One of the most feared diagnoses that a physician can deliver to a patient is that of a neurodegenerative disease. Whether the disease is Alzheimer’s, Parkinson’s, frontotemporal dementia (FTD), chronic traumatic encephalopathy (CTE), or Creutzfeldt-Jakob (CJD), there are no drugs available to stop or slow down the relentless spread of brain destruction. For some neurodegenerative diseases, there are drugs that provide temporary relief from some of the symptoms, but nerve cell death continues unabated.
Over the last year, a remarkable consensus of scientific evidence has shown that neurodegenerative diseases are caused by prions. Prions are infectious proteins that are devoid of DNA and RNA and are formed when healthy proteins become abnormally folded. Once this misfolding occurs, prions multiply in an ever-accelerating, self-propagating, autocatalytic process that is toxic to neurons. As prions move from one nerve cell to another, this destructive process spreads throughout the brain. While prions and their precursor proteins are different in each disease, the mechanism of brain degeneration is similar.
Dr. Stanley Prusiner was awarded the Nobel Prize in 1997 for the discovery of prions that cause mad cow disease and CJD. It has taken more than a decade to apply the principles of prion biology to other neurodegenerative disorders, resulting in a growing consensus that most, if not all, neurodegenerative diseases are caused by prions. The importance of this unification of neurodegenerative diseases cannot be overestimated with respect to the development of informative diagnostics and effective therapeutics.
We now have drug targets that will allow us to develop medicines that not only block the formation of new prions but also clear the existing ones. Animal studies of mad cow prions showed that lowering the number of prions halted the destruction of brain tissue. At UCSF, we have developed rapid, high-output screening procedures and sophisticated brain imaging in animal models, both of which are crucial for drug development—but our pace of discovery is much too slow. To scale up the process, we propose a nationwide effort to develop drugs to prevent and cure neurodegenerative diseases. Let us hasten the day when a neurologist who gives a patient the diagnosis of a neurodegenerative disease also has the drugs to effectively treat it.
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