Research seeks to reduce dose and cost of treatment against neuroblastoma

A campaign for Pedro's health, son of indigenous man Bruno Pereira and anthropologist Beatriz Matos, drew attention to the need for oncological treatments that are not yet available in the Unified Health System (SUS). Pedro, just five years old, was diagnosed with stage 4 neuroblastoma. He must undergo a bone marrow autotransplant and, after the procedure, he will need medication that, at the recommended dosage, can cost R$2 million.

This medicine is betadinutuximab, whose commercial name in Brazil is Qarziba. The substance is being studied at the University of São Paulo (USP) and at Hospital Israelita Albert Einstein. The research attempts to reduce the dose of medicine currently imported for treatment to 20%. One of the doctors involved in the study is childhood cancer specialist Vicente Odone Filho, from Albert Einstein Hospital.

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According to him, neuroblastoma challenges scientists, who are looking for new solutions through conventional cancer treatment resources, such as chemotherapy, surgery, radiotherapy and, more recently, bone marrow transplants. The introduction of immunotherapy resources has had a very significant impact on the disease in advanced stages.

In the case of neuroblastoma, immunotherapy is called anti-GD2, an antibody that is present in the affected cells and in some other cells as well. “This allows this antibody to act directly on this substance and destroy the tumor cells that harbor these substances.” Odone assured that this is a fundamental feature. The drug is already registered by the National Health Surveillance Agency (Anvisa) in Brazil, but is not yet on the SUS list of special medicines, due to its high cost.

“This has caused a huge problem for those who work with this disease, in terms of fighting to make it available to all the children who need it”, explained the doctor. “There are three commercial presentations available for this medication. The three forms have analogous effects. Neither is superior to the other.”

Researchers at Hospital Israelita Albert Einstein and USP are working with an investigation protocol that aims to use this medicine in a slightly different way. “If this protocol is successful, we will be able to use a much lower dose of Qarziba than is normally used, more or less 20% of that dose. It will still be a very expensive medicine, but more easily, since we would use 20% of the conventional sweet.”

Odone Filho stated that the signal for dose reduction, based on ongoing studies, will largely depend on the possibility of carrying out research on a regular basis. “Because obtaining the medicine is very expensive. Even for studies that employ a lower dose, it is very difficult to carry out, because of the costs involved.” The estimated time to obtain a substantial scientific response is approximately two years. “It’s a long time for those who urgently need treatment, but it’s what science has to do to provide correct information to everyone.”

According to Albert Einstein's doctor, a child like Pedro, who has stage 4 neuroblastoma, overcomes all the initial stages, continues treatment and undergoes a bone marrow transplant, has a chance of survival 20% higher than that of would have with more conventional resources.

The doctor admitted that there is a very long road to be covered beyond this Qarziba path, which is to increase the number of children who can use the drug and benefit from it. There are already trials to use the drug in the initial stage. “It is not a panacea drug, which can be applied in any situation”, warns the specialist. “It needs to be applied at a time when the patient has already responded to other treatments and is complemented by this drug.”