The New Medicine

Posted |

 

A Conversation with Dr. Jude Samulski 

By: Tom Griffen

Dr. Samulski

In the world of genetics, he is a Wright brother. Besides being a professor in the department of Pharmacology and the former director of the Gene Therapy Center at UNC Chapel Hill, he’s also the founder of Chapel Hill-based Bamboo Therapeutics—a manufacturing facility formed to advance his three decades of genetic pioneering. You’ve probably never heard of Dr. Jude Samulski, but he doesn’t mind. He just keeps plugging away in his efforts to cure the incurable.

When I arrived at his reception-less office, dozens of screen-lit researchers popped up like meerkats. They squinted when I said I was there for a meeting with Dr. Samulski.

“Doctor who?” someone asked. Made me think I said his name wrong.

A lengthy silence ensued. “It’s Jude, someone said, “Dr. Samulski is Jude.” Then everyone shook their heads like, oh yeah, we totally knew that.

I sat with Jude in the conference room. On the wall hung a white board covered in colorful diagrams, reminding me of science classes I didn’t take in college.

Jude patiently explained his work to me in layman’s terms: he isolates a virus, extracts its unhealthy gene and replaces it with a healthy one, then turns the virus into a vector that, when introduced to the patient, potentially changes the course of their disease. Full disclaimer—this is my nutshell version of gene therapy—a promising treatment that, according to Jude, will be the new medicine.

“And it will be phenomenal,” he said. Youthful blue eyes widening behind metal glasses.

When Jude needs a break from the endless stream of professional and political rejection, he spends time in his garden. A place where people aren’t telling him no as he pushes the scientific envelope.

More than 30 years and 20 patents later, Jude continues to build on innovative research he started a lifetime ago as a grad student at the University of Florida.

He traces his motivation to 1982. During a conference in Cambridge, England, he learned the son of his bed and breakfast host had muscular dystrophy. The boy’s palliative “treatment” floored him—the family was building a first-floor bedroom so he wouldn’t have to climb stairs.

“Though I knew MD was a progressive disease with no treatment, her response hit me like a ton of bricks,” Jude said.

His efforts were further propelled by another parent who donated $3 million to his research saying, “No parent should have to go through the same thing as me.”

“These days we take pills to modify our blood pressure, our cholesterol,” Jude said. “Eventually we’ll use therapy to modify genetically integral processes that lead to these rare diseases. One day, adults will look back and say they were born with a genetic defect that was corrected because of this kind of therapy.”

Jude believes it’s all about timing. History is wrought with moments that draw a line between past and present. The eradication of scourges such as plague, smallpox, and polio helped shape a healthier world.

“I’ve seen enough to know how misunderstood these diseases are. So I keep working at it.” Jude said.

Recently Jude told the state legislature that 1 in 9 North Carolina residents have a genetic disease. This accounts for nearly 900,000 potential taxpayers unable to live their lives to the fullest because the state doesn’t recognize genetic therapy as a viable modality.

“Those numbers hit them hard. More importantly, we were able to call a session to further discuss the details,” Jude said.

Teaming up with pharmaceutical giant Pfizer gave Jude the necessary muscle to scale his research for practical applications.

Last month, after 9 years in development, a vector treatment was delivered to a young woman suffering from a neuromuscular genetic disorder. Jude showed me the 10-second video of the procedure—a basic syringe administering the solution into the patient’s spinal column—he then pointed out how trivial and unspectacular it all seemed.

“On some level when you watch this happen, you wait for the crowds to gather in fanfare. But there are no crowds—there’s really nobody out there to celebrate the accomplishment.”

Sort of like at Kitty Hawk, when two unknown men took flight, thus altering the historical paradigm. Which is exactly what Dr. Samulski’s doing— right here in Chapel Hill.

And what about his garden?

“Right now my figs are coming in,” he said. “Scuppernongs and blackberries. Roses too. And even when I find my plants drooping, it doesn’t take much to get them thriving again.”