Stanford-led study offers relief to children with dangerous food allergies, leading to FDA drug approval
Medication reduces potentially deadly reactions to allergy-triggering foods like nuts, milk and wheat.
For Anabelle Terry, 12, even the most joyful gatherings were potentially dangerous.
Born with a severe peanut allergy, “I had to watch every little thing I was eating — at friends’ houses for dinner, at parties, on Halloween when I was trick-or-treating” to prevent a medical emergency, she said.
But the first-ever medication to treat food allergies, recently approved by the FDA after a successful study of volunteers like Anabelle, offers the hope of a safer future.
Regular use of the medication, called omalizumab (Xolair), reduces the risk that an accidental exposure to small amounts of an allergy-inducing food will trigger a life-threatening reaction, according to the research published in Sunday’s issue of the New England Journal of Medicine.
About 67% of people who received the injection could tolerate the equivalent of two or three peanuts without moderate to severe allergic reactions, reported the study’s senior author Dr. Sharon Chinthrajah, associate professor of medicine and pediatrics, and the acting director of the Sean N. Parker Center for Allergy and Asthma Research at Stanford Medicine. That compares to only 7% of people who had been given a placebo powder.
It works to combat symptoms caused not just by peanuts, but other common allergens such as milk, eggs, wheat, cashews, hazelnuts and walnuts.
“There is a layer of protection for our food allergy patients so that they can live their lives with a little more normalcy,” said Chinthrajah. “This is something that our food allergy community has been waiting a long time for.”
The drug doesn’t cure the allergy. Patients must still avoid the food, and keep epinephrine nearby to treat a sudden allergic reaction.
But a simple misstep — unwittingly taking a bite of a contaminated food, for instance — is no longer catastrophic. Symptoms are less serious and have a slower onset. There’s time for treatment.
“I still had a reaction,” said Anabelle, a spunky Burlingame middle-schooler who loves science. “But it didn’t come as quickly. I could withstand more.”
There’s another benefit: easing the anxiety and stress that many families experience as they struggle to cope with a child’s allergy.
“You always have to be very conscientious and wary at any kind of social engagement where there’s food,” said Anabelle’s mother, Victoria Terry. “You’re constantly asking the host: ‘Who’s making the food? Will there be nuts? What’s in the cake?’”
Almost 8% of children and 10% of adults in the United States have a food allergy, according to the U.S. Centers for Disease Control and Prevention.
Food allergies happen when the immune system — the body’s defense against infection — mistakenly treats proteins found in food as a threat, said Chinthrajah.
It responds by releasing allergic antibodies, which activate chemicals. It’s these chemicals that cause the symptoms of an allergic reaction, such as anaphylaxis. This acute physiological response — that includes lowered blood pressure, shock and constriction of the airways — can be deadly.
The new medication works by binding to the antibodies, taking them out of circulation, said Chinthrajah. “It’s interrupting the inflammatory cascade,” she said.
Until now, the most reliable strategy is to avoid any risky foods.
The only other approach to reduce the risk of food allergies, called oral immunotherapy, involves desensitization. Patients eat small amounts of allergy-triggering foods under medical care to build tolerance. But it can take months, even years, to work. And the treatment can trigger an allergic response.
“There is a real need for treatment that goes beyond vigilance and offers choices for our food-allergic patients,” Chinthrajah said.
Omalizumab, which the Food and Drug Administration originally approved to treat diseases such as allergic asthma and chronic hives, is produced by drugmakers Novartis and Roche and is distributed by a Roche subsidiary, Genentech. The list price ranges from about $2,900 a month for children to $5,000 a month for adults, according to Genentech, but is generally covered by insurance.
Without volunteers like Anabelle, the medicine would not have moved forward.
The FDA decision to approve the drug was based on the study of 177 participants, all children. Of these, about 20 lived in the San Francisco Bay Area and were treated at Stanford. The rest were treated at other trial sites in Baltimore, Washington D.C., New York City, Boston, Philadelphia, Atlanta and beyond.
Two-thirds of the children were randomly assigned to receive omalizumab injections, and one-third received an injected placebo.
The trial, which started in 2019 and is ongoing, is a major commitment for families, which need to schedule it around school, work and family, said Chinthrajah.
Injections were given once every two or four weeks, depending on the dose needed, for four months. Then the research volunteers were “challenged” by small amounts of powders made from the allergenic food, mixed in with applesauce or pudding, to see how much of each allergy-triggering food they could safely tolerate.
About 80% of patients taking omalizumab could eat small amounts of at least one allergy-triggering food without inducing an allergic reaction, 69% of patients could eat small amounts of two allergenic foods and 47% could eat small amounts of three allergenic foods.
Omalizumab was safe and did not cause side effects, other than some instances of minor reactions at the site of injection, said Chinthrajah.
Although the drug is now approved and can be ordered by family physicians, Stanford is continuing its study of the drug to answer many unresolved questions. For instance, they hope to learn how long the protection lasts, which people have the strongest response, and whether people with other allergies could also be protected.
“The injection hurts, but it is way more helpful than painful, knowing that there could be a positive outcome,” said Anabelle.
“It’s really awesome to have been a part of this amazing study that helped me, but not only me,” she said. “There are millions of other people across the world who have food allergies, too.”