Connecticut’s 10-year, $100 million investment in stem-cell research has spawned some promising medical treatment breakthroughs and business opportunities, including one Farmington biotech startup trying to tackle treatments for multiple sclerosis.
In early June, ImStem Biotechnology said it successfully treated multiple sclerosis in a mouse using a unique stem-cell treatment, and is now looking to raise more money to begin clinical trials based off its research findings.
ImStem, which was awarded more than $1 million in state money last year and is a UConn technology incubator member, said it worked with UConn Health Center scientists and Massachusetts’ Advanced Cell Technology Inc. on research that found mesenchymal stem cells (MSCs) derived from human embryonic stem cells are more effective at treating MS in mice, than MSCs derived from adult bone marrow.
In fact, the researchers said they found unexpectedly that the use of adult bone marrow stem cells to treat MS is highly variable and may carry a previously unrecognized risk of poor outcome.
ImStem’s findings suggest MSCs — a type of cell that can differentiate into other cell types like bone and cartilage cells — may promote nerve-cell regeneration in multiple sclerosis, for which scientists have yet to find a cure. If they’re right, their research could potentially help the 2.3 million people worldwide affected by the disease, which debilitates the central nervous system.
“Current therapies don’t cure the disease,” said Xiaofang Wang, ImStem’s cofounder and chief technology officer. “[They] only provide temporary relief — and side effects. We hope stem-cell [therapy] can give patients new options with fewer side effects.”
Wang launched ImStem alongside Ren-He Xu, the former head of UConn’s Stem Cell Institute and Michael Men, an angel investor who is CEO. Xu, who is now a professor at the University of Macau in China, and Wang deduced the potential application of their MSC research as a treatment for multiple sclerosis.
“There are quite a few groups trying stem-cell [therapies] on MS,” he added. “This is a tough disease to deal with.”
The National Multiple Sclerosis Society invested $29 million nationwide in research last year to help develop MS cures and treatments. The society currently has $4.5 million invested in 15 projects by researchers at UConn and Yale University, investigating everything from the earliest stages of immune system attacks to the impact high-salt diets have on the development and severity of MS.
UConn Health Center researcher Joel Pachter has won two of those grants and has collaborated with ImStem on the MSC research, terming it an “extraordinary” development.
Stem cells have drawn intense research due to their ability to develop into mature cells with differing functions — for instance muscle tissue or red blood cells — and divide without limit, making them a valuable tool to repair damaged tissue.
In 2005, under former Gov. M. Jodi Rell, Connecticut committed to investing $100 million in stem-cell research over 10 years, with investigators having since won an equivalent amount in federal funding to support those projects. On June 24, Gov. Dannel P. Malloy released the latest round of funding, with ImStem founder Xu among the recipients.
Last year, ImStem won a $1.1 million Connecticut Stem Cell Research Program grant, followed by a $150,000 pre-seed investment from Connecticut Innovations, the state’s quasi-public venture capital arm.
Without the state investment, ImStem would not exist, Wang said. ImStem became the second private company to secure funding under the state’s stem-cell program, following Chondrogenics, a UConn spinoff that is developing a stem cell-based treatment for cartilage repair.
ImStem is now seeking approval for Phase I clinical trials and looking to raise further outside investment. Additionally, the company is looking to take on several other ailments, starting with inflammatory bowel disease and spinal cord injuries.
“If everything goes as planned, in five years we should have finished Phase II clinical trials on multiple sclerosis and started Phase I trials on other diseases,” Wang said. “The company can grow to around 30 to 50 people.”
Wang said funding remains ImStem’s biggest challenge, with pre-clinical trials mandated by the Food and Drug Administration commanding a steep price. “It’s hard for a startup biotech company to raise funding before Phase I,” Wang said. “There is a big funding gap between grant support and venture capital.”
Earlier this year, the Malloy administration won legislative approval to couple Connecticut’s Stem Cell Research Program with a new Bioscience Innovation Fund, renaming it the Regenerative Medicine Research Fund and extending the window for making grants from 2015 to 2020.
The proposal, however, didn’t win unqualified support: Yale University researcher Lawrence Rizzolo and Wesleyan University’s Laura Grabel expressed worries the new emphasis on regenerative medicine could move Connecticut away from the gains it has made in building a cluster of stem cell science research.
