CF-012
A potential first-in-class, ETV6 inhibitor for the treatment of Ewing sarcoma
In collaboration with:
Target:
ETV6
Modality:
Small molecule inhibitor
Indication:
Ewing sarcoma
CF-012 aims to address a critical unmet need in relapsed and metastatic Ewing sarcoma, a rare and aggressive bone and soft tissue cancer primarily affecting young adults. The disease is driven by an abnormal gene fusion found in patients’ tumours, which has proved extremely difficult to target with current therapies. As a result, outcomes remain poor once the cancer spreads.
CF-012 targets ETV6, a newly identified and critical transcriptional dependency of the fusion oncogene that defines the vast majority of Ewing Sarcoma, whose activity is essential for tumour growth and metastasis. Our aim is to develop a potential first-in-class, mechanistically precise inhibitor with a precise mechanism to disrupt tumour growth and metastasis in patients.
Research partner leads
John Bushweller, PhD
Professor of Molecular Physiology and Biological Physics
University of Virginia
Kimberly Stegmaier, MD
Chair of the Department of Pediatric Oncology
Dana-Farber Cancer Institute
Miguel Rivera, MD
Associate Professor of Pathology
Massachusetts General Hospital
C-Further project lead
Louise Tonkin, PhD
Group Leader
Cancer Research Horizons
It took a long while for Harry to recover from his treatments. An MRI revealed that the proton beam therapy he received has scarred his left kidney, and it is now functioning at only 10%. For us, and for many families, it is crucial to develop kinder cancer treatments for children, to make the side effects easier to manage, to make them less vulnerable to infections, and to reduce the long-term impact on vital organs such as the kidneys and the heart.
Lyndsay, Mother of Harry
Patient advocate
About Ewing Sarcoma
Ewing sarcoma is a rare but aggressive cancer of the bone and nearby soft tissues that mainly affects children and young adults.
Standard treatment with chemotherapy, surgery, and radiation has improved outcomes for patients whose disease is localised, with survival rates exceeding 70%. However, the disease remains devastating once it spreads or comes back.
Around 25% of patients have detectable metastases at diagnosis, and nearly all are thought to have hidden micrometastatic disease. This helps explain why five-year survival drops to under 30% in relapsed or metastatic cases, and is often closer to 13–20%.
A major clinical challenge is the underlying biology of the disease. Ewing sarcoma is driven by an abnormal gene fusion present in the tumours of patients with this disease, that is extremely difficult to block with existing drugs. This leaves children and young people with relapsed disease few effective and often highly toxic treatment options.
This project takes a unique approach. It targets ETV6, a helper protein that Ewing sarcoma cells depend on for growth and spread. ETV6 was once considered undruggable.
By using a novel inhibitor to block ETV6 function, this strategy aims to slow tumour growth and reduce metastasis, offering a more precise and potentially safer option where standard treatments fall short.
Upcoming deadline: 13 March 2026
We welcome expressions of interest at any time, but to be reviewed in this round, they must be submitted before the closing date at 23:59 GMT. Early submissions are encouraged.
