On March 7, 2016, International Stem Cell Corporation (OTCQB: ISCO) announced that the Melbourne Health Human Research Ethics Committee (HREC) had approved its application to initiate phase I clinical trials of ISC-hpNSC® (human parthenogenetic neural stem cells) for the treatment of moderate to severe Parkinson’s disease. With that approval, ISCO has begun enrolling patients for those phase I clinical trials in what marks a major milestone for the company.
International Stem Cell Corporation is researching new ways to increase the quality and length of human life spans through regenerative technologies that augment or replace organ transplants, developments made possible by the discovery of the structure of deoxyribonucleic acid (DNA) in 1953 by American biologist James Watson and English physicist Francis Crick. DNA was first identified (http://dtn.fm/Elyc9) by the Swiss physiological chemist Friedrich Miescher in 1869. The knowledge that the DNA molecule exists in the form of a three-dimensional double helix has been hailed as one of the ‘two radical developments over the past sixty years’ in medicine in What Happened to the Future (http://dtn.fm/FqxA9), a lament on the slow pace of present technological development.
The What Happened to the Future jeremiad penned by the venture capitalist Bruce Gibney while at Founders Fund tells how it used to be. ‘Less than twenty-five years after Watson and Crick published the structure of DNA, venture capitalist Robert Swanson and biochemist Herbert Boyer founded Genentech, now a subsidiary of Roche (OTCQX: RHHBY), which went on to synthesize insulin far faster and more cheaply than almost anyone believed possible. And in a great revolution in the FDA approval process in the 1980s following pressure from the AIDS lobby, the agency acted almost nimbly to approve a huge number of important new drugs for many maladies.’
However, since then the drug development process has become notoriously tortuous and costly. It starts with drug discovery, when new insights into how a disease progresses allow researchers to design a product to stop or reverse its effects. Alternatively, a number of molecular compounds may be tested to determine what effect, if any, they may have against a range of medical conditions. Sometimes, good fortune may come of the ubiquitous side effects. A drug prescribed for one condition may be found to have benefits in other areas. And then, of course, there are the new technologies like ISCO’s human parthenogenetic stem cell (hpSC) technology.
After a particular compound has been identified, it must be subjected to pre-clinical trials. Pre-clinical trials, or, as they are sometimes called, non-clinical testing, may be pharmacodynamic (examining what the drug does to the body); pharmacokinetic (examining what the body does to the drug); may examine how the compound is absorbed, distributed, metabolized and excreted by the body; or may attempt to determine its toxicity. In some instances, animals may be employed in pre-clinical trials.
It is only after greater knowledge of the compound has been obtained through pre-clinical testing that it will be administered to humans in clinical trials. Clinical trials typically may have four phases, each of which is designed to answer a research question. In phase I, researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. This is the point that ISCO has reached with its human parthenogenetic stem cell ISC-hpNSC® technology.
In phase II clinical trials, the compound is given to a larger group of people to see if it is effective and to further evaluate its safety. In phase III studies, the drug is administered to an even larger group of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely. Phase IV testing is conducted after the drug has been marketed to gather information on the drug’s effect in various populations and any side effects associated with long-term use.
Before a drug can be marketed, it will be reviewed by the FDA when the sponsoring company submits a new drug application (NDA). And for many years to come, the FDA will continue its post-market safety monitoring. It’s quite a journey for a drug discovery company to take, but mindful of Gibney’s complaint, International Stem Cell Corporation is forging ahead with the phase I trials, which will be performed at the Royal Melbourne Hospital in Australia.
For more information, visit www.internationalstemcell.com
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