Death of patients using polylaminin leads Anvisa to reinforce the requirement for clinical studies in Brazil

The use of the experimental substance polylaminin in patients in Brazil, under the compassionate use modality, has resulted in the deaths of seven people since February, the period in which Laboratório Cristália began offering the treatment outside the scope of clinical studies. The news was released on August 7, 2026, after a presentation that detailed the patients’ evolution. Despite the deaths, Anvisa (National Health Surveillance Agency) has stated, so far, that the assessment does not indicate a direct relationship between the deaths and the product. However, the regulatory agency reiterates the need for rigorous studies to prove the safety and effectiveness of the substance, a fundamental step for any medicine.

Experimental substance: what is compassionate use and why it raises debate

Polylaminin is a substance still in the research phase, investigated by the Cristália Laboratory for its potential in helping to recover from spinal cord injuries. Compassionate use, a modality that has allowed access to patients in Brazil, is a legal mechanism that offers individuals with serious illnesses and no proven treatment alternatives the possibility of accessing experimental therapies. This occurs before the completion of clinical studies and definitive approval by regulatory bodies such as Anvisa. In Brazil, this permission is granted voluntarily, but always with prior authorization from the agency, ensuring a minimum of monitoring and control.

The relevance of this mechanism lies in offering a chance to patients in critical conditions and high suffering. However, this route also carries inherent risks, as the safety and efficacy of the treatment have not yet been fully established. This duality between hope and uncertainty underscores the imperative need for robust clinical trials. Such studies are the basis for determining whether the benefits of a new therapy outweigh its potential harms, a non-negotiable principle of evidence-based medicine.

Cristália data on patient evolution and the AIS scale

Since February, when compassionate use applications began, just over 100 people have received polylaminin in the country. Among these patients, seven deaths were recorded, with the last notification on August 6th. Researcher Tatiana Sampaio, leader of studies with polylaminin, presented data that indicate some clinical evolution in ten of the 17 patients who have already completed the minimum period of six months of follow-up.

Evolution was assessed using the AIS scale (ASIA Impairment Scale), a tool used to classify the severity of spinal cord injuries. The presentation slides showed that at least ten patients progressed from AIS A, which denotes complete injury, to AIS C or B. It is essential to clarify that this improvement in the scale, although positive, generally indicates the recovery of sensory or motor functions in the anal region, and does not imply the resumption of broader movements, as occurred in the case of patient Bruno, who gained notoriety. This distinction is important to avoid unrealistic expectations about the effectiveness of treatment to date.

Anvisa refutes questions about the need for clinical studies

In her presentation at Rio Innovation Week, Tatiana Sampaio expressed doubts about the real need for an in-depth clinical study for polylaminin, considering the volume of data already observed. “If you are working with human beings and you already have this volume of data, why are you going to do a clinical trial?” asked the researcher during her speech.

Anvisa reacted strongly to this statement. The regulatory agency refuted “any statement that conducting a clinical study is harmless and will not produce useful data.” In an official note, the entity stated that “any statement in this regard demonstrates a complete lack of knowledge of the scientific process and protocols used around the world for the development of medicines and whose central objective is to guarantee the safety of research participants.” This firmness from Anvisa reinforces the global commitment to science and the protection of public health, highlighting that validating a treatment requires formal and proven steps.

Cristália and Anvisa investigate deaths and adverse events

Laboratorio Cristália, through its pharmacovigilance sector, responsible for collecting data on adverse events, analyzed death cases. The company stated that the majority of patients did not experience any adverse events. Among those who manifested any symptoms, most were classified as not related to substance use. Less than 10% of patients had any adverse event considered possibly linked to polylaminin, and none of them were categorized as serious. Given this panorama, the laboratory maintains that “so far, the administration of polylaminin does not promote significant adverse events.”

Anvisa, which received notifications about the deaths, also confirmed that its preliminary assessment does not indicate a direct relationship between the deaths and the experimental product. A seventh case of death was recently reported, and its data is awaiting formal submission for analysis by the agency, ensuring continued regulatory oversight.

Restrictions and processes for authorizing the use of polylaminin

Anvisa announced that it received 128 requests for compassionate use of polylaminin. Of these, 71 were judicial requests and 57 were non-judicial. The balance shows that:

• 110 requests were authorized
• 18 requests were not authorized

The regulatory agency clarified that the first authorizations for the use of the substance were granted even before clinical research was approved. However, after the clinical study design was approved, the new permissions began to follow the preliminary safety and efficacy data provided by the company. There was even a change in restrictions: before April, patients with injuries lasting up to 90 days could have access; subsequently, the limit was reduced to just 3 days, more in line with the criteria of the polylaminin research itself, which does not include use in old lesions.

Anvisa emphasized that, “so far, the preliminary data presented by the company only indicate the possibility of use in cases of thoracic injury between the T2 and T10 regions and within the 72-hour window after the injury.” The agency complemented the statement by highlighting that “even these preliminary data are limited and incomplete, as the product has not had a clinical study of any phase initiated anywhere in the world.” This lack of global clinical trials and the rigorous specifications of existing data reinforce the necessary caution around the substance.

Next steps for polylaminin: focus on clinical studies

For polylaminin to advance and be considered an approved medicine, it is essential that it unequivocally demonstrate its safety for use in humans and its proven efficacy for patients with spinal cord injury. Currently, the clinical study that was already approved by Anvisa at the beginning of this year has not yet started.

According to Tatiana Sampaio, the first stage of the research involves recruiting five volunteers and will focus mainly on validating the substance’s safety. This study will be conducted by teams from the HC (Hospital das Clínicas) of USP (University of São Paulo) and Santa Casa de São Paulo. Successful completion of these phases is an essential requirement and a critical milestone for polylaminin to eventually gain definitive approval as a treatment. The scientific community and patients eagerly await the results of these formal investigations.