When Alessandro Barbera was rushed to a California hospital with infant botulism in October, his father had heard almost nothing about the disease, let alone the rare and expensive treatment that likely saved the newborn's life.
However, Tony Barbera is now deeply grateful to BabyBIG, the only antidote to the paralyzing and potentially fatal diseases associated with contaminated ByHeart infant formula.
“It's extremely remarkable,” said Barbera, 35, whose son is slowly recovering.
botulism outbreak tethered to ByHeart formula sickened at least 39 children in 18 states. from August — and showed the value of treatments derived from blood plasma donated by a small group of scientists and other volunteers.
“It's almost a miracle,” said Dr. Vijay Viswanath, a pediatric neurologist at Children's Hospital Los Angeles who has treated several children with botulism in his career, including one during the current outbreak.
“Before BabyBIG opened, some of these hospitalizations would have taken two or three months,” Viswanath said, if infected children had recovered at all.
Licensed in 2003, BabyBIG is a trademark for Human Botulism Immunoglobulin, an intravenous drug that uses antibodies from volunteers vaccinated against botulism to help children too young to fight the disease on their own.
The treatment was the brainchild of the late Dr. Stephen Arnon, who was a scientist at the California Department of Public Health. In 1976, Arnon and his colleagues identified a rare form of botulism that affects children under 1 year of age and then devoted their 45-year career to figuring out how to treat it. The disease occurs when children ingest botulism spores, which germinate in the intestines and produce a dangerous toxin that attacks the nervous system.
More than 3,700 children worldwide have been treated with BabyBIG since Arnon and his team conducted a landmark clinical trial in California in 1997 that showed the drug could shorten hospital stays and reduce the need for breathing machines.
BabyBIG is produced in small batches every five years and costs nearly $70,000 per treatment, according to the California Infant Botulism Treatment and Prevention Program, which Arnon founded. According to state law, fees from the sale of the drug are used only to fund the botulism control program.
The cure depends on donors like Nancy Shine, a 76-year-old retired biochemist in California who was vaccinated against botulism because she worked with the deadly microbe in a laboratory. Arnon first recruited Shine and other scientists to the BabyBIG project two decades ago because their blood produces high levels of antibodies, blood proteins that neutralize botulism toxin.
An early protocol required volunteers to receive booster doses of an investigational botulism vaccine, also used by the U.S. military, and then undergo a procedure to collect blood plasma containing antibodies against botulism types A and B.
“It was not very pleasant to get the vaccine,” Shine recalled. “There were a lot of side effects, such as large scars from the vaccine and some pain.”
However, Shine was involved in the production of three batches of the antitoxin produced between 2008 and 2019.
“Probably the highlight of my career was actually being able to participate in this project and become a plasma donor,” Shine said. “We've created a product that can save babies' lives.”
Because infant botulism is rare, with fewer than 200 cases reported annually in the U.S., finding funding and other resources to develop BabyBIG's treatment took nearly 15 years and $10.6 million—and faced significant obstacles, Arnon noted. in a 2007 article.
Today, about 30 people on average provide plasma for each batch of BabyBIG, California health officials said. Lot 8, the latest version, is currently being manufactured at Takeda Pharmaceutical Co.'s plant, according to Giles Platford, president of the company's plasma therapy division. near Los Angeles. The company contracts with California officials to produce BabyBIG on a “not-for-profit basis,” Platford said.
Some of the first donors, like Shine, have already dropped out of the BabyBIG program, which collects blood from adult volunteers under the age of 70. New donors are accepted, but they must enroll in a clinical trial run by the California Department of Public Health and agree to receive a booster dose of another experimental botulism vaccine.
California officials estimate they have enough BabyBIG in reserve to last until next summer, based on current projections. The ByHeart outbreak is part of an alarming rise of at least 107 cases of infant botulism treated in the U.S. since August, said Dr. Jessica Khoury, senior medical officer for the government program.
Shine recently received a booklet with photos and letters from families whose children have recovered from botulism after taking BabyBIG.
“It's really wonderful. I read a couple of books every day,” she said. “Each one of them makes you want to cry.”
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