Tiny cardiac cells could be sick, but that is a good thing, at least for researchers. The cells match those of a patient suffering from a rare and deadly heart condition.
"We can actually make heart cells right in a dish that's genetically identical to a person, then we can give drugs to those cells and have the heart speed up or slow down and so we're doing clinical trials in a dish right now," Dr. Bruce Conklin said.
Conklin and his team at the Gladstone Institutes at the University of California, San Francisco, helped pioneer a revolutionary method of creating and testing new drugs.
Their technique uses induced pluripotent stem cells or IPS cells cultured from the skin of patients. Those cells are then grown into specific human tissue for testing.
"To be able to work directly on human tissue, directly on human heart tissue, directly on human neuronal tissue is a fantastic leap forward," Conklin said.
That is because for decades researchers have relied on lab animals to conduct the early phases of drug testing; but that has led to many false starts because their physiology is significantly different from humans.
But now, South San Francisco-based Ipierian believes it is on the verge of developing new treatments for neuro-degenerative diseases using drugs tested first on human nerve cells created in their lab.
"We have started with skin cells from patients that already have the disease, turned those skin cells into stem cells and then turn those stem cells into neurons that also have the disease," Ipierian senior scientist John Dimos said.
Using the diseased neurons, researchers can test dozens of samples at a time to see which drugs are working, with potentially far more accurate results than in animal models.
"What we believe we have the opportunity to do is take actual cells from patients and to develop an understanding of actual human disease in the dish," Ipierian CEO John Walker said.
Walker believes testing specific drugs on human cells first, instead of animals, could ultimately cut the time it takes to develop new medicines by years.
"We hope to be in the clinic with two different agents to treat, we hope, both spinal muscular atrophy and ALS, within the next four year time frame," Walker said.
Researchers also believe the technique could have an impact on animal testing, perhaps someday dramatically reducing the need for lab animals.