As 2020 comes to an end, I am both hopeful for better days ahead, and, at the same time, deeply grateful for the gains this community has made together in our mission, particularly against the backdrop of these uniquely challenging times. From patients having responses on four ongoing CF-supported clinical trials, to big strides in the quest to develop the first drugs against chordoma’s Achilles’ heel, brachyury, to the launch of our Chordoma Survivorship Initiative, it has actually turned out to be a year of truly encouraging progress (see key accomplishments of 2020 below).
Amidst all of the uncertainty in the world, to me, these successes provide important reminders of things that we can bank on: the ability of good science to combat disease; the incredible power of human ingenuity to solve the seemingly unsolvable; and the strength that comes from a community united in a common purpose.
Thank you for being a part of this community.
Wishing you health and happiness this holiday season and in the year ahead!
Donations to our ALL IN campaign to enabled significant and tangible progress this year in each of our five current focus areas.
Brachyury drug discovery
Developing drugs that strike at the Achilles’ heel of chordoma
Made significant chemistry advances toward compounds that bind to brachury with drug-like strength and precision
Four drug companies now actively exploring the application of their technologies to brachyury
Created open access research tools to accelerate all brachyury drug discovery projects
Applying powerful new technologies to harness the immune system to fight chordoma
New markers found on the surface of chordoma cells which could serve as targets for immunotherapy
Initiated and funded research to (1) identify new ways to break down chordoma’s defenses against immune attack and (2) apply an emerging form of immunotherapy, which works by boosting a patient’s own cancer-killing white blood cells
Systematically identifying existing drugs that can help chordoma patients in the near term
Responses (tumor shrinkage or stabilization) seen in some patients on 4 ongoing clinical trials – afatinib, nivolumab, palbociclib, and pemetrexed
Tested 18 drugs and combinations in mouse models through our Drug Screening Program, several of which significantly inhibited tumor growth
Drug supply and partial funding secured for a planned trial of cetuximab
Improving the patient experience
Helping patients and their families navigate life with chordoma and get the best care possible
Launched the Chordoma Survivorship Initiative to holistically address the needs of those living with, through, and beyond chordoma
Created a Survivorship Specialist Directory to help those affected by chordoma find care to manage quality of life challenges
Revamped and expanded our Peer Connect Program so that more patients, survivors, and caregivers can connect one-on-one with others who have walked in their shoes
Learning from each patient
Understanding how different treatments impact patient outcomes to continually improve patient care
Partnered with the National Cancer Institute on a natural history study to better understand the course of the disease and improve treatment options
Created a blueprint for a definitive study on the benefits and best use of radiation for treating chordoma with leading treatment centers (start pending funding)