Friday, December 9, 2011

I’ve never been to a horse race, so I don’t know what it’s like to see your horse win. But right now I’m feeling that maybe I’ll get to found out.

For months on the IWMF talk-list I’ve been reading about how Dr. Treon’s team has identified a gene mutation in 90% of 30 cases analyzed in his genomic study. Today Dana-Farber issued the press release in advance of Dr. Treon’s presentation on Monday at the 2011 annual meeting of the American Society of Hematology: http://www.dana-farber.org/Newsroom/News-Releases/Researchers-identify-genetic-mutation-responsible-for-most-cases-of-Waldenstroms-macroglobulinemia.aspx.

The mutation they identified is in gene MYD88. Dr. Treon explains in the press release: “The mutation causes the cells to produce a distorted protein, which switches on the IRAK complex pathway, leading to activation of NF-kB, a protein that is essential for the growth and survival of Waldenstrom’s tumor cells. . . .When we shut down the pathway by blocking the abnormal protein with drug molecules, the tumor cells entered apoptosis [i.e., die].”

What’s significant for drug development is that the tested molecules had no adverse effect on normal cells. One case study patient reports that target therapy research for this specific gene mutation is now the Bing Center’s highest priority, and during my recent visit, his office said they were way ahead of where they thought they would be in coming up with a definitive drug.

As Waldenstrom’s is a so-called “orphan disease” – one that is so rare that there’s little financial motive for governmental and pharmaceutical investment in its research – I want to point out that funding for this research was provided by patients: contributors to the International Waldenstrom's Macroglobulinemia Foundation and Peter Bing and his family. I decided this past year to stop donating to other cancer and broader lymphoma organizations, and contribute directly to the IWMF. I like the idea that a small group of patients worldwide have banded together to fight for our own cure.

Not long after my first visit last Thanksgiving, Dana-Farber opened its Yawkey Center for Cancer Care. I got to experience this beautiful new facility this past visit. On my way out, I passed by lovely meditation gardens and this interfaith chapel with a wall that shimmered of stained glass. I stopped in to give thanks for my many blessings and particularly for those who dedicate themselves to cancer research. And for the insistence that I bet on the Dana-Farber horse, I am especially grateful.