MILA TO MILLIONS

In 2016, my seemingly healthy, outgoing daughter Mila was diagnosed with a fatal genetic disease with no cure. In a race against time together with Dr. Tim Yu and Boston Children’s Hospital, Mila became the first person in the world to receive a drug tailored to just one person, called milasen. Remarkably, during the first two years of treatment, Mila’s medicine stopped her disease and improved some of her symptoms. However, too many genetic dominoes had already fallen by the time she began her treatment. After three years on milasen, her disease had progressed and Mila died at just ten years old.

Despite losing a lot before beginning treatment, Mila was given an unprecedented second chance at life. Mila’s fight was not in time for her, but her story is now giving enormous hope to children across rare diseases that otherwise would have none.

THe problem

A Global Health Crisis

Tens of millions of children will die of a rare genetic disease before they turn five. While some genetic diseases are common, nearly all are individually rare with no treatments. Together, these rare diseases impact more people than cancer and AIDS combined, yet few to no treatments are available.

Today, access to treatments depends on luck. Only large, commercially viable diseases or herculean one-off efforts by parents and doctors benefit.

But what about the millions of others?

THe solution

Individualized Medicines

Science is no longer the limiting factor. We now have the technology to find children like Mila early enough and rapidly design treatments which could halt their conditions.

Mila’s story showed what’s possible — that a medicine can be made to target the underlying genetic cause of disease, even if unique to one person. Instead of one drug for thousands, milasen points to thousands of drugs for one or a few, setting the stage for a new paradigm of individualized medicines.

OUR VISION

Today, we have millions of dying children and the science to help them. What’s missing is the infrastructure and processes to connect them. While academics and industry perfect the diagnostics and therapeutics, we launched Mila to Millions to bridge the gap by piloting the change that is needed, shifting the mindset, and adding the necessary urgency.

OUR FOCUS

Mila to Millions is focused on Strategic Pilots to prove new models of access and an Impact Campaign to change the framing and influence the critical decisions. Our Mila to Millions Committee of expert advisors from around the world serves as a consistent group dedicated to strategically driving this new field forward. Success of our campaign will be a rapid and reasonable path to treatment access for millions of children with rare disease.

Strategic Pilots

We are initiating transformative pilots by carefully convening the right people in the right places around ideas critical to the individualized medicine paradigm.

UK Rare Therapies Launch Pad: we launched this groundbreaking pilot endorsed by the gov’t to prove a new scalable, sustainable model for access to individualized treatments, shifting the regulatory and reimbursement pathways.

US Individualized Medicines Pilot: we are strategically working toward a similar pilot with the California Institute of Regenerative Medicine, FDA, ARPA-H & US Senators in the HELP Committee.

Additional Pilots: we are collaborating with other countries, such as Australia, to launch similar pilots, implementing consistent concepts across the world.

Impact Campaign

We are pushing the moral imperative as history has shown us that this is what leads to transformative change. Our message is that we must ensure access to treatments for every child amenable to technology that exists today, no matter how few. We use consistent, strong messaging to thousands following us via:

Presentations at strategic meetings and conferences around the world

Photo journal exposing life behind closed doors of rare disease families

Short films using Mila’s story and my ongoing fight to engage stakeholders

Parent voice to capture families' risk tolerance and view of meaningful benefit

OUR IMPACT

Milasen gained global attention for being the first medicine designed for one person. Now, we’re determined to make similar treatments possible for many more children with rare genetic diseases. Speaking at presentations and meetings worldwide, we’re creating awareness and opportunity for the future of individualized medicines to lead the way forward… from Mila to Millions.