US-based clinical-stage biotechnology company Oncternal Therapeutics has begun dosing patients in its Phase I clinical trial of TK216 to treat Ewing sarcoma.
Jointly developed by Oncternal and Georgetown University, TK216 is a small molecule that has been designed to inhibit the biological activity of ets-family transcription factor oncoproteins in a variety of tumour types, resulting in the stoppage of cancer cell growth and tumour formation.
In Ewing sarcoma, TK216 targets a single and well-characterised genetic mutation responsible for the disease and inhibits the downstream effects of the chimeric protein EWS-FLI1 transcription factor.
The open label, first-in-human Phase I trial will be undertaken to evaluate the safety and efficacy of TK216 when administered to patients with relapsed or refractory Ewing sarcoma.
Ewing sarcoma is a type of tumour appearing in bone or soft tissue triggering symptoms such as swelling and pain near the tumour.
Oncternal president and CEO James Breitmeyer said: “Ewing sarcoma is a very serious bone cancer affecting children and young adults, and the effects can be devastating.
“These patients have an urgent need for additional treatment options and TK216 represents the first potential new therapy for Ewing sarcoma in many decades.
“We are delighted that enrolment and dosing are now underway in this important clinical trial.”
Oncternal and Georgetown University are planning to conduct clinical studies of TK216 to explore its efficacy in treating glioblastoma, prostate cancer and leukaemia.
Image: MRI displays Ewing's sarcoma of the left hip. Photo: courtesy of National Cancer Institute.