Critical questions that must be answered to cure JMML.
The JMML Research Roadmap is a vehicle through which affected families, new researchers, and donors alike can visualize the way forward for JMML research. It shows some of the most important current questions about JMML. Answering these questions now will help doctors better understand how JMML works and improve their care for patients.
The JMML Foundation Medical Advisory Committee, chaired by Dr. Christian Kratz of the University of Freiburg, Germany, divided the many possible roads to a cure into four categories: Pathophysiology, From bench to bedside, Murine models, and Clinical management.
Eighteen critical questions were identified as starting points for future research, and potential strategies for addressing each question related to JMML are presented (see table on next page). As noted by Professor Henrik Hasle of the Aarhus University Hospital, Denmark, “it is very appropriate and useful for putting the different scientific questions into perspective.”
Researchers cannot focus solely on the questions in the Roadmap, however, since their research may lead them into unforeseen directions that deserve follow up. Likewise, individual researchers may choose different strategies to answer these questions according to their areas of expertise and their institutions’ resources. This model, therefore, simply estimates the work that lies ahead on the path to a cure for JMML and is designed for ease of update as new questions, and answers, emerge.
What’s next? The JMML Foundation is using this JMML Research Roadmap now to create a research grant process. Through this process, donated funds will be directed to high pay-off targets on the road to a cure. Estimating the cost to answer any particular research question is impossible, though, as it may take many approaches to find the one that works. For an idea of how much it costs to execute a research project, consider how other nonprofits fund leukemia research:
- Leukemia Lymphoma Society (LLS) Career Development Grants: $55,000, $65,000, or $110,000 per year for 3 to 5 years
- Leukemia Research Foundation New Investigator Grants: Up to $100,000 per year
- LLS Translational Research Grants: Up to $200,000 over 3 years
- LLS Specialized Center of Research Grants: Up to $6,250,000 over 5 years
Clearer direction for JMML research. Less uncertainty and greater confidence in the research process. A rallying point for research funding. Increased international coordination. Faster progress to a cure. These are some of our aims, supported by the JMML Research Roadmap. We welcome your thoughts and urge your support to make these goals a reality.