Gene therapy: The breakthrough that inspired Spider-Man | Euronews Tech Talks

If you are a science nerd and a Marvel fan, you might have noticed that gene therapy, a promising medical approach to treat diseases by targeting their genetic causes, made an appearance in the latest iteration of the arachnid superhero franchise, Spider-Man: Brand New Day. ADVERTISEMENT ADVERTISEMENT In this film, Peter Parker or Spider-Man feels weird. His DNA mutates, and his spider side starts taking over, providing him with new and sometimes difficult-to-control powers. To manage them, Parker resorts to RNA interference, a real-world gene therapy technique that silences specific gene activity. But how does gene therapy work? And what are its limitations? To answer these and more questions, Euronews Tech Talks interviewed Alberto Auricchio, director of the Telethon Institute of Genetics and Medicine in Naples, professor at the University of Naples and president of the European Society of Gene & Cell Therapy. How does gene therapy work? Originally used to treat monogenic diseases, gene therapy is now employed and studied to find a treatment for a wide range of illnesses, including cancer and HIV. “Gene therapy basically targets the root cause of a genetic disease,” Auricchio explained. This medical approach essentially consists in the introduction of nucleic acids (DNA and RNA) into target cells and tissues for therapeutic purposes. Nucleic acids can be introduced into target cells either in vivo (directly within the body) or ex vivo (in cells extracted from an organism and then transplanted inside the body). In both cases, delivery is achieved using either viral or non-viral vectors. Types of gene therapy include gene addition and gene editing, with the latter being further divided into gene silencing and gene correction. As the names suggest, gene addition consists of adding a gene; gene correction is the fixing of a dysfunctional gene; and gene silencing deactivates a gene. What’s the state of gene therapy in Europe? Gene therapy is a complex and evolving field, but it is not only material for science-fiction stories. “We are beyond clinical trials now. There are approved [gene therapy] products that are available to patients,” Auricchio told Euronews Next. The first in vivo gene therapy approved in Europe was Glybera, which received the European Commission marketing authorisation in 2012 and offers a medical treatment for patients with severe or multiple pancreatitis attacks due to lipoprotein lipase deficiency. The first ex vivo gene therapy approved in Europe was Strimvelis, which received authorisation in 2016. This drug treats severe combined immunodeficiency due to adenosine deaminase deficiency and was developed at the Hospital San Raffaele in Milan. What makes gene therapy promising? Among others, gene therapy has a crucial advantage: it is a durable treatment. “There are approved gene therapy drugs, for which we have decades of follow-up in patients where we know that the therapeutic effect is stable over decades, and in theory for the lifetime of the individual,” Auricchio explained. As a matter of fact, gene therapy drugs are designed to be a once-in-a-lifetime treatment, meaning that a single administration should last for a long time. On the other hand, beyond the scientific difficulties, the main limitation of gene therapy is the economic obstacle. “These are very expensive drugs to be developed, and those that get to the market have very high prices, some of them matching millions of euros,” Auricchio explained. For instance, the cost of Strimvelis amounts to €594,000 excluding VAT. “These drugs are developed for rare diseases in many cases, which are not a very attractive market for typical developers like pharmaceutical companies; the high development cost of this drug is a challenge,” Auricchio said. Spider-Man: Brand New Day does not explore the economic problem behind gene therapy. Peter Parker is lucky to know the brilliant scientist Bruce Banner or Hulk, who helps him develop his therapy. But real-life science is often more challenging than a superhero movie. Euronews Tech Talks is a podcast produced, written, presented and published in English.