HUB Madness 2019 presented by BNY Mellon is in full swing! We caught up with Dr. Timothy Yu, the lead researcher behind the Children's Programmable Medicine Initiative, a program that developed a personalized therapy in record time to save a child fighting a rare genetic disease. Read on to find out more about this groundbreaking project, and what Dr. Yu and his team have planned for 2019.
Some background on the project:
Rare genetic diseases disproportionately affect children, and because we don't have treatments for the vast majority of them, three in ten afflicted kids won't live to see their fifth birthday. Dr. Yu's team at Boston Children's Hospital is developing a pathway for offering tailor-made treatments — individualized medicines — to help these most vulnerable patients.
Their first case, Mila, is an eight-year old girl diagnosed with Batten disease — an ultra-rare childhood disorder that causes brain degeneration, is rapidly progressive, and ultimately fatal. Using a promising type of "programmable medicine" called antisense oligonucleotides, the team designed and manufactured a brand-new drug, customized to her unique genetic mutation, tested it in her own cells, and begin treating her, all within one year. A world's first for truly personalized medicine, the team is now working to extend this pathway to offer hope to many other children who, like Mila, suffer from rare, orphan diseases.
What has been the biggest challenge of the project? How have you overcome it?
Designing brand-new drugs for our patients in a matter of months instead of years. To do this, we have to coordinate and focus the efforts of hundreds of people across academia, industry, and government. We make it work because everyone recognizes it's about the patients — children with fatal, rapidly progressive diseases — so everyone involved knows what’s at stake.
What makes your project so revolutionary?
Most of the time, precision medicine refers to choosing the right medicine for the right patient. This is certainly a worthwhile thing to do. In our case, we are not picking the right medicine — we are designing and manufacturing entirely brand new medicines for each patient, based on their genetic code. These are the world’s first examples of truly personalized medicines.
What's next for the initiative? What are you looking forward to accomplishing in 2019?
We’re working with the FDA and other partners to launch a pilot to develop brand-new treatments for another dozen patients, each with an orphan genetic disease.
What did you think was the coolest thing to come out of Greater Boston last year (aside from your project, of course)?
You know, there’s a ton of cool stuff going on. But I honestly can’t think of any other project I’d rather be working on right now.