New funding initiative will speed disease research

A colony of iPS cells, courtesy of Kathrin Plath at the University of California, Los Angeles.

Yesterday was an exciting day at CIRM headquarters, especially for some of our science officers who have been hard at work putting together a new three-part funding initiative. We posted those requests for applications yesterday and expect to start receiving applications in September. (You can read those requests here.)

Admittedly, the initiative doesn’t have an exciting sounding goal: we’re planning to fund groups to create, store and distribute stem cell lines with disease characteristics.

I know, storerooms full of frozen cells doesn’t have the cache of a novel disease research. But wait: those frozen cells are more exciting than they might sound.

We’ve blogged about so-called disease-in-a-dish research, most recently in Parkinson’s disease and Huntington’s disease. The idea is that in order to develop therapies for complex diseases we need to know more about what goes awry in people who get the disease. Since it’s often difficult to directly study the brain cells of someone with, say, Alzheimer’s disease or autism, scientists have turned instead to reprogrammed stem cells.

The scientists take skin cells from people with the disease they want to understand, reprogram the cells to an embryonic-like state, then mature those cells into the ones they want to study. In the case of both Parkinson’s disease and Huntington’s disease, the resulting neurons show signs of the disease. Scientists can then study those cells to understand what goes wrong, and also expose those cells to different drugs in an attempt to find a drug that eliminates symptoms.

It’s important work and is already pointing to potential drugs for several diseases. CIRM’s goal is to help speed that critical work along by funding people to collect tissue from patients, create stem cell lines and store the cell lines. That way, if a scientist wants to study a particular disease, he or she can turn to CIRM’s repository of frozen cells and get to work.

We wrote about this initiative in our annual report. You can read that story here.

A.A.