Gene Therapy: A Medical Breakthrough Beyond Science Fiction
Gene therapy, a medical approach that targets the root causes of diseases at their genetic source, has moved from the realm of science fiction into clinical practice. While the latest Spider-Man film features the technique as a plot device, the reality is that approved gene therapy treatments are now available to patients across Europe.
In Spider-Man: Brand New Day, the superhero Peter Parker uses RNA interference, a genuine gene therapy technique, to manage his mutating DNA. The film's portrayal, while imaginative, draws on real scientific advances that have been decades in the making.
How Does Gene Therapy Work?
Gene therapy involves introducing nucleic acids, either DNA or RNA, into target cells for therapeutic purposes. This can be done in two ways: in vivo, where the treatment is delivered directly within the body, or ex vivo, where cells are extracted, modified, and then transplanted back.
Delivery methods use either viral or non-viral vectors. The three main types of gene therapy are gene addition, gene silencing, and gene correction. Gene addition introduces a new gene, gene silencing deactivates a problematic gene, and gene correction repairs a dysfunctional one.
Alberto Auricchio, director of the Telethon Institute of Genetics and Medicine in Naples and president of the European Society of Gene & Cell Therapy, explained the approach:
Gene therapy basically targets the root cause of a genetic disease.
What Is the State of Gene Therapy in Europe?
Gene therapy has progressed significantly since its early days. We are beyond clinical trials now, Auricchio confirmed. Several products have received marketing authorisation in Europe.
The first in vivo gene therapy approved in Europe was Glybera in 2012, which treats severe pancreatitis due to lipoprotein lipase deficiency. The first ex vivo therapy, Strimvelis, received authorisation in 2016 and treats severe combined immunodeficiency.
What Makes Gene Therapy Promising?
The primary advantage of gene therapy is its durability. These treatments are designed to be administered once and provide long-lasting effects. Auricchio noted that some approved drugs have shown stable therapeutic effects over decades of patient follow-up.
However, significant challenges remain. The economic burden is substantial, with some treatments costing millions of euros. Strimvelis, for example, carries a price tag of €594,000 excluding VAT.
Auricchio highlighted the market difficulty:
These drugs are developed for rare diseases in many cases, which are not a very attractive market for typical developers like pharmaceutical companies.
While Spider-Man's fictional world offers convenient solutions through brilliant scientist allies, real-world gene therapy requires sustained investment and scientific dedication. The field continues to evolve, offering hope for patients with conditions that were once considered untreatable.
This article is based on an episode of the Euronews Tech Talks podcast.