Cortex Pharmaceuticals Inc., an emerging California-based neuroscience company, has been focusing on the development and future commercialization of Ampakines since the mid-1990s. Discovered in 1993 at the University of California in Irvine, CA, by Professor Gary Lynch, Ampakines are small molecules that are synthesized in the lab and are designed to be taken orally in the form of a pill. Once ingested, they get absorbed into the blood and enter the brain.
“The brain is filled with connections – trillions of connections,” says Mark Varney, Ph.D., CEO and President of Cortex. “Ampakines work by enhancing the communication between the connections in the brain. In doing so, they are able to facilitate learning and memory, and to overcome some of the chemical imbalances that can occur in the brain with certain diseases.”
In patients with psychiatric disorders, such as schizophrenia and ADHD, for example, certain areas of the brain fail to function as they would in mentally healthy individuals. Ampakines, Dr. Varney says, “allow the brain to recruit these malfunctioning areas and overcome these imbalances.”
The innovative aspect of Ampakine technology is the mechanism by which it operates in the brain. Unlike some anti-depressant drugs which modulate the serotonin neurotransmitter, or drugs that are used to treat Parkinson’s Disease, which modulate the dopamine neurotransmitter, Ampakines modulate the glutamate neurotransmitter system. Glutamate is, in fact, the most widely used neurotransmitter within the brain and is central to learning, memory and other cognitive tasks.
The innovators at Cortex consider Ampakines, as a glutamate modulator, well-suited to treat diseases involving cognitive deficits. They can also facilitate the production of growth factors within the brain.
“These are proteins that our brains make, and they actually are sort of nurturing agents to brain cells that are not functioning very well; they also help stabilize memory formation,” said Mark Frasier, Ph.D., Associate Director/Team Leader of Research Programs at the Michael J. Fox Foundation for Parkinson’s Research.
One particular protein brain-derived neurotrophic factor (BDNF) is turning out to be a very important player in neuroscience today, he explained, “and Ampakines can facilitate the release of BDNF. Based on animal data support, we found that when brain cells are stressed from a certain insult to a particular part of the brain, like during a stroke, or with some of the peptides that exist in Alzheimer’s Disease, BDNF may be able to rescue them.”
Cortex is not alone when it comes to its faith in the promise of these revolutionary drugs. The Michael J. Fox Foundation, where Dr. Frasier works, recently awarded Cortex a grant. The money will be used to fund a year-long preclinical efficacy study of Ampakines. The study involves inducing pathology of Parkinson’s in mice and treating them with Ampakine molecules, and then looking for the drugs’ physical effects on the animals’ brain cells.
“If successful and everything goes as hypothesized,” said Dr. Frasier, “[Ampakines] will provide what we would call a ‘neuroprotective effect,’ whereby the compounds will preserve the loss of particular brain cells that are lost in Parkinson’s Disease – [in effect] protecting them.”
Unlike current drugs used to treat Parkinson’s and which treat only the symptoms, Frasier added, “Ampakines actually have the potential to protect the brain cells from further damage and death by increasing the neuroprotective factors of the affected brain cells. That’s exciting!”
BDNF can also help stabilize immature neurons that are seen in autism patients – Cortex’s most recent area of interest. The company was recently granted exclusive worldwide rights by the University of California to develop a molecular combination of Ampakines and metabotropic glutamate receptor 5 antagonists (mGluR5 antagonists) in order to study their potential to treat Fragile X, the most common genetically proven cause of autism.
“Obviously, autistic children and adults have very significant cognitive impairments,” stated Varney. “They typically have lower IQs and low sociability.” Ampakines, he explained, can improve sociability, emphasizing how excited he is about the prospect of helping to facilitate incorporating autistic people into mainstream society.
Of course, the road from animal labs to human testing to FDA approval, and finally to pharmacy shelves, is a long and arduous one. “We would not expect to be on the market before 2014-15,” said Varney. “Part of the problem is that we’re a small company, and we haven’t as many resources as larger companies.”
Varney is devoted to guiding Cortex’s development of this novel class of drugs. He believes Ampakines will not only benefit people suffering from disease, but will also have deeper implications for society at large. “Consider Alzheimer’s, for example,” he offered. “The Alzheimer’s Association reports that currently over five million Americans suffer from the disease. It’s going to be a tremendous burden on our healthcare system.”
Hefurther pointed out that not only does the disease affect patients, but often family members who sacrifice their own lives and jobs to care for their loved ones. “Ampakines really have the potential to slow down or to delay these people going into nursing homes,” he added, “And if they can do that, it will have a really huge effect.” Certainly,it’s a worthy endeavor.
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