Cleveland scientists say they’ve created the first known treatment that, in tests so far, reverses Parkinson’s disease.
A team at Case Western Reserve University (CWRU) School of Medicine has created a protein, called CS2 for short, that helps in mice, human cells and human tissues to undo a brain malfunction behind the progressively debilitating disease.
Parkinson’s attacks the nervous system, affecting movement, balance, and more. Exercise and other medicines ease its symptoms, but no treatment available to patients yet reverses or even slows the disease’s progress, according to the CWRU study’s senior author, Xin Qi, professor of brain sciences.
The breakthrough was announced on Dec. 22 in the peer-reviewed journal Molecular Neurodegeneration by Qi and research colleagues Di Hu and Xiaoyan Sun. In eight months of tests, the team found that CS2 improved stricken cells with no apparent side effects.
If all goes well, Qi says, it will still take at least five years to develop CS2 for human trials and at least another six to make it available for prescriptions.
More than 10 million people are believed to have Parkinson’s, including 2 percent of people over age 60. Some cases have genetic or environmental links, but most have no clear cause.
The first symptoms may be tremors, a weakened sense of smell, dizziness, fainting, stooping, hunching or a steadily grave, depressed or angry expression. Later on, Parkinson’s may hurt balance, coordination, motivation and other functions, contributing indirectly to death.
Since doing the published research, the team has been trying, among other things, to discover a biological marker — something measurable in the brain that reflects the disease’s state and the treatment’s success. Qi says a marker might also allow diagnosis before symptoms appear. “That could be an important window for treatment.”
Qi has co-founded two local companies to optimize and test CS2 as well as two drugs from her lab aimed at Alzheimer’s disease and Huntington’s disease.
A protein called alpha-synuclein normally helps communication within the brain. In Parkinson’s patients, though, it tends to become part of clumps known as Lewy bodies. The CWRU scientists discovered that it binds in those bodies with a normally helpful enzyme called ClpP. Then each substance causes the other to harm a common structure within cells called a mitochondrion.
“A mitochondrion is a powerhouse,” says Qi. In human brains, it boosts energy, pleasure, movement and more.
The team’s protein acts as a decoy, luring the alpha-synuclein from the enzyme, rendering both harmless. CS2 was the team’s second try at a decoy; hence the “2” in its name.
During 15 years at CWRU, Qi has won about $37 million in grants to study Parkinson’s from foundations and the National Institutes of Health. She holds seven patents, including one for CS2. She has contributed to more than 70 published studies and given nearly 60 academic presentations. She is co-director of the medical school’s Center for Mitochondrial Research and Therapeutics and interim chair of its department of physiology and biophysics.
Asked about her latest findings, James Beck, executive vice president and chief scientific officer of the Parkinson’s Foundation, says, “This is a thorough study that nicely advances Parkinson’s disease research on both a biological and therapeutic level… The results of this study establish an intriguing foundation for future PD therapies.”
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