CALR MUTATION: FOCUS AREA ONE

By Sam Klepper

As a board member of the MPN Research Foundation for more than 10 years, it has been encouraging to see the progression of the scientific community’s understanding of the MPNs and the development of an initial set of targeted therapies. One of the challenges we face as a foundation is determining which set of promising research and talented researchers to fund. It can be a hit and miss exercise and we want to use the funds we raise from patients, their families and corporate sponsors as thoughtfully as possible.

We took a chance back in 2011 on Dr. Robert Kralovics, PhD, Research Center for Molecular Medicine of the Austrian Academy of Sciences, who noted that not all MPNs were explained by a JAK2 positive result. He proposed looking at an alternative pathway called Calreticulin, or CALR, to understand if it played a role. Our scientific advisory board thought it was a promising path to follow, but nothing was guaranteed. The Foundation decided to issue Dr. Kralovics a grant to study this pathway. The work paid off. In 2013 Dr. Kralovics revealed the discovery of the CALR mutation in 30%-45% % of MPN patients who do not have the JAK2 or MPL mutation. His paper is published here.

Given the important potential for the CALR discovery to lead to new diagnostic tests and treatments, the MPN Research Foundation made further study of the CALR pathway a focus area for its 2014 grant program. We are excited to announce that four grants are being made to fund CALR related research:

• Michael W. Deininger, MD, PhD; Thomas O’Hare, PhD; Djordje Atanackovic, MD; Tim Leutkens, MD of University of Utah Cancer Center
• Robert Kralovics, PhD of CEMM, Austria
• Jean-Luc Villeval, PhD of INSERM (France); Sandra Pellegrini PhD and Stefan Constantinescu MD, PhD of Universite Catholique de Louvain (Brussels)
• Leonard Zon, MD of Boston Children’s Hospital

For more information on these grants please visit our 2014 MPN Challenge Grants Award Page.

It is only the generosity of your donations that allows us to take a risk on new research that can lead to an important discovery. We hope you will continue to fund the MPNRF, or if you are not yet a donor, consider making your first donation now. This will enable us to continue to take these type of calculated risks that can lead to important discoveries.

Sam Klepper
MPNRF Board Member
PV patient since 2001