Stem cells can screen drugs for sometimes-lethal side effects

CIRM grantee Bruce Conklin recently spoke at the California Academy of Sciences about using reprogrammed skin cells (so-called iPS cells) to test drugs for lethal side effects in the heart. You can see a 4 minute excerpt of that talk here. One of the most common reasons for a drug to fail clinical trials is that it causes a heart rhythm defect called long QT syndrome. He argues that it could save money in clinical trials—and save lives—if stem cells could be used to screen the drugs to eliminate those that cause long QT syndrome before they are tested in people. There is currently no effective way of testing drugs for heart-related side effects before the drug enters clinical trials.

Conkin, who is a Senior Investigator at the Gladstone Institutes in San Francisco, has a CIRM New Cell Lines grant to develop iPS cells and mature them into heart cells. The idea would be to then put drugs in the dish with those beating heart cells and screen the cells for signs of long QT syndrome.

In the talk Conklin said:

What if you had just been prescribed a new medication and you'd gone to Walgreens and picked it up and taken the medication and then suddenly fainted and passed out. One possibility would be that you fainted because you'd had one of these [long QT] episodes and that you'd had a near-lethal arhythmia. One possibility to determine whether that drug caused the arhythmia would be of course to take the drug again and see whether or not it reoccurred. Now, although I'm sure you are supportive of medical research this is probably not advisable.

However, now one could have a different alternative. Perhaps we could recommend that we take a skin biopsy, we take that skin biopsy and grow iPS cells. WE grow heart cells from those iPS cells and we test the drug on those cells. That indeed would be something that you could potentially take part of and be supportive of.

We also produced a video of Conklin discussing this work.



A.A.