Headlines
As reported by The Hartford Courant, April 2, 2009.
State Earmarks $100 Million to Fund Stem Cell Research
By Arielle Levin Becker
The key to understanding and treating a severe form of mental retardation could lie in Stormy Chamberlain's petri dishes.
Using some skin cells and a technique that was unthinkable just a few years ago, Chamberlain can take skin cells from someone with a neurological disorder, transform them into stem cells, and turn those into brain cells with the same disorder. The technique could allow researchers to see how diseases develop and what interventions or drugs can treat them.
"It's one of the once-in-a-lifetime scientific breakthroughs," said Chamberlain, a postdoctoral fellow at the University of Connecticut Health Center.
On Tuesday, the project got a major boost, becoming one of 24 projects to receive funding as part of the state's 10-year, $100 million effort to finance stem cell research.
State officials established the grants in 2005 in response to a federal ban on funding for human embryonic stem cell research, allowing Connecticut researchers to continue their work in a field that scientists believe could hold the key to treating a host of diseases. The ban has since been lifted by President Barack Obama, and Connecticut scientists say the state funding has given them a leg up in qualifying for federal research money.
This year's $9.8 million in state grants was awarded to researchers at UConn, Yale and Wesleyan investigating topics including leukemia, spinal cord injuries, repairing heart damage and epilepsy.
UConn Professor Linda Shapiro received $500,000 to study what guides stem cells to repair damaged human hearts. Yale Professor Jun Lu is studying the mechanisms that turn specific genes on and off, which could have applications for understanding and treating cancer. He received $500,000.
The largest grant, $1.9 million, went to the UConn-Wesleyan Stem Cell Core, run by UConn Professor Ren-He Xu. The money will allow the core to continue its work, which includes training scientists and creating lines of human embryonic stem cells, the raw material for the research.
Chamberlain's work focuses on a disease called Angelman syndrome, a form of severe mental retardation in children. Children with it often have happy dispositions and are prone to outbursts of laughter, but typically do not speak. They can have seizures and difficulty walking.
Angelman syndrome occurs when a child fails to inherit a specific gene, known as UBE3A, from the mother. The gene also has implications for some forms of autism, which can occur if the maternal copy of the gene appears twice in a child.
In her research, Chamberlain begins with skin cells from people with Angelman syndrome and adds to them ingredients that turn them into stem cells. Because stem cells are capable of becoming any type of human cell, Chamberlain can then coax those cells into becoming neurons.
Unlike most neurons made from stem cells, these are expected to have the same genetic characteristics of someone with Angelman syndrome.
That would allow researchers to see how Angelman syndrome develops, potentially identifying ways to intervene. It would also give them brain cells on which to test potential drug therapies or ways to make the father's copy of the gene active in the brain, replacing the absent maternal version.
The same process could be used for other disorders, Chamberlain said, with "endless" possibilities. The technique is particularly useful in neurological disorders for which research on mice falls short, such as diseases that affect a person's ability to speak.
"How do you study a disorder that mice can't really have?" Chamberlain said.
For years, researchers were limited to studying human brains from people after they died, making it difficult to test drug therapies or other interventions to see what might work on a brain disorder, said Marc Lalande, director of UConn's Stem Cell Institute and the principal investigator in Chamberlain's laboratory. Having the ability to create any human organ, including ones with a particular disorder, is nothing short of revolutionary, he said.
"This is a whole new way of thinking of human disease," he said.