Date: Jul 05, 2012 Author: Luke Timmerman Source: xConomy (
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Matthew Scholz has been told many times that his idea for reprogramming the body's immune cells to create drugs was impossible. Maybe dangerous. Maybe just dumb.
Scholz, a computer scientist with no formal biology training, could easily have been written off as a quixotic dreamer until this spring, when he got his breakout moment. The foundation started by Peter Thiel, the billionaire co-founder of PayPal, decided that Scholz's startup, Immusoft, just might be onto something, granting it close to $400,000.
Seattle-based Immusoft has gone through a pretty amazing journey the past couple years. The company, which began as Scholz's nights-and-weekends passion while he held down a day job doing tech support at a nonprofit, has grown into a bootstrapped biotech company. It has no venture capital backing and just four employees, but in April, the company was one of six startups to win a grant from the Thiel Foundation, which aims to support what it calls "radical scientific innovations."
Scholz has scraped together about $900,000 now through angel investment, a federal small business grant, and foundation support. Armed with a technology license from Nobel laureate David Baltimore's lab at Caltech, some more intellectual property from Europe, and a couple of scientific collaborations at the Fred Hutchinson Cancer Research Center, Immusoft has put together a plan to see if can do the unprecedented--program immune system cells to create therapeutic proteins on their own, rather than have them be delivered the traditional way through repeat injections.
"I want to turn your own cells into drug factories," Scholz says.
Immusoft is still very much in its infancy, and may never get close to fulfilling its vision. But it is preparing for its first mouse study at the Hutch this summer. This will be the first test in mammals to see if Immusoft can do a blood draw, filter out the resting B cells of the immune system, and genetically modify them in the lab to secrete a specific therapeutic protein. The newly programmed B cells would then be injected back into the body, where Immusoft hopes they continue to pump out the protein drug, and settle into the bone marrow, where cells can live for years. That's important, because if the newly injected, reprogrammed cells die after a few hours, then such a drug would require repeat injections, essentially defeating the purpose.
If all goes according to plan in this key experiment, Immusoft hopes to move on to monkeys, set up a meeting with the FDA, and hopefully prepare to run its first human clinical trial in about three years, Scholz says.
Alan Leong, a professor of entrepreneurship at the University of Washington Bothell, who got to know Scholz as a student a decade ago, says he was skeptical when his former student called him to ask for advice about this new biotech venture. Leong, who's also an analyst with Seattle-based Biotech Stock Research, knows the odds are terribly slim, even for folks with the best scientific and business pedigrees. "I had to ask, is he really, truly bright, or is this hubris? Maybe it's a combination of both that it takes to make this happen," Leong says.
Scholz's previous entrepreneurial experience was with Point B Telematics, a fleet logistics company that helped companies find the fastest routes around urban areas like Seattle. The company was acquired in 2009 by TIE Technologies for an undisclosed sum. Before Immusoft consumed much of his attention, Scholz dabbled a little with making iPhone apps "which made a little money" and had a steady gig doing tech support at the Discovery Institute.
Scholz got his inspiration to start Immusoft in 2009 after making a basic observation that many computer scientists know quite well--the human immune system adapts to fight pathogens a bit like how software and computer viruses square off. If the immune system is ultimately an information system, he wondered, then shouldn't it be possible to reprogram cells to help them confront a virus like HIV, or some other cellular irregularity that leads to disease?
Scholz sought to learn about whether this was possible, reading lots of journal articles, and then tracking down many of the scientists by phone, by email, and at conferences to ask them about their work. He learned that 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: 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."
