How Stem Cells Can Play a Major Role in Developing New Therapeutics

Stem cells can create systems to mimic healthy cells or diseased cells and by using these systems, companies can evaluate cell response to different drug candidates early in development. Studies using stem cells can identify new therapies, detect toxicity, and can help in evaluating effective dosing.

Last month Shinya Yamanaka won the Nobel Prize in Medicine for his research on transforming skin cells into induced pluripotent stem cells (iPSCs). Induced pluripotent stem cells are particularly valuable because they can differentiate into any cell type thereby creating model systems for drug discovery and testing. A recent article in Bloomberg Businessweek described how GlaxoSmithKline is currently using iPSC derived heart cells to test various compounds for cardiovascular safety. In the article, Jason Gardner, Head of Glaxo’s early stage regenerative medicine research, stated, “about half of all experimental drugs fail for safety reasons, and half of those failures are due to toxic effects on the heart. Finding that risk even before animal testing could potentially save drug makers millions of dollars in clinical trial costs and better protect patients.”

While using stem cells for toxicity testing is becoming more commonplace, this is just the beginning of their value in creating new medicines. A recent article in Drug Discovery and Development, “Bringing Stem Cells to the Forefront,” describes other ways stem cells can be used in drug development. In addition to creating heart cells to test cardiovascular toxicity, iPSCs can be used to generate liver cells to test liver toxicity. In the article, Amr Abid, PhD, general manager for cell technologies at GE Healthcare Life Sciences, states, “cardiotoxicity and hepatotoxicity (liver) cover about 60% of why drugs are withdrawn from the market.” Identifying toxicity issues before extensive pre-clinical work allows companies to save money by halting work on drugs that have serious toxicity issues. It also reduces the rate of drugs that drop out of clinical studies later due to toxicity issues previously undiscovered.

The Drug Discovery and Development article goes on to describe how eventually stem cells could be used to conduct virtual clinical trials for a number of drug candidates, which would save time, money, and potential adverse events. There are also opportunities to make advances in cancer therapies by identifying and studying cancer stem cells. Cancer stem cells are thought to be the reason why cancer often returns after surgery, chemotherapy, and radiation. The idea is that even though cancer cells are removed or killed, the cancer stem cells are not and they will generate more cancer cells. The article points out that if cancer stem cells can be identified and cultured, you can find something to kill them and prevent its return.

In addition to benefits in pharmaceutical development, stem cells are also at the center of a number of innovative new cell therapies. In the previous blogs titled “Ten Companies With Innovative Stem Cell Therapies in Clinical Trials Part I” and “Part II,” we discuss companies that have cell therapies currently in clinical trials. Human neural, muscle, embryonic, and mesenchymal stem cells are being developed as therapies and represent a new frontier in medicine.

While there are still challenges associated with stem cells in drug discovery and as drugs themselves, there is no question that the use of stem cells is opening up new avenues that will have a major impact in shaping the future of medicine.

We want to hear from you. What do you think are the most exciting uses for stem cells?

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