Stem cell research is exploring new applications for treatment everyday. In the not-too-distant future, it will be possible to repair heart tissue damaged by myocardial infarction (heart attack), to replace nerve cells lost in Parkinson?s and Alzheimer?s diseases, to transplant new insulin producing cells for patients of diabetes, and to replace bone and cartilage lost through aging and inflammatory disease. This newly emerging field of regenerative medicine will fundamentally alter clinical medicine and significantly influence our perceptions of aging, health and disease, with a myriad of consequences for society at large.
Stem cell technology is also making inroads in drug discovery. Excitement has been spreading for years about the potential of stem cells and stem cell-derived models to offer to the drug development process. That?s hardly surprising given the pharmaceutical industry?s problems with research and development (R&D) productivity.
Despite the ever increasing investment by the pharmaceutical industry into R&D, the number of new drugs that reach the market place has been decreasing annually. A major hurdle lies at the juncture between the preclinical and clinical phases due to failures in the translation of preclinical efficacy and safety data into humans. This clearly indicates that preclinical research and screening need to be modified to ensure improved relevance to human diseases.
Recent advances in stem cell research demonstrate the ability to derive fully differentiated human cells from stem cells, raising the exciting possibility that infinite supplies of specific types of human cells may soon be available for preclinical studies. It is therefore increasingly recognised that the first major utility of stem cell technology is likely to be in drug discovery.
Stem cell technology offers great potential for understanding disease mechanisms and identifying targets, and for effective toxicology screening. A strong advocate of this new-age concept is Stephen Minger, global head (research & development) at Cell Technologies, a division of GE Healthcare. His venture is looking at India to commercialise its stem cell drug discovery technologies. His rationale: stem cells are better suited to help screen for drugs and to investigate how different diseases damage the body. The GE Healthcare division is aggressively promoting its analysers for pharmaceutical companies in a bid to help speed up the screening of drug compounds to assess its safety standards. Minger says these technologies would enable drug companies in India to reduce the cost of failure.
Minger has been making efforts to create an awareness about the range of automated technologies to profile drug candidates, cellular imaging in bio-medical research and cell therapy systems. He says, ?Without the introduction of automated technologies, we cannot expect improvement in R&D productivity or in the discovery for more effective therapies.?
GE Healthcare is engaged in discussions with hospitals, pharmaceutical companies and research institutions for its automated technologies that can help drug developers test the efficacy of their medicines on humans at the earliest stage that can help bring a good, efficient drug for the patients and save billions of dollars from drug failures.
Company officials reckon that when a compound undergoing rigorous pre-clinical analysis fails in phase III clinical trials, the investment lost is over $1 billion. ?The pharmaceutical industry cannot afford another Vioxx that was pulled out of the market to cost Merck $4 billion. This is where instrumentation for high throughput screening is a boon for drug companies,? they reveal.
According to Minger, stem cell technology is cost-effective and target-oriented too, as opposed to the discovery process where huge is money is lost when the drug fails in clinical trials. GE Healthcare and another American drug discovery firm, Geron, have an agreement in place to develop and commercialise cellular assay products derived from human embryonic stem cells (hESCs) for use in drug discovery, development and toxicity screening.
GE Healthcare launched the first products from its alliance with Geron? Cytiva cardiomyocytes (human heart muscle cells)?for predictive toxicity testing. Minger believes that embryonic stem cells, from which all the body?s mature cells are made, is a powerful research tool that provides better predictability. He gained one of the first licenses in the United Kingdom for the derivation of human embryonic stem cells, and generated the first human embryonic stem-cell line. However, he feels that the Indian market is still nascent and needs more awareness.
Going by the large number of birth rates in the country, Minger believes that there is an opportunity for harvest of stem cells from cord blood. Public banking of cord blood cells can benefit a larger population with advanced technologies today. There is a need to create public cord blood banks in government medical centres through public private partnerships to set up facilities. The best opportunity lies in storing waste umbilical cord cells to benefit patients.
Cell-based therapies are useful in tissue engineering, regenerative medicine and gene therapy. The availability of a new generation of human cell lines derived from embryonic stem cells represent a significant advance that will change the way in drug discovery.