Science journalists are cautioned never to use to the four letter word “cure.”
But when it comes to gene therapy, clinicians are daring to use that word.
They talk of witnessing “biblical results.” Children with congenital blindness are seeing; three year olds with spinal muscular atrophy (SMA) who should have died by the age of two, are walking and skipping.
And it’s not just clinicians who are frothing at the mouth. Big pharma is diving into this space. Last May, Novartis spent $US 8.7 billion to purchase AveExis a gene therapy company that treats SMA. This February, Roche paid $4.8 billion to buy the start-up company Spark, whose gene therapy product treats blindness.
As an author of two books about the stem cell and genome revolutions, there are some remarkable features to the gene therapy revolution.
For one thing, it arrived without me (and many others) noticing. We all seem to have been dazzled by the hype around CRISPR, a technology that has the power to readily alter the DNA of everything from mosquitoes to mankind, though it has yet to prove itself in the clinic.
For another, gene therapy seems set to disrupt the way we deliver and pay for medicine.
The costs are staggering. Novartis has suggested a price tag of $4-5 million for its one-off SMA cure – and that it would be well worth it! We should find out soon as the FDA is set to approve the treatment this May.
In this talk allow me to guide you through the gene therapy revolution and how Australian researchers are trying to get into the game.
Dr Elizabeth Finkel is the recipient of the 2019 medal from the Australian Society for Medical research, the first time it has been awarded to a journalist.
After a PhD in Biochemistry from the University of Melbourne, she spent five years at the University of California, San Francisco. Returning to Melbourne she turned to freelance journalism. In 2005 she co-founded the popular science magazine, Cosmos, and from 2013 to 2018 served as Editor in Chief.
She has written two books: ‘Stem Cells: Controversy at the Frontiers of Science’ and ‘The Genome Generation’.
She now writes for Cosmos, Science, and The Monthly and contributes to the ABC Science Show.