good tissue samples (who really wants to do brain biopsies on a large scale?). By taking adult cells, and making induced pluripotent cells, iPierian can make all the heart cells and liver cells they want to see if a given drug might be too toxic, but also make the motor neurons and brain glial cells that would suggest whether a given drug hits its target and might actually work.
If you could bank enough cells from enough individuals with a neurodegenerative disease, it’s possible you could use all that data from the lab dish to predict which individual patients might respond to a drug in a clinical trial. By doing all this testing on effectiveness in human cells upfront, it’s possible that drugmakers will be able to eliminate the need for testing drug efficacy in animals, Walker says. Instead, researchers might just test whether a given drug candidate is too toxic in animals before it leaps into clinical trials. Given that only about one out of 10 drugs that enter clinical trials ever make it through the FDA approval process, and big pharma spends an estimated $1 billion on every drug candidate, it sure sounds appealing.
The drugmakers are certainly kicking the tires, but none of them have struck a big partnership yet with iPierian, Fate, or anybody else yet. Walker predicts that he’ll close at least one deal, and maybe more, before the end of 2010.
Selling Big Pharma on this idea is important, but so is developing drugs in house. iPierian’s management and board figured they’d never really get rewarded by investors just for providing technology to others, but only if they developed their own drugs.
So, partly because neurodegenerative diseases are such big markets, and scientists think they have gotten pretty good at creating differentiated neurons from induced pluripotent stem cells, this is the place iPierian is going. It has moved first into spinal muscular atrophy, a rare disease in which a single gene is malfunctioning. There are no effective therapies now, it’s a serious disease, and a small enough patient population that a small company like iPierian could probably commercialize a drug for this group on its own, Walker says. The company hopes to use its stem cell platform to help it pick a drug candidate by the first half of 2011, and then dash off an application to the FDA to start its first clinical trial in early 2012, Walker says.
Spinal muscular atrophy was thought to be ideal, partly because it’s a single gene disorder, which reduces the number of variables that scientists have to deal with, Walker says. The next program, amyetrophic lateral sclerosis, can’t be traced to a single gene defect, and neither can Parkinson’s or Alzheimer’s. So the sledding will only likely get harder.
Walker knows how hard drug development can be, and how long the odds are. His last company, Novacea, stumbled with a prostate cancer drug that failed in the third and final stage of clinical trials after all kinds of positive signals for years.
But biotech is infused with can-do optimism by its nature. When I asked him about what’s in store for the next 12 months, he wasn’t afraid to say he said he expects to close a Series B venture financing before the end of year. And this wasn’t by making some headline-grabbing claim about miracles of regenerative medicine—just about creating a reliable, informative tool that can help researchers increase their odds of success by learning more of the basic human biology of an individual’s cells before ever trying to give that patient a new pill or injectable drug.
“If we are able to use this new technology in a way that provides better insights, and a truly does provide approach to identifying therapeutics that people haven’t been able to use to date, then we feel we have a leg up,” Walker says.
