The California Cure Small Is Beautiful: Dr. Leonard Rome

Leonard “Lenny” Rome is working to crack a vault. No, not the kind found in a bank, the kind a biologist or biochemist wants to breach: a barrel-shape capsule made from multiple copies of three proteins, the vault is found by the thousands within every cell of the body. They’re beyond miniscule, measured in nanometers, a billionth of a meter, and are, quite possibly, the future of drug-delivery systems.

Since the mid ’80s, when Rome and Dr. Nancy Kedersha, who was a post-doctoral fellow in Rome’s lab, discovered the vaults, he has been among the researchers trying to figure out what Mother Nature intended the little things to, well, do.

“They must be doing something, but we’ve been somewhat frustrated for many years not being able to crack that riddle,” says Rome, associate director of the California NanoSystems Institute and a senior associate dean of research at the UCLA David Geffen School of Medicine.

About nine years ago, researchers decided that since they knew enough about how these vaults acted within the body, they could actually start to engineer them and give them functions, putting drugs inside the capsules for targeted delivery to damaged cells, perhaps even correcting gene mutations in DNA to head off disease.

So vast is the potential use of vault particles that since it began in 2007, it has helped the Nanosystems Institute garner some $350 million and counting in grants from businesses like the L.A.–based Abraxis, makers of the breast-cancer-treatment drug Abraxene.

Potential is the word Rome stresses: “A boulder sitting up on a mountain top is not going anywhere, but if you could move it just a few feet and get it to start coming down that mountain, it would convert into incredible kinetic energy. Vaults are sitting on very high mountains. They have a lot of potential for doing good, and we are working very hard to move them along.”

The promising research looks like all systems are a go. The son of a feed-store owner from Youngstown, Ohio, Rome is the first to say he couldn’t have imagined what a big future lay inside the tiny world he chose to study. “Even 10 years ago, I probably didn’t have idea I’d be doing this.” —Samantha Dunn