Positive clinical trial results
A trial we supported at St. John’s Cancer Institute suggests that the chemotherapy pemetrexed is effective in some patients.
Onward and upward
A letter from our Executive Director, Josh Sommer
In 2023, a critical mass of scientific advances, emerging technologies, and research partnerships, coupled with new capabilities provided by Chordoma Foundation Labs, converged to provide unprecedented opportunities to advance chordoma research. And this community stepped up in a major way to seize those opportunities, contributing more than $7M to propel our shared mission and fuel even greater progress to come!
Notably, that included the largest single contribution to chordoma research yet: a landmark $4M gift from Oaz Nir motivated by his mother’s experience with chordoma and a desire to maximize the chances of finding effective treatments in time for those currently facing this disease. Paired with the generous support of others, this catalytic investment is bringing a new level of speed and scale to our quest for better treatments.
As you’ll read in this report, 2023 marked the beginning of that inflection with record strides advancing clinical trials, testing promising therapeutic concepts in the lab, supporting innovative research projects to identify the next wave of potential therapeutic approaches, and more. But it is truly just the beginning, setting the stage for us to aim higher and move even faster in the years ahead.
With a heightened sense of urgency following my recurrence in 2022, there are no words to adequately describe the optimism the developments of the past year have provided, nor the gratitude I feel to all those who made them possible.
One day individuals affected by this disease will no longer have their lives threatened by it, and, when that happens, it will be because people like you believed that this is a solvable problem and did what you could to solve it.
Until that day comes, I look forward to forging ahead together to ensure everyone in our community, no matter where they live or what their circumstances, gets the support and care they need and has ever more reason for hope.
Onward!
Josh
Dedication
As we forge ahead, we remember those whose lives were cut short by chordoma, knowing that their experience and their contributions to our community are forever embedded in the improving outlook for those who face this disease.
We dedicate this year’s annual report to our longtime friend and supporter Rodolfo Roye who passed away at the end of 2023 after a 25-year fight with this disease. Over the past ten years, Rodolfo and the Roye Family invested substantially in research that was instrumental to uncovering new therapeutic targets, building our Drug Screening Program, and supporting a number of clinical trials that are now bearing fruit.
We will always appreciate Rodolfo’s wholehearted support of our mission, his constant encouragement, and the outsized role he and his family have played in bringing about the advances from which we now derive so much hope.
(Photo: Rodolfo Roye [R] with wife Lourdes and Stuart Schreiber, Ph.D, Broad Institute of MIT and Harvard)
Research advances | 2023
Progress across the R&D continuum
2023 saw significant research advances with the potential to improve treatment options on multiple time horizons.
For patients needing better treatments in the near term, we advanced a record 8 clinical trials testing existing drugs approved or in development for other cancers. In parallel, we focused on filling the pipeline of future therapies by testing 13 more promising drugs in the lab to generate data needed to justify new clinical trials, and supporting 14 basic and translational research projects with the potential to reveal new, more personalized therapeutic options.
This year we also continued to set the stage for more research progress through additions to our research enabling-infrastructure, like the first mouse model of sacral chordoma, a tool that enables scientists to study 95 tumors from our Biobank in parallel, and more publicly available data.
Together, these efforts, along with previous research investments, culminated in results shared in 9 peer-reviewed publications and posters.
(Photo: NYU researchers Drs. David Zagzag, Chandra Sen, Kristyn Galbraith and Matija Snuderl, along with Josh Sommer, and Northwestern researcher Dr. Catalina Lee-Chang)
Research advances | 2023
Clinical research milestones
Most notable on the clinical research front were results from the pemetrexed clinical trial we supported: Of the 14 patients on the trial, 10 had their tumors stabilize or shrink, and the majority experienced symptomatic improvements — the best result of any therapy tested to date.
Building on the encouraging findings in this small trial, we began studies in Chordoma Foundation Labs to identify drugs that cans synergize with pemetrexed, and we awarded a nearly $600K grant to St. John’s Cancer Institute to enable a larger phase II trial testing pemetrexed in combination with the immune checkpoint inhibitor pembrolizumab (Keytruda).
Additionally, preliminary results from the ongoing cetuximab trial we’re supporting were presented at this year’s International Chordoma Research Workshop, indicating that a number of patients are benefitting from this therapy. Based on several responses in the first stage of the trial, we provided funding to expand the trial to a total of 29 patients.
Rapid accrual and expansion of the cetuximab trial was facilitated by our new Clinical Trial Assistance Program, which reduced the financial burden of trial participation for 11 patients in 2023. Funding permitting, we hope to be able to offer this program to additional patients in future trials.
Finally, based on data generated through Chordoma Foundation Labs and our Drug Screening Program, five new trials opened to chordoma patients in 2023 and planning advanced for three future trials.
(Photo: Clinical trial participant Tom Giarratana and his daughter Lily)
Testimonial
Research advances | 2023
Chordoma Foundation Labs
Chordoma Foundation Labs (CF Labs) continues to bring a new level of speed and efficiency to the treatment development process. This year, our scientists uncovered new insights about drugs (and drug combinations) that may be effective against chordoma, and how these treatment concepts could be applied in a personalized way.
In one notable example, a research collaborator hypothesized that a phenomenon called replication stress could be exploited in chordoma and turned to CF Labs to quickly explore it. Within months, we confirmed that drugs targeting replication stress are highly active in chordoma cells and effective at stabilizing or shrinking chordoma tumors in mice. As a result, our Medical Advisory Board endorsed two relevant concepts for clinical testing.
In another important project, we identified a possible biomarker of resistance to a class of drugs called EGFR inhibitors, which are demonstrating antitumor activity in some chordoma patients in the ongoing cetuximab trial and recently completed afatinib trial. Such a predictive biomarker could help identify the patients most likely to benefit from this widely used class of drugs, while sparing other patients from a treatment unlikely to work for them.
Results from both of these projects were shared in posters at the American Association of Cancer Research (AACR) annual meeting— the largest cancer science conference in the world — and were posted to our public data repository to enable other researchers to rapidly build on our findings.
(Photo: CF Labs’ Director Dan Freed, PhD, at our International Chordoma Research Workshop)
Research advances | 2023
Brachyury drug discovery
Brachyury is thought to be chordoma’s greatest vulnerability, and discovering drugs to target it continued to be a top priority. This year, we developed four new testing capabilities in CF Labs to evaluate brachyury-targeting compounds and enable researchers and companies to more easily pursue brachyury drug development. In parallel, we launched our first internal drug discovery program aiming to create compounds that could be incorporated into an emerging class of drugs called protein degraders to home in on and destroy brachyury in cells.
(Photo: colonies of bacteria that are engineered to produce brachyury in CF Labs)
Top research stories
We greatly appreciate our grantees, collaborators, and all of the researchers worldwide whose energy and dedication are changing the treatment landscape for chordoma.
Here are some of the most notable stories resulting from our work together in 2023.
Six new grants awarded
Our investments will open the door to the next wave of treatment options.
A test to guide personalized care
With our support, a new multinational consortium is studying whether a new test can predict which patients are likely to benefit from radiation after surgery.
Identifying potential therapeutic options
Working with partners at Massachusetts General Hospital and Duke University, CF Labs scientists uncovered two new drug classes with great potential for chordoma.
Building community to drive progress
In July, we hosted our latest International Chordoma Research Workshop and Community Conference in Boston, which brought together nearly 200 researchers, clinicians, and patients and family members from around the world. Both events were deeply energizing, and a testament to how special this community is. Check out the recap here.
A particular highlight was our Uncommon Awards ceremony, an uplifting tradition recognizing community members who’ve made remarkable contributions to our mission in a wide variety of ways.
Testimonial
Education and resources | 2023
Helping patients get the best outcomes
This year we continued to serve as a trusted resource for chordoma patients and caregivers, bringing expertise, connection, and hope to what could otherwise be a bewildering and lonely journey.
Our Patient Navigators provided free, personalized guidance to 577 patients and caregivers — our largest annual case volume to date, and a nearly 30% increase in cases served over 2022.
We also vetted and added nearly three dozen new chordoma experts from across the world to our Doctor Directory.
(Photo: Marie Perillo with husband and patient Jack Dworkin; both are also members of our Community Advisory Board)
Education and resources | 2023
Connecting the chordoma community
Chordoma Connections, our online community, is a vibrant, private forum for chordoma patients and family members to connect with one another 24/7. In 2023 we re-launched Chordoma Connections on a more modern and user-friendly platform, featuring a member directory, direct messaging and chat, discussion threads, and more. We also made the platform available as a mobile app (download: Apple or Google Play).
Additionally, we added four new trained Peer Guides to our Peer Connect program, in which patients and caregivers can sign up to be matched with someone whose experiences with chordoma are similar.
Education and resources | 2023
Expanding our support globally
Our ultimate goal is to provide support for every individual impacted by chordoma regardless of geography, and in 2023 we made big strides in that direction. For example, we launched our website in six additional languages and debuted our Chordoma Community Map, including 4,122 patients worldwide. Additionally, our new Chordoma Connections platform introduced dedicated discussion groups for members in six countries outside the US, with India being the latest addition. And we welcomed three new volunteer Ambassadors: Misty Bland from Australia, Wally van Laarhoven from the Netherlands, and Carolina Herzfeld from Germany.
As a result, we've significantly increased our global reach: over half of new visitors to our website and almost half of all Patient Navigation cases now originate from outside the US.
(Photo: Program Manager Kimberley de Haseth and German Ambassador Irene Badura)
Our Team | 2023
Increased capacity to drive progress
To proactively address growing opportunities and needs in clinical research, this year we were thrilled to recruit Greg Cote, MD, PhD, one of the world’s leading chordoma medical oncologists, as our Lead Clinical Research Advisor. In this new part-time role, Dr. Cote will lead our worldwide efforts to streamline and accelerate clinical development of new chordoma treatments.
Additionally, we welcomed Dani Pike, a chordoma survivor, as our first Development and Engagement Officer to help connect members of our community with opportunities to get involved in our mission.
We also added terrific new voices to our Community Advisory Board: Frankie Cordero, Allen Cragin, Jack Dworkin, Nancy Forte, Karen Hermanson, Marie Perillo, and Tyler Simmet.
Finally, we celebrated the extraordinary contributions of our longtime Director of Operations, Tammy Silverthorne, and Board members Ingemar Lanevi and Julie Song as they concluded their time on our team. Their many years of service, hard work, and wise counsel were instrumental in creating a strong footing for our ongoing growth and sustainability.
(Photo: Team members Dani Pike and Kaitlin Slepian)
2023 Donors
Thank you for powering our progress!
Thanks to the generous support of our community, 2023 marked a new high-water mark of $7.4M in funding for our mission. We sincerely appreciate every gift, and are in awe of what this small but mighty community is capable of achieving together. Expand each section below to view the many individuals and organizations whose gifts added up to make 2023 a year of significant, long-lasting progress.
(Special thanks to donors whose multi-year pledges make possible the long-term term investments that are crucial to the success of our mission. Consistent with our accrual-based accounting method, the full amount of multi-year pledges is recognized in the year in which the pledge is made. Pledge payments are denoted in subsequent-year annual reports with an * but do not contribute to revenue in that year.)
It is very important to us to acknowledge each gift accurately and in accordance with each donor's wishes. If we have made any errors or omissions in the lists above we apologize and we are eager to make them right. We welcome you to contact us at development@chordoma.org with any questions, comments or corrections.
Our IRS Form 990 and audited financial statements will be added to this report when finalized. Previous years’ 990s and financial statements can be found here.
(Photo: Patient Richard Westin with wife Karen [both center] and Chordoma Foundation Labs team members, whose work their family supported)
Taking Action | 2023
A new level of giving, a new level of speed
After supporting his mother through her diagnosis and treatment, Oaz Nir wanted to ensure that his mom, and chordoma patients everywhere, had access to more effective treatments as quickly as possible. “As I learned about the Chordoma Foundation, it became clear that their work was catalyzing research progress faster than the norm, and that with additional resources there was an opportunity to get urgently needed therapies to patients even more quickly," Oaz says.
Based on this realization, Oaz committed an extraordinary $4M to help boost our community’s collective efforts to a new level. These funds, alongside the contributions of others, are allowing us to pursue many of the most promising current research opportunities on an accelerated timeline, while also opening the door to a vast array of new opportunities, and laying the groundwork for even more significant progress in the near future.
We are inspired by this transformative gift to aim higher and move faster, and we can’t wait to partner with all who are similarly motivated.
Testimonial
Taking action | 2023
Paying it forward
When Zach Martin was diagnosed with sacral chordoma in 2021, he reached out to our Patient Navigators for guidance. They connected him with a Peer Guide: Susan Hall, a fellow sacral chordoma survivor. Susan provided Zach with valuable insights about what to expect during his treatment journey and offered much-needed empathy during a difficult period in his life.
Following his surgery, Zach was inspired to give back to the chordoma community. He completed our training to become a volunteer Peer Guide, eager to offer the same support to others that Susan had given him.
Zach was also motivated to help improve treatment options for chordoma. Recognizing this need, he and his wife, Lauren, have made significant contributions to our Clinical Trial Assistance Program. This program not only facilitates chordoma patients' access to clinical trials but also contributes to the trials' success, benefiting future patients.
“Our family is thankful for the Chordoma Foundation’s research, information, and the personal connections that not only have helped us but so many others,” Zach says. Heartfelt thanks to the Martin family for their generosity.
(Photo: Zach Martin and family)
Community fundraisers
Individuals hosting online and in-person fundraisers play a crucial role in advancing our mission to develop new therapies and provide essential services for those navigating this disease.
Stephanie Finley, a long-time supporter and survivor whose family has raised over $125,000, says, “Our annual fundraiser is a chance to share my chordoma story with my friends and family, and give them the opportunity to help ensure that chordoma patients like me will have better treatments available in the future."
Here are just a few examples of the creative events organized by our community this year. We're sincerely thankful for every fundraiser, whose eagerness to enlist the generosity of their networks is making a difference for everyone affected by chordoma.
Chloe Fights Chordoma
Drinks, dancing, and dogs! The Finley family’s creative annual birthday bash for their pup, Chloe, raised $60,000 thanks to the generous support of party attendees.
#MissionMatteo
In memory of their son Matteo, who passed away from chordoma, the DiFolco family hosted their inaugural #MissionMatteo Charity Golf Classic to support pediatric chordoma research.
One-in-a-Million Party
Chordoma survivor Mick Potempa and his wife, Noreen, recently celebrated their 50th wedding anniversary. In lieu of gifts, they requested donations toward better chordoma treatments, bringing their lifetime fundraising total over $375K.
Pub Crawl for Cameron
This spirited tradition hosted by the Riccomini family raised a record amount in its 11th year running. The event is focused on moving the needle for pediatric chordoma research in memory of Cameron Riccomini.
Sloane Swanton Research 5K
After losing their young daughter, Sloane, to chordoma, the Swanton family has undertaken several critical fundraising efforts. In 2023, hundreds of people in Ohio laced up their sneakers to raise over $25K for pediatric chordoma research.
Steppin' Out for Chordoma
John Mainey has been hosting events in memory of his son, Chuck, for many years. This year he walked 71,000 steps in a single day, raising awareness and funds for chordoma treatment development.
Swing Fore the Cure
Maggie and Jon Bates united their friends and family for a day of camaraderie on the golf course, continuing a decade-long tradition started by their friend and late chordoma patient Jeff Schilling.
Team Chordoma
Chordoma survivor Steven Mandel’s beloved and growing race tradition has raised over $450,000 to date. This year’s Team Chordoma fundraisers also included Frankie and Marea Cordero, Andrew Davidson, Jack Dworkin, Alex Olsson, Marie Perillo, and Matt Sauerhoff.
Virtual fundraisers
People like Irene Badura, Sharon Berlan, Frankie Cordero, Beth Cullinan, Jessica Horton (pictured), Tim McCormack, and many more hosted online fundraisers — the easiest way to raise funds for cures.
Testimonial
Taking action | 2023
Every gift accelerates cures
A new wave of chordoma therapies is on the horizon: treatments that could shrink tumors, prevent recurrence, and preserve patients’ quality of life.
Your gift today will enable us to take the immediate next steps toward those treatments: like conducting additional experiments in our lab, initiating new clinical trials, expanding our Clinical Trial Assistance Program, and more.
Together, our continued investments could dramatically change the outlook for this disease in our own lifetimes.
(Photo: Mick and Noreen Potempa)
Testimonial