Little-tested spinal cord treatment triggers hope, hype and lawsuits in Brazil

Cristalia Lab in Brazil. (Reuters)

SAO PAULO — An experimental treatment for spinal cord injuries has ignited a frenzy in Brazil, drawing patients from as far off as Mexico to a small-town laboratory, mobilizing the government to expedite approval and turning a scientist into a national celebrity – all before its safety has been proven in a clinical trial.

The buzz around polylaminin, a placenta-derived protein aimed at promoting nerve regeneration, has lured interest from thousands of potential patients worldwide, with dozens hiring lawyers to gain access to the drug.

Absent clinical proof of safety and efficacy, experts are urging caution while trying to separate facts from hope.

Animal studies since the early 2000s suggest polylaminin can stimulate regeneration of damaged nerve fibers.

Though Phase 1 clinical trials are still underway, public interest in polylaminin exploded after a 2024 preprint of a study was promoted last year by Cristalia, the Brazilian pharmaceutical company that acquired the patent.

The lead researcher, Brazilian scientist Tatiana Sampaio, told Reuters a peer-reviewed study of polylaminin is close to publication but declined to name the journal.

The public response in Brazil has been far-reaching, with popular Brazilian musician Joao Gomes calling out Sampaio at a Carnival parade this year: “You are the biggest celebrity here,” he said.

The enthusiasm is a matter of national pride.

“A female scientist made a groundbreaking discovery for the world, and she is Brazilian,” Sampaio said.

Government bullish, scientists wary

If proven safe and effective, polylaminin would be a big win for a Brazilian industry that has long prioritized generic drugs and a country that invests just 1.2 per cent of GDP in research and development, according to 2023 World Bank data.

The government of President Luiz Inacio Lula da Silva, who is running for reelection this year, has seized on the optimism.

“It is something so important to us that it was the first product analyzed by the newly created Innovation Committee at Anvisa,” Health Minister Alexandre Padilha said of the state health regulator. “If the clinical study yields good results, it can be made available to the population as quickly as possible.”

Some experts warn not to let enthusiasm get ahead of science.

Marco Baptista, chief science officer at the Christopher & Dana Reeve Foundation, called polylaminin “innovative” and “promising,” but noted it was one of many experimental treatments undergoing early testing.

Baptista said the foundation, founded by the “Superman” actor after a 1995 equestrian accident left him paralyzed, follows promising science around the world but does not pin too much hope on preliminary findings.

“It is not the only approach out there and we need to see if it’s safe and efficacious,” Baptista said.

Prominent voices in Brazil’s scientific community also urge caution.

“We want the research to be successful... but we need to meet certain criteria,” said Delson Jose da Silva, president of the Brazilian Academy of Neurology.

Mixed results

The first human polylaminin study involved eight patients with complete spinal cord injuries, half paraplegic, half tetraplegic, treated within days of injury.

Two died due to their injuries. But all six survivors regained some degree of voluntary motor control and one, who suffered a broken neck in a car accident, made a full recovery after two years.

“I believe polylaminin was crucial for my recovery,” said Bruno Drummond de Freitas, 31, who is now able to walk without assistance.

Freitas, who was 23 when he was injured and received polylaminin within 24 hours, has become a prominent voice for the treatment.

Some experts said hype over the promise of the drug could lead to disappointment.

Luiz Fernando Mozer, a 38-year-old auto painter, received the treatment a few days after a motocross accident in early December. The only improvement he has experienced was the return of sensation in his legs.

The slow recovery has left him frustrated. “We are eager to get results, but they just don’t happen.”

Lofty legal fees

Brazil’s legal system allows patients to secure access to experimental treatments through the courts. Since news of the drug became public, dozens of lawsuits seeking access to polylaminin have been filed, Anvisa said.

The hype could leave desperate patients open to exploitation. Health law specialist Vanessa Patricia said she has seen cases where lawyers charge chronic patients for access to a treatment they are not eligible for.

Sampaio said animal studies and early evidence from humans suggest polylaminin works best within 72 hours of injury and up to three months after.

Some patients have turned to lawyers as Anvisa can take up to 45 days to rule on such requests.

Anvisa has begun fast-tracking approvals in cases where there was a clear indication for the treatment, making legal intervention less necessary, Patricia said.

So far, 84 patients have received Anvisa’s authorization for compassionate polylaminin use, 44 of them through court orders, the regulator said.

Legal fees to represent patients seeking polylaminin access can range from 15,000 to 300,000 reais (US$3,000 to $60,000).

Clinical trial approved

Sampaio said she can no longer keep up personally with all of the cases, which may come with 24-hour legal injunctions requiring treatment before researchers have time to fully assess each case. But she said she remains committed to the work and hopes trials will prove the drug’s clinical worth.

Rogerio Almeida, vice president of research and innovation at Cristalia, said it is donating the drug to acute patients following Anvisa’s green light.

Cristalia, based in the countryside of Sao Paulo state, has invested over 110 million reais (US$22 million) developing polylaminin.

The Phase 1 trial authorized by Anvisa will assess the safety of polylaminin in five patients aged 18 to 72 who were treated within 72 hours of acute complete thoracic spinal cord injuries requiring surgery.

Natalia Pasternak, who runs a non-profit promoting science-based public policy, said public documents do not explain Anvisa’s decision to authorize the study. She is seeking further documentation in court.

“Well-detailed clinical trials that follow a solid protocol take time,” she said, “and rushing them can compromise both quality and safety.”

Reporting by Luciana MagalhaesEditing by Brad Haynes and Bill Berkrot, Reuters