a-syn – NIH Director's Blog (original) (raw)
Could Repurposed Asthma Drugs Treat Parkinson’s Disease?
Posted on September 19th, 2017 by Dr. Francis Collins
Thinkstock/ia_64
I had asthma as a child, and I still occasionally develop mild wheezing from exercising in cold air or catching a bad cold. I keep an inhaler on hand for those occasions, as this is a quick and effective way to deliver a medication that opens up those constricted airways. Now, an NIH-supported team has made the surprising discovery that some asthma medicines may also hold the potential to treat or help prevent Parkinson’s disease, a chronic, progressive movement disorder that affects at least a half-million Americans.
The results, published recently in the journal Science, provide yet another example of the tremendous potential of testing drugs originally intended for treating one disease for possible use in others [1]. In this particular instance, researchers screened a library of more than 1,100 well-characterized chemical compounds—including drugs approved by the Food and Drug Administration for treating asthma—to see if they showed any activity against a molecular mechanism known to be involved in Parkinson’s disease.
Tags: a-syn, alpha-synuclein, approved drugs, asthma, beta blocker, beta2-adrenoreceptor, brain, clenbuterol, drug repurposing, drugs, genomics, inhaler, metaproterenol, motor system disorder, neurology, Norwegian Prescription Database, Parkinson's disease, Propranolol, repurposing drugs, salbutamol, SNCA gene
Yeast Reveals New Drug Target for Parkinson’s
Posted on November 5th, 2013 by Dr. Francis Collins
Caption: Left, yeast sick with too much α-synuclein, a protein that is implicated in Parkinson’s disease. Right, the same yeast cells after a dose of NAB, which seems to reverse the toxic effects of α-synuclein.
Credit: Daniel Tardiff, Whitehead Institute
Many progressive neurodegenerative disorders like Alzheimer’s, Huntington’s, and Parkinson’s disease, are characterized by abnormal clumps of proteins that clog up the cell and disrupt normal cellular functions. But it’s difficult to study these complex disease processes directly in the brain—so NIH-funded researchers, led by a team at the Whitehead Institute for Biomedical Research, Cambridge, MA, have turned to yeast for help.
Now, it may sound odd to study a brain disease in yeast, a microorganism long used in baking and brewing. After all, the brain is made up of billions of cells of many different types, while yeast grows as a single cell. But because the processes of protein production are generally conserved from yeast to humans, we can use this infinitely simpler organism to figure out what the proteins clumps are doing and test various drug candidates to halt the damage.
Posted In: Science
Tags: a-syn, alpha-synuclein, Alzheimer’s disease, brain, drug candidates, genetic mutations, Huntington's disease, induced Pluripotent Stem cells, N-aryl benzimidazole, NAB, National Institutes of Health, neurological diseases, neurons, NIH, Parkinson's disease, protein, protein clumping, Rsp5, ubiquitin tag, yeast