Kate Andrews

Will the pandemic put gene therapy advancement at risk?

Will the pandemic put gene therapy advancement at risk?
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When Boris Johnson delivered his first speech as Prime Minister on the steps of Downing Street, we were living in a different world: Brexit and a looming general election were the issues of the day, and a pandemic that would come to dominate every aspect of public policy was still six months off our radar.

The Prime Minister was far more comfortable in last year’s territory — negotiating with Brussels and wooing voters — and is loath to give up the agenda crafted before Covid. But even as vaccine updates bring some hope that we could be at the ‘beginning of the end’ of this pandemic, there’s no doubt that Johnson’s plans have been thrown off course — and it’s unlikely every big idea will get back on track before the next election.

We are faced with trade-offs: which non-Covid issues will be prioritised, now that so many resources will be redirected to our virus recovery? From the levelling-up agenda, to supporting a struggling arts sector, to investing in medical innovations and technology, there is no shortage of sectors competing to be top of the list.

On those Downing Street steps in July last year, the Prime Minister paid lip service to the world-leading developments in life science. ‘It is here in Britain,’ he boasted, ‘that we are using gene therapy, for the first time, to treat the most common form of blindness.’ While Johnson has found himself in hot water recently for making claims about ‘world-beating’ technology — concerning the UK’s struggling Covid Test and Trace system — he wasn’t off the mark back then: Britain is known for its advancements and investments in gene therapy.

The treatment — inserting genes, instead of medicines or drugs, into the cells of sick patients — has the potential to transform the lives of those suffering from a wide range of diseases, including immune deficiencies, neurodegenerative diseases and certain types of cancer. Patients with chronic or even fatal conditions, such as muscular dystrophy, can see a lifetime of hospital visits drastically reduced thanks to one-off treatments, dramatically improving their quality of life.

But while thousands of trials have taken place in the past 20 years, only a handful of therapies have made it and become readily available for patients. The road to gene therapy advancement is promising but long. And with focus currently directed towards pandemic planning and vaccine production, there is rising concern that other medical innovations will be sidelined.

Does that put gene therapy advancement at risk? The best indicator is to follow investment. In September the government took further steps to deliver on its manifesto promise to ‘make the UK the leading global hub for life sciences’ by launching the National Genomic Healthcare Strategy, or Genome UK. Its ambition is to create the ‘most advanced genomic healthcare system in the world’, which will include offering genome sequencing as part of routine care. For patients, this could eventually mean knowing the health status of their DNA like they might know their height, weight or cholesterol.

The government is optimistic that this will advance gene therapy development and access to new levels, with life sciences minister Lord Bethell of Romford telling The Spectator: ‘The Genome UK strategy will help create the most advanced genomic healthcare ecosystem in the world, where government, the NHS, research and technology communities work together to harness the full potential of genomics to give patients the very best predictive, preventive and personalised care.’

The launch of a defined strategy has come alongside cash investment. Rishi Sunak used his first Budget in March to increase Research and Development spending from £9 billion to £22 billion by 2024/5, which stands to benefit the gene therapy sector — though Britain still falls behind other developed countries in what it invests in R&D. Meanwhile the Cell and Gene Therapy Catapult — a centre in Westminster dedicated to advancing research and manufacturing of gene therapy — has been provided with more than £70 million of additional funding to support its 200 gene therapy experts, according to the Life Sciences Industrial Strategy Update published in January.

Still, there are lingering questions as to whether these investments go far enough, and how accessible treatments will become. Cost and reimbursement barriers still stand between patients and gene therapies that have come to market (explored by Dr Roger Henderson on p8) and Covid-19 has created problems as well: hospital waiting lists have tripled since last year.

Covid means we’re set to wait a while for further financial updates from the government, but this has not put gene therapy advancement out of sight or mind. Across government departments, the contribution of the sector is becoming better recognised, with the Department for International Trade pledging on social media to help direct foreign investment to Stevenage, Hertfordshire — which is becoming a hub for cell and gene therapy. The Department of Health continues to advocate for gene therapy, with Lord Bethell also telling The Spectator that he is ‘immensely proud of the UK’s position as a global leader in genomics, with the highest number of cell and gene therapy commercial developers in Europe, as well as the most cell and gene therapy clinical trials’.

Gene therapy is clearly not the medical or scientific phenomena to dominate the news headlines this year — but the building blocks are being put in place to make it the conversation leader in years to come. Given its potential, the headlines it generates are likely to be far more optimistic than the ones we’ve had this year.

This article first appeared in a Spectator supplement: The evolution of gene therapy. Sponsored by bluebird bio.