An innovative cancer therapy, which can cost up to US$500,000 and has already been shown to cause disease remission in patients, should be made available to the Brazilian population by the Unified Health System (SUS). The CAR-T cell therapy program is an initiative by Butantan, the School of Medicine of the University of São Paulo (FMUSP), the School of Medicine of Ribeirão Preto (FMRP-USP) and the Ribeirão Preto Blood Center, supported by the State of São Paulo. Comprised of Nutera/SP (São Paulo, SP) and Nutera/RP (Ribeirão Preto, SP), the centers will be the first facilities to produce CAR-T therapy on a full scale in Brazil.
“This new program is proof of the maturity and excellence of Brazilian science, and will make our country the largest producer of advanced cancer therapies in Latin America. We are the only Brazilian public institutes working in this area, in a work that involves a team of more than 50 researchers. And we will oversee it from beginning to end, with the aim of delivering this treatment to the Brazilian Unified Health System (SUS) and benefiting the population”, says the hematologist leading the initiative, Dimas Covas, president of Butantan.
Hematological neoplasms (or blood cancers) are the target of CAR-T technology, which “reprograms” the patient’s own defense cells so that they identify and eliminate the cancer. The therapy developed at the Ribeirão Preto Center for Cell-based Therapy has already been tested in a compassionate way, when the patient runs out of treatment options with no results. Now, it is moving to phase 1 of the clinical study, which will be submitted to the Brazilian Health Regulatory Agency (ANVISA) and should start in October with 30 patients with B-cell non-Hodgkin's lymphoma. If proven safe, the therapy will move to phases 2 and 3 with a greater number of volunteers.
The partnership between Butantan, USP and the Ribeirão Preto Blood Center aims to expand access to CAR-T cell therapy for the Brazilian population. The 100% national production by non-profit institutions is one of the great advantages of the new center to allow cost reduction and facilitate the inclusion of such treatment in the SUS. “The Ribeirão Preto Center for Cell-based Therapy has more than 20 years of experience in the field. This helps to reduce costs, since we don't have to spend high amounts for licensing or for a technology transfer, for example, because we already have researchers with the necessary expertise", says Butantan's Innovation manager, Cristiano Gonçalves, responsible for the Innovation and Tech Licensing Office of the institution.
In March, Anvisa approved the registration of the first cell therapy for cancer in Brazil, also aimed at hematological neoplasms: Kymriah, developed by Novartis. It is recommended for pediatric and adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia and for adult patients with diffuse large B-cell lymphoma who have undergone other treatments with no improvement. However, few are able to access this therapy, which costs around US$ 500,000, equivalent to R$ 2.5 million. The T cells extracted from the patient are exported to the United States, where they are processed in the laboratory and then returned to the donor.
CAR-T emerged in the United States and has been experimentally applied to end-stage cancer patients since 2010. In 2017, the positive results led the Food and Drug Administration (FDA) to approve unanimously the use of this therapy to fight cancer. Since then, another six CAR-T technologies have been approved in the country, all focused on blood cancers – leukemia, lymphoma and multiple myeloma. The cost also reaches US$ 500,000 per patient.