Anvisa authorizes import of experimental medicine for treatment of influencer Lito Sousa

Anvisa authorizes import of drug in testing phase for treatment of influencer Lito Sousa

Lito was included in the company's compassionate use program and will receive the substance in Brazil. He will be the first person in the world to test the product for the disease.

By Poliana Casemiro, g1

Lito will be the first patient to test the experimental substance ALN-6457, released on an exceptional basis for the treatment of the rare and fatal Creutzfeldt-Jakob disease.

The treatment will be carried out through a compassionate use program, which allows patients without available therapeutic alternatives to have access to substances that are still in the experimental phase.

Creutzfeldt-Jakob disease is a rare, progressive and incurable brain disease, which causes the majority of patients to die within a year of the first symptoms.

Lito Sousa in a video published this Friday (4) — Photo: Reproduction

The National Health Surveillance Agency (Anvisa) authorized this Friday (4) the import of a medicine still in the testing phase for the case of influencer Lito Sousa, in the treatment of Creutzfeldt-Jakob disease.

The substance, called ALN-6457, from Regeneron Pharmaceuticals, has no commercial registration in Brazil and no legal representative in the country — therefore, the release followed an exceptional rite, outside the standard medicine import flow.

The product is still in the preclinical stage, with no studies formally initiated in humans for prion disease. In other words, Lito will be the first person to test the substance.

The import request was made by Einstein Hospital Israelita, responsible for monitoring the patient, after Lito was accepted into a compassionate use program for the medicine.

Family request

According to Mila Seidl, the influencer's wife, it was she who requested the inclusion and organized the joint action for import authorization, which involved the Ministry of Health, hospital, Anvisa and FDA.

"The impossible is becoming reality! We are ready to receive treatment! We expect to arrive in Brazil in 7/9 days", stated Mila Seidl, Lito's wife, on her Instagram account.

COMPASSIONATE USE: Compassionate use allows patients without available therapeutic alternatives to have access to substances that are still in the experimental phase, even before the completion of clinical tests that would prove their safety and effectiveness on a large scale.

According to Anvisa, the assessment considered that it is a disease with rapid and irreversible evolution. Furthermore, authorization on an exceptional basis was adopted due to the absence of a sponsor or responsible representative in Brazil.

There is still no information on how the treatment will be carried out.

A rare, rapid and incurable disease

CJD is a prion disease — caused by the abnormal alteration of a protein called a prion, which multiplies in the brain and destroys neurons, leaving a pattern of holes in the brain tissue that gives rise to the term "spongiform." The disease is rare, progressive and, to this day, there is no treatment capable of reversing or stopping its progression.

According to neurologist José Bauab, the prion settles inside the neuron and progressively intoxicates it. The effect is not restricted to the isolated cell: toxicity also compromises the glia, the tissue responsible for supporting neurons.

"The protein particles enter the neuron, intoxicate the neuron, and have an absolutely destructive and toxic behavior in the cell", describes Bauab.

Understand Lito Sousa’s illness — Photo: Arte/g1

In Brazil, the disease mainly affects older people: the 55 to 74 year old group accounted for 60.2% of notifications, with an average age of 66 years old among suspected cases — a different profile from Lito's case, as the sporadic form usually affects people between 60 and 80 years old.

The literature used by the Ministry of Health indicates that around 90% of patients die between six months and one year after the onset of symptoms, with an average survival of five months — a fact that reinforces the severity of the condition reported by Lito's family. Among the notifications analyzed, there were 290 records of deaths due to the disease, although the bulletin points out flaws in filling out and monitoring the data.

According to a survey by the Ministry of Health, Brazil registered 547 confirmed cases of the disease between 2005 and 2021 — a period that corresponds to the first 17 years since CJD became part of the list of compulsory notifications.