When the state of Connecticut began awarding grants for stem cell research in 2005, only a handful of scientists were investigating embryonic or adult stem cells. Today the number is well over 150. The $68.8 million in state funding that launched the program has attracted an additional $300 million from outside Connecticut. This stimulus has created more than 300 jobs and four new companies, and the explosion of research by state scientists is evidenced by 641 papers, 192 invention disclosures, 132 patents, 26 licenses, and two clinical trials using stem cell-based therapy.

This overview of the state of stem cell research in Connecticut came on November 7 at the Connecticut Stem Cell Retreat, held at the UConn Stem Cell Institute. Stem cell research in the state is “doing fantastically well,” said Milton Wallack, D.D.S., a member of the Connecticut State Stem Cell Research Advisory Committee, which awards grants for stem cell research.

Following Wallack’s introductory remarks, scientists from Yale, the University of Connecticut (UConn), and Wesleyan University gave updates on their institutions’ stem cell research.

Diane Krause, M.D., Ph.D., associate director of the Yale Stem Cell Center, said that Yale has hired 13 new faculty members who focus on stem cells. The center has also recruited within Yale—another 73 investigators from 23 departments are now researching stem cells, and the labs of 43 investigators are working with human embryonic stem cells (hESC) and/or induced pluripotent stem cells (iPSC). This activity has created a strong stem cell community, said Krause, and seminars, monthly forums, and faculty “chalk talks” keep scientists in the loop and encourage collaborations. Krause mentioned two examples of recent research by two new faculty members: an analysis by Jun Lu, Ph.D., assistant professor of genetics, of the genetics of leukemia using stem cells, and an investigation by Matthew Rodeheffer, Ph.D., assistant professor of comparative medicine and of molecular, cellular, and developmental biology, into the stem cells where body fat originates.

Yale has also expanded its ability to do stem-cell clinical trials. The latest enhancement, said Krause, is the center’s soon-to-open “clean room,” a lab that will help researchers “accelerate novel stem-cell therapies.”

Laura Grabel, Ph.D., the Lauren B. Dachs Professor of Science and Society, and director of the stem cell program at Wesleyan, said that funding from the state’s initiative not only boosted her lab’s ability to research stem cells, but also allowed it to combine efforts with two other Wesleyan faculty members to design a stem-cell based therapy.

The Wesleyan researchers looked for a way to suppress seizures caused by temporal lobe epilepsy, by using stem cells to replenish inhibitory neurons. Using embryonic stem cell material, they transplanted inhibitory interneuron progenitors into mice with epilepsy—and the seizures decreased. Recently, working with hESCs, they have made large quantities of inhibitory neuron progenitors and are now analyzing transplants using these cells to see if they function in the brain to suppress seizures.

Marc E. Lalande, Ph.D., director of the UConn Stem Cell Institute, said that until late 2006 the university had no stem cell research at all. Now more than 40 labs and 70 investigators are working with stem cells. Lalande noted that induced pluripotent stem cells (iPSCs) have revolutionized stem cell biology by allowing scientists to make patient-specific stem cells. The Stem Cell Institute has generated more than 100 of these stem cell lines and now sends them all over the world.

“The next transformational technology,” said Lalande, “is the ability to edit the genome of human embryonic stem cells and other human cells. It was not possible until recently.” The institute managed this feat within the past year. Lalande mentioned one of the first applications: Ren-He Xu, Ph.D., director of UConn’s Stem Cell Core, has used this breakthrough to correct a genetic defect called Marfan syndrome by making iPSC lines that are genetically identical except for the edited mutation. “This is very powerful technology,” said Lalande.