Former CIRM board member John Reed, Professor and CEO of Sanford-Burnham Medical Research Institute, recently interviewed Francis Collins for Medscape one-on-one. Collins is Director of the National Institutes of Health (NIH), and a vocal supporter of funding stem cell research.
The interview covers Collins’ own beginnings in science and advances over the last decade in genome sequencing as well as the role of the internet in the doctor-patient relationship. It’s a fascinating conversation and well worth listening (the full text of the conversation is also available).
Collins also discusses the importance of modeling diseases using stem cells reprogrammed from adult cells, something we’ve blogged about frequently. Collins says:
If you’re studying Parkinson disease, for instance, and you want to know what’s really going on in the neurons, you probably don’t want to do that by performing brain biopsies in people with Parkinson disease, but you could take a skin biopsy, convert those cells to IPS cells, and then add the appropriate cocktail, and differentiate those into dopamine-producing neurons; then, you have a pretty good model of what’s going on in the brain. You could then use those neurons, and people like you are doing this, to be able to assess among a library of possible drugs what would be the one that would be most likely to make those neurons happy. That is now being done for dozens of diseases, using this as a model of doing drug screens without having to depend on animal models (which sometimes have misled us), without having to just hope for the best and expose patients to drugs with less evidence. This is profound, with lots of potential there.
Collins goes on to talk about using stem cells—adult, iPS or embryonic—to directly treat disease:
At NIH, we have just started a new center for regenerative medicine on the campus, which has the use of our clinical center with its 240 research beds as a real possibility of pursuing cell therapy using stem cells. I don’t know for which diseases this is going to work, but I bet it’s going to work for some, and I bet it will be pretty transformative in the array of opportunity we have to treat diseases that are pretty frustrating right now.
Collins also made a point about the importance of basic research in the development of new therapies. CIRM has put significant funds into the later stages of research attempting to bring new therapies to clinical trials. But we also fund the basic research that is the engine for new discoveries and new ideas for how to treat disease. Collins says:
The work done by Elizabeth Blackburn and Carol Greider on telomeres, for which they won the Nobel Prize. Something that seemed so abstract, something that they studied in slime mold, of all places, and which now is profoundly important in our understanding of cancer and of aging, is just breathtaking as an example of a molecular insight into conditions that we never dreamed would have that particular connection.
A.A.
The Stem Cellar 
Those doctors are audacious, cheat taxpayer’s money with their stupid theory, steal from stem cell research. The disease is in the brain, not the skin. Disease in a dish they made from the healthy skin has nothing to do with the sickness in the brain, just like biopsy fruitfly to fool patients. And no wonder Francis Collins cannot get NIH budget to increase, buy such audacious claim.
The disease is in the brain, not the skin. Disease in a dish they made from the healthy skin has nothing to do with the sickness in the brain. Those doctors are audacious, steal from stem cell research with their stupid theory. And no wonder Francis Collins cannot get NIH budget to increase, buy such audacious claim.
Dear Mr. Anonymous,
Before attacking Dr. Collins, do your homework!
This is a novel approach which has captured worldwide attention to able to model human disease using human tissues. It is the low hanging fruit of stem cell research, of which translatable findings could find their way to the clinic in record time. As for the disease in hand, it is a GENETIC disorder, i.e., the faulty gene are present in every cell in the body and is silenced during development, but the gene it self is expressed in neurons. So basically, if you were to generate neurons with such procedures,you can activate the faulty gene, and have the chance to do a battery of testing to determine the mechanism of pathogenesis, leading to novel therapuitic strategies.
P.S: research groups in US, UK, Japan, and Europe are already using these technologies to model disease.