The ‘Ozempic for the brain’ pill that could tackle sleep disorders, ADHD and depression…and why scientists believe it’s the biggest breakthrough in 30 years

First we had GLP-1s that not only help people lose weight and manage diabetes, but have since been found to help with other conditions even if those taking them don’t slim down.

Now scientists are exploring various uses for another drug, which it is hoped will prove to be an ‘Ozempic for the brain’, able to target several brain-related conditions.

GLP-1 agonist drugs work by mimicking the action of the hormone glucagon-like peptide-1 (GLP-1), which is released after we eat.

The new drug is designed to boost levels of another peptide, orexin, a kind of chemical messenger made in a part of the brain called the hypothalamus, which regulates when we sleep and when we wake up.

Now a twice-daily pill called oveporexton has been developed to ward off day-time sleep ‘attacks’, and could help thousands of people in the UK with narcolepsy – the neurological condition that makes those affected collapse into a sudden deep sleep without warning.

And just as GLP-1s have been found to help not only with weight loss but heart health, kidney disease and more, it is hoped that orexin-boosting medication will help with narcolepsy but also ADHD and sleep apnoea, which makes sufferers stop breathing briefly during sleep.

Orexin is also needed for attention, reward processing and motivation, for example – so the hope is that orexin-boosting medications may have other uses, including treating depression and addiction.

Oveporexton is the biggest breakthrough in narcolepsy treatment in decades and is the first drug to target the root cause of the condition rather than just the symptoms. It works by increasing production of orexin. Last month, the US Food and Drug Administration approved the use of oveporexton for narcolepsy – and now the UK’s National Institute for Health and Care Excellence (which vets the cost-effectiveness of NHS treatments) is evaluating whether it’s worth the estimated £120,000 per patient a year. It’s expected to reach a decision by April 2027.

The treatment targets type-1 narcolepsy – the most common form, accounting for about 75 per cent of cases – which is thought to develop when the immune system goes haywire and attacks healthy brain cells. This damages their ability to produce orexin.

The drug stimulates the receptors on the outside of brain cells that are responsible for increasing orexin production, boosting levels enough to bring sleeping and wakefulness under control.

Narcolepsy affects around 30,000 people in the UK. Most are children or young adults when it first strikes.

It’s thought this is because the immune system is at the peak of its powers early in life, so any abnormal reaction – where it attacks healthy tissue – is likely to be more destructive.

Sufferers can suddenly fall into a deep sleep several times a day, even in mid-conversation.

Very often these sleep attacks are accompanied by cataplexy – a sudden brief loss of all muscle control, which results in patients collapsing. Cataplexy can also be triggered by strong emotions, such as laughter, surprise, excitement and even anger.

Each bout of sleep lasts up to 20 minutes and can be followed by a short period of terrifying sleep paralysis – being completely unable to move or speak for several minutes afterwards.

Current drug treatments include modafinil, a stimulant which prompts the brain to pump out stress hormones such as adrenaline to try to stay awake during the day.

But the drug has a limited effect and, according to the NHS, has been linked to irregular heartbeats and dangerous spikes in blood pressure.

The other main treatment is sodium oxybate, a liquid medicine that patients drink before bedtime to promote deep sleep. This deep rest then helps reduce drowsiness in the day.

But the drug can also cause bad side-effects, ranging from dizziness, nausea and bedwetting to confusion, memory loss and sleepwalking.

According to one recent study, narcolepsy can heighten the risks of a premature death. A team of international researchers from five countries tracked thousands of narcolepsy sufferers over a 25-year period and found they were up to 30 per cent more likely to die at a younger age than those without the condition, according to results published in the Journal of Clinical Sleep Medicine in May.

Illnesses brought on by years of sleep disruption – including heart disease, type 2 diabetes and mental illness – were mainly to blame.

The search for a drug that could tackle the lack of orexin has been ongoing for almost 30 years. Oveporexton is the first in a new class of narcolepsy drugs – called orexin receptor-2 agonists – likely to come on stream over the next few years.

In clinical trials involving more than 270 narcolepsy patients who took the drug twice a day for three months, daytime alertness improved significantly, according to results released by the drug’s manufacturer, Takeda Pharmaceuticals.

Meanwhile, cataplexy rates fell by an average of 80 per cent. Side-effects were mostly mild and included night-time insomnia and more frequent urination.

Guy Leschziner, a professor of neurology and sleep medicine and a consultant neurologist at Guy’s and St Thomas’ Hospital in London, said a drug that replaces or stimulates orexin in the brain has been regarded as the holy grail of narcolepsy treatment for years.

He told Good Health: ‘This drug is the first agent to come to market, and trial data are extremely encouraging.

‘It is potentially very exciting for those who experience this debilitating neurological disorder, and we look forward to having access to it in the UK.’

Meanwhile, pharmaceutical companies are testing drugs that target orexin for sleep apnoea and ADHD (after narcolepsy patients treated with the new drug reported better attention). Researchers are also looking at an orexin receptor linked to reward and motivation – blocking it might help reduce addictive cravings while activating it could help patients with some forms of depression.