Each month CIRM President Alan Trounson gives his perspective on recently published papers he thinks will be valuable in moving the field of stem cell research forward. This month’s report, along with an archive of past reports, is available on the CIRM website.
I am very proud that two of the articles this month’s report discusses are from the new journal CIRM supports financially, Stem Cells Translational Medicine. Just a little more than a year ago CIRM solicited proposals from publishers to launch a journal that would give a home to and consolidate articles on translational research—those papers focused on pushing our field into clinical trials and beyond to routine care. Too often, these sorts of very practical papers cannot get accepted at routine research journals. Yet, it is critical this information spread to other teams in the field quickly. Our solution, provide the seed money to jumpstart a journal that would publish in a free online format with rapid peer review turnaround. Our ultimate publishing partner, AlphaMed Press, also publishes Stem Cells, the oldest journal in the field.
The two articles I cite both deal with the critical issue of scaling up growth of cells to produce sufficient quantities to supply large clinical trials and to do this using methods that limit as many cumbersome and expensive steps as possible. Especially important is avoiding anything that would reduce the chances of satisfying the FDA’s requirements for Good Manufacturing Practice (GMP), which is needed for cells being transplanted into people. (This short video has more information about GMP.)
The first paper details a method to get around the tendency of pluripotent stem cells, either embryonic or reprogrammed iPS cells, when grown in culture to want to mature into adult cell types. Early methods for keeping cells in their embryonic, or pluripotent, state had the double problem of being difficult to scale up and requiring animal feeder cells, which would greatly complicate creating GMP grade cells. Numerous reports have shown various ways to eliminate the animal cells, but those systems are still quite complex involving several human-derived proteins. The CIRM-funded team in this study was able to eliminate two of those human proteins with a single small molecule, greatly simplifying the process.
The second paper looks at the exploding use of Mesenchymal stem cells (MSC), now deployed in several hundred early stage clinical trials. The problem is that the usual source of these cells, bone marrow and fat tissue, have miniscule numbers of MSCs. This could make scaling up those small early-stage clinical trials into large definitive clinical trials difficult and costly. So, the Australian team in this study tried using iPSCs as the source, which has been done before but with slow and cumbersome techniques. They succeeded in shaving off about half the time to go from iPS pluripotent cells to MSCs and reduced the number of steps in the process.
My full report goes on to discuss one of my favorite topics, using iPSCs for modeling disease, but for the first time an infectious disease, hepatitis C. My full report is available online [pdf], along with links to my reports from previous months.