Game-changing new capabilities in our in-house laboratory
A key purpose of the Chordoma Foundation has always been to give those of us affected by this disease a way to propel the search for better treatments at a speed that matches our personal sense of urgency.
For me, facing a recurrence this year brought that sense of urgency to a new level. Thankfully, over the past four months I’ve bounced back from a successful surgery, along the way experienced the joy of meeting our second child, and this week got the good news that my latest scan shows no evidence of disease (longer story here). There are really no words to describe how lucky and grateful I feel.
I’m ending this year with a heightened sense of hope for the future thanks to a number of encouraging milestones at the Chordoma Foundation. Here are some of the many reasons for optimism coming out of 2022:
- Three CF-supported clinical trials were completed testing the drugs pemetrexed, afatinib, and nivolumab; results should be forthcoming next year.
- Two new clinical trials opened to chordoma patients based on data generated through the Foundation’s Drug Screening Program: a Phase 1 trial testing an experimental drug called FHD-609 for patients with poorly differentiated chordoma, and a Phase 2 trial at MD Anderson testing the approved drug cetuximab.
- Our new Clinical Trial Assistance Program is helping to alleviate the financial burden of trial participation and allow trials to progress faster (eight of ten patients on the cetuximab trial have relied on it).
- Strong preclinical efficacy of two drugs studied in our Drug Screening Program was presented at the American Association of Cancer Research annual conference, possibly paving the way for future clinical trials.
- Landmark findings were reported by CF grantees and collaborators, including the most complete catalog of chordoma’s potential therapeutic targets to date, and foundational insights (here and here) into how chordoma interacts with the immune system.
- We made new investments to bring immunotherapy to chordoma including the validation of a new mouse model for testing immunotherapies, initiation of the first experiments in that model, and grants in partnership with the Cancer Research Institute to support two high-impact immunotherapy research projects.
- We launched Chordoma Foundation Labs — the first laboratory 100% dedicated to chordoma research.
Together, these advances foreshadow a coming expansion of the toolbox available for patients whose tumors can’t be controlled with surgery and radiation, and lead me to believe that patients like me have a real chance to outrun this disease.
Most significantly, having our own in-house laboratory is proving to be a game changer for the Foundation’s ability to bring urgency to the treatment development process. In the lab’s first year, we set up the ability to quickly, efficiently and reliably run a variety of important experiments in a panel of more than 20 chordoma cell lines — a capability that exists nowhere else in the world. Already, we’re seeing how this is enabling ideas to be pursued and questions to be answered with unprecedented speed.
As one example, in the span of a few weeks and for just a few thousand dollars we were able to answer a question posed by our Medical Advisory Board which was important for greenlighting a clinical trial concept. Previously, answering this question would have taken many months — if it happened at all. In another instance, our lab validated an unpursued hypothesis generated several years ago by one of our grantees, at last, opening the door to a company partnering with us to test one of their drugs in chordoma mouse models through our Drug Screening Program. And in collaboration with investigators at Massachusetts General Hospital, we’ve generated initial evidence that many chordomas could be particularly vulnerable to therapies that exploit a phenomenon known as replication stress — an insight which we’re immediately and vigorously building on with further studies.
But this is just the beginning for our lab. This month, we added a second scientist at the bench, and have begun building capabilities to test immunotherapies and emerging drugs targeting chordoma’s main Achilles’ heel, brachyury. Going forward, this is a platform that can scale to meet the growing demand from academic and industry collaborators, and nimbly pursue the clues emerging from various ongoing target discovery projects we’re supporting.
Thanks to this and other investments we’ve made in recent years, and the unfailing generosity of the many supporters who make our work possible, the coming year promises to bring even more cause for hope. In addition to scaling up the capacity of the lab, some of the Foundation’s top priorities in the year ahead include:
- Funding research to identify proteins on the chordoma cell surface that can serve as targets for a variety of powerful therapeutic approaches
- Partnering with companies to advance development of brachyury drugs
- Initiating clinical trials for several drugs that have shown compelling activity in cell and mouse models
- Building more capacity to increase the speed and efficiency of clinical research, including by expanding our Clinical Trial Assistance Program, and hiring our first Head of Clinical Research
- Continuing to expand our support and resources for patients and families across the world
Who knows how much more time I’ll have to run this race, but I feel really lucky to get to continue to stay in it for now, and I’m more fired up than ever for the work ahead.
I’m also emerging from this year with a profound new sense of appreciation: for the members of the Chordoma Foundation’s capable team who shined this year like never before (by the way, we’re hiring!), for the doctors and nurses who care for patients with this disease, for all the resources now available to survivors like me, and for the strength and comfort that comes from connections with others in this community. I cherished the return of our in-person Chordoma Community Conference this fall, and am eagerly looking forward to reconnecting with many friends and colleagues at our upcoming International Chordoma Community Conference and Research Workshops in Boston next summer.
In the meantime, I want to wholeheartedly thank everyone who is standing with us in this quest. Ultimately, you are the reason I have hope.
If you’re considering a year-end gift, there’s never been a higher impact time. Contributions this time of year make an outsized impact on our ability to hit the ground running on our 2023 priorities. Thanks for propelling our mission in 2023!