Five deaths had already been reported to the National Health Surveillance Agency (Anvisa) when only 19 patients had received polylaminin through compassionate use, a modality that allows access to a substance still being studied outside of clinical research. The notifications were made between January and March 2026 and are equivalent to approximately one death for every four patients treated.
The Cristália laboratory, responsible for clinical research, disputes the calculation. According to the company, one of the five people died before receiving the substance, which would reduce the death toll to four deaths among 19 patients, or one in every five.
In the following months, Anvisa received another four notifications, bringing the total to nine. Cristália recognizes eight cases of patients who were treated, all of which were notified to the agency and analyzed by the pharmacovigilance area.
After investigating the deaths, the laboratory claims to have found no evidence of a causal relationship with polylaminin. The company attributes the deaths to common complications in those who suffer severe spinal cord injuries, such as sepsis, pneumonia, pulmonary embolism and cardiac arrests, and remembers that mortality in this population depends on the severity and level of the injury.
Among the patients who died, there were people with an old lesion located in the cervical region, a profile that was outside the criteria of clinical research authorized by Anvisa.
The absence of a relationship indicated by the laboratory, however, does not equate to proof of safety. Polylaminin remains experimental, and only clinical studies will be able to say whether it is safe and effective.
Medical team applying polylaminin — Photo: Santa Casa de Araçatuba/Disclosure
Deaths and surge in orders
The decision that limited compassionate use, announced this week, was based on a new assessment of risks and benefits carried out by Anvisa.
In a note to g1, the agency cited three factors: the deaths of patients who received the substance outside the study criteria, the still insufficient evidence on safety and an increase in orders never seen for any other product under investigation.
According to Anvisa, the exposure of many patients to an experimental product usually happens in expanded access programs, which require research from phase 3 onwards. In the case of polylaminin, the phase 1 study has not even started.
Before deciding, the agency consulted a technical panel with representatives from the Federal Council of Medicine (CFM) and neurologists and neurosurgeons from Brazil and abroad. Unanimously, experts recommended not expanding access, because of the reported deaths and the way the substance is applied.
With the change, only patients with an injury that occurred a maximum of 72 hours ago and located in a specific region of the spine can request compassionate use, the same criteria as the clinical study. Anvisa emphasizes that there is not enough safety evidence for this group.
Paths to receive polylaminin
Polylaminin is obtained from laminin, a protein present in tissues that helps support cells. Researchers are investigating whether, when applied to the injured area, it stimulates the regeneration of nerve connections and restores movement to paraplegic or quadriplegic people. The procedure is complex and carried out directly on the spinal cord.
Today, the substance follows two paths.
Phase 1 study. In January, Anvisa authorized a phase 1 study, the initial stage focused on safety, but it did not get off the ground: according to Cristália, it was not possible to recruit patients within the defined criteria.
Compassionate use. The other path is compassionate use, in which the laboratory provides the substance to those who obtain authorization, and the surgery, medical team and hospitalization are covered by the Unified Health System (SUS) or the private network. Cristália has 115 applications so far, including court decisions and agency authorizations.
Provided for in Brazilian regulations since 2013, compassionate use is intended for critically ill patients with no treatment alternatives. Each request comes with medical documentation and is evaluated individually by Anvisa, without automatic approval.
The research published so far deals with recent injuries in a specific region of the spine. Use on old injuries is also investigated, but testing on dogs has not been completed.
Results do not allow attributing improvement to the substance
In August, during Rio Innovation Week, researcher Tatiana Sampaio, who participated in the development of polylaminin, presented data from 17 patients, among more than 100 who had already received the substance.
Ten of them changed their classification on a scale that measures the severity of spinal cord injuries: they went from category A, complete injury, to B or C, which indicate some sensitivity or preserved motor function below the point of injury. The change may signal recovery of certain functions, but it does not mean that the patient can walk again. None of the 17 returned.
Bruno is a separate case. The researcher publicized her motor recovery and attributed it to the substance, which helped popularize polylaminin on social media.
Neither his history, however, nor the changes recorded in other patients allow us to conclude that polylaminin was responsible for the improvement. This would require studies capable of separating the effect of the substance from other factors that influence recovery after a spinal cord injury.
Researcher accuses agency; Anvisa and laboratory counter
Cristália's consultant in the clinical study, Tatiana Sampaio reacted to the new rules in a video on social media. He accused Anvisa of acting illegally and making access difficult for patients, calling the decision evil and describing director Daniela Marreco as "cold and emotionless". According to her, access now depends on a race against time that few can complete.
To illustrate the criticism, he cited Tiba Camargos, who became quadriplegic in February 2025, when trying to save his son from drowning in the interior of Rio Grande do Sul. His request was denied because the cervical injury is more than a year old.
Tatiana advocates its application to old injuries based on research carried out on dogs. Cristália, however, informs that the tests on animals have not yet been completed and that, without the results, there is not enough evidence to move forward with research on humans with this injury profile.
The researcher also argues that data collected in compassionate use could lead to changes in clinical study design. In the video, she did not mention the reported deaths or the fact that authorized research has not yet begun.
Anvisa repudiated the attacks. In a statement, he said that his decisions follow technical criteria and the protection of the population's health and that terms like those used in the video are unacceptable, especially coming from researchers or representatives of the pharmaceutical sector, as they move the debate outside of regulation and science.
Cristália also came out in defense of the agency. He said he was unaware of any persecution, reaffirmed his trust in Anvisa and declared his support for the director. The company considered that compassionate use and expanded access are important humanitarian instruments, but they do not replace the production of robust clinical evidence.
