IMiD's Department of Oncology has launched Project BUTTERFLY, a non-commercial clinical trial evaluating the efficacy of naxitamab. It is a novel treatment for Ewing's sarcoma. The aim of the study is to improve the diagnosis and treatment of children with refractory and recurrent forms of this cancer. Eligible for the program are patients between the ages of 2 and 21 for whom previous medical management has failed. The first patient - 12-year-old Jacob - received immunotherapy on October 25.
Groundbreaking clinical trial on novel treatment for Ewing's sarcoma at Mother and Child Institute
Published Nov. 8, 2023 08:29
Ewing's sarcoma is a malignant tumor, the second most common bone tumor in children and young adults. It mostly develops in bones, such as the femur, pelvis or ribs, but can also appear in soft tissues - such as muscle tissue. The peak incidence is between the ages of 15 and 19, although younger children are often affected. Unfortunately, more than half of patients also have metastatic lesions at the time of diagnosis. Therefore, it is important to diagnose quickly and implement appropriate treatment. However, about 20-30% of patients face treatment failure - in order to minimize this percentage, the Mother and Child Institute has launched Project BUTTERFLY, a study of a novel treatment method for Ewing's sarcoma patients.
Groundbreaking study
Project BUTTERFLY is one of the first immunotherapies in bone tumors and the first naxitamab therapy in Ewing's sarcoma worldwide. The trial aims to improve the diagnosis and treatment of patients with refractory and relapsed disease, as well as standardize and unify diagnostic and therapeutic approaches. It will also enable the creation of a biobank to store patients' biological samples. This, in turn, will help further the development of personalized therapy, tailored to the individual needs of each patient.
- Patients with Ewing's sarcoma with disease dissemination and negative prognostic factors have not had improved treatment outcomes for more than 30 years. Preliminary data show that in about 60-80% of patients, GD2 ganglioside is detected on the surface of tumor cells. The clinical efficacy of naxitamab in destroying them has been demonstrated in children and adolescents with neuroblastoma treated with the drug. In conjunction with other issues, this justifies undertaking a study of naxitamab in combination with chemotherapy in Ewing's sarcoma. A key objective of the study is to test the efficacy and safety of advanced personalized chemoimmunotherapy. It assumes the integration of standard chemotherapy with the availability of a new antibody," explains Prof. Anna Raciborska, head of the BUTTERFLY project carried out at the Department of Oncology and Oncologic Surgery of Children and Adolescents at the Mother and Child Institute.
The first patient receiving naxitamab therapy is 12-year-old Jacob, who has already received two doses of the drug since October 25.
Opportunity for modern therapy
Currently, the treatment of Ewing's sarcoma is combination. Its basis is chemotherapy applied both before and after surgery. Radiation therapy is also used in the treatment of this type of cancer - usually after surgery. Sometimes, if the body does not respond to initial chemotherapy, radiation therapy is used together with induction chemotherapy. Such treatment leads to recovery in 60-65% of patients, but in the case of metastatic lesions this percentage is much lower[2]. It happens that despite the traditional treatment pathway, some patients fail to stop the disease or the disease returns. That is why it is so important to develop modern methods of diagnosis and treatment of cancer in children and young adults.
- Non-commercial clinical trials are primarily designed to optimize treatment. They provide an opportunity to compare new regimens with and/or incorporate additional drugs, their dosage or diagnostic and therapeutic procedures. They enable the use of more precise and often less invasive diagnostics. Thanks to their development, doctors can create new standards of management, personalize treatment, and increase cure rates. Those who qualify for IMID's trials have no treatment costs, receive ongoing care from a team of doctors and nurses, and have access to the highest quality medical procedures and diagnostics. This gives child patients and their families a sense of security and is a confirmation that the Clinic provides medical services at the highest level," explains Professor Anna Raciborska, head of the Department of Oncology and Oncologic Surgery of Children and Adolescents at the Institute of Mother and Child.
Patient recruitment has begun
The study under the BUTTERFLY project will run until the end of July 2028. Patients between the ages of 2 and 21 with progression or recurrence of Ewing's sarcoma can participate. You can be invited to participate by a physician-researcher or apply on your own at the study center. Eligibility is decided on the basis of the patient's medical history, current results and meeting certain inclusion criteria. For patients for whom standard treatment has not been successful, participation in the study offers the opportunity to benefit from state-of-the-art therapies, innovative drugs and treatments.
Pediatric malignancies are rare diseases - there are about 1100-1200 new cases per year in Poland. Their curability depends on many factors, but on average it is estimated at about 80%. However, there is no shortage of pediatric patients refractory to traditional treatments or those who experience recurrence or metastasis. A chance for them to recover is through non-commercial clinical trials. In addition to BUTTERFLY, other clinical projects are underway at the Department of Oncology and Oncologic Surgery for Children and Adolescents at the Mother and Child Institute. POLHISTIO includes 3 research efforts (HISTIOGEN, BRAVO, TRAM), targeting patients with histiocytic cell proliferations, and REGBONE for patients with malignant primary bone tumors (Ewing's sarcoma, osteosarcoma) that are resistant to standard therapy. All of these projects are being carried out with funding from the Medical Research Agency.
Source: press release
[1] https://projektbutterfly.pl/informacje-o-projekcie
