Clinical Trials
Additional Information
Currently, in these clinical studies, the American DFMO maintenance therapy for high-risk children has made great breakthroughs, and bivalent vaccine research is also ongoing, for relapsed or refractory children have efficacy, the future is promising, but there are no related drugs and studies in China, so stage 4 high-risk children still have a 40% chance of relapse after finishing all treatments in China, and the study found that DMFO Research has found that DMFO treatment can reduce the recurrence rate of these children to 15% when used within two months of the end of regular treatment, and the bivalent vaccine needs to be used within six months of the end of regular treatment for the best results. However, domestic families of children with disabilities have very little knowledge of these treatments, and lack of information on the clinical use of these drugs in the United States, and don’t know about the process of applying to the U.S. hospitals for clinical treatments. Of course, because of language barriers, there may be some difficulties in the application process, thus missing the opportunity to effectively improve the long-term survival and improve the prognosis of the child, which is very regrettable.
DFMO Treatment
DFMO is a long-marketed drug for African sleeping sickness, which was not widely produced and used due to its single indication. Later, DFMO was found to have hair reducing properties and was used in some female hair removal beauty products. With the discovery that DFMO can effectively break down polyamines and inhibit the reproduction of tumor cells, it attracted the attention of pediatric oncologists, who began the research on the therapeutic effect of oral DFMO drugs on children with neuroblastoma. Dr. Sholler of the United States spent nearly 10 years to carry out strict phase I/II clinical trials, and the research results confirmed that DFMO treatment is effective for children with high-risk neuroblastoma, especially for high-risk children with gene amplification, which greatly improves the long-term survival rate of high-risk children and has no effect on normal tissues, and the side effects of DFMO are very small, and the side effects of a small number of children are controllable and reversible. The side effects are controllable and reversible. Moreover, DFMO has been proved to be effective in breaking through the blood-brain barrier and blocking the proliferation and spread of tumor cells with head metastasis, so it has a very good therapeutic effect on difficult-to-treat children with head metastasis. The 5-year progression-free survival rate of 85% and 5-year overall survival rate of 95% for children with high-risk DFMO who have been taking DFMO orally for 2 years after conventional treatment is very encouraging, and will be a boon for children with high-risk DFMO and their families. At present, DFMO treatment has been evaluated as a breakthrough therapy for children with high-risk mother of God by the US FDA, and large-scale efficacy validation is being done in various states in the US. Meanwhile, oncologists are studying the effects of DFMO in combination with chemotherapy drugs in induction chemotherapy treatment, and preliminary findings have shown that DFMO works better in combination with some chemotherapy drugs. The U.S. is also currently conducting a phase II clinical study of DFMO + etoposide combination regimen for relapsed and refractory high-risk children with the mother of God, and has begun to accept foreign children into the enrollment.
GD2/GD3 Bivalent Vaccine
Neuroblastoma Bivalent Vaccine is a drug developed by Sloan Hospital in the United States for use in the maintenance treatment of children with Mother of God who have ended conventional treatment. The bivalent vaccine was used in 120 children in the previous Phase I and Phase II clinical trials, and the results of the studies showed that the bivalent vaccine was safe to use and effective, but the results were not very satisfactory. In October 2022, the Sloan clinical team readjusted the clinical regimen based on the data from the studies, and the new regimen is currently undergoing a Phase II clinical trial, which is set up with three clinical experimental groups (three different drug with the regimen), which are randomized, and the trial will end in 2025.
CAR-T Therapy
CAR-T therapy is a breakthrough therapy in the field of tumor treatment in recent years, which has been proved to be effectively used in the treatment of high-risk hematological tumors and lymphomas. Therefore, some oncologists at home and abroad have conducted clinical trials to study the therapeutic effect of CAR-T therapy on solid tumors, among which, the research of CAR-T therapy for neuroblastoma is also underway. In China, there are some CAR-T clinical trials for high-risk neuroblastoma, mainly focusing on CAR-T therapy for GD2 target. However, the effect of CAR-T therapy for GD2 target is not satisfactory in neuroblastoma treatment, so the research of CAR-T therapy for other targets or dual targets has been started in the last 1-2 years.
