I’ve just returned from the EWOG-MDS/JMML conference in Denmark, an opportunity afforded to me by the Merilyn Hester Scholarship Award, which I have written about previously. I had a wonderful experience and again wish to express my deepest gratitude to the IUSCC for granting me the award, the EWOG-MDS/JMML group for the opportunity to present my research, and to Rebecca Chan and Merv Yoder whose mentorship has been so unfailing these past 2+ years.
The conference was attended by ~200 people united by the goal to treat and cure children with myelodysplastic syndromes and JMML. The attendees were mostly clinicians from the large European MDS/JMML centres —in Germany, Italy, Denmark—but there were also large contingents from as far away as Brazil and Japan. The diversity of this group highlights the importance of collaborative international research when focusing on rare (1 in 2 000 000 births) diseases. It also highlights the need to reduce and eliminate all barriers – be they language, cultural, financial – that may inhibit free discourse. As such, it was an eye-opening experience for me as a young trainee: the groups that study such diseases are small and tight-knit. There is little tolerance of egos; bridges can easily be burned by flippant comments. With this translational research there is a simple mantra: the patient always comes first. All research must prioritize what is best for the patient.
Therefore it comes as no surprise that – with one exception – all of the research presentations were based on patient data: results of ongoing clinical trials, proposals for new therapeutic strategies, updates on the building of clinical centres, etc. The exception was my own talk. My talk proposed a novel cell of origin for JMML given data from a mouse model of the disease. Despite its difference, my talk was favourably received. Several clinicians approached me afterwards and were eager to discuss developmental hematopoiesis—a topic that must rarely emerge in their clinic.
My presentation did strike a negative chord with one researcher. They said: ‘you study mice; how do you know your work is relevant to my patients?’ The point is well taken. A mouse is not a human. I use mouse models because my experiments cannot be performed on humans. However, unless I aim to treat leukemias in mice, I must always remain vigilant to ensure the clinical relevancy of my animal model.
Fortunately, the evidence remains strong that mouse hematopoiesis fairly accurately represents that of the human: blood cells first arise in the yolk sac, hematopoietic stem cells emerge from endothelium, macrophages in both organisms resist myeloablation and replacement following stem cell transplantation. I made this clear in my presentation, but the criticism reinforces the translational research mantra: the patient always comes first.
As I progress in my career as a clinician-scientist I anticipate relying more and more on patient data for my studies. This will ensure the clinical relevancy of my work. But in the meanwhile, I will continue to use the animal models at my disposal: performing studies that are impossible or unethical in patients while remaining fully cognizant of my models’ limitations.
As an aside: make a trip to Denmark a top priority. The country and its culture are both truly fascinating. Cyclists outnumber motorists in the streets. Pedestrians always wait for the walk signal to turn green. But they make up the time by walking faster than the most fleet-footed New Yorker. And art plays a large role in their life.
Stefan & The Boy
