Experts urge health regulators to approve ‘turning point’ dementia drugs | Alzheimer’s

Health regulators are being urged to rapidly approve two gamechanging dementia drugs in order to ensure millions of people who could benefit are not left in limbo, after more stunning trial data.

The final results from a landmark study confirmed that donanemab, made by Eli Lilly, slowed cognitive decline in Alzheimer’s patients by 35%. Trial results published last year showed that a second drug, lecanemab, made by Eisai and Biogen, reduced the rate by 27%.

Health leaders hailed the arrival of the drugs as a turning point in the fight against Alzheimer’s, through which the disease could become just as manageable as conditions such as diabetes or asthma.

Dr Susan Kohlhaas, the executive director of research and partnerships at Alzheimer’s Research UK, said it was critical for regulators to act promptly to avoid patients experiencing frustrating waits for clinically effective treatments.

“We now have two potentially life-changing Alzheimer’s treatments on the horizon and we need to see rapid regulatory decisions so people who could benefit from these treatments aren’t left in limbo,” Kohlhaas said. “After 20 years without new Alzheimer’s medicines, people affected by this disease deserve to have answers about new treatments as quickly as possible.”

Eli Lilly has applied for approval for donanemab in the US and will do so in the UK in the coming weeks, according to a person familiar with the matter. “Submissions to other global regulators are currently under way,” they added.

On Monday evening, the health spending watchdog in England, the National Institute for Health and Care Excellence, said it had begun assessing whether donanemab could be used in the NHS.

Before Nice can give the drug the green light for NHS use, Eli Lilly must also gain approval from the UK medicines regulator, the Medicines and Healthcare products Regulatory Agency.

Lecanemab received the green light in the US earlier this month but it is still awaiting approval in the UK and Australia.

Alzheimer’s is the most common cause of dementia, one of the world’s biggest health threats. The number of people living with dementia globally is forecast to nearly triple to 153 million by 2050, and experts have said it presents a rapidly growing threat to future health and social care systems in every community, country and continent.

Final trial results of donanemab, published in the Journal of the American Medical Association and presented to the Alzheimer’s Association International Conference in Amsterdam on Monday, concluded that after 76 weeks of treatment it had slowed clinical decline by 35.1% in people with early Alzheimer’s whose brain scans showed low or medium levels of a protein called tau.

When the results were combined for people who had different levels of this protein, there was a 22.3% slowing in disease progression.

That means people with the disease could still go about performing day-to-day tasks including shopping, housekeeping, managing finances and taking medication.

The final results of the trial – known as Trailblazer ALZ-2 – examined the safety and efficacy of the drug. Researchers studied almost 1,800 people with early-stage Alzheimer’s. Half of them received a monthly infusion of donanemab – which works by removing a protein called amyloid that builds up in the brains of people with Alzheimer’s – and the other half were given a placebo over 18 months.

Researchers found that among a small number of people in the study there were some serious side-effects such as brain swelling. Three deaths in the donanemab group and one in the placebo group were considered “treatment-related”.

David Cameron, the former prime minister and president of Alzheimer’s Research UK, described the results as a breakthrough.

Mike Colley, who has been on the global trial of donanemab for two years, said: “I am one of the luckiest people you will ever meet, just for this.”

The 80-year-old, whose memory and ability to process information have been affected by the illness, told BBC News: “I seem to get more confident every day and I am sure this is going to be successful.”

Eli Lilly said it was ready to work with health regulators worldwide on the “appropriate regulatory next steps”.

Anne White, an executive vice-president at Eli Lilly and president of Lilly Neuroscience, said: “If approved, we believe donanemab can provide clinically meaningful benefits for people with this disease and the possibility of completing their course of treatment as early as six months once their amyloid plaque is cleared.”

Dr Richard Oakley, an associate director of research and innovation at 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 towards a future where Alzheimer’s disease 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.”

As well as approval by regulators, diagnosis would be key, Oakley said. “We can’t have a situation where treatments are approved for use … but people aren’t diagnosed early or accurately enough to be eligible.”

He added: “These trials show [these drugs] remove a protein called amyloid from the brain really effectively, and that seems to slow down the progression of the disease.

“It keeps people being able to do things like drive a car, manage finances, talk about current affairs, recognise family members for longer. We really believe this is the beginning of a new era for Alzheimer’s disease.”