Custom cures: tailor-made drugs for rare diseases

定制疗法:针对罕见病的量身定制药物

Babbage from The Economist

2026-01-22

42 分钟
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单集简介 ...

A teenage girl in Britain recently received a custom-made drug to fix a fault in her genetic code. Doctors hope that it will treat her ultra-rare neurodegenerative condition. It is also part of a medical trial that could transform the way that customised medicines are made available to people who otherwise have no alternatives.  Host: Alok Jha, The Economist's science and technology editor. Guests: Natasha Loder, The Economist's health editor; Julia Vitarello of EveryONE medicines; Paul Gissen of UCL Great Ormond Street Institute of Child Health. Transcripts of our podcasts are available via economist.com/podcasts. Listen to what matters most, from global politics and business to science and technology—subscribe to Economist Podcasts+.
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  • Julia Vitarello and her daughter Mila lived in Colorado where they enjoyed hiking and skiing in the Rocky Mountains.

  • And then really quite drastically changed when my daughter was diagnosed with a rare 100% fatal genetic disease.

  • At the age of six, Mila was diagnosed with baton disease.

  • It causes neurons in the brain to stop working and it eventually leads to an early death.

  • She had already lost her vision.

  • She had already lost her words.

  • She was starting to slump over and it was very hard for her to sit up straight.

  • Swallowing was also difficult, so she was eating pureed foods and then had a G-tube put in,

  • so her foods were primarily through a tube into her stomach.

  • She was not responding and laughing at the funny parts of the books and the songs that she normally had.

  • Doctors told Julia there was nothing they could do about Miller's ongoing neurological damage.

  • But that didn't stop Julia from trying.

  • She was missing one of two mutations that would cause her disease and no lab could find it.

  • And in my quest to find that, that led me down a very different path that was quite unique.

  • The doctor who eventually discovered the genetic cause of Mila's condition had a theory.

  • He suspected that her disease might be paused by using a drug specifically tailored to her.

  • That led Julia to start frantically raising money to test out that hypothesis.

  • I started a nonprofit foundation and had to raise millions of dollars selling what felt like,

  • you know,

  • lemonade and cookies and trying to find doctors to come together and create a treatment for my rapidly declining daughter.