Dr. Xianmin Zeng, associate professor at the Buck Institute for Research on Aging answers your questions about Parkinson’s disease and stem cell research. Zeng has a CIRM research grant to develop a stem cell treatment for Parkinson’s disease.
Parkinson’s disease is a neurodegenerative disorder, which leads to tremors, slowness in movement, impaired balance, and stiffness. There is no cure for Parkinson’s. And although drugs can help reduce symptoms, they eventually lose their effectiveness. Zeng has developed methods for transforming those stem cells into dopamine-producing nerve cells, the same cells that are lost in Parkinson’s disease. The hope is that by transplanting these cells into the brain, they will replace the lost cells and restore function in the brain.
For more information about CIRM-funded stem cell research related to Parkinson’s research, see fact sheet.
By Brooke Adams, The Salt Lake Tribune
The mice at the top of a column of stacked plastic bins at Q Therapeutics are shivering so hard they seem to be jumping.
Their nonstop shivering and seizures are caused by a genetic defect that robs the mice of the crucial myelin sheath that surrounds nerve cells and helps them send signals. Because of the defect, the mice are soon paralyzed and die prematurely.
It is a related problem — loss of this myelin sheath — that in humans causes the progressive loss of function in multiple sclerosis and several other diseases that can cause paralysis in humans.
And that’s why what has happened to the mice is so promising: After being treated with an adult stem cell therapy developed at Q Therapeutics, they are no longer shivering.
The product, called Q-Cells, also may be applicable to such neurodegenerative diseases as Parkinson’s, Alzheimer’s and amyotrophic lateral sclerosis, or ALS — better known as Lou Gehrig’s disease.
Now, the National Institutes of Health have awarded a $5 million grant to Q Therapeutics, the University of Utah’s Cell Therapy Facility and Johns Hopkins University School of Medicine, which as a team has had success in animal models of ALS.
The funding will help support work needed to get permission from the Food and Drug Administration to start human clinical trials at Johns Hopkins. If efforts to raise additional funds are successful, those trials would begin next year.
“This type of therapy can bring about a major change in modern health care,” said Deborah Eppstein, CEO of Q Therapeutics. “It’s not just a little step. It’s a pole vault change, a going to the moon change.”
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‘Chemical’ programming avoids problems genetic manipulation poses, study finds
April 23 (HealthDay News) — Scientists have converted adult cells into embryonic-like stem cells by using chemical programming instead of genetic manipulation.
Gene manipulation is an older method that has posed the risk of serious health problems such as cancer, the researchers explained.
The ability to make stem cells without genetically altering them could lead to the development of many new types of therapies for a wide range of diseases, including type 1 diabetes and Parkinson’s disease, the team noted.
“We are very excited about this breakthrough in generating embryonic-like cells from fibroblasts [cells that give rise to connective tissue] without using any genetic material. Scientists have been dreaming about this for years,” research leader Sheng Ding, an associate professor at the Scripps Research Institute in La Jolla, Calif., said in a Scripps news release.
Ding and his colleagues reprogrammed adult cells by engineering and using recombinant proteins, which are proteins made from the recombination of fragments of DNA from different organisms. They experimented with these proteins until they found the exact mix that enabled them to gradually reprogram the adult cells.
The reprogrammed embryonic-like cells from fibroblasts behaved the same as embryonic stem cells in terms of molecular and functional features, including differentiation into various cell types, such as neurons, pancreatic cells and beating cardiac muscle cells.
The study, published online April 23 in the journal Cell Stem Cell, was supported by Fate Therapeutics.