Mila Seidl, wife of influencer Lito Sousa, denied that the authorization to import an experimental drug against Creutzfeldt-Jakob disease was the result of a privilege granted to her husband. She stated that she intends to explain the procedure followed by the family to help other patients with rare diseases and no alternative treatment options.
In videos published on social media, Mila said that the request followed the rules for the compassionate use of medicines and that contact with the pharmacist was made by the family itself, with the participation of Lito's doctors.
"This is not our privilege. It is the right of anyone who is in a similar situation. The difficulty is that this information is not clear", he stated.
The National Health Surveillance Agency (Anvisa) authorized on Friday (4) the import of ALN-6457, an experimental substance produced by the pharmaceutical company Regeneron Pharmaceuticals.
The product has no commercial registration or legal representative in Brazil and is still in the pre-clinical stage for the treatment of prion diseases, with no studies formally initiated in human beings. Therefore, imports were authorized on an exceptional basis.
The exceptional rite, however, does not mean that a specific rule was created for Lito. The authorization was granted within the compassionate use mechanism, which allows patients with serious illnesses and no therapeutic alternatives access to still experimental medicines.
According to Mila, she and the doctors accompanying the influencer gathered exams, scientific documents and information provided by the pharmaceutical company to support the request.
"We presented a lot of documentation, it was very robust. I prepared a lot. We were well equipped with the documents that the pharmacist gave, with the exams, with everything scientific", he said.
The import request was formalized by Einstein Hospital Israelita, responsible for monitoring Lito, after the influencer was accepted by the pharmaceutical company in a compassionate use program.
Mila said that the family initially tried to include Lito in an experimental study, but was refused because there were no more places available. After that, he started looking for another way to access possible treatment.
"We participated in the studies and were denied. Not because it didn't meet the requirements, but because everything was closed. So, we followed another path, which is the compassionate use of the medicine", he stated.
In the videos, Mila also refuted publications according to which authorities or businesspeople had interfered to obtain the medicine. She stated that contacts with the pharmaceutical company were made by the family itself, with the participation of Lito's doctors.
"There are people saying that it took a minister's hand to get the medication, that a rich businessman financed it, that Anvisa got it. This is the last time I'll explain: it was me and Lito who got the medication", he said.
According to Mila, after contacting another pharmacist, the family participated in a meeting and presented Lito's case. The company then allowed access to the substance through compassionate use.
From then on, according to her, the necessary authorizations were requested in the United States and Brazil, in a process that involved the pharmaceutical company, the hospital, Anvisa, the Ministry of Health and the North American regulatory agency, the FDA.
"We managed to contact another pharmaceutical company by our own means, we had a meeting with them and, because Lito's case is very interesting, they approved compassionate use. So I went after the entire process", he stated.
Mila also said that there has been no use of public resources or financing by third parties so far.
"There isn't a cent of public money, there isn't a cent from anyone else in this. You didn't see me opening a crowdfunding or anything", he declared.
Family wants to explain the path to other patients
Lito's wife said she intends to publicize details of the procedure to help other families facing rare diseases and seeking access to experimental treatments.
According to her, although compassionate use is provided for in the regulations, information about the necessary documents, the bodies involved and the processing of the request is not easily found.
"This is not our privilege. It is the right of anyone who is in a similar situation. The difficulty is that this information is not clear. I had to work hard to get this", he stated.
Mila also said that the bodies involved recognized the urgency of the case and accelerated the analysis. Even so, he stated that the process was distressing due to the rapid evolution of the disease.
"Even this faster flow took about a week. And, in a week, we lost some things. But I understand that they also have to be assured that they are doing the right thing. They can't just write, they need to analyze", he said.
For her, the case can help encourage the creation of faster procedures for patients with similar conditions.
“Perhaps this will bring light to, from now on, review these processes and create slightly faster flows for other families”, he stated.
The drug has not yet been tested on humans
ALN-6457 remains in the preclinical stage for prion disease. This means that there are still no results from studies in humans that prove its safety or effectiveness in stopping or slowing the progression of the disease.
According to Anvisa, the analysis of the request took into account the rapid and irreversible nature of the disease, the absence of therapeutic alternatives and the fact that the pharmaceutical company does not have a sponsor or representative responsible for the product in Brazil.
No details have yet been released about how the medicine will be administered or when treatment will begin. When announcing the authorization, Mila stated that the expectation was that the product would arrive in Brazil within seven to nine days.
Rare, rapid and incurable disease
Creutzfeldt-Jakob disease is a rare, progressive neurological disease and, to date, there is no approved treatment capable of reversing or stopping its progression.
It is caused by the abnormal alteration of a protein called prion, which accumulates in the brain and destroys neurons. As the disease progresses, it can cause memory failures, changes in behavior, tremors and difficulty moving.
Lito is 59 years old and left Einstein on Tuesday (1st), after more than 20 days in hospital. He began receiving palliative care at home, accompanied by nurses and a multidisciplinary team.
The family emphasizes that palliative care does not mean that the influencer is in a terminal phase. This type of assistance seeks to control symptoms, reduce suffering and preserve quality of life while the search for treatment alternatives continues.
Data from the Ministry of Health indicate that Brazil confirmed 547 cases of the disease between 2005 and 2021. The literature gathered by the department indicates that approximately 90% of patients die between six months and one year after the onset of symptoms, with an average survival of five months.
