Gene Therapy Moves Beyond Rare Disease to Target Diabetes

A child destined to lose the ability to walk, talk, and see is now thriving in school, living proof that rewriting human DNA can save lives.

Dr. Alessandra Biffi will never forget her first gene therapy patient. He was the fourth child in a family where three siblings had already died from metachromatic leukodystrophy, a devastating genetic disease. The treatment she developed at Milan's San Raffaele Telethon Institute saved his life.

Today, that same revolutionary approach is getting bigger. After decades focused on ultra-rare genetic conditions, gene therapy is expanding to tackle diseases that affect millions of people worldwide.

Biffi, now chief of pediatric hematology at Padua University Hospital in Italy, is leading a clinical trial called Immunostem. The study investigates whether gene therapy can prevent type 1 diabetes from fully establishing and progressing. The first phase launched just months ago.

"It could be another demonstration that gene therapy could be applicable to diseases that affect a large number of individuals," Biffi explained. That shift represents exactly what researchers have been working toward.

Gene therapy works by altering a person's DNA to treat or prevent disease. What started as a technique for single-gene disorders now treats certain cancers too. The spectrum keeps growing.

The Ripple Effect

The patient Biffi saved with Libmeldy, now an approved drug on the market, represents more than one life rescued. His survival opened doors for thousands of other children with the same condition. Now, expanding gene therapy to common diseases like diabetes could help millions more people worldwide.

Artificial intelligence is accelerating the field even faster. Scientists can use AI to design new molecules essential for editing DNA. In September, AI company Anthropic announced its Claude model helped discover a potential new gene editing mechanism.

Biffi sees AI as a powerful tool when properly managed. "In every setting, AI could be a great asset for advancement," she said.

The researcher stumbled into gene therapy almost by accident when a professor noticed her during medical school. Now she's a world leader in the field. "I have a lot of passion for what I do, and it's renewed every day," she told Euronews Next.

From a chance invitation to a laboratory decades ago, Biffi's career has touched countless lives. The first patient she treated remains deeply connected to her emotionally, a healthy reminder that science fiction is becoming everyday medicine.