JMML research is sometimes put on hold because orphan disease funding from the government and major non-profit grantmakers is very limited. In order to further our mission of curing this disease, the JMML Foundation has asked researchers into the disease to keep us updated on promising projects that have been written but temporarily shelved due to lack of available funding. These “wish list” research projects, as well as emerging opportunities for novel therapies as identified by researchers, will be listed below as received and reviewed.

Please contact these researchers directly for more information and if you would like to directly support their research.

New! (November 2015):  Potential Clinical Trial using Fasudil

Description:  As part of our studies on CBL proteins, we have used in vitro and mouse model studies to look for potential drugs that might have an ameliorative effect on JMML disease. We have identified Fasudil, a ROCK inhibitor that is clinically safe (used in vascular conditions), to have significant activity and an initial report of this findings has just been accepted in Hematology. We would like to develop this track further in order to open a clinical trial in collaboration with colleagues that treat JMML patients at various centers.  ...we do not have resources to undertake it with any existing funds.... [Click here for a copy of the accepted manuscript]. This work was done by a Hem/Onc physician Basem William and other students in my lab. Basem has taken a faculty position at Case and will continue to be involved in this research on the clinical side.