Q: What prompted the creation of the Life Raft Group?
The Life Raft Group (LRG) started in 2000, when a handful of patients in the early Gleevec trials began sharing their experiences online. Many patients had been misdiagnosed with leiomyosarcoma and were just now finding out they had an entirely different cancer – gastrointestinal stromal tumor, or GIST. Sharing their stories and experiences online helped them in a variety of ways, including gaining much-needed support while facing a rare disease and managing the side-effects of a new drug. In the next two years, this group formed a newsletter and Web site to share information with an even wider audience, before formally incorporating in June 2002. In the last 10 years, the Life Raft Group has grown immensely, meeting the challenges facing GIST patients and caregivers and the research community every step of the way.
Q: What were the challenges in GIST research that you felt LRG could help address?
GIST is one of many fast-growing, deadly cancers, so time is of the essence. Despite the billions spent on biomedical research, cancer death rates over the last 50 years have hardly changed. Based on 2010 data from the National Cancer Institute, death rates have gone down only 11 percent since 1950. With the traditional research and drug development process taking as long as 15 years to produce new therapies, LRG decided to take action. We set out to identify the barriers to success and how to overcome them.
One hindrance to the process is the high overhead costs charged by academic institutions. Rare cancer organizations like the Life Raft Group simply cannot afford to pay a proportionate share of administrative overhead rates ranging from 50 to 75 percent. If the total amount of money on the table is limited, as is the case with GIST, we could not rely upon this trickling down of funding to put enough in the hands of the actual researchers.
Additionally, the traditional way of distributing available money to cancer researchers is to issue a call for proposals by individual investigators and to have an expert board review. Although this process has merit, it is time-consuming and not at all guaranteed to fund those researchers with the best chances of success in finding effective cancer treatments. Finally, it is not driven by any strategic plan and is almost guaranteed to foster competition rather than collaboration among the various researchers.
Q: How has LRG tried to overcome those barriers?
Based on this information, the LRG decided not to follow more traditional rules and simply hand over money to cancer centers for the purpose of GIST cancer research. Determined to hasten the research process, the LRG established a clear research philosophy regarding these challenges: planning replaces disorganization, cooperation replaces competition, and urgency replaces complacency.
The LRG research project, "Pathway to a Cure," would be driven by a leading group of GIST researchers with complementary expertise, whose combined personalities would permit them to work together synergistically – with the patient included at the decision-making table. The primary focus would be on finding a cure and beginning with the creation of a comprehensive blueprint for understanding the mechanisms of treatment.
Rather than request research proposals, we challenged the team to create a strategy that consisted of specific prioritized projects with the greatest chance of success and to commit to our collaborative rather than a competitive philosophy. We also created a supportive grants process that capped all administrative overhead at 10 percent and eventually eliminated it altogether, requiring both financial and project accountability in the form of detailed progress reports. These are presented and data are shared via teleconference and in-person meetings. Additionally, we created the GIST Collaborative Tissue Bank, allowing researchers access to tissue from patients all over the world, along with their de-identified medical history, provided by the LRG.
Q: What do you consider LRG's greatest accomplishments to date?
In our 10-year history as a nonprofit organization, the LRG is proud of each and every accomplishment, large and small. However, the creation and strengthening of the Life Raft Group GIST Patient Registry is considered one of our greatest by the LRG community. This registry provides benefit to GIST patients and researchers alike and in a variety of ways.
The registry provides a comprehensive look at a patient's care at a glance, as well as providing up-to-date analysis of more than 1,400 GIST patients worldwide. The data from this tool have been of unparalleled importance in assisting patients with GIST in making informed choices paramount to their survival. It also provides current evaluations of which therapies a patient has tried – medications, dosages, procedures, and surgeries – as well as what treatments have worked and for how long.
The benefit to the researchers studying this disease is immeasurable; for the first time they are able to gain access to both the patient tissue and the patient's clinical history, which cross institutional boundaries, thanks to a process developed by the LRG and Research Team members at Stanford University and Oregon Health & Science University. In addition, because of this process, participants are able to receive free mutational testing, which can further refine their best treatment options.
Q: What are your top research goals for 2012, and what will it take to reach those goals?
In July 2010, the LRG launched a second phase of our research – "Project D-Day," which focused on four areas: sequencing the GIST genome; gene knockdown studies or screens that examine pathways essential for GIST cell survival; drug screening of GIST cells against large libraries of drug compounds, including drugs in late stage development and approved drugs; and validation studies where discoveries are investigated for relevance. Each of these four "teams" has made immense progress, including identifying new critical mutations and possible therapeutic drug compounds.
However, this research is now at a critical juncture, as we still have much to learn. While we now have a clearer understanding behind the mechanisms of GIST and the development of resistance, we are in the process of identifying and validating potential therapeutic targets based on this newly gained knowledge. After these targets have been validated, the goal is for our researchers to work together with industry to develop the next generation of drugs, while we move to create a new clinical trial process, thus bringing us that much closer to our ultimate goal – a cure for GIST.