Mila's legacy
One mother’s battle to save her daughter's life may change the way that medicines are made in Britain, and the rest of the world
Mila’s Legacy, a new BBC Radio 4 documentary on March 29th at 11am.
Presented by Natasha Loder and produced by Sandra Kanthal.
How many medicines can you think of created for just one person? The likelihood is none - which is why the world hasn’t heard of milasen yet. But its creation, and the efforts behind it, could build a pathway towards some of the greatest advances in genomic medicine, and a new initiative being trialled in Britain has a huge role to play in making this happen.
At the age of seven, Mila Makovec became the first person in the world to be treated with a medicine created just for her. A bubbly young girl from Colorado, Mila suffered from a very rare genetic disorder called Batten’s syndrome, which leads to a painful early death in children. Mila’s mother, Julia Vitarello, resolutely sought out scientists to try to discover a way to save her daughter. One doctor, Timothy Yu from Boston Children’s Hospital, imagined a possible treatment for Mila. The challenge was that it involved making a completely unique treatment for Mila’s specific genetic mutation. It would be novel and very expensive - but it was her only option. Julia raised the millions of dollars required through a charity she set up in her daughter’s name, and in 2018 Mila became the one patient in the world to receive the drug milasen.
Initially, it worked, and Mila’s condition stabilised and improved. However, the treatment was given after the disease had done a great deal of damage to a small child and Mila died when she was ten years old. Now Julia is on a mission to bring customised therapies such as the one that Mila received to the millions of children who are born with genetic diseases. Because so many rare diseases have only a few patients, and time is short, it is often impossible to do the sorts of clinical trials that are usually conducted to approve medicines for use. This also looks to be costly and economically uninteresting to firms.
Julia’s efforts to make customised medicines available to other children with rare diseases has taken a huge step forward in Britain. The launch of the Rare Therapies Launch Pad is bringing together efforts from Mila’s Miracle Foundation, the medicine’s regulator the MHRA, Genomics England and Oxford University in an world leading attempt to build a new streamlined regulatory pathway to allow one-off drugs to be designed and approved for use in individual patients with rare diseases. Listen to the documentary Mila’s Legacy at 11am on Friday, March 29th on BBC Radio 4, or later on BBC Sounds.
NL’s previous work on customised treatments.
Congenital diseases reveal a lot about human biology. And some can be treated—even if there is only one patient. The Economist, March 2020.
The world’s first pathway for individually designed drugs. Britain commits to finding a regulatory route for customised genetic medicines. The Economist, November 2023