In 2015, physician-bioengineer and entrepreneur Thomas Neumann, M.D., suspected he had a solution to a long-term problem in the stem cell research field: the need for a fast, accurate test that could gauge the quality of stem cells. According to many scientists, the ability of stem cells to transform into practically any type of cell may hold the key to treating a range of diseases, including cancer and Parkinson’s disease.
Researchers at Nortis, Inc., are developing a tissue chip to grow stem cell-derived kidney cells to study kidney function. (Nortis, Inc.)
A few years earlier, Neumann, along with some of his University of Washington (UW) colleagues, had founded the Seattle-based biotechnology company, Nortis, Inc., and had then been awarded support through NCATS’ Small Business Innovation Research (SBIR) program, which fosters the growth and development of small companies. As the Nortis chief executive officer, Neumann also participated in an NIH SBIR Innovation Corps (I-Corps) training program to help company leaders navigate the challenges of product development. I-Corps is supported by a $50,000 supplement to existing SBIR or Small Business Technology Transfer (STTR) support.
The NIH I-Corps program is a collaborative effort with the National Science Foundation to support the development and commercialization of biomedical technologies. In August 2015, the White House announced that NIH would expand its I-Corps program to focus on moving technologies developed through SBIR/STTR funding to the marketplace. NCATS is among 17 participating Institutes and Centers from NIH and the Centers for Disease Control and Prevention.
“In many cases, scientists in academic laboratories lack the experience and know-how to take their discoveries to the marketplace,” said Lili Portilla, M.P.A., director of strategic alliances at NCATS. “I-Corps helps train them to think more like businesspeople, such as to ask, ‘Does my product or technology really solve a problem?’; engage with and learn from potential customers; and ultimately make decisions that will help deliver biomedical innovations to the market more efficiently.”
Participants are asked to interview a minimum of 100 potential customers for their product and have the opportunity to speak with regulatory agencies, insurance companies and others. Experienced trainers, who are often entrepreneurs themselves, provide advice and guidance on what the participants can learn from customer interviews. The NIH SBIR I-Corps training curriculum consists of three separate tracks: diagnostics, therapeutics and devices. Companies commit to eight weeks of training and assemble a three-person team, including a principal investigator, a company officer and a key opinion leader who knows the company and the field. Every team member must spend 20 hours a week on the program.
Learning what customers want
I-Corps has been a perfect fit for Nortis and for another NCATS SBIR-supported company, CrossLife Technologies, a six-year-old biotechnology company in Carlsbad, California. Scientists at CrossLife are developing a quick and easy bedside test to determine whether a person has dengue fever, a potentially life-threatening mosquito-borne illness caused by a virus. Detecting the virus early can lead to more effective treatment.
Dengue fever is found primarily in tropical climates in Southeast Asia, Africa and Central and South America. CrossLife founder and chief executive officer HyunDae Cho, Ph.D., a biochemist, said that current tests — often unavailable in areas affected by dengue virus — do not always provide an accurate diagnosis. CrossLife researchers are developing a simpler technology that uses a paper dipstick probe to detect the virus in the blood, enabling a diagnosis in 30 minutes.
Cho and his colleagues originally thought their customers would be the companies developing dengue fever vaccines. But after interviewing more than 110 potential customers — including village hospital doctors in Southeast Asia, Central America, and South America, as well as vaccine developers and distributors — they quickly realized they had made a mistake. Vaccine companies weren’t interested, but nongovernmental organizations and physicians in those regions wanted a test to diagnose and treat patients as quickly as possible. Cho and his team had to rethink their product idea and focus on a new customer base.
Cho said the company learned a great deal from I-Corps training, including the complexities of taking a product from the lab to the marketplace, and how markets and regulatory landscapes can differ from country to country. “The I-Corps program helped us discover who our customers are and what they want. It also provided a better understanding of the translational path for our product,” he said. Cho has already applied for a second SBIR grant, which would enable CrossLife scientists to develop and evaluate the test further.
Switching tracks
Neumann and his Nortis colleagues were also surprised to discover that they needed to adjust their product plan to meet customer needs because there was little interest in the product they were proposing. Nortis researchers, who develop microchips with three-dimensional chambers to grow miniature human tissues, organs and systems — including the liver, kidney, brain and immune system — have collaborated with UW as part of the NCATS Tissue Chip for Drug Screening program to develop the kidney-on-a-chip project. The program supports the development of tissue chips to better predict the safety and effectiveness of candidate drugs.
The Nortis scientists planned to use the tissue chip technology to create a human tissue environment that allowed them to study stem cell quality. Scientists typically measure the quality of stem cells by injecting them into animals and watching for the development of different cell types that come from three embryonic cell layers, while making sure the stem cells don’t have cancer-forming effects. But the current test can be expensive and requires a large number of animals, and it may not always be accurate. Neumann and his team developed a business plan and received state funding to replicate the animal test on a tissue chip, which they said would be faster and less expensive.
However, when they interviewed more than 100 potential customers, including academic researchers and industry experts, Nortis researchers were disappointed to learn that few people were interested in a new test.
“Most people said that while the current test wasn’t perfect, it was good enough for now,” Neumann said.
But in conversations with their I-Corps mentors and with colleagues at NCATS and in the tissue chip field, Nortis researchers discovered that there was a need for a tissue chip with a stem cell-based model to study kidney function. “If successful, stem cells transformed into kidney cells could replace the current source of kidney cells in Nortis’s chips, which is cells harvested from patients,” Neumann said. These cells vary from patient to patient. In contrast, stem cell-derived kidney cells offer a more uniform and renewable supply.
When they polled potential customers again, Neumann and his team found a very different response: 99 percent of those interviewed said they would use such a tissue chip. The new product is aimed at providing a steady source of stem cell-derived kidney cells and allowing researchers to test the effects of various concentrations of drugs on the kidneys.
Portilla sees the I-Corps program as a win-win situation for NCATS and NIH more broadly, as well as participating SBIR- and STTR-supported companies. “It’s great because our agency has opportunities to fund projects that truly have a customer base,” she said. “What’s more, the I-Corps program also supports an NCATS goal to train translational investigators who ultimately will lead the way in translating great science more efficiently to patients.”
Posted December 2016