New medicine could prevent disease before it begins — but at a cost

As hay fever season arrives, once again, for allergy and asthma sufferers in the northern hemisphere, researchers in California are working on a new treatment that could prevent childhood asthma entirely — through a slight tweak to bacteria in the gut.

Scientists at Berkeley and San Francisco universities have found a link between these microbes and the chances of suffering from childhood asthma. So, now, using Crispr gene editing — a new technology that allows precise changes to be made to DNA — they are exploring how to alter the bacteria to remove the risk of children developing the lung condition.

“We’re at the stage where we have target organisms, and even target genes, that we know are contributing to a wide range of clinical outcomes,” says Susan Lynch, head of San Francisco’s Benioff Center for Microbiome Medicine and the lead researcher on the asthma project.

This is one of several technological and pharmaceutical advances that could prevent diseases before they even begin — enabling long-term savings on healthcare provision and avoiding suffering for patients.

And these preventive treatments are becoming ever more important, as health systems strain under the weight of ageing populations and increasing levels of chronic disease.

Lynch first identified a small molecule in infant gut bacteria linked to the risk of childhood allergies and asthma back in 2019. This led to a project to remove the bacterium’s ability to create the molecule, rather than removing the bacterium itself, explains Lynch. “It’s precision engineering as opposed to taking things out in a non-specific way,” she says.

Treatments like this come with large upfront research and development costs but, by preventing chronic diseases, could lead to long-term savings: the yearly costs of asthma in the US alone are $56bn, mainly linked to hospital stays, according to the Asthma and Allergy Foundation of America.

In addition, tackling common diseases such as asthma with gene editing could help reduce antimicrobial resistance linked with the chest infections that are more common in asthma sufferers, Lynch adds.

The project will use tools developed by the Innovative Genomics Institute, founded by Nobel-prize winning-scientist Jennifer Doudna who was one of two discoverers of the gene editing potential of the DNA sequence Crispr-Cas9.

While Lynch’s work is linked to asthma, the Institute is also exploring how to apply Crispr to other challenging disease areas. Brad Ringeisen, executive director of the IGI, has identified links between gut inflammation and Alzheimer’s disease.

“Wouldn’t it be great if you could turn those mechanisms off, or shift the microbiome away at an early time point where you’re now preventing those diseases, or pushing them off further in life, rather than trying to treat something that’s really recalcitrant?” says Ringeisen.

But the technology remains in its infancy and rolling it out more broadly will not occur for many years. There are also huge upfront costs to gene editing treatments that may outweigh later cost savings. The only approved Crispr-based treatment to date, Vertex Pharmaceuticals’ Casgevy, which can cure sickle cell disease, is complex to deliver and costs $2.2mn per treatment.

Cost is also a contentious issue with new weight-loss drugs known as GLP-1s, which can treat obesity and diabetes, as well as lowering the risk of heart attacks and liver issues, according to clinical trial data.

One of the drugs, Novo Nordisk’s Wegovy, received approval in the US for prevention of heart attacks and other cardiovascular events in March. But the drug has faced political criticism over its cost, with Senator Bernie Sanders labelling Wegovy’s list price of $1,349 a month as “outrageously high”.

High costs have to be assessed against the increasing problem of chronic diseases, notes David Wheeler, a professor of kidney medicine at University College London. Levels of advanced-stage chronic kidney disease (CKD), for example, are forecast to rise 60 per cent by 2032 in eight countries, including the US, China and Brazil, according to a study by scientists at UCL and drugmaker AstraZeneca.

The findings are of clear interest to AstraZeneca: its Forxiga drug developed for diabetes and heart failure has also demonstrated effectiveness in preventing severe CKD. It sold nearly $2bn in the first few months of 2024, making it the company’s highest-selling drug.

However, potential buyers have to be convinced of the value of preventative medicines, as the resulting medicines can often be very expensive. For stretched public health budgets, investments in public health could be better value for money.

Properly funded public health interventions are a good example, says Dave Buck, a senior fellow in public health and inequalities at the King’s Fund, a British health think-tank.

In the UK, the most effective prevention measures have been local authority schemes for smoking cessation and promoting healthy eating. These are funded by public health grants from local government, which are 3-4 times more cost-effective than spending by the national health service, which funds most treatment in the country, according to a 2020 study by University of York academics.

But, while NHS budgets have been mostly protected in recent years, real spending per person from the public health grants has fallen 28 per cent since 2015/16, the Health Foundation has found.

“Obviously, I’m not against the high-tech stuff but you need to get the basics right,” says Buck. “You need to keep pushing at the boundaries but also focus on what’s behind it, as well.”