Parkinson's researchers focus on 'designer' cells

University of Florida regenerative medicine researchers have received a $1.6 million federal grant to study whether "designer" cells can be used to rescue the brain from Parkinson's and other neurological diseases.

Using cell cultures and a rodent model of Parkinson's disease, scientists want to study whether stemlike cells from mice and from adult human brains and bone marrow can be adapted to deliver a potentially protective protein to the brain.

"Certain cells derived from brain or bone marrow may have the potential to be engineered to release therapeutic factors in Parkinson's disease," said Dennis Steindler, Ph.D., principal investigator of the five-year grant and executive director of the Evelyn F. and William L. McKnight Brain Institute. "The possibility of using a person's own cells to slow or perhaps even halt the course of devastating neurological disorders offers a tremendous advantage, because there is less chance the therapy will be rejected."

The new, five-year study is funded by the National Institute of Neurological Disorders and Stroke. Scientists want to dose the brain with engineered cells capable of producing growth factors that have shown promise for replacement and preservation of neurons.

The idea is to nourish and protect brain cells that produce dopamine, a substance essential for normal movement that is depleted in Parkinson's patients.

About 1.5 million Americans currently have Parkinson's disease, according to the National Parkinson Foundation. The condition usually develops after the age of 65.

"It takes a great deal of dopamine-producing brain cells to die before symptoms appear," said Ron Mandel, Ph.D., a professor of neuroscience in the College of Medicine. "Our strategy is to protect these cells to slow or halt the progression of the disease."

Scientists will first genetically engineer the ability to produce two particular growth factors into immature human cells that haven't quite finished developing, and then introduce the modified cells into the models of Parkinson's disease.

"The idea is to obtain a few cells of the needed type from a patient, grow those cells, modify them to produce growth factors that protect at-risk dopamine neurons, and then put them back in the patient in a reasonable time," said Kenneth Berns, M.D., Ph.D., a collaborator on the project and director of the UF Genetics Institute. "It will be a challenge, but it will be a terrific application of human gene therapy in adult human stem and progenitor cells as well as differentiated cells."

Also collaborating on the project are Lung-Ji Chang, Ph.D., a professor of molecular genetics and microbiology, and Eric Laywell, Ph.D., an assistant professor of anatomy and cell biology.

Previously, McKnight Brain Institute studies revealed that so-called differentiated cells ' adult brain cells that have reached the last stage of their development ' may be able to morph into different cell types, a previously unknown characteristic. They can also be coaxed to produce large amounts of new brain cells in culture, making them a readily available and noncontroversial source for cell-replacement strategies.