Influencer Lito Sousa Receives Experimental Treatment for Rare Brain Disease

Lito Sousa, a 59 year old influencer and pilot, has received an experimental substance to treat Creutzfeldt Jakob disease, a rare and progressive neurodegenerative condition. His wife, Mila Seidl, announced in a video on the YouTube channel Aviões e Músicas that Sousa remains stable in the intensive care unit at Einstein Hospital Israelita in São Paulo. He had been hospitalized since September 8 due to complications from the disease. The substance administered is ALN 6457, a genetic silencer developed by the pharmaceutical company Regeneron. This compound has shown promising results in animals but has not been formally tested in humans for this specific disease. Its safety and efficacy remain unknown. The family sought inclusion in clinical trials but could not secure a spot, leading to the approval of compassionate use. This regulatory path allows patients with grave illnesses and no other therapeutic alternatives to access experimental treatments. The Brazilian Health Regulatory Agency, Anvisa, authorized the exceptional import of the substance on September 4. The logistics of delivering the medication involved a complex operation. Parts of the substance were sent from Switzerland and the United States. Due to a flight cancellation from New York and heavy rain causing flooding in São Paulo, the family coordinated with aviation partners to transport the medication from Guarulhos International Airport to the hospital via helicopter. This urgency was necessary because the therapeutic potential of the substance tends to decrease over time. Seidl stated that the medical team will monitor Sousa over the next six weeks to compare samples and evaluate the evolution of the case. She noted that while the outcome of this specific case will not be enough to conclude if the substance is effective against the disease, other pharmaceutical companies have shown interest in bringing experimental studies to Brazil.