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February 2011

2010 Executive Director's Report

The year 2010 was a bittersweet year built upon extreme highs and lows.

This year we celebrated a decade of treatment with Gleevec and Sutent, commemorated by an extraordinary gathering at our Life Fest weekend in New Jersey that brought ten year GIST survivors together with family and friends and with the clinicians, researchers and pharmaceutical companies that were responsible for this medical milestone. Luminaries of the GIST medical world were inducted into the GIST Hall of Fame; Drs. Demetri and Vasella were honored with Humanitarian of the Decade Awards; Dr. Trent was honored with the Clinician of the Year Award and hundreds of GIST patients and their families were honored for countless acts of kindness and of courage. (You can read more about Life Fest 2010 here)

On the research front, the critical work of the Life Raft Group’s research team was dramatically expanded due to a very generous donation by a Dutch patient helping to create “Project D Day”. This project seeks to accelerate research into the cure by creating teams focused on the following areas: Sequencing, Gene Knockdown, Drug Screening and Validation. We believe this strategy is our best hope for finding the cure. (Read more about Project D Day here)

In addition to our ongoing international networking and support, a Latin American coalition, Alianza GIST, was created at the first meeting of Latin American GIST patient advocates from ten countries held in Monterrey, Mexico. This alliance is focused on four key principles facing Latin American patients:

 To improve the knowledge of patients and physicians.

 To increase patient access to adequate treatment and resources.

 To support local patient support organizations, including the creation of new ones.

 To encourage collaboration and coordination with the physician community.

(Learn more about Alianza GIST here)

While these initiatives were taking front row, we continued to improve our patient registry and tissue bank, to expand our educational, outreach and advocacy efforts and to provide one-onone consultation to patients and oncologists seeking treatment options.

Perhaps the most poignant statement of the year was delivered at the Life Fest Meeting by Dr. Jonathan Fletcher, the head of our research team:

“In Dallas, we (the LRG research team) were asked by Norman to get up on the platform in front of this incredible group and say we were going to cure GIST and I was concerned with saying this because we can’t cure it...but, I am delighted to say and in a clean conscience, four years later, that our understanding of GIST has grown such in the last four years that now we can stand up on this stage and say with determination and conviction that we are going to cure GIST.”

However, we cannot dwell only on the year’s highlights. In spite of these successes, many GIST patients continued to endure disease progression and too many continued to die because they ran out of treatment options.

Even more frustrating were those GIST patients who died because they could not access treatment or because medical providers failed to use current knowledge appropriately.

The sad irony is that we could dramatically increase the survival rate of GIST patents simply by using what we currently know and without the introduction of a single new drug. Yes, we patient advocates do know that in a perfect medical world all knowledge would be created by randomized double blind clinical trials, reported in prestigious medical journals and provided with the seal of approval of oversight agencies like the United States Food and Drug Administration (FDA). But the reality of rare cancers such as GIST is that not everything can be investigated by such clinical trials and in the interim we really do have to use what we currently know to try to stay alive.

As my “New Year’s Resolution”, I would like to tackle some pressing questions with the help of the medical and GIST community.

To oncologists: Tests are currently available to determine the mutational status of GIST patients; these tests have been available for over eight years. About 15 percent of GIST patients will have an exon 9 mutation and clinical trials have shown that patients with exon 9 mutations receive far more benefit from a higher dose of Gleevec. Why are so few GIST patients in the United States receiving such mutational tests?

To the US FDA: Gleevec has been clearly demonstrated to shrink tumors, both primary and metastatic. If you administer Gleevec for a few months prior to surgery for a primary tumor, you are likely to shrink the tumor and make it easier for the surgeon to remove it and just as importantly, doctors can ascertain whether the cancer is responsive to Gleevec should there be recurrence following surgery. Can we accept this without a randomized clinical trials requirement, particularly when such a trial is unlikely to happen?

To the pharmaceutical companies: The LRG accepts that drug companies must make a profit and must navigate the complicated clinical trial process to do that. But there is not necessarily a conflict between good science and good business.

 The current clinical trial criteria for evaluating whether a drug is effective against GIST are called RECIST. Many GIST specialists and respected members of the medical community have commented that it is outdated and misleading. Why is the more relevant CHOI criterion or just plain disease stability not being used?

 Including mutational testing within your protocol may mean that you will find that some mutations respond to your drug while others do not. Why are you not routinely including this testing when, as a result, what may have become a clinical trial failure may now become a success?

 A number of pharmaceutical companies have begun consulting with patient advocacy organizations in order to garner help designing more effective clinical trials. Why isn’t this common practice?

It may seem as if I’m coming down a bit hard on the medical community. But in order to make 2011 a better year than 2010, we owe it to ourselves to push the boundaries of convention—to focus not on what is familiar, but what is right.

It is the patients, caregivers and advocacy groups who must stand by and watch our friends leave this world. If we know something is wrong, we owe it to their memories’ to try and make it right.

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