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 addresses a critical unmet need in relapsed and metastatic Ewing sarcoma, a rare and aggressive bone and soft tissue cancer primarily affecting children and young adults. Driven by an abnormal gene present in almost all patients and extremely difficult to target with current therapies, this disease has poor outcomes once it spreads.
CF-012 inhibits ETV6, a newly identified transcriptional dependency that is essential for tumour growth and metastasis. As a potential first-in-class, mechanistically precise inhibitor, CF-012 offers a novel strategy to disrupt tumour progression and metastasis, providing a promising therapeutic option where current treatments are limited and often highly toxic.
Research partner leads
John H. Bushweller
Professor of Molecular Physiology and Biological Physics
University of Virginia
Kimberley Stegmaier
Chair of the Department of Pediatric Oncology
Dana-Farber Cancer Institute
Miguel Rivera
Assistant Molecular Pathologist
Massachusetts General Hospital
C-Further project lead
Louise Tonkin
Group Leader
Cancer Research Horizons
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 present in all patients, which 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.
