The new drug could prevent the disease before it starts, but at a cost

As hay fever season approaches once again for allergy and asthma sufferers in the Northern Hemisphere, California researchers are working on a new treatment that could prevent childhood asthma altogether through a slight adjustment to the bacteria in the gut.

Scientists from the universities of Berkeley and San Francisco have found a link between these microbes and the chances of 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 bacteria to eliminate the risk of children developing lung disease.

We were at the stage where we have target organisms, and even non-target genes, that we know contribute to a wide range of clinical outcomes, says Susan Lynch, head of the Benioff Center for Microbiome Medicine in San Francisco and principal investigator on the project about asthma

This is one of several technological and pharmaceutical advances that could prevent diseases before they start allowing for long-term savings in healthcare delivery and patient suffering.

And these preventive treatments are increasingly important, as health systems strain under the weight of an aging population and rising levels of chronic disease.

Susan Lynch, head of San Francisco’s Benioff Center for Microbiome Medicine and principal investigator of the asthma project

Lynch first identified a small molecule in children’s gut bacteria linked to the risk of childhood allergies and asthma in 2019. This led to a project to remove the bacteria’s ability to create the molecule, instead of removing the bacteria itself, Lynch explains. Its precision engineering rather than pulling things off in a non-specific way, he says.

Treatments like this carry huge up-front research and development costs, but by preventing chronic disease, they could lead to long-term savings: the annual costs of asthma in the US alone are $56 billion, mostly related to hospital stays , according to the Asthma and Allergy Foundation of America.

Also, addressing common diseases like asthma with gene editing could help reduce antimicrobial resistance linked to chest infections that are more common in asthmatics, 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 Crispr-Cas9 DNA sequence.

How DNA defects can be edited.  Graphic that explains how the Crispr-Cas9 technique can fight the disease from its origin

While Lynchs work is related 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 intestinal inflammation and Alzheimer’s disease.

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

But the technology is still in its infancy and wider deployment won’t happen for many years. There are also huge upfront costs for gene-editing treatments that can outweigh the cost savings later on. The only approved Crispr-based treatment to date, Vertex Pharmaceuticals Casgevy, which can cure sickle cell disease, is complex to deliver and costs $2.2 million per treatment.

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

One of the drugs, Novo Nordisks Wegovy, received approval in the US for the prevention of heart attacks and other cardiovascular events in March. But the drug has faced political criticism over its cost, with Sen. Bernie Sanders calling Wegovys list price of $1,349 a month outrageously high.

The high costs must be weighed against the growing problem of chronic disease, says David Wheeler, professor of renal medicine at University College London. For example, levels of advanced chronic kidney disease (CKD) are predicted to increase by 60% by 2032 in eight countries, including the US, China and Brazil, according to a study by scientists at UCL and the pharmaceutical manufacturer AstraZeneca.

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

However, potential buyers must be convinced of the value of preventive drugs, as the resulting drugs can often be very expensive. For large public health budgets, public health investments could be better value for money.

Properly funded public health interventions are a good example, says Dave Buck, senior fellow for public health and inequalities at the Kings Fund, a British health think tank.

In the UK, the most effective prevention measures have been local authority schemes to stop smoking and promote healthy eating. These are funded by local government public health grants, which are 3 to 4 times more cost-effective than spending by the National Health Service, which funds the majority of treatment in the country, according to a 2020 study by academics at the University of York

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

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

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