Welcome back to Montagna, 2015! The conference keeps getting better and better!
The morning session, chaired by John McGrath, focused on stem cell-based therapies and innovative reprogramming technologies. In this session, it seemed almost possible to glimpse the future — a future where monogenic disorders of the skin like epidermolysis bullosa might be treatable! John reported on remarkable responses to cell-based therapies which led to life-changing improvements in the quality of life for his patients. Angela Christiano followed with a story about the next generation of 3-D skin models that she is developing that include not only keratinocytes and fibroblasts, but also melanocytes, nerve cells, and even rudimentary hair follicles. Jakub Tolar, a bone marrow transplant (BMT) expert from the University of Minnesota then showed truly amazing responses in recessive dystrophic epidermolysis bullosa with BMT. He demonstrated that stem cells derived from the BMT donor were able to differentiate and populate the skin and that these cells were able to produce collagen VII and improve clinical results in patients. Finally, Tony Oro presented clinical trial results from the Stanford dystrophic EB trial and showed new advances being made in adenoviral-driven reprogramming of autologous cells for therapeutic benefit. Taken together, these talks demonstrate the progress that is being made on all fronts, through the use of stem cells, for the benefit of these patients. It is one of the most optimistic and hopeful times I have seen for this class of disease.
The meeting ended with a bang – in keeping with tradition. Xiao-Jing Wang, from University of Colorado, revealed her new mouse model for patient-derived xenografting. One of the major limitations of the PDX mouse models is the need to use immunosuppressed mice in order to prevent rejection of the human tumor grafts. The immunocompromise in the traditional model eliminates the possibility of investigating the role of the immune system in the cancer progression and does not permit the investigation of immunotherapies. Xiao-Jing has now not only xenografted immunocompromised mice with human squamous cell carcinomas, but also used stem cells harvested from the same donor to reconstitute (or humanize) the mouse immune system. The same patient’s cancer graft is then removed and re-transplanted into the matched humanized mouse. In short, this is a new personalized model of the patient’s own tumor and immune system and opens up the possibility for testing novel drugs and drug combinations to predict response of the tumor to therapy, even immunotherapy. This is truly a preview of personalized medicine.
The last elements of the meeting included a panel discussion that was very lively. Topics included how to compete more successfully for grants, how to publish successfully, and how to position yourself for getting the job of your dreams after you finish your post-doctoral fellowship. I hope the young students and investigators in the room “felt the love” – those more senior members in the room were clearly committed to helping them become a successful next generation of investigators!
The finale of the meeting was a Potlatch Northwest-Style Salmon Barbeque at the home of Drs. Diane and Jim Baker. Just like the science at the meeting, life doesn’t get much better than this!