Researchers from South Korea, Sweden, and the United States have collaborated on a project to restore neuron function to parts of the brain damaged by Huntington’s disease (HD) by successfully transplanting HD-induced pluripotent stem cells into animal models.
Induced pluripotent stem cells (iPSCs) can be genetically engineered from human somatic cells such as skin, and can be used to model numerous human diseases. They may also serve as sources of transplantable cells that can be used in novel cell therapies. In the latter case, the patient provides a sample of his or her own skin to the laboratory.
In the current study, experimental animals with damage to a deep brain structure called the striatum (an experimental model of HD) exhibited significant behavioral recovery after receiving transplanted iPS cells. The researchers hope that this approach eventually could be tested in patients for the treatment of HD.
UC Davis Health System researchers who are working to speed therapies to patients suffering from critical limb ischemia, osteoporosis and Huntington’s disease received approval today for three separate research grants from the state’s stem cell agency totaling more than $53 million. Each of the research studies that can now begin at UC Davis are specifically designed to lead to U.S. Food and Drug Administration approval of human clinical trials using stem cells and regenerative therapies.
At today’s meeting of the California Institute for Regenerative Medicine (CIRM) in San Francisco, the agency’s 29-member governing board approved five other grant proposals as part of its $150 million Disease Team Research Awards program. Funding goes to a highly select group of scientific collaborations to accelerate the state’s most promising stem cell research.
“Today’s Disease Team awards reflect the expertise and experience that UC Davis brings to the world of regenerative medicine,” said Jan Nolta, professor of internal medicine and director of the UC Davis Stem Cell Program and its Institute for Regenerative Cures in Sacramento. “Our teams are as eager as patients and families themselves to begin the type of clinical research that will allow us to discover new therapies and cures for people suffering from chronic and life-debilitating diseases. These generous grants allow our researchers to focus as never before on finding the best answers to the most challenging questions in regenerative medicine.”
Scientists at the UCSF-affiliated Gladstone Institutes and an international team of researchers have generated a human model of Huntington’s disease — directly from the skin cells of patients with the disease.
For years, scientists have studied Huntington’s disease primarily in post-mortem brain tissue or laboratory animals modified to mimic the disease. Today, in Cell Stem Cell, the international team shows how they developed a human model of Huntington’s disease, which causes a diverse range of neurological impairments. The new model should help scientists better understand the development of Huntington’s — and provide better ways to identify and screen potential therapeutics for this devastating disease.
This new model comes at a time of concentrated federal efforts to accelerate solutions for diseases — including a number of debilitating conditions that touch only small percentages of the population. Last year, the National Institutes of Health consolidated its efforts to attack rare diseases under the new National Center for Translational Sciences.