Stem cell agency awards $1.97 million to Stanford autism, heart disorder research

Joseph Wu

Two School of Medicine researchers have been awarded a total of $1.97 million by the state stem cell agency — the California Institute for Regenerative Medicine — to collect tissue samples from patients with dilated cardiomyopathy and from children with autism.

The grant recipients — Joseph Wu, MD, PhD, associate professor of cardiovascular medicine and of radiology, and Joachim Hallmayer, MD, associate professor of psychiatry and behavioral sciences — will use tissue samples, as well as control samples from unaffected people, to generate induced pluripotent stem cells for disease modeling and drug discovery.

CIRM also awarded $16 million to Wisconsin-based Cellular Dynamics International to fund a facility in California to derive induced pluripotent stem cells from patients, and $10 million to the New Jersey-based Coriell Institute for Medical Research to establish a pluripotent stem cell repository in California to safely store and distribute the cells to researchers worldwide. Together, the three grants, announced March 19, make up the agency's Human Induced Pluripotent Stem Cell Initiative.

Joachim Hallmayer

"Access to high-fidelity iPSC lines from a wide range of complex diseases will be an important accelerator of research," said CIRM president Alan Trounson, PhD, in a press release. "This initiative will provide scientists with access to multiple cell lines that should have much of the genetic variations that represent the variety within any human disease such as Alzheimer's, heart disease, lung fibrosis and autism. Scientists and companies can use these cells to discover the nature and causes of the underlying human diseases in a way not feasible before."

The initiative is expected to create and store a total of 9,000 cell lines from 3,000 people with 11 different diseases, according to CIRM.

The agency is giving $1.44 million to Wu, co-director of the Stanford Cardiovascular Institute, to collect blood and skin samples from several hundred patients with idiopathic familial dilated cardiomyopathy — that is, members of families with a predisposition to develop enlarged and weakened hearts without an obvious cause. The tissue samples will be used to make induced pluripotent stem cells, which will then be used to generate heart cells in the laboratory. Wu and his colleagues plan to study how heart cells from patients with the condition differ from those without it in order to search for novel genetic causes of the disease and to test potential drug treatments.

Hallmayer will receive $530,000 to collect blood and skin samples from children with autism spectrum disorders. Induced pluripotent stem cells made from the tissues will be used to generate neurons in the laboratory. These neurons will then be used to study cellular development and function in affected children; the researchers also will study neuron samples from a control group of children who don't have the disorders. In addition, Hallmayer and his colleagues will investigate the neurons' responses to environmental factors and to possible therapeutic drug treatments.

The governing board of CIRM's Independent Citizen's Oversight Committee also voted March 19 to adopt a number of amendments to its bylaws involving voting on grants, the role of patient advocates and the separation of the duties of the president and the chair of the oversight committee in response to a number of recent recommendations by the Institute of Medicine.

"Our goal now is to have the focus be on the work that we do, work that the IOM had great praise for, saying it had helped establish California as a world leader in stem cell research," said committee chair Jonathan Thomas, PhD, JD, in the release. "There is so much progress being made, and so many promising developments in stem cell research, and we want that to be the center of attention from now on."

With these most recent grants, Stanford has received a total of about $269 million from CIRM.

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions and required setting up a state agency to oversee allocation of the money.