An experimental spinal cord treatment using a placenta-derived protein called polylaminin has created a frenzy in Brazil, drawing patients from across Latin America, spurring government fast-tracking, and turning the lead scientist into a national celebrity-all before its safety has been proven in a clinical trial.
Brazilian scientist Tatiana Sampaio says a peer-reviewed study is close to publication. But experts urge caution, noting the hype may outpace the science.
Marco Baptista of the Christopher & Dana Reeve Foundation called polylaminin “innovative” and “promising,” but stressed it is one of many early-stage treatments. “We need to see if it's safe and efficacious,” he said.
The drug aims to stimulate nerve regeneration. In an early human study of eight patients with complete spinal cord injuries, two died from their injuries, but all six survivors regained some voluntary motor control. One patient, Bruno Drummond de Freitas, 31, who broke his neck in a car accident, made a full recovery and now walks without assistance.
But others have not fared as well. Luiz Fernando Mozer, 38, received polylaminin after a motocross accident and has only regained sensation in his legs, leaving him frustrated. “We are eager to get results, but they just don't happen,” he said.
Brazil’s legal system allows patients to sue for access to experimental treatments. Anvisa, the health regulator, said dozens of lawsuits have been filed since the drug became widely known. Legal fees can range from $3,000 to $60,000.
Health law specialist Vanessa Patricia said some lawyers charge chronic patients for access to treatments they are not eligible for, raising concerns of exploitation.
Cristalia, the Brazilian pharmaceutical company that acquired the patent, has invested over $22 million in developing polylaminin. The company is donating the drug to acute patients under Anvisa’s compassionate-use authorization.
A Phase 1 clinical trial has been approved to assess safety in five patients treated within 72 hours of acute complete thoracic spinal cord injuries.
Natalia Pasternak, who promotes science-based public policy, said public documents do not explain Anvisa’s decision to authorize the study. “Well-detailed clinical trials that follow a solid protocol take time,” she said, “and rushing them can compromise both quality and safety.”