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I’ve always been in awe about how much we know about the immune system. In fact, one of my earliest mentors impressed upon me that our knowledge can be attributed to how easy it is to isolate, separate and classify immune cells. Same goes for pluripotent cells (like embryonic stem cells). We know so much about how to grow them in the lab and how to turn them into different types of cells that labs all over the world are able to set up shop and start replicating each other’s data. When protocols are reproducible, and you know exactly what you’re working with, you are able to test hypotheses and draw conclusions.
Cell models of FSHD fall short in many of these regards. Different labs use different sources of cells that are isolated from patient muscle biopsies. Although these cells can be expanded somewhat, they are poorly characterized, incredibly variable and finicky to deal with in the lab. It therefore becomes difficult to extrapolate the data that is generated to the bigger picture. Sure we may identify a potential lead compound or two, but that won’t suffice for the long-term.
To that end, Friends of FSH Research and FSHD Canada have issued a continuation of funding for Dr. Maura Parker who is seeking to identify a more reliable source of cellular material for the FSHD field. Ideally, she will develop the methodology to extract, maintain, and define more stem-like cells from muscle biopsies. These cells could then be used for drug discovery and creating animal models for FSHD, in which the human stem cells would re-populate the muscle of a mouse. Thinking further down the pipeline, a properly characterized cell line could also form the basis of a therapeutic, transplantable stem cell.
Having an entire field of research dependent on a finicky cellular model has proven troublesome in other fields, and there is no reason why ours is different. Not having a cellular model is not an option. There is a lot we have to learn from muscle cells, and the experiments proposed by Dr. Parker are going to send us in the right direction.
The grant will fund Dr. Parker’s research program for two years at $50,000/year. Progress reports will be evaluated biannually by our scientific advisory board.