Mila to Millions: A New Era of Individualized Medicines

MHRA foreword

Mila's story is one of the most powerful you will ever read. It is both heart-breaking and also inspiring when you learn how her mother, Julia, has turned her own personal tragedy into a global mission to save millions of other children and their families from going through similar ordeals in the future. Where she finds the strength, we will never know, but it surely demonstrates the power of a mother's love and her determination to create a legacy to change the world in Mila's name. We were privileged to have Julia speak to our leadership team at the MHRA, which was both deeply moving and also energising. It provided us with even more motivation and drive to put patients with rare and ultra-rare diseases at the heart of our work and to ensure the UK leads the way in bringing the new possibilities of gene therapies to those patients safely and expediently, and that this country can be a torchbearer for others to follow.

Lawrence Tallon, Chief Executive of the Medicines and Healthcare products Regulatory Agency (MHRA)Guest blog: Julia Vitarello

Ten years ago this year, my daughter Mila was diagnosed with a rare form of an already rare condition, Batten disease. In just one minute, I was told that my previously healthy, outgoing little girl, who loved singing, skiing, and swimming, would lose all her abilities and die in the next few years. No child with this condition had ever survived. And there were only a few dozen known patients in the world. Within just one year from her diagnosis, she rapidly lost her vision, said “mommy” for the last time, had a feeding tube surgically inserted into her stomach and began having up to 30 seizures per day.

In many ways, my life as I had known it ended the day she was diagnosed. But as I cried on my closet floor, I watched my daughter smiling and laughing despite the dark and scary world she was entering, and I told myself, “If she can fight, so can I”. So I pulled myself up and raced against time to save her life, starting Mila's Miracle Foundation and turning to the only thing I had to offer, our story of devastation and hope to raise millions of dollars needed to start and fund a treatment to stop her disease.

I was incredibly fortunate to find Dr. Tim Yu who, together with his team at Boston Children's Hospital, raced against time with me and created an antisense oligonucleotide or ASO that targeted the root cause, a single mutation, of Mila's horrendous disease. The catch was that no one else in the world shared her mutation, resulting in a medicine tailored just for her. In less than one year from her diagnosis, the team designed, tested, manufactured and received the greenlight from the FDA to proceed with the medicine, called milasen. Our family moved across the country to Boston and Mila began receiving the highly personalized treatment, making history as the very first person in the world to receive a medicine designed for one person.

For the first year, milasen halted my daughter's downward spiral, allowing for nearly seizure free days, giving her strength to lift her legs higher as she walked with my support from behind, giving her the ability to eat pureed food by mouth again and bringing back alertness and responsiveness as seen through her more frequent smiles and laughter. But it was unclear in the second year if her disease had stopped or slowly progressed. By the third year we accepted the painful reality that too many genetic dominoes had already fallen when she began treatment, as her brain atrophied and her abilities were slowly lost. When Mila was just ten years old, I was forced to hold her in my arms and kiss her goodbye.

Milasen wasn't in time for my daughter, but her story has since kicked off an entirely new era of individualized medicines. Her story has shown that we now have the ability to find the root cause of patients' diseases and make highly precise medicines that target them. Mila's story has since opened up a pathway for 85 other patients across diseases who have received individualized ASOs, and more recently for Baby KJ who received the first individualized CRISPR. Yet despite the promise of this hyper targeted approach, it's harder, longer and more expensive  today to get these medicines to patients than it was eight years ago when Mila opened this field. Herculean efforts are still required, resulting in access for only a tiny fraction of the millions suffering around the world. In many ways, hope for children with genetic disease is nearly as distant as it was in 2016 when Mila was diagnosed.

The status quo is simply not acceptable. Ground-breaking technologies offer patients enormous hope today, but our system was designed for population medicine, making it nearly impossible for the hundreds of millions with small, or “rare”, genetic diseases to access them. Science is no longer the limiting factor, it's the system. In 2023, I saw the success of Genomics England and felt a willingness to change by the MHRA, so I came to the UK and helped form the Rare Therapies Launch Pad, an infrastructure pilot led by a team of people across all stakeholders.  Focusing initially on the regulatory pathway, we recognized we could learn from the highly personalized fields of surgery and stem cell transplants which led us to propose a shift from Product to Process Approval, where the regulator would test and approve a standardized way of making medicines rather than approving one medicine at a time.

After many years fighting for what has felt like a logical solution to a changing reality in science and medicine, it feels like the tides are finally turning. In October 2025, EveryONE Medicines, the biotech I co-founded with Tim Yu to create a sustainable engine to make individualized medicines for children like Mila at scale, received approval from the MHRA to start a single Master Protocol trial crossing diseases, mutations and medicines – with parameters set to include small numbers of children with fatal and life-threatening neurological diseases using intrathecal administration of ASOs.

On the 13th of January 2026, Patient A in our Master Protocol trial was treated, representing a monumental step in the direction of Process Approval. Our hope is that after treating up to ten children with different diseases using the same process each time and collecting the data, the MHRA will do the right thing and become the first regulator in the world to officially give Process Approval, paving the pathway for many other groups in many other countries. I can now see the day where children like my daughter are diagnosed at birth and soon after treated with a highly precise individualized medicine, stopping their diseases before they ever begin so that they and their families never know life with disease.

This year has just begun, but it's already pivotal - one big step to getting from Mila to Millions.