How a Rare Drug Made from Scientists' Blood Saves Babies from Botulism
When Alessandro Barbera was rushed to a US hospital with infant botulism in October, his father had barely heard of the disease, let alone the rare and costly treatment that likely saved his newborn's life. Now, Tony Barbera is deeply grateful for BabyBIG, the sole antidote to the paralyzing and potentially deadly illnesses linked to contaminated ByHeart infant formula.
"It's incredibly remarkable," said Barbera, 35, whose son is slowly recovering. The botulism outbreak tied to ByHeart formula has sickened at least 39 babies across 18 US states since August, highlighting the value of the treatment made from blood plasma donated by a small group of scientists and other volunteers.
"This is almost like a miracle," said Dr. Vijay Viswanath, a pediatric neurologist at Children's Hospital Los Angeles, who has treated several children with botulism during his career, including one in the current outbreak. "Prior to the discovery of BabyBIG, some of these hospital stays could have lasted two or three months, and recovery was far from guaranteed."
Licensed in 2003, BabyBIG is the brand name for human botulism immune globulin, an IV medication that uses antibodies from volunteers vaccinated against botulism to help babies too young to fight the disease on their own. The treatment was the brainchild of the late Dr. Stephen Arnon, a scientist with the California Department of Public Health, who identified the rare infant botulism form in 1976 and spent 45 years developing a treatment.
The disease occurs when babies ingest botulism spores that germinate in the intestine, producing a dangerous toxin that attacks the nervous system. More than 3,700 children worldwide have been treated with BabyBIG since Arnon's team conducted a pivotal clinical trial in California in 1997, demonstrating its effectiveness in shortening hospital stays and reducing the need for breathing machines.
Produced in small batches every five years, BabyBIG costs nearly $70,000 per treatment, according to the California Infant Botulism Treatment and Prevention Program, which Arnon founded. Fees from the drug's sales fund the botulism program under state law. The treatment relies on donors like Nancy Shine, a 76-year-old retired biochemist in California who was vaccinated against botulism due to her lab work with the lethal germ. Arnon recruited Shine and other scientists two decades ago because their blood produced high levels of antibodies that neutralize the botulism toxin.
The early protocol required volunteers to receive booster doses of an investigational botulism vaccine and undergo a procedure to harvest blood plasma containing antibodies against botulism types A and B. "It wasn't pleasant, but it was worth it," Shine recalled, having contributed to three batches of the antitoxin between 2008 and 2019. "Participating in this project and donating plasma was the highlight of my career. We created a product that can save infants' lives."
However, developing BabyBIG faced significant challenges due to the rarity of infant botulism, with fewer than 200 cases reported in the US annually. Finding funding and resources took nearly 15 years and $10.6 million, and the process faced substantial hurdles, as noted by Arnon in a 2007 article. Today, about 30 people provide plasma for each batch of BabyBIG, with Batch 8 currently being manufactured at a Takeda Pharmaceutical Co. plant near Los Angeles.
The company contracts with California officials to produce BabyBIG on a not-for-profit basis, according to Giles Platford, president of the plasma-derived therapies unit. Some early donors, like Shine, have aged out of the program, which accepts adult volunteers up to age 70. New donors must enroll in a clinical study directed by the California health department and agree to receive a booster dose of a different investigational botulism vaccine.
California officials estimate their reserves of BabyBIG will last until next summer, based on current projections. The ByHeart outbreak is part of a worrisome rise in infant botulism cases treated in the US since August, according to Dr. Jessica Khouri, the senior medical officer for the state program. Shine recently received a booklet filled with photos and letters from families whose children have recovered from botulism after receiving BabyBIG, finding the experience deeply moving.
