Tuesday, February 25, 2014

Crafting the MPN environment

The World Orphan Drug Congress recently put out an independent study of the most researched rare diseases. We were shocked to see that the MPNs (MPDs on the list) ranked No. 10 of 25. 

This was not the world we knew in 2000. 

Ten years ago there was no infrastructure for driving research into PV, ET, and MF. There was no buzz around MPNs. No organization existed that was solely focused on funding research into MPNs. 

We had to build our own house. 

There were already some big names invested in MPN (at the time MPD) research who did foundational work. But there was no buzz around MPNs, the kind that attract and keep the interest of young researchers. The kind of buzz that will get the attention of pharmaceutical companies. 

The JAK2 discovery changed all that. 

In 2006 the MPN Research Foundation initiated the MPD Research Alliance, a program that formalized collaboration among researchers at Harvard, UIC and Mayo Clinic who were tasked with translating the discovery of a genetic marker for the majority of PV patients and half of all MF and ET patients into something that helped them.  As a result, today we have one FDA approved drug for symptoms of MPN - Jakafi - with many more drugs in clinical trials. 

But the Foundation didn't stop with the JAK2 discovery. In 2008 we opened up our research programs to any ideas that showed promise in combating PV, ET, and MF. Through this work we've helped discover yet another genetic mutation - CALR - that explains nearly all the non-JAK2 MPN. The CALR discovery has been characterized as a "game changer". 

Just as we didn't stop with the discovery of JAK2 or CALR, we see no reason to stop now. We are in the middle of the war on MPNs, not at the end, and we won't rest until all those with PV, ET, and MF have the answers and the medications they need.