Earlier this year, we launched our own research lab to bring increased speed, efficiency, and nimbleness to chordoma research. This is the story behind the new lab and our vision for it.
The Chordoma Foundation has always been driven by a deep sense of urgency — urgency to find treatments that not only help patients in the future, but that also offer hope to those of us currently affected by this disease.
That sense of urgency motivates us to continually pursue ways to make research move faster.
Naturally, funding is critical to making research go. But so too, we’ve learned, are a host of other factors: a strong research community, rapid data sharing, access to scientific resources like tumor tissue and models, and patient participation in research, to name a few.
Putting all of those things in place over the past fifteen years has sparked a surge in chordoma research — hundreds of researchers have joined the field, many potential treatment approaches have been identified, seven of which have moved into clinical trials, and the stream of new discoveries which could lead to even better treatment options is steadily growing.
But, as encouraging as all this is, the rate at which these discoveries are translated into better treatments is still too slow to match the urgent needs of patients.
A big reason is the way biomedical research is currently organized.
For example, companies and scientists with the specialized expertise or technologies critical to advancing new cancer treatments seldom start with any experience doing chordoma research. For many, taking on a chordoma project can be a heavy lift, if it’s feasible at all. It can be expensive. There’s a learning curve. There are competing priorities. Sometimes they don’t have the necessary facilities. Even for established chordoma researchers — and, thankfully, there are now quite a few — certain critical experiments are slow to start up, inefficient when not performed routinely, or sometimes just don’t line up with their areas of expertise or interest.
In this configuration, no matter how much is invested, there’s a structural limit to how fast research can move, just as there’s a limit to how fast an engine can go. At a certain point, to go faster, you need a faster engine — and that’s what we’re determined to build for chordoma research.
As a first step, seven years ago, we set out to create a faster and more economical mechanism for testing promising therapies in chordoma mouse models — experiments that are needed to justify, prioritize, and rationally design human clinical trials, but which are often prohibitively expensive or difficult for individual researchers to perform. Through the creation of our Drug Screening Program, which performs these experiments on behalf of various collaborators within a specialized contract lab, we’ve been able to test more than 70 drugs, compared to just a couple that had been tested previously, and far more than have been tested for most rare cancers.
The impact has been massive, with results providing justification for five ongoing clinical trials (plus at least two more that are being planned) and attracting investments by pharmaceutical companies in these trials worth millions of dollars.
Yet gaps in the current research ecosystem continue to stymie translation of discoveries into clinical trials.
Seeing a need and an opportunity to bring similar increases in nimbleness and efficiency to other areas along the chordoma R&D continuum, early this year, we launched CF Labs: the first lab 100% dedicated to chordoma research. To our knowledge, it’s also the first lab of its kind for any form of cancer. But the idea is not completely without precedent. Our aim is to follow in the footsteps of the Cystic Fibrosis Foundation Therapeutics Lab and the ALS Therapeutic Development Institute, both of which have played a catalytic role in advancing multiple treatments for their respective diseases.
Located at BioLabs, a fully equipped life science research facility in Durham, North Carolina, CF Labs specializes in a variety of critical experiments in chordoma cells — from validating new therapeutic targets, to determining the effects of various treatments on cell behavior, to identifying ways in which cells become resistant to certain drugs. This specialization produces economies of scale, which drives down the time, cost, and difficulty of conducting experiments relative to most individual academic or industry labs. Over time, it will also enable the accumulation of deep expertise and knowhow, which we expect will increase the quality, reliability, and reproducibility of resulting data, especially compared to labs that don’t routinely perform the same experiments in chordoma models.
The idea is to bridge the gaps between the capabilities and core strengths of academic and industry partners, making it more feasible for them to apply their specialized biology, technology, and therapeutic expertise to this disease.
In the past few months, the lab has gotten off to a great start under the leadership of our Head of Target Discovery and Translational Research, Dr. Dan Freed, who draws on more than fifteen years of basic and translational cancer research and drug development experience across academia and industry. Already, we’ve hired our first full-time lab scientist, imported 21 chordoma cell lines, established a number of experimental capabilities, and began work on our first three projects, none of which were feasible prior to CF Labs.
One involves testing a hypothesis about biomarkers that predict sensitivity to a certain class of drugs on behalf of Dr. Greg Cote, a Massachusetts General Hospital oncologist and CF Medical Advisory Board member. Another is validating a potential vulnerability identified by one of our grantees as an inducement for a biotech company developing drugs against that pathway. And the third is an internally-driven project to identify what causes certain chordomas to be sensitive or resistant to afatinib and other EGFR inhibitors. We are planning to begin two additional projects shortly, and already more are queuing up.
Nindo Punturi and Dr. Dan Freed at CF Labs
Initially, CF Labs’ central focus is on accelerating repurposing of existing drugs for chordoma — drugs that are already approved for other diseases or that have advanced to the point of human clinical trials. But this is just the beginning. The next phase for the lab will be to establish capabilities that are needed to streamline the development of new drugs targeting chordoma’s main Achilles’ heel, brachyury. From there, we aim to continue scaling up the capacity of the lab to address the most critical questions and needs across the full spectrum of preclinical research and therapeutic development. Ultimately, our vision is to configure the lab as an engine for discovery limited in speed not by logistical factors but solely by biology and the resources to make it go.
This is the chordoma community’s lab and it gives those of us affected by the disease much greater power than we’ve ever had before to influence the search for the cures we desire — and, potentially, our own futures. For me, there is no greater source of hope right now.
Our team at the Foundation looks forward to partnering with researchers, companies, and all those who share our fervent desire for faster progress towards cures to make the most of the lab’s catalytic potential. We hope you’ll join us in this endeavor and we look forward to providing opportunities to follow the progress as this new research engine revs up.
To discuss research collaborations, contact Dan Freed (firstname.lastname@example.org). To help propel the lab’s work through philanthropic support, contact Kenny Brighton (email@example.com) or contribute directly here. Media inquiries can be directed to Sara Nick (firstname.lastname@example.org).
We recently held a webinar about CF Labs, including more about the research currently ongoing and the opportunities that lie ahead. You can view it here.