February 2012

2009 Executive Director’s report

Norman Scherzer There are a number of reasons to put the year 2009 behind us. The global financial crisis put retirement plans on hold, and lost jobs and homes added to the stress of families struggling to survive cancers like GIST. Fundraising for critical research was set back, and the hopes of patients for easier access to quality medical care were far from realized. Americans continued to argue over the universality of health care, British GIST patients continued to fight for access to secondline treatments, Canadian GIST patients waged their battle for adjuvant treatment, and too many GIST patients in too many countries struggled for access to any treatment at all. GIST patients continued to die either because they could not access treatment or because known treatments had failed them.

Despite these setbacks, the Life Raft Group continued its work on many fronts. We maintained our funding to the ten investigators on our world-class research team which continued to make progress in a number of areas including preclinical validation of KIT switch pocket inhibitors and improved understanding of cell mechanisms that normally destroy activated KIT and PDGFRA. In-person meetings in Boston and Portland continued the ongoing collaboration and information sharing that distinguish the Life Raft Group team from the unfortunate reality of competition and disunity that characterizes most of the cancer research world.

We rebuilt the data structure of our patient registry and published a breakthrough study on the relationship of Gleevec dosage to progression-free and overall survival (Go to http:// springerlink.com/content/346011445r10um38/ to see the published article). In collaboration with Stanford University and a group of key research laboratories we launched a comprehensive tissue bank which enables researchers to access and share research results on hard to obtain GIST tissue and to connect these results with the clinical histories maintained within the registry. To provide patients and their doctors with diagnostic and treatment summaries, we began to distribute medical histories aptly named GISTories.

Toward the end of 2009 we formally launched our Latin American Initiative to facilitate the flow of information about GIST throughout Latin American in both Spanish and Portuguese and to help develop viable patient organizations to create culturally relevant grass roots support to patients, their families and their doctors. I’ve just arrived in Monterey, Mexico where I’ll join a planning group of patient representatives from ten Latin American countries.

Not a week went by that we did not provide one-on-one assistance to numbers of GIST patients and caregivers seeking the latest information on treatment, including clinical trials. Our websites, webcasts and pamphlets continued to provide information to patients and medical professionals around the world. Our local groups continued to meet and provide personal support through a combination of hugs and critical information. And we continued to advocate both publicly and behind the scenes on behalf of GIST patients.

In 2010 we plan to accelerate our battle to find a cure for GIST and to keep patients alive until we do so.

In addition to maintaining our core commitments to our world-class research team, we plan to build upon their unique coordinated approach to implementing a continuously refined strategic plan by providing new incentives to exploit promising new ideas to identify and overcome pathways of treatment resistance.

We will also continue to expand our efforts to make sure that no patient dies because of the ignorance or inexperience of his or her physician or because of a lack of urgency in applying cutting-edge knowledge to support the battle for survival. We will insist that every GIST patient receives a mutational test for his or her primary tumor and, if on Gleevec, a plasma level test on an ongoing basis.

We remain concerned that 400mg of Gleevec may be inadequate over the long term to provide maximum protection to many patients against the development of resistance. We believe that it is currently easier to prevent resistance by optimizing Gleevec dosage levels than it is to reverse resistance by raising such levels after the fact. We have gained a great deal of experience in avoiding serious side effects on higher doses of Gleevec by employing gradual dose escalation and a growing array of techniques to manage side effects should they occur.

Although we do not yet have accepted reference levels for plasma testing, we think nonetheless that such testing is a critical tool for the physician to utilize now, particularly in the event that the patient demonstrates downward trends. At a minimum, this should create an occasion for the physician to discuss treatment compliance and to do so in a non-threatening manner. The issue of patients not taking all of their oral medications remains an ongoing concern for us, and we plan to intensify our efforts to address this issue. Exec Direc 09 quote

We will continue to utilize every method at our disposal for providing information, education and support to create empowered patients who seek a mutually respectful partnership with their physicians. This combination of well-informed patients and physicians reinforces the battle for survival. We believe that competent physicians will welcome this partnership.

Given the rapidly growing amount of information about GIST and the fact that it is a comparatively rare cancer, we will encourage patients to seek out physicians who have practical experience gained through seeing a significant number of such rare patients. We will continue to expand our online directory of GIST specialists and are planning to better define and identify physicians and centers of excellence.

Should our tone seem a bit strident or impatient we submit that our intention is to encourage thought and collaboration and that our urgency is driven by the very human struggle for survival.

The struggle to find a cure, and to help patients stay alive until then, continues.

Too many candles have been lit not to succeed.