Researchers from Yale School of Medicine have discovered a new drug compound that has reversed the effects of Alzheimer's disease in mice.
The researchers found a compound that inhibited the effects of a protein that is key to regulating memory and learning. The research has led to cautious optimism among Alzheimer's research advocates.
"Further studies have now begun to see if the compound leads to cognitive improvement in other species," including monkeys, said Dr. Paul Lombroso, lead author and professor in the Yale Child Study Center and the neurobiology and psychiatry departments at Yale.
Lombroso cautioned that it is too soon to know if the treatment would be effective in humans.
"I would be hard-pressed to say that this is the drug (that will be used to treat Alzheimer's), but we're much closer," Lombroso said.
Lombroso and the other researchers have found the compound, TC-2153, alters the effects of a protein called striatal-enriched tyrosine phosphatase, also known as STEP.
If the body's STEP levels get too high, it can prevent synaptic strengthening in the brain -- a process that's required to turn short-term memories into long-term memories. When STEP is elevated, it can impair cognitive function. High levels of the protein are associated not just with Alzheimer's, but a number of other disorders, including schizophrenia, Parkinson's disease, and the developmental disorder fragile X syndrome.
Lombroso said the role of STEP in Alzheimer's has been known for years, and there's long been interest in finding something that keeps the enzyme from affecting brain function. He and his co-authors studied thousands of small molecules, looking for one that would inhibit STEP. They found that treating mice with a single dose of TC-2153 improved their cognitive function.
Basically, the medication "binds very tightly to the STEP molecules and keeps (the protein) from doing its job," Lombroso said.
According to the state chapter of the Alzheimer's Association, there are more than 5 million Americans living with Alzheimer's, including 72,000 in Connecticut.
The findings -- published in the Aug. 5 issue of the journal PLoS Biology -- are encouraging, said Christy Kovel, senior director of communications for the Connecticut chapter of the Alzheimer's Association.
"It is an exciting and busy time in Alzheimer's disease research, with hundreds of potential therapies being tested at various stages of the research process, and many more being developed," Kovel said in a statement. "We need to create more treatment targets for Alzheimer's disease and the only way to do that is with more research, especially basic research into the causes and progression of the disease. This new data will help the scientists determine the likelihood and viability of moving forward with testing this potential therapy."
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