Joseph Gold, Ph.D.
, didn't set out to become chief overseer of a sprawling, state-of-the-art manufacturing facility cranking out mass quantities of carefully engineered stem cells aimed at curing the most dangerous and debilitating diseases of our time.
He had a slightly different path in mind.
“I wanted to be a doctor,” he said.
But then, a little boy died.
It happened while Gold, still in college, was volunteering at Children's Hospital of Philadelphia.
“I was in the emergency room,” he recalled, “And they brought in a 2-year-old in cardiac arrest. Everything in that ER changed in an instant. All these people sprang into action, working together, laboring, struggling to save this child's life.
“And then, as if on cue, they all stopped, and looked at each other. There was nothing more they could do.
“I'd never seen a child die before. It really affected me. All those talented, highly skilled people in that room, working so hard ... and they couldn't help him.
“I decided I wanted to do more, to become an expert in the biology of things like heart defects, and maybe play a role in eliminating them.”
His decision led to a Harvard doctorate in cell and developmental biology, followed by more than two decades of investigating and developing the potential of stem cells, first in the private sector, then at Stanford University's Cardiovascular Institute and, since April of 2016, as manufacturing director at City of Hope's Center for Biomedicine & Genetics (CBG)
, one of three campus facilities producing drugs and components used in clinical trials.
Ask Gold what he does at CBG, and he answers simply, “I make weapons. In a biological forge.”
This requires a little explaining.
Most people familiar with City of Hope already know its “all under one roof” philosophy: By grouping together clinics, research labs and manufacturing facilities on the same campus, lifesaving treatments can rapidly progress from theory to prototype to clinical trial to real treatments for real patients.
Perhaps there's no better illustration than the production of CAR T cells
, which are immune cells taken from a patient, reprogrammed with viruses to seek out and destroy cancer cells, then re-introduced into the patient. The treatment has shown tremendous promise in battling blood cancers and, more recently, in brain tumors.
Gold's CBG facility produces those viruses to exacting quality control standards, in the large quantities necessary to supply clinical trials at City of Hope and also at outside institutions.
But City of Hope's people always come first.
“Our mission, our mandate is clear,” asserted Gold. “And that is to advance City of Hope's work. Our investigators always take priority,” getting their CAR T cell orders filled within weeks, compared to the year-long wait for outsiders.
Still, the hardest part of his job, Gold says, is triage: choosing among the multiple requests for CBG's limited resources and capacity.
The task is more challenging than ever, because City of Hope is committed to reach beyond cancer work, and deploy some of its manufacturing capability for critical research in regenerative medicine: using stem cells to repair damaged or malfunctioning human components – like a little boy's defective heart muscle – rather than just destroy tumors.
Quietly, City of Hope has become a major player in this arena, producing the “weapons” that may someday conquer everything from Alzheimer's to retinitis pigmentosa to ALS (Lou Gehrig's disease).
“The technology bridges the different domains,” said Steven T. Rosen, M.D.
, City of Hope's provost and chief scientific officer and the Irell & Manella Cancer Director's Distinguished Chair. “We are working to ensure that we have the capacity for both City of Hope-based projects and revenue generating relationships with external entities.”
One of those “external entities” is presenting CBG with its biggest challenge yet.
Brainstorm Cell Therapeutics, an Israeli company, needed a facility to manufacture its specialized adult stem cell product for an upcoming phase 3 study of ALS patients. Brainstorm asked CIRM, the California state agency promoting regenerative medicine for help. CIRM made the professional match, and now City of Hope is the clinical supplier for all U.S. medical centers participating in the trial.
“It's the biggest trial we've ever done,” said Gold.
Running CBG also happens to be the biggest job Gold's ever done and, one year in, he says he's still learning. He loves the collaborative atmosphere and enthusiasm at City of Hope, qualities he found lacking in previous gigs. He revels in the professional cross-pollination on campus, and he's passionate about playing his part on the manufacturing side, especially when it comes to quality control.
“CBG, like any good manufacturing facility, leaves nothing to chance. Not in the materials, not in the process, not in the final testing,” he said. Haunted by horror stories of what can and has gone wrong when other facilities cut corners, Gold brushes off any notion of “speeding things up” or skipping any steps in the process, no matter how small they may seem.
“Until somebody shows me proof that every raw material is perfect, human beings are infallible and everything works the first time, facilities like CBG will continue to do our job.”