Country begins strategy to expand the supply of gene therapies in the SUS

The Oswaldo Cruz Foundation (Fiocruz) signed, this Tuesday (26), agreements to start its Strategy for Advanced Therapies, which aims to make it cheaper and, thus, expand the supply of gene therapies through the Unified Health System (SUS). The project is financed by the Ministry of Health, through the Growth Acceleration Program (PAC).

Gene therapies involve the manipulation of genes to combat diseases such as cancer, autoimmune diseases, AIDS and genetic syndromes. There are several treatments currently in use, 15 of which are authorized by the National Health Surveillance Agency (Anvisa) for use in Brazil. There are hundreds more being studied around the world.

The big problem with these therapies is the cost. A therapy for Duchenne muscular dystrophy, for example, a progressive and irreversible disorder of the muscles, can cost up to R$15 million, a similar amount to therapy against hemophilia A.

According to Fiocruz, it is estimated that over the next five years between R$4 billion and R$14.5 billion will be spent on these treatments, if the amount charged by pharmaceutical companies is paid.

The provision of these treatments in private health units is out of reach for people who do not have that kind of money. The free provision provided by the SUS is very expensive for public coffers.

The idea is that Fiocruz, through its Institute of Immunobiological Technology (Bio-manguinhos), will be able to develop and offer gene therapies at a cost of around 10% of what is spent today on the same treatment.

The initial development of these treatments will have the support of the North American non-profit organization Caring Cross, which will transfer technology to the Brazilian institute.

Minister Nísia Trindade highlights the importance and innovation of gene therapies – Tomaz Silva/Agência Brasil

“The guidance is that [essas terapias desenvolvidas pela estratégia da Fiocruz] be incorporated into the SUS”, stated the Minister of Health, Nísia Trindade. “This is not just a very important and innovative therapy. There is a tenfold reduction in the cost of treatments available today. Which means multiplying access to this therapy tenfold.”

Cancer

The first project to be developed by the Strategy for Advanced Therapies involves the reprogramming of the body’s defense cells to combat some hematological cancers, such as lymphomas and acute leukemias. It will be a partnership between Fiocruz and the National Cancer Institute (Inca), which is already carrying out research in this area.

“It is a process of re-education of the cell. We collect the patient’s cells and, in the laboratory, we will make a genetic modification to them, placing a gene that leads to the expression of a protein that will make this cell start to recognize these tumor cells”, explained the Inca Martin researcher Bonamino. “And, after all quality control, this cell is returned, as if it were a transfusion, to the patient.”

The therapy called CAR-T consists of inserting, into patients’ T lymphocytes (defense cells), lentiviruses that carry a specific gene to fight the tumor. With the new gene, the lymphocyte becomes more effective in finding cancer in the body and recognizing it as something to be eliminated, sending substances that will cause the death of the cancer cell.

For this specific treatment, whose cost is around R$2 million, there is a possibility that the value will drop to R$200,000. In addition to the development of CAR-T therapy itself, there will be the production of lentiviruses.

Clinical tests to validate the Fiocruz and Inca therapy should be carried out from the end of this year, in the United States and Brazil.

With the therapy validated, the idea is to install laboratories in container modules, which can be installed in hospitals in various parts of Brazil, enabling the decentralization of these treatments.

“We here at Bio-Manguinhos will take advantage of the knowledge we acquired during the pandemic [de covid-19] to produce the lentivirus, which is the viral vector. And there will also be modules where the CAR-T process will be carried out. And these modules will be certified by Anvisa and Bio-Manguinhos”, explained the director of Bio-Manguinhos, Mauricio Zuma.

According to Minister Nísia Trindade, making gene therapies cheaper will not only benefit Brazil, but could also help other countries in Latin America and Africa.