Nobel Prize-Winner’s Stem Cells Help Glaxo Pinpoint Risks

GlaxoSmithKline Plc (GSK) is applying Shinya Yamanakas Nobel Prize-winning discovery in stem cells to identify heart risks linked to experimental drugs earlier in the development process.

Yamanaka was awarded the Nobel Prize in medicine this month for his work in turning ordinary skin cells into induced pluripotent stem, or iPS, cells with the potential to become any cell in the body. That breakthrough has enabled the London-based drugmaker to study iPS cells transformed into heart-muscle cells that may be used to test compounds for cardiovascular safety, said Jason Gardner, head of Glaxos early-stage regenerative medicine research.

About half of all experimental drugs fail for safety reasons, and half of those failures are due to toxic effects on the heart, Gardner said. Finding that risk even before animal testing could potentially save drugmakers millions of dollars in clinical-trial costs and better protect patients, he said.

I call this a low-risk, must-do approach to stem cells, Gardner said in an interview in Upper Providence, Pennsylvania, where Glaxo has been building on research using iPS cells that began in the U.K. in 2010. Its on the verge of being used now for decision-making.

Other applications include using the test, also called an assay, to help discover new medicines that improve heart function, where there is a huge need, he said. The iPS cells can also be used to create motor-neuron cells, aiding development of treatments for conditions such as Parkinsons disease, and test for drugs damage to the nervous system.

At a laboratory in Upper Providence, Glaxo scientists are working with iPS-derived heart-muscle cells supplied from Cellular Dynamics International of Madison, Wisconsin. In a petri dish, individual cells can be seen beating like a heart under a microscope.

The new safety test may bring drugmakers closer to clinical trials in a dish and enable them to reduce the number of animal studies, Brian Donovan, an investigator of platform technology science, said during a tour of the lab.

Drug testing may be one of the key applications of Yamanakas work, said John B. Gurdon, the British scientist who shared the Nobel Prize with him.

If you can take cells from a patient, like a patient with a neurodegenerative disease, and grow those in culture in the laboratory, you can test drugs on them easily, Gurdon said at an Oct. 8 press conference in London. But you wouldnt be allowed to test drugs that might have some health implications on the individual, on humans. To be able to derive cells that reflect a disease, in culture, is immensely important.

Glaxo and its peers have an ongoing dialogue with the U.S. Food and Drug Administration as they work to validate the tests effectiveness, and it will probably be used across the industry within a year or two, especially for development of diabetes and cancer treatments, Gardner said.

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Nobel Prize-Winner’s Stem Cells Help Glaxo Pinpoint Risks