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.
Of the six papers I discuss in this month’s full report, I just want to highlight two here. Both combine cell therapy with gene therapy to produce results that suggests the approach can reverse the damage of disease, at least in animal models.
This work is important to me and to California’s stem cell agency because I, and many of my colleagues, believe that this combination of gene therapy and cell therapy will produce some of the most dramatic results in regenerative medicine. In 2009 when we issued our first round of Disease Team awards five of 14 involved this combo. All of our disease teams have the goal of getting to a clinical trial within four years. Last week, in our second round of Disease Team grants, half of the eight grants awarded so far involved gene therapy with stem cells. The press release describing those awards is here. These therapies fall neatly into CIRM’s niche; they have high potential impact and they would be difficult to fund through normal channels. Such approaches, known as “dual modality” therapies, require passing through two different regulatory channels at the Food and Drug Administration, and this scares off many traditional investors.
The first paper discussed Huntington’s disease (HD) work at the CIRM-funded Buck Institute in Novato (although we did not fund this particular project). The team there started with iPS cells, in this case made by reprogramming skin cells from Huntington’s patients that had been made previously by another team. Then they replaced the defective Huntington gene with a correct copy using a clever technique that involves an artificial bacteria chromosome. When they matured those gene-corrected stem cells into the type of nerve cells destroyed in HD and transplanted those cells into a mouse model of the disease, they saw nerves grow that seemed to function normally.
The second paper involves work with muscular dystrophy (MD) done jointly at University College London and the San Rafaele Institute in Milan. They too, began with iPS cells, ones they created from the skin of patients with a specific form of MD called limb-girdle muscular dystrophy. They developed a way to push those stem cells to become large quantities of the precursor cells that can become muscle. Then they corrected the gene that is mutated in LGMD using a virus to carry the new gene into the cells. When they injected those cells into mice with a form of the disease, the cells homed to the damaged muscle and formed new muscle fibers.
A few of our disease teams have similarly produced early data in animal models suggesting their gene-therapy-plus-stem-cell combinations can correct disease in animal models (we blogged about some of that work in HIV/AIDS here). Collectively these results make us feel good that we may be going down a path that will be productive and help eliminate suffering caused by many diseases. My colleague blogged about the most recent Disease Team awards here.
My full report is available online, along with links to my reports from previous months.