Over the past year, we’ve heard from more than a few partners, advisors, and supporters that it feels like the Foundation is starting to “fire on all cylinders.” We are feeling it too, and the progress is energizing. For example, our patient services are growing increasingly robust and utilization has increased by more than 50% in the past year. More research discoveries are moving from the lab into the clinic, unimpeded by barriers that once stood in the way of progress. And we’re now working with twelve drug companies to test or develop drugs for chordoma – a feat that would have been hard to imagine just a couple years ago.
When the Foundation started, the road to better treatments was littered with hurdles – from a significant lack of research resources to a sparse and underfunded scientific community – and patients faced a lonely and bewildering journey to get the care they needed. With your help, we’ve now knocked through many of these roadblocks and are heading into 2017 with our foot on the accelerator.
Several important milestones in 2016 contributed to this growing momentum in research and improved experience for chordoma patients:
Top research accomplishments
- Alleviated scarcity of disease models
The longstanding scarcity of cell lines and mouse models that once held back research is being overcome. This year, we surpassed our initial goal of developing 10 valid chordoma cell lines, launched the first chordoma xenograft repository, and initiated a pipeline of more than 30 new xenograft models in development. Perhaps most remarkably, our longtime collaborators at Duke University have developed the first genetically engineered mouse model of chordoma – mice that develop chordomas of their own.
- Revealed chordoma’s vulnerabilities
Our research partners at the Broad Institute of Harvard and MIT, Dana Farber Cancer Institute and Baylor College of Medicine used powerful technologies to systematically identify genetic dependencies – genes upon which chordoma depends on for survival – revealing several new potential drug targets.
- Launched chordoma Drug Screening Pipeline
This new program enables fast and cost-efficient evaluation of drugs in preclinical models of chordoma, greatly speeding the process of moving discoveries from the lab to the clinic.
- Expanded pipeline of clinical trials
Over the past two years we’ve put mechanisms in place to initiate and support clinical trials for highly promising treatments identified by our research partners. One clinical trial is currently underway, three are nearing launch, and planning for three more began this year.
- Strengthened the chordoma research community
Our fifth International Chordoma Research Workshop, held this summer in Boston, attracted 70 first time participants, including prominent researchers from the Broad Institute, Dana Farber, the NIH, MD Anderson, and as far away as Beijing. Notably, scientists from five biotech and pharmaceutical companies were represented. In total, the field of chordoma research has now grown to more than 300 investigators across the world.
Top patient services accomplishments
- Developed first Expert Recommendations guidebook
This year we released the first plain-language booklet for patients and caregivers that summarizes best practices for the diagnosis and treatment of chordoma based on recommendations published in The Lancet Oncology by an international group of more than 40 leading chordoma experts. It is currently available in English, Spanish, Dutch, Italian, and a Mandarin translation is on the way.
- Launched Expert Answers Video Series
Now patients can get straightforward answers to common questions about chordoma directly from leading physicians. This year we released the first six videos in this series and have plans for an additional fifteen videos to come.
- Enhanced our Patient Navigation Service
This year we provided one-on-one assistance to more than 450 patients and families in more than 40 countries, helping them overcome the medical, emotional, and practical challenges brought on by chordoma. We also made important process improvements to be able to provide more in-depth and valuable assistance, and to handle growing demand.
To continue increasing the Foundation’s impact, this year we added several new team members to our ranks with the expertise needed to take on the opportunities that lie ahead. They include:
- Five new members of our Board of Directors with expertise in law, venture capital, drug discovery, cancer research, and medical philanthropy.
- A Community Advisory Board which formalizes the mechanism by which patients and family members provide input and direction to the Foundation, and complements our Medical and Scientific Advisory Boards in steering the Foundation’s activities.
- A talented Development team that is here to give everyone who shares our desire to bring about a brighter future for those affected by chordoma a way to get involved.
- AND – we’ve begun searching for a Director of Research to head our research programs and oversee our growing portfolio of research investments. (Interested in this position? Click here to apply or learn more.)
As our team continues to grow and professionalize, we are setting our sights higher and making plans to invest significantly more in Research and Patient Services in the years ahead.
Looking ahead to 2017
2017 will be a year of doubling down on our programs that serve the patient community (e.g. our Patient Navigation Service) and catalyze research (e.g. our model repositories and Drug Screening Pipeline), while also pushing boldly into new frontiers of research. We have ambitious plans for 2017 across all areas of our work, with a focus on five leading initiatives:
- Clinical trials
Over the next 12-18 months, we will work to provide patients who have advanced disease with six new clinical trials endorsed by our Medical and Scientific Advisory Boards – bringing us more than halfway toward our goal of 10 new trials by 2020. These trials will test a variety of targeted therapies and immunotherapies that have shown promise in slowing or stopping the disease in the lab. (Look for details about these trials once they open on our Clinical Trials list.)
- Brachyury drug discovery
We will take aim at the key driver and chief vulnerability of chordoma by beginning a concerted, multi-year effort to develop drugs that target brachyury. This will include making investments in multiple technologies, including: small molecule inhibitors, therapeutic vaccines, antisense drugs, and others.
- Leveraging advances in cancer immunotherapy
Immunotherapy is showing tremendous promise in other cancers, and it seems that each month a new immunotherapy success story makes the news. To position chordoma to benefit from these advances, we need to better understand how chordoma interacts with and evades the immune system. To do so, in 2017 we plan to release a request for proposals and aim to fund several grants to support projects in top cancer immunology labs.
- Better educational resources
In the coming year, we will round out our library of educational resources designed to help patients find appropriate care and make informed treatment decisions. Coming soon: new stage-specific guides to managing chordoma, a second Expert Recommendations booklet focused on managing recurrence, translations of existing resources into more languages, a new set of videos in the Expert Answers Video Series, user-friendly website updates, and more.
- Organizational growth
As our annual investments in research approach $2M and we embark on new clinical trials and drug discovery efforts, we are eagerly looking forward to hiring the Foundation’s first Director of Research to head our growing research program. Additionally, to fulfill our ongoing commitment to meet the needs of the patient community, we will add an Education and Outreach Coordinator to our Patient Services team and plan to launch a new Patient Services Committee of our Board. Finally, our development team will begin putting plans in motion to double the Foundation’s annual budget from ~$2.5M to $5M over the next several years.
As I reflect back on this most incredible year, and look forward to what lies ahead, the feeling I am left with is one of great appreciation. The support, involvement, and generosity of many friends and partners has brought us to this pivotal moment, and made it possible for patients to begin benefitting from advances in research in a matter of years. From all of us at the Foundation: thank you for helping us create a brighter future for everyone affected by chordoma. We wish you a healthy and prosperous 2017 and look forward to continuing to make great progress together.