SAO PAULO – A controversial experimental therapy for spinal cord injuries has created a medical phenomenon in Brazil, attracting patients from countries like Mexico to a small laboratory while propelling a researcher to celebrity status – all without completed safety trials.
The experimental therapy uses polylaminin, a protein derived from placenta tissue designed to encourage nerve tissue regrowth. This treatment has captured worldwide attention from thousands of prospective patients, with many securing legal representation to obtain access to the medication.
Without completed clinical evidence proving both safety and effectiveness, medical professionals are advocating for careful consideration while attempting to distinguish between scientific reality and patient optimism.
Research conducted on animals beginning in the early 2000s indicates polylaminin may encourage damaged nerve tissue to regenerate.
While Phase 1 human trials continue, widespread public fascination with polylaminin surged following promotion of a 2024 preliminary study by Cristalia, the Brazilian drug manufacturer that owns the patent rights.
Brazilian researcher Tatiana Sampaio, who leads the research team, informed Reuters that a peer-reviewed polylaminin study nears publication but would not identify which medical journal will publish it.
Brazil’s public reaction has been extensive, with well-known Brazilian performer Joao Gomes acknowledging Sampaio during this year’s Carnival celebration: “You are the biggest celebrity here,” he declared.
This excitement represents national pride for Brazil.
“A female scientist made a groundbreaking discovery for the world, and she is Brazilian,” Sampaio stated.
**OFFICIALS OPTIMISTIC, RESEARCHERS CAUTIOUS**
Should polylaminin prove both safe and effective, it would represent a significant achievement for Brazil’s pharmaceutical sector, which has traditionally focused on generic medications, and for a nation investing only 1.2% of its economic output in research and development, based on 2023 World Bank statistics.
President Luiz Inacio Lula da Silva’s administration, currently seeking reelection, has embraced this positive outlook.
“It is something so important to us that it was the first product analyzed by the newly created Innovation Committee at Anvisa,” stated Health Minister Alexandre Padilha regarding the national health regulatory agency. “If the clinical study yields good results, it can be made available to the population as quickly as possible.”
Several medical professionals caution against allowing excitement to outpace scientific rigor.
Marco Baptista, chief science officer at the Christopher & Dana Reeve Foundation, described polylaminin as “innovative” and “promising,” while emphasizing it represents just one among numerous experimental therapies in early development phases.
Baptista explained that the foundation, established by the “Superman” actor following a 1995 horseback riding accident that caused his paralysis, monitors encouraging research globally but avoids placing excessive confidence in initial findings.
“It is not the only approach out there and we need to see if it’s safe and efficacious,” Baptista commented.
Leading figures within Brazil’s research community also recommend caution.
“We want the research to be successful… but we need to meet certain criteria,” stated Delson Jose da Silva, president of the Brazilian Academy of Neurology.
**VARYING OUTCOMES**
The initial human polylaminin research included eight patients with complete spinal cord damage, with four experiencing paraplegia and four experiencing tetraplegia, all treated within days of their injuries.
Two patients died from their injuries. However, all six surviving patients recovered some voluntary movement ability, and one patient who sustained neck injuries in an automobile crash achieved complete recovery within two years.
“I believe polylaminin was crucial for my recovery,” stated Bruno Drummond de Freitas, 31, who now walks independently.
Freitas, injured at age 23 and treated with polylaminin within 24 hours, has become an advocate for the therapy.
Some medical professionals warned that excessive excitement about the treatment’s potential could result in disappointment.
Luiz Fernando Mozer, a 38-year-old automotive painter, underwent treatment several days following a motocross crash in early December. His only improvement has been restored feeling in his legs.
His gradual progress has caused frustration. “We are eager to get results, but they just don’t happen.”
**EXPENSIVE LEGAL COSTS**
Brazil’s court system permits patients to obtain experimental treatments through legal proceedings. Following public disclosure of the medication, dozens of legal cases requesting polylaminin access have been submitted, according to Anvisa.
This excitement could expose vulnerable patients to financial exploitation. Health law expert Vanessa Patricia reported observing situations where attorneys charge patients with chronic conditions for treatments they cannot receive.
Sampaio explained that animal research and preliminary human evidence suggest polylaminin functions most effectively within 72 hours of injury and up to three months afterward.
Some patients have sought legal assistance because Anvisa may require up to 45 days to decide on such applications.
Anvisa has started expediting approvals when clear treatment indications exist, reducing the need for court involvement, Patricia noted.
Currently, 84 patients have obtained Anvisa’s permission for compassionate polylaminin treatment, with 44 receiving approval through court decisions, the regulatory agency reported.
Attorney fees for representing patients seeking polylaminin access range from 15,000 to 300,000 reais ($3,000 to $60,000).
**CLINICAL STUDY AUTHORIZED**
Sampaio acknowledged she can no longer personally manage all cases, which may include 24-hour court orders requiring immediate treatment before researchers can thoroughly evaluate each situation. However, she remains dedicated to the research and hopes trials will demonstrate the medication’s clinical value.
Rogerio Almeida, vice president of research and innovation at Cristalia, confirmed the company provides the medication at no cost to patients with acute injuries following Anvisa’s approval.
Cristalia, located in rural Sao Paulo state, has invested more than 110 million reais ($22 million) in polylaminin development.
The Phase 1 study approved by Anvisa will evaluate polylaminin’s safety in five patients between ages 18 and 72 who received treatment within 72 hours of complete thoracic spinal cord injuries requiring surgical intervention.
Natalia Pasternak, who directs a nonprofit organization promoting evidence-based public policy, noted that public records do not clarify Anvisa’s rationale for authorizing the study. She is pursuing additional documentation through legal channels.
“Well-detailed clinical trials that follow a solid protocol take time,” she explained, “and rushing them can compromise both quality and safety.”
($1 = 5.0109 reais)
