When it comes to modern medicine, stem cells are seen as playing a role unmatched by anything else. Used in a growing number of cell therapies for certain cancers, various diseases related to the immune system, and other treatment applications, in addition to cell research, stem cell technology is opening an increasing number of doors for medical researchers and practitioners. Today it’s a market rapidly closing in on $1 billion, and, in spite of controversy surrounding some aspects of stem cell use, the funding for stem cell research continues to grow.
Stem cells, including pluripotent stem cells (non-embryonic adult cells that are artificially reprogrammed to mimic embryonic stem cells), are unique in their ability to differentiate into specialized cells, such as heart or muscle or nerve cells. As a result, they provide an important tool for medical research, treatment, and drug development.
For example, stem cells can play a substantial role in the discovery of new medicines that are more effective and safer for patients. They can also be used in the field of regenerative medicine, where various tissues and organs can be generated to replace damaged ones, for application to spinal cord injuries, Parkinson’s disease, diabetes, and perhaps even Alzheimer’s. In addition, given their ability to duplicate the functions of a whole host of other cells, stem cells are becoming important in all types of advanced biomedical research, helping us understand the most basic workings of the human cell and human body.
But one of the lesser known, though potentially huge applications of stem cell technology, involves the early stage testing of drug candidates. The pharmaceutical industry can spend vast amounts of money developing and introducing a new drug, only to find out later that it has negative effects on the heart, liver, or other organs. As a result, the drug must be limited or withdrawn, greatly reducing, or even eliminating, the anticipated profit potential. The numbers involved are telling. It can cost over $1 billion to bring a new drug to market, and it can all be lost if the drug is later found to have toxicity issues, such as causing heart problems. In addition to the time and money spent on developing and marketing, companies can face a long string of associated lawsuits.
In the case of Avandia, an anti-diabetic drug produced by GlaxoSmithKline, concerns that the drug may lead to an increased risk of heart attack, even though not firmly established at the time, still led to a major reduction in sales, and a slew of lawsuits. Given that the drug had been bringing in over $2 billion in annual sales prior to the trouble, it was a significant blow, and gives an idea of how much big pharma might be willing to pay for a workable solution.
It’s a problem for which California based VistaGen Therapeutics believes it has the answer, and it all revolves around stem cells. Using advanced stem cell technology, VistaGen has produced functional human cardiac cells that can be used early on in the drug development process to test for cardiotoxicity. Cardiotoxicity has been a factor in over 30% of drug withdrawals, and addressing it is seen as a major market. The use of real human heart cells in pre-clinical testing offers important advantages over traditional testing methods, such as animal testing.
First of all, it can be performed at the earliest stages of development, reducing the risks of developing the wrong drug. It’s also more accurate, since traditional testing involving animals can fail to detect potential risks in humans. And it’s far easier than the large number of patients and lengthy testing required in human trials. By identifying cardiotoxicity issues early in the process, drug developers can take steps to rescue the drug candidate, developing variants that are both functional and safe. Given that stem cells, including non-embryonic stem cells, can be pointed in many different directions, their potential to transform drug development has no clear limit.
But VistaGen is only now beginning to show up on many investors’ radar screens. Its recent strategic drug rescue-related collaboration agreements with Synterys, a medicinal chemistry and drug discovery services company, and Cato Research Ltd., a leading contract research and development organization, were further indications of the recognition VistaGen has earned for the exciting potential of its stem cell technology-based drug rescue initiatives.
The collaborations are intended to help develop safer drug rescue variants by leveraging VistaGen’s drug rescue initiatives with Synterys’ medicinal chemistry expertise and Cato Research’s drug development and regulatory capabilities. In the case of its key collaboration with Synterys, the idea is to combine VistaGen’s human pluripotent stem cell technology platform, called Human Clinical Trials in a Test TubeTM, with modern medicinal chemistry, to generate new safer variants of once-promising drug candidates discontinued in development due to heart toxicity concerns.
VistaGen sees itself as essentially transforming drug development by bring human biology to the front end of the process, attacking cardiotoxicity issues early in the cost curve, and removing much of the risk and uncertainty typically involved in bringing new drugs to market. Perhaps more importantly, it lessens the chance that patients will be asked to play the role of unsuspecting guinea pig, taking drugs that may cause them far more harm than good.
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