Innovator Uses CMU Education To Combat Rare Diseases
By Heidi Opdyke
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Carnegie Mellon University alumnus Jeff Milton has his eye on the horizon in more ways than one.
An avid pilot who flies vintage military aircraft and other types of planes, he also is looking for ways to provide therapies to patients with rare diseases.
According to the U.S. Government Accountability Office, only 5% of the nearly 10,000 identified rare diseases have Food and Drug Administration-approved treatments.
As co-founder for the drug discovery company La Jolla Labs, Milton is on a mission to find RNA therapeutics to address rare and complex diseases through a combination of artificial intelligence, next-generation sequences and RNA biology.
"Pharmaceutical companies often neglect these diseases due to high costs related to research and development for a small market," he said. But La Jolla's screening technology reduces costs and time for patients.
La Jolla uses RNA as a platform because it plays a crucial role in various cellular processes, and many of the rare diseases come down to specific molecules that need to be targeted.
"We target RNA because it contains a human's genetic makeup and it contains the mutations. We can target those specific mutations at the genetic level. Every therapy is genetically focused down to the nucleotide," he said.
His Carnegie Mellon education is helping him create to support patients and their medical teams.
"I wanted fundamental knowledge of life, and chemistry was my focus. I spent so much time in the Mellon Institute at all levels," said Milton, who graduated from of Carnegie Mellon in 2004 with a bachelor's degree in chemistry. "Faculty were so open to exploring and supporting what I wanted to do that they thought was novel and interesting." He said the entrepreneurial and coaching opportunities available at Carnegie Mellon today are stronger than they've ever been.
Among the professors Milton interacted with was Krzysztof Matyjaszewski, J.C. Warner University Professor of Natural Sciences and director of the Center for Macromolecular Engineering. Milton was visiting his lab when he first heard of atom transfer radical polymerization (ATRP), a novel method of polymer synthesis that revolutionized the way macromolecules are made.
"Every day I dive into science and technology that I can directly link to the skills I learned at Carnegie Mellon," Milton said.
Milton also worked with David Yaron, who recruited Milton to design the first Department of Chemistry website. It's when Milton first started developing software writing skills. Among the projects that Milton worked on with Yaron was Chem Collective, the precursor to Real Chem, courseware for introductory chemistry classes that marries in-person instruction with online instruction and practice.
Prior to co-founding La Jolla Labs, Milton served as senior director and head of data science at Arcturus Therapeutics, where he led the development and implementation of data-driven strategies for mRNA therapeutics. Additionally, he served as director and associate director at Ionis Pharmaceuticals.
He also is an executive fellow at St. Jude Children's Research Hospital in Memphis, where he is helping to optimize rare disease therapies. He is hoping Carnegie Mellon might help with that effort.
"I've been talking to the Department of Chemistry, and Department Head Bruce Armitage, specifically, about building a research-grade synthesis lab to make the compounds needed," he said. "It's one thing I'm super excited about and I think it would be great for Carnegie Mellon and the Mellon College of Science."
Milton, who was a student-athlete on the Men's Swimming and Diving team, is now giving back to the place that gave him so much as a member of the Mellon College of Science Dean's Council.
"CMU defined my life. It made me who I am. I had support from research faculty who saw my passion and drive," he said. "I'm going to dedicate a significant portion of the second half of my life to CMU to help make sure others receive that same support."
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