Alzheimer’s charity chief: FDA approval for new drug is a historic moment

The chief executive of a leading Alzheimer’s charity has hailed a “historic moment” after US regulators approved the first new drug to treat the disease in almost 20 years, offering fresh hope for millions of patients worldwide. 

Hilary Evans, chief executive at Alzheimer’s Research UK, told Reaction that the FDA’s approval was the source of “cautious optimism” and that while more data was needed, “overall this gives great comfort to people who have been diagnosed with a disease where there are no treatments”.

Unlike existing drugs for Alzheimer’s, Aducanumab targets the underlying cause of the disease rather than the symptoms and its approval is a major step forward in the hunt for preventative treatment. 

The drug works by targeting amyloid, a protein that forms abnormal clumps in the brains of people with Alzheimer’s that can damage cells and trigger dementia.

The US Food and Drug Administration (FDA) said there was “substantial evidence that Aducanumab reduces amyloid beta plaques in the brain” and that this is “reasonably likely to predict important benefits to patients”.

Despite the FDA approval, the effectiveness of the new drug is disputed by some scientists, who have called for more research.

In March 2019, late-stage international trials of Aducanumab, involving about 3,000 patients, were halted when analysis showed the drug was no better at slowing the deterioration of memory and thinking problems than a placebo.

But later that year, the US manufacturer Biogen analysed more data and concluded the drug did work if it was given in higher doses. The company also said it significantly slowed cognitive decline.

The FDA’s approval for Aducanumab is conditional, which means that Biogen will need to complete a trial to confirm that removing the amyloid plaques offers cognitive benefits.

Despite the controversy, the approval of the drug is still seen as a crucial step forward in treating people with early Alzheimer’s.

According to the World Health Organisation, an estimated 30 million people globally suffer from Alzheimer’s. In the UK, some 500,000 people are thought to be living with the disease.

Aducanumab could be available for patients in the US in as little as a few months. But use in the NHS would require the Medicines and Healthcare Products Regulatory Agency (MHRA) to approve the drug as safe and effective, and the National Institute for Health and Care Excellence to agree that it represented value for money. 

Alzheimer’s Research UK has written to Matt Hancock, the health secretary, to prioritise and accelerate research into Aducanumab, which is administered via monthly infusion, and could pose significant logistical challenges to an NHS system that is not currently set up to deal with monthly infusion treatment on this scale.

The MHRA’s decision is expected this autumn but the whole process of approval and cost analysis for the UK health system could take around 12 to 18 months. 

Shorter term, Evans says the FDA’s decision will “invigorate” the dementia research community in the coming months – bringing companies and pharmaceuticals into efforts to find more and better treatments for other diseases that cause dementia.

Like any drug that is first to the market, Evans said Aducanumab is not going to be the best treatment in the world and is likely to help only a small number of people. She said: “I think we can take this as an opening of the floodgates to a better understanding about how we can treat dementia.”