Jan 2016

A model approach to treating disease

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When most of us think about using stem cells to cure disease, we picture these building block cells being injected into damaged tissues or organs to help repair and rebuild them.

According to a recent Wall Street Journal article, there are more than 300 clinical trials underway around the world to test stem cells’ ability to treat diseases such as heart attack (read about a Canadian study here), stroke and amyotrophic lateral sclerosis — among others. The general idea is to get the stem cells to where the disease is doing damage so they can affect healing.

But, as the WSJ article points out, there is another approach in which stem cells are used to create models of disease outside the human body, in a Petri dish. These models not only provide researchers with a closer look at the molecular makeup of a disease, they offer the opportunity to test drugs that might be effective against it. The article highlights research at the University of California, San Diego where stem cells are being used to make Alzheimer’s neurons to test the safety and effectiveness of potential drug therapies.

There are real benefits to this approach. For one, it’s a supplement to using animals as disease models. Sometimes animals aren’t susceptible to the same diseases as people. In Alzheimer’s, for example, researchers use mice that have been altered to carry different genes or combinations of genes associated with the dementia.

The stem cell model approach also can save time and money. If scientists can rule out a compound as ineffective before moving to clinical trials, it can prevent years of work being done and millions of dollars being spent to travel down a research road that ultimately is a dead end.

Also, advances in technology mean scientists can use high-throughput screening to test hundreds, even thousands, of compounds against model tissue or organs to find a good candidate to shut down the disease — the research equivalent to finding a therapeutic needle in a haystack.


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