Stem cell grants target multiple sclerosis, epilepsy, cancer

The nearly $10 million in state stem cell research grants awarded this year will fund researchers at three Connecticut universities exploring topics including chronic pain, tissue engineering and ways to develop vaccine-like treatments for cancer.

The grants represent the seventh installment of the state’s 10-year, $100 million commitment to funding research on stem cells, which scientists think hold the key to major breakthroughs in understanding a host of diseases and conditions.

Some of the money will fund continued operation of the stem cell “core” facilities at Yale University and the University of Connecticut Health Center, which is also used by scientists from Wesleyan University.

Wesleyan professor Janice Naegele will receive $1.49 million to continue her research on epilepsy, using brain cells derived from human embryonic stem cells.

One of the largest awards, $1.13 million, went to a startup company, ImStem Biotechnology, which is based on research by Dr. Ren-He Xu, director of UConn’s Stem Cell Core, and a postdoctoral fellow in his lab, Xiaofang Wang. Xu declined to discuss the company’s work in detail because a patent is pending, but said it’s aimed at exploring the potential for cells derived from human embryonic stem cells to be developed into treatments for multiple sclerosis.

Other grants went to researchers just beginning their own research careers, including Kevin Lo, who is working on ways to use stem cells and other particles to regenerate bone tissue. He came to UConn in 2008 when the leader of his lab, Dr. Cato Laurencin, took a job as dean of UConn’s medical school. Now he has his own lab and, as of Monday, his first grant.

“I’m quite surprised,” he said.

Stem cells are valuable to researchers because they carry the potential to turn into any other type of cell. Scientists can study them to better understand how normal cells and those with diseases develop. They could also be used to generate new, specialized cells.

Arvind Chhabra, a professor at the UConn Health Center who received a $600,000 state grant, is using stem cells in his search for ways to develop cancer treatments.

The idea is to develop a way that the mechanisms of the body’s immune system can work against cancer, potentially providing treatment without the toxicity or side effects of chemotherapy or radiation.

The immune system protects the body against foreign micro-organisms. Specialized cells recognize invaders like bacteria and viruses, and attack them. A healthy immune system doesn’t attack the cells of its own body.

Unlike infectious diseases, though, cancer develops inside a person’s body. And that renders the immune system generally ineffective against it.

But what if you could get the body’s immune system to fight a tumor? That’s the goal behind Chhabra’s work. That could be done, he said, by “teaching” the immune cells to attack a specific tumor, or to engineer those cells to kill tumors. He’s trying to develop an approach to turning human stem cells into immune cells that can fight specific tumors.

“Connecticut’s investment in stem cells continues to position the state on the cutting edge of biomedical research,” Gov. Dannel P. Malloy said in a statement released by his office. “These grants are critical to keeping and recruiting top scientists and help make possible innovative research that serves to improve people’s lives.”

Public Health Commissioner Dr. Jewel Mullen said the research holds “great promise for clinical applications that will help address a number of significant health challenges.”

“Connecticut’s stem cell program continues to fund some of the best basic and advanced stem cell research,” Mullen said in a statement.

There have been several major changes in the stem cell research field since the state began the grant program in 2005. At the time, federal funds for research on human embryonic stem cells were limited to work on a small number of existing cell lines. Since then, that restriction was lifted. Researchers at UConn have since derived lines of human embryonic stem cells that can be used in federally funded research by scientists across the country.

And since 2005, researchers have gotten another key tool for stem cell work, the ability to create “induced pluripotent stem cells,” or iPS cells. Instead of using cells from embryos that haven’t matured — “differentiated” — into a specific type of cells, they can take cells that have already developed — say, from an adult human — and cause them to revert back to their undifferentiated states.

If you want to study a genetic disease, you can create stem cells from the cells of a person who has that disease, making it possible to study how cells with the genes for that disease develop. You wouldn’t be able to do that with stem cells from an embryo that doesn’t have the genes for that disease.

Some of the grants are going to projects that use human embryonic stem cells, while others use iPS cells.

And Marc Lalande, director of UConn’s Stem Cell Institute, said some of the recently awarded grants fund projects involving another new technique: Gene editing.

It allows researchers to correct a mutation in a cell, or create a mutation in a cell that doesn’t have one. That means scientists can compare two cells that have the same genetic background except for a mutation in one, making it possible to better determine the effects of the mutation.

“This, for stem cell technology, for understanding the disease mechanism, for testing drugs and so on, I think is a transformational technology,” Lalande said.