussion between patients, carers, and doctors about the benefits and risks associated with this type of treatment.’

It is not known how much donanemab will cost but lecanemab is around £21,000 a year in the US.

Kate Lee, Alzheimer’s Society chief executive, said: ‘This is a defining moment for dementia research. But new treatments could mean nothing if we don’t fix dementia diagnosis.

‘We estimate around 720,000 people in the UK could potentially benefit from these emerging new Alzheimer’s disease treatments if they’re approved for use here. But the NHS is simply not ready to deliver them.

‘Timely, accurate diagnosis is key, and currently only 2 per cent of people in England and Wales receive their diagnosis through the specialist investigations needed to be eligible for these treatments.’

Secretary of State, Steve Barclay, said: ‘These results represent a real step forward in the treatment of Alzheimer’s and will be welcomed by those living with the disease and their families. 

‘To protect patients all medicines have to go through the MHRA and NICE’s regulatory processes, but NHS England has in place a dedicated team to ensure the quickest possible rollout for if and when donanemab is approved.’

Some Brits are already seeing the benefits of donanemab.

Mike Colley started having problems with his memory and decision making a couple of years ago due to Alzheimer’s.

But he is one of only a few dozen UK patients to be part of the global trial, receiving a monthly infusion of donanemab for the past two years.

The 80-year-old, from Kent, said he feels ‘incredibly fortunate’ to be taking the immunotherapy drug.

Visiting a London clinic for treatment, he told the BBC: ‘I seem to get more confident every day and I’m sure this is going to be successful and they’re going to get all this rubbish off my brain.’

His son, Mark, added: ‘I never thought that I would see my dad so full of life again. Now we have hope and two years ago, we didn’t. That’s just an incredible difference.’ 


What is donanemab?

Donanemab teaches the body’s immune system to target harmful proteins, called amyloids, and destroy them. Amyloid build-ups are thought to be toxic to brain cells, eventually causing them to die, leading to the symptoms of Alzheimer’s disease. Alongside lecanemab, the drug is one of the first treatments designed to tackle the underlying disease and stop it worsening.

How is it given?

The treatment is given once a month, intravenously, via an infusion or drip. Patients would therefore need to be treated in hospital or a suitable ‘infusion’ centre. During the trial, people were treated for up to 18 months but were often found to be free from amyloids within six months.

Any side effects?

Researchers found some serious side effects, such as brain swelling and bleeds. Three participants died, which researchers said was linked to their treatment. Deaths have also occurred in trials of other similar, rival drugs.

Who would be eligible?

Patients would need to be in the early stages of Alzheimer’s disease and have amyloid protein build-up in the brain. Around 720,000 people could be eligible but currently only 2 per cent receive the necessary diagnosis through specialist investigations.

When might the drug be available?

Eli Lilly, the US pharmaceutical company behind the drug, said it would be applying to European regulators within six months. This includes the Medical and Healthcare Products Regulatory Agency which must give the legal go-ahead before any drug can be considered for use in the UK. It would then need to be reviewed by the National Institute of Health and Care Excellence to consider its cost-effectiveness, alongside its clinical benefits and side effects. It means the earliest donanemab could be available on the NHS is 2025.

Are we ready for it?

Not as it stands. Experts say the resource implications of delivering treatments like this are enormous. The NHS would need to train a skilled workforce to deliver the drugs, with investment in many more brain scans and infusion suites. It has just 88 PET scanners that can detect amyloid plaques, one of the lowest rates in the developed world.

What does this mean for prevention or cure?

‘Finally there is some hope,’ said John Sims of Eli Lilly when presenting the new results yesterday. This hope has been decades in the making and there is now significant promise in tackling the root causes of the disease. But while it is right this is hailed as a major breakthrough, there is still a long way to go before we can say we have beaten dementia.

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