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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