A Computer Guy’s Dream, Immusoft, Turns Cells Into Drug Factories

nobody in biology had really approached the problem his way, that many in biology are siloed off from many of their peers, and that quite a few people thought this idea wouldn’t work. One common objection was that he might unwittingly coax cells into a cancerous state, for example. When Scholz heard objections, he didn’t hear the sound of discouragement, he heard the sound of opportunity. He pressed the scientists to explain the problem as they saw it in detail, and when they were done, he sought out another expert in a related discipline who knew more about how to get around that specific roadblock.

Essentially, he wouldn’t take no for an answer. “Every time I ran into a question I couldn’t answer, I’d recruit a new advisor,” Scholz says. “I did it over and over again.”

Still, biotech history is littered with companies that had lots of great advisors and never amounted to anything. What Immusoft has done so far, through a collaboration with Seattle Biomedical Research Institute, is show that it can take the DNA code for making a “broadly neutralizing” antibody against HIV, insert it into cells in a petri dish, and get those cells to produce the antibody repeatedly.

The next step scientifically is a big one, going from the lab dish, what’s known as the controlled “in vitro” environment to the messy, complicated and unpredictable world inside the body, what’s known as “in vivo.”

HIV might be an interesting test case for Immusoft, but Scholz has heard from numerous advisors that it’s a non-starter as a business proposition. HIV is already well-treated by daily antiviral pills that keep people alive for decades, which can be produced cost-effectively, and which will eventually become generic. Even if the Immusoft approach were able to coax the body to produce broadly neutralizing antibodies against HIV on a single shot, or maybe with one or two boosters, proving that to the satisfaction of the FDA would take many years, and many millions of dollars.

So Immusoft is shifting gears to another disease that looks like a better business proposition. It’s mucopolysaccharidosis I (MPS I), a genetic disease in which people don’t produce enough of a key metabolic enzyme, which means they end up with toxic buildups of carbohydrates in the lysosomes of the cell. Today, patients with this disorder can take an enzyme replacement therapy through a regular injection of lironadase (Aldurazyme) from Novato, CA-based BioMarin Pharmaceuticals (NASDAQ: [[ticker:BMRN]]). Since this is a very rare disease, and it’s deadly, Immusoft can realistically take on a clinical trial program that will be much shorter, smaller, and cheaper than the kind of program needed for a new HIV drug.

Scholz gives off the impression that such short-term business strategy is necessary, but not really the thing that rocks his world. He’s most animated talking about all the things you could do when you have a technology that can turn cells into drug factories. “It could change the way we think about disease. We’ll treat a disease with information instead of a drug,” Scholz says.

He takes the analogy a bit further in a statement on his website. “Within 50 years, we will program human cells like we program computers. Envision a stand-alone device capable of modifying a patient’s cells to manufacture biologic-based therapies for a wide range of disease including cardiovascular disease, cancers, infectious diseases, and lysosomal storage diseases.”

Clearly, that’s the kind of talk that folks at the Thiel Foundation eat up. Now it will be Immusoft’s job to see if it can deliver on this computer-science inspired approach toward biology. No one can say for sure how far this idea will go, but it’s already gone pretty far for a guy who freely admits “I knew nothing about biotech” three years ago.

Leong, the advisor, says Scholz has deployed a characteristic mix of self-confidence, resourcefulness, tenacity, and can-do spirit to get his company this far. And there’s also a quirkiness, too. In a field full of very sober people who speak in technical jargon, Scholz has an irreverent streak, joking at one point during an interview about getting cells “to do my evil bidding.” No question, Scholz is enjoying this adventure, wherever it leads.

“The odds are against him probably getting this far, frankly, and it has taken someone with that kind of personality,” Leong says. “If you didn’t have that kind of entrepreneurial personality, you wouldn’t even try.”

Author: Luke Timmerman

Luke is an award-winning journalist specializing in life sciences. He has served as national biotechnology editor for Xconomy and national biotechnology reporter for Bloomberg News. Luke got started covering life sciences at The Seattle Times, where he was the lead reporter on an investigation of doctors who leaked confidential information about clinical trials to investors. The story won the Scripps Howard National Journalism Award and several other national prizes. Luke holds a bachelor’s degree in journalism from the University of Wisconsin-Madison, and during the 2005-2006 academic year, he was a Knight Science Journalism Fellow at MIT.