Q: What prompted you to start your own foundation?
Initially, I searched the country to find an advocacy organization that would provide support for chordoma patients and fund researchers. Unfortunately chordoma did not fall under the purview of any other medical research foundation's mission. So after an extensive search I incorporated the Chordoma Foundation in February 2007 to unite researchers around a coherent plan, to facilitate communication and collaboration between their respective labs, and to make sure that they had the resources and funding they need to be successful.In 2006, when my son Josh was 18, he was diagnosed with chordoma, a rare cancer with no effective chemotherapy and an average survival of five to seven years. Josh and I became resolved to do everything in our power to bring about new treatments, and ultimately a cure. We first set out to learn everything known about the biology of the disease, and map out all active chordoma researchers. Although there was only one NIH-funded chordoma research project at the time, we identified about a dozen researchers across the globe who were interested in chordoma but were working in isolation and with only very limited funding.
At the same time, we recognized that navigating the medical system, especially for a rare cancer, was exceedingly difficult and lonely. We then united a community of patients, families, and caregivers to work collaboratively with the medical and research community to advance chordoma research and improve the quality of care for chordoma patients.
Our mission is to rapidly develop effective treatments and ultimately a cure for chordoma, while improving the diagnosis, treatment, and quality of life for people affected by this devastating bone cancer.
Q: What do you think has been your biggest achievement?
In three years we have cultivated a new field of chordoma research, bringing together over 100 investigators from eight countries, most of whom were not previously studying chordoma. Doing so has sparked over two dozen new research projects, and has led to three new clinical trials.
Q: What are your goals for 2010 and what will it take to get you to realize these goals?
Our top three goals are: enrolling 100 participants in the Chordoma Foundation Biobank and registry, completing Phase I of the Chordoma Genome Project, and developing new validated cell lines and animal models. We have an ambitious funding goal of $5 million.
Q: Is there anyone you haven't been able to collaborate with yet that you'd like to?
Overall we have been met with overwhelming support and cooperation. We would like to collaborate with:
- Philanthropists who are interested in partnering with us to fund our innovative, project-management approach to find improved treatments for chordoma, which can ultimately be applied to other cancers.
- Pharmaceutical companies that are interested in rare cancers. We would like to determine if there are new drugs in the pipeline or ones that have not made it to market that could potentially be effective for chordoma.
- Marketing, communications, and public relations firm(s) to help us raise awareness of the need for more treatment options for patients with chordoma and garner greater visibility and support for the foundation's research portfolio.
Q: What are among your most significant research achievements?
- While we reviewed the literature in 2007 we postulated that a particular pathway could play a role in chordoma. This led to collaborations between multiple researchers across the globe and treatment with an existing drug that is currently being studied in a clinical trial.
- We identified all the existing cell lines in the literature and had them sent to Duke University, where Josh was working in the lab to discover his own cure. He revealed that in fact there was only one valid chordoma cell line that existed in the world and it was located in a freezer in Germany. Astonishingly it was not being used for new research since 2001. I obtained the legal rights for the Chordoma Foundation to conduct research on this cell line and subsequently we have distributed this to over 25 researchers internationally, sparking new and exciting research efforts.
- We initiated over 12 new research projects, including:
- cutting-edge robotic high-throughput drug screening project to determine if there are any existing FDA-approved drugs that may be effective for chordoma patients. We believe that repurposing existing drugs will be the fastest way to get new treatments into the clinic.
- funded an innovative genome project that will make chordoma one of the few cancers in the world that will have undergone state-of-the art genetic sequencing to identify new cancer-causing genes that can be used to develop new treatments for chordoma and other types of cancer
- funded research projects to develop model systems for chordoma that have served as seed money to allow researchers to obtain large grants from NIH and pharmaceutical companies
- funded the researchers who identified a gene associated with some of the cases of familial chordoma