Gene therapy: a treatment for more than rare diseases

After decades developing gene therapies for ultra-rare conditions, Alessandra Biffi is researching how altering a patient’s DNA could help treat more common diseases like diabetes.

Alessandra Biffi went into gene therapy by chance. A professor of haematology noticed her during her medical degree and invited her to do her thesis work in his laboratory.

Back then she was just a student; now she is a world-leading expert in gene therapy, a therapeutic approach altering a person’s DNA to treat or prevent certain conditions.

“I have a lot of passion for what I do, and it’s renewed every day, that’s really the driver,” she told Euronews Next.

A former director of the gene therapy programme at the Dana-Farber Cancer Institute in Boston, United States, Biffi is now chief of the paediatric haematology, oncology and stem cell transplant department at Padua University Hospital, in northern Italy.

She joined Euronews Tech Talks to discuss the state of gene therapy, what it means to use this technology to treat patients, and how artificial intelligence (AI) is changing this field.

Gene therapy: not just for rare diseases

Although gene therapy might sound like science fiction, it is a widely studied technique used to cure multiple diseases. It started being used as an approach to treat monogenic diseases, pathologic conditions caused by mutations in a single gene, but it is now also used to treat certain forms of cancer.

“The spectrum of conditions that are amenable to treatment by this kind of approach is enlarging to non-inherited conditions,” said Biffi, who is currently working on Immunostem, a clinical study investigating how gene therapy could prevent type 1 diabetes from fully establishing and progressing.

The first phase of the clinical trial for this treatment started a few months ago, and Biffi is hopeful about the results it could yield.

“It could be another demonstration that gene therapy could be applicable to not ultra-rare conditions, to diseases that affect a large number of individuals, which is really the kind of opportunities that the field is looking for right now,” Biffi explained.

Despite her efforts to develop gene therapy to treat common diseases, Biffi spent years of her career using gene therapy to cure patients affected by complicated conditions, and there is one that she particularly struggles to forget: “The patient that is mostly in my real emotional life is the first one I treated,” Biffi said.

“He was the fourth of a family where his previous siblings, unfortunately, were affected by the same genetic disease and succumbed because of the disease,” she continued.

Diagnosed with metachromatic leukodystrophy, a rare genetic disease that progressively robs patients of the ability to walk, speak and see, the patient was saved by a gene therapy drug developed by Biffi and her colleagues at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, where she previously worked.

This patient was the first one to receive such a treatment, a product called Libmeldy that is now available on the market.

“He [the patient] is now living in Italy, having a very healthy and successful life. He’s attending school, and he’s growing up. And clearly, I’m very, very emotionally bonded to him,” Biffi said.

Gene therapy and AI

Gene therapy is moving quickly, and as many other scientific fields, it is being impacted by AI.

For instance, at the end of September, AI giant Anthropic announced that its Claude model helped discover a new enzyme system that, the CEO Dario Amodei said on X, “could represent a new gene editing mechanism”.

Although experts are sceptical about the actual outcome of this discovery, Biffi has, in general, a positive view of AI applied to gene therapy.

“It has to be properly governed and managed, but I truly believe that in the field of discovery that is needed to develop new drugs, including gene therapy, AI could be of great relevance”.

According to Biffi, AI could help scientists design new molecules essential to edit DNA.

“In every setting, AI could be a great asset for advancement,” she said.

Euronews Tech Talks is a podcast produced, written, presented and published in English.

Source: www.euronews.com