On July 16, more than 120 patients and caregivers from five countries and 21 states gathered in Boston for the 2016 International Chordoma Community Conference, with over 100 more watching the proceedings remotely via live stream. Throughout the day, 20 speakers presented on topics ranging from the latest advances in chordoma research to the status of new therapy development to the most up-to-date support services available to patients and their loved ones. Hope and perseverance were the mantras of the day, and helping patients and family members become as informed as possible about their disease and how to manage it was the anchor of every discussion.
Heather Lee, one of CF’s founding board members and recipient of the 2016 Uncommon Commitment Award, welcomed participants to the conference, noting what an exciting moment this is for chordoma science, and how far we have progressed since the community first gathered together in 2008. Back then, “we had no basic science to speak of, and the few researchers that were working on chordoma were doing it in silos,” she recalled. Heather also pointed out that while the Foundation knew it wanted to focus on both advancing research and supporting the patient community, the infrastructure to do that effectively didn’t exist yet.
Fast forward eight years, and we now have a much greater understanding of the biology of chordoma, a wealth of new disease models, several clinical trials preparing to launch, a vibrant community of researchers and specialists at top institutions around the world, and patient services that have helped thousands across the globe. “This is a movement,” said Heather. “It’s going places. It’s on a rocket ship. And it’s up to all of us to be part of the solution. If not us, who?”
Patient services and research
Chordoma Foundation staff members Shannon Lozinsky (Patient Services Manager) and Josh Sommer (Executive Director) provided an overview of the Foundation’s patient services and research programs, highlighting milestones from the past year and offering insights into what lies ahead.
“Improving life for chordoma patients and their families has always been a part of the Foundation’s mission,” said Shannon. “It has led to the creation of resources like the peer connect program, the doctor directory, and these community conferences. But the patient services program really started taking off in April of last year.”
Since then, she noted, the Foundation has:
- Developed Expert Recommendations for the Diagnosis and Treatment of Chordoma, an easy to understand booklet adapted from consensus guidelines published in the Lancet Oncology in 2015;
- Added 50 new profiles to the Doctor Directory and launched a more interactive, mobile friendly version of that tool;
- Formed the inaugural Community Advisory Board, to keep the perspective and needs of the patient community front and center in the Foundation’s work; and
- Served more than 450 chordoma patients and their families all around the world through the newly launched Patient Navigation Service, which offers one-on-one assistance to patients and caregivers wherever they are in their journey, offering resources, support, and information to help them overcome any barriers in the way of getting the best care possible.
On the other side of the Foundation’s dual mission is the goal of advancing research toward new therapies and cures. “We take a proactive approach to driving research forward so we can get the whole job done,” said Josh. “It’s not enough just to have interested researchers. To find treatments for any disease requires a lot of research to be done across a lot of different domains. Our research roadmap, created in partnership with our Scientific and Medical Advisory boards, was designed to help ensure that if we’re taking a shot on goal, we’re taking the right shot on goal with the greatest chance of success.”
Josh went on to walk participants through the five steps of the research roadmap – 1) resource development, 2) target discovery, 3) therapeutic discovery, 4) preclinical research, and 5) clinical research – and explain how the Foundation is optimizing its scientific and financial resources to accelerate the pace of progress across the entire research continuum.
- Exceeding the goal of creating 10 validated cell lines (tumor cells that grow in a petri dish), and making them available to over 100 labs and companies across the world, leveraging millions of dollars in research funding to focus on chordoma.
- Supporting the development and validation of 5 xenograft models (human tumors that grow in mice), with 22 more currently in varying stages of development.
- Participating in the creation of the very first engineered mouse model for chordoma (a recapitulation of the human disease in a mouse).
- Supporting research in over 20 labs around the world to assess the vulnerabilities of chordoma, and determine how best to attack it, leading to the identification of over a dozen targets which are being tested against existing compounds.
- Creating the first drug screening pipeline for chordoma, a centralized facility where drug screening can be done in mouse models at a fraction of the cost and time of an individual lab, and testing 12 drugs and drug combinations in the first year, with the capacity to test up to 15 drugs each year moving forward.
- Holding the 5th and largest International Chordoma Research Workshop, increasing the number of collaborations and density of our research network, and allowing information to flow more freely between labs.
- Funding/supporting three new clinical trials set to launch in the next six months.
Following Josh’s presentation, Dr. Adrienne Flanagan of University College London gave an update on recent advances in our understanding of chordoma. She explained how evidence has accumulated from her lab and others that brachyury is a key driver and prime vulnerability of the disease. Highlighting a paper she and her colleagues published in the Journal of Pathology last month, she also explained how chordoma cells appear to have a particular sensitivity to compounds that inhibit a protein called epidermal growth factor receptors (EGFR), and how that finding led to a clinical trial starting later this year.
Additionally, Dr. Tanaz Sharifnia of the Broad Institute updated participants on the Chordoma Target Discovery Project – a multi-institution collaboration spearheaded by the lab of Dr. Stuart Schreiber to identify new ways to treat chordoma. Employing three powerful technologies, the Schreiber lab and colleagues are systematically identifying vulnerabilities in chordoma which could point to new treatment possibilities. “There is a defined path forward that we’re traveling along to identify new targets for chordoma,” she said, emphasizing that her “best allies come in the form of patients.” To underscore this point, she told the story of Brittany and Todd Fuchs who have not only generously funded her work, but have actually come to work in her lab.
Immunotherapy, personalized medicine, and clinical trials
Dr. Chris Heery of the National Cancer Institute provided participants with
an introductory lesson on immunotherapy, how it works, and what the implications for chordoma appear to be. He noted that while immunotherapy is showing promise in melanoma and other cancers, research is still underway to determine its applicability to chordoma. “We’re still trying to understand immunogenic cell death and modulation in chordoma,” he noted. “We know brachyury is highly expressed in chordoma tumors, so studies are currently testing if a yeast-based brachyury vaccine will fight chordoma cancer cells.”
Dr. Greg Cote of Massachusetts General Hospital then walked participants through the three phases of clinical trials, and explained how to decide which trials are right for which patients. In short, trials at each phase are intended to answer a different question:
- Phase 1 – Is it safe?
- Phase 2 – Is there any sign of activity?
- Phase 3 – Does it work for sure?
When considering participation in a trial, he encouraged patients to make sure they know the subtype of their tumor, its rate of growth, and prior therapies that have been attempted, to help assess eligibility to different studies.
He also spoke about the potential of personalized tumor profiling to identify new treatment options, a topic which has received a lot of attention in the oncology field, but he urged caution. Tumor profiling, he explained, is promising in theory, but has limitations, and the utility in chordoma remains unknown. Currently, information gained from tumor profiling is really only relevant for patients with advanced disease – for them results could point to a potential off label treatment or a relevant clinical trial.
Dr. Silvia Stacchioti of Instituto Nazionale Dei Tumori in Milan explained recently released expert recommendations for diagnosing and treating chordoma which she developed along with 40 leading doctors from 12 countries who specialize in caring for chordoma patients. Published in 2015 in the Lancet Oncology, it represents the first in what will become a series of efforts by the Foundation and the European Society for Medical Oncology to create consensus guidelines for chordoma care, with the next focus being on treatment for advanced and metastatic disease.
Ask the experts
Moderated by chordoma survivor, Dr. Ed Les, a physician and former Board member of the Foundation, this 90-minute session provided an opportunity for patients and family members to ask pressing questions of a panel of renowned chordoma experts representing each of the primary specialties involved in treating chordoma. Questions covered topics ranging from diagnosis to treatment to follow up.
The panel included:
- Greg Cote, MD, PhD – Medical Oncology
- Tom DeLaney, MD – Radiation Oncology
- Paul Gardner, MD – Neurosurgery
- Chris Heery, MD – Medical Oncology
- Fran Hornicek, MD, PhD – Orthopedic Surgery
- Silvia Stacchiotti, MD – Medical Oncology
Several questions were submitted in advance via social media, and many more were taken live onsite. Here is a full video of that session (beginning at 1:38).
Being your own best advocate
Throughout the day, we heard from expert after expert – researchers, surgeons, nurses, care coordinators – all of whom noted that success in treating chordoma is dependent not just on using the right tools (surgery or radiation), but also on having an experienced care team who knows how to use those tools effectively for chordoma.
“Be prepared to advocate for yourself,” counseled Al Ferreira of
Massachusetts General Hospital. “Stand up for yourself and question your doctor if you need to. Ask until you understand what they’re talking about. Find out how many other chordoma patients they’ve treated and what their outcomes were. Learn the specifics of your tumor – location, size, rate of growth – and which of the four types of chordoma it is – conventional, chondroid, dedifferentiated, or poorly differentiated. And bring an advocate, someone who can listen and process the information on your behalf.”
Being persistent fighting for the care you need is crucial for chordoma patients. “Don’t be afraid to ask for a second opinion,” Al reminded participants. “This is a serious disease and it is important to understand all your options before deciding on a care team.”
Also, be aware of all of the support services available to patients and families. From the Foundation’s Patient Navigation Service and Peer Connect Program to the palliative care teams that work with most major hospitals. “A great misperception about palliative care is that it is just for the dying,” noted Dr. April Zehm of Massachusetts General Hospital. “The truth is, palliative care is a medical specialty for anyone facing serious illness, is appropriate at any stage of illness, and can be provided together with curative treatments.”
Having a support system in place to help manage not just the physical pain, but the emotional strain and medical decision making process is tremendously important for chordoma patients. As we heard time and time again, it’s not something anyone should have to go through alone.
Celebrating those who have made our progress possible
The exciting achievements of the past few years could not have happened without the involvement of an informed and engaged chordoma community. To have come so far in such a short time is a testament to the tireless dedication of researchers, volunteers, donors, and champions who have committed themselves to propelling the field forward.
At a dinner that bridged the Research Workshop and Community Conference, the Foundation recognized a group of individuals whose contributions have had an outsized impact on advancing chordoma science and care. The recipients of the Fourth Annual Uncommon Awards include:
- Uncommon Collaboration Award: Adrienne Flanagan, Susanne Scheipl, and Fabrice Turlais of the University College London; and David Drewry and Bill Zuercher of UNC Chapel Hill and the Structural Genomics Consortium
- Uncommon Action Award: Adriane Rothstein
- Uncommon Champion Award: Beth Cullinan
- Uncommon Commitment Award: Heather Lee
Read more about the 2016 award recipients
The next community conference will be held on Saturday, October 15 at the Mayo Clinic in Rochester, Minnesota. REGISTRATION IS NOW OPEN, and we hope to see you there.
Anyone interested in reviewing conference materials and photos from Boston can find them here: