Zolgensma, the most expensive drug in the world, costs R$ 6.5 million, and is applied in Brazil to children with Spinal Muscular Atrophy (SMA), a rare paralyzing disease: but the procedure, in addition to being costly, involves secrecy and security, and the drug was even transported in an armored car.
The Pequeno Príncipe Hospital, in Curitiba, is a reference in pediatric care and has been providing Zolgensma since 2020, being the second in the country to perform the procedure. In an interview with UOLneuropediatrician Adriana Banzato, responsible for the application, tells more about the security scheme involved in the process.
How is Zolgensma applied?
Among the complications, the price is certainly the worst: the drug is so expensive that it can derail the health budget of entire cities. Families who need it often go to court, which often passes the cost on to the state or federal government. Novartis, the pharmaceutical company responsible for the drug, made a launch event in the Brazilian market in May. In it, Banzato discussed details of patients treated with other medical professionals.
According to her, Zolgensma comes in small bottles of 5.5 ml or 8.3 ml: each patient receives from 40 ml to 80 ml, which is calculated based on size and weight. Arms, although already used for transport, have already been discarded due to the attention they attract – but the secrecy remains, and it was never informed where the medicine is stored in hospitals.
It arrives frozen at minus 60ºC and takes 24 hours to reach a liquid state for application, at which point the vial can be opened and placed in syringes, then finally taken from the hospital pharmacy to the patient. Employees are even apprehensive: “What if I drop it? What if I lose a drop?” The doctor, however, guarantees that they are used to the work and certified for it.
The Zolgensma is then inserted into the body via a catheter, just like an IV. For the application, the patient needs to be hospitalized: there are hospitals that require one-day hospitalization, others, one hour. The application is unique, and the patient can be discharged even on the same day. The application cost varies according to the hospital protocol and patient evolution, but it does not exceed 1% of the cost of the drug, which, even so, is a lot: it can reach the mark of R$ 100 thousand.
How is the medicine made?
As with the cost of the drug, the application is generally not paid by patients, funded by the state from court decisions. And not every SMA patient can receive it: The treatment is genetic, as patients do not have the SMN1 gene, which produces the SMN protein, responsible for muscle functions, including breathing. Without it, neurons begin to die and atrophy the patient.
The drug is then encapsulated in a virus, AAV9, present in the air and harmless to humans. Its contents are removed in the laboratory and the gene that produces the protein is put in its place. When injected into the body, the modified virus binds to cells and, when attacked by antibodies, releases the content, which binds to DNA and causes the body to produce the protein.
The problem is that, as AAV9 circulates through the air, we tend to develop antibodies against it, which usually happens until the age of two. After that, the body fights the virus and kills it before releasing the gene. The age, then, is considered a limit for the treatment, although it has already been done in patients up to 7 years old.
The treatment generates rapid changes, being felt in up to two or three days after application, and at 30 days, the gain in movement is remarkable. But although Zolgensma prevents neurons from dying due to the lack of the protein, it does not recover cells that have already died: the sooner SMA can be treated, the better.
Permanent side effects are not known — some patients have liver inflammation within days of application, but this can be addressed with other medications. SMA, on the other hand, is controlled for the rest of life: after treatment, the body produces the protein forever.
There are controversies about the drug, given its cost: SUS could pay for many other treatments with the value of the drug, for example, as several critics point out, while Zolgensma already has a very high value to treat just one patient. Banzato responds, asking: “How much is a life worth? A child with SMA remains in full treatment for life, costing society and family much more.”
Source: UOL 1, 2