VistaGen Therapeutics (VSTA) Helps Get the Drug to Where It’s Needed

The pharmaceutical industry has invested billions of dollars developing drugs that can do truly amazing things once they are successfully applied. However, one of the historic challenges faced by the healthcare industry has been to figure out better ways of getting the drugs to the places where the problems are. It can be wasteful and potentially harmful to flood the entire body with a substance in order simply to ensure that enough of it gets to the right locations. A good example of such a challenge is the blood-brain barrier.

The blood-brain barrier consists of an especially tight alignment of cells lining the capillaries that permeate the brain and central nervous system. The structure prevents many substances from passing into the brain and central nervous system. It’s the body’s effective way of protecting the system from bacteria and damaging chemicals. The problem, of course, is that the barrier can also be effective in preventing potentially beneficial drugs from making it to the central nervous system where they could do important work, such as treating pain or disease.

VistaGen Therapeutics, best known as a developer of new generation, stem cell-based bioassay systems for the superior drug development, including predictive and liver heart toxicity testing, is developing a small molecule drug candidate with potential to overcome the challenging blood-brain barrier. VistaGen’s AV-101 (L-4-chlorokynurenine or simply “4-Cl-KYN”) is a novel, oral prodrug candidate designed to treat neuropathic pain, a condition of chronic pain affecting millions worldwide. In preclinical studies, AV-101 demonstrated the ability to be rapidly transported across the blood-brain barrier, at which point it converts into 7-CKYNA at the site of seizures and potential neural damage in the brain and spinal cord. Like the Trojan Horse, AV-101 infiltrates as one thing, and then converts into another. Once across, its converted form has the potential to be used for treating neuropathic pain and potentially depression and other neurological conditions.

The company has already been awarded over $8.5 million from the U.S. National Institutes of Health (NIH) for development of AV-101. VistaGen expects to complete its Phase 1 clinical development program for AV-101 in fall 2012.
For additional information, visit the company’s website at

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