An experimental drug reduced the likelihood of a serious allergic reaction to peanuts in some children and adolescents with peanut allergies, according to clinical trial results presented Sunday.
Researchers say the findings could lead to the first-ever FDA-approved oral immunotherapy regimen that could reduce children's allergic response to peanuts in the case of an accidental exposure.
The trial will be published Thursday in the New England Journal of Medicine.
Peanut allergies have grown in prevalence
According to the American College of Allergy, Asthma, and Immunology, the incidence of peanut allergies in children in the United States has increased by 21% since 2010. Almost 2.5% of children in the United States have a peanut allergy. For some children, the allergies can be deadly—especially in cases of unexpected exposure.
Doctors say that the only way to prevent severe allergic reactions is to avoid peanuts entirely. But, a new late-stage clinical trial conducted by Aimmune Therapeutics reveals that oral immunotherapy, or slowly introducing foods with small amounts of peanuts into children's diets, can reduce the chance of a potentially life-threatening allergic reaction.
The clinical trial tested the effectiveness of Aimmune Therapeutics' "peanut-derived investigational biologic oral immunotherapy" drug AR101 at reducing the severity of allergic reactions to peanut protein, according to the New York Times. The drug is made out of peanut flour and packaged in capsules or foil sachets so the contents can be mixed with unheated food.
The study included 551 people with peanut allergies—most of whom were between the ages of four and 17. At the start of the study, all of the participants had allergic reactions upon exposure to 100 milligrams of peanut protein—the equivalence of one third of a peanut.
The participants in the test group consumed a three-milligram dose of the drug every day for two weeks. After two weeks, the participants increased their daily dose for six months with the goal of reaching the maximum dose of 300 milligrams, which is equivalent to one peanut. After six months, the participants on the drug regimen took a maintenance dose of 300 milligrams for an additional six months.
At the conclusion of the maintenance phase, researchers tested how much peanut protein the participants could consume before experiencing a severe allergic reaction.
After treatment, many participants could tolerate a small amount of peanut
After one year, 67.2% of the children on the AR101 regimen experienced mild symptoms after ingesting 600 milligrams of peanut protein—about two peanut kernels—and 50% were capable of consuming 1,000 milligrams of peanut protein without severe symptoms. Only 4% of children in the placebo group were able to consume 600 milligrams without experiencing a severe reaction.
The researchers observed severe adverse reactions in 4.3% of participants in the test group and less than 1% of the control group.
The researchers did not find the treatment effective among the adults enrolled in the study.
Nearly all of the participants had an adverse allergic reaction to the peanut protein over the course of the trial. Among the active treatment group, 34.7% had mild reactions, 59% of participants had moderate reactions, and 4.3% had severe reactions. By contrast, among the placebo group, 50% had mild reactions, 44% had moderate reactions, and less than 1% had severe allergic reactions.
A new hope for children with peanut allergies
According to Brian Vickery, the lead author of the study and former employee of Aimmune, the majority of research on oral immunotherapy is "flawed, and small, or uncontrolled." But the new late-stage clinical trial "has generated the highest quality of evidence to date about whether oral immunotherapy works and how safe it is," he said. He added, "[the study] is the most definitive look at whether this treatment is right for patients."
While the study revealed that children are at risk of having a moderate or mild reaction to the medication regimen, Vickery said the benefit is protection against a life-threatening reaction. "You exchange that uncertain, unpredictable risk of having an accidental reaction that spirals out of control for these sort of lower level, mostly mild or moderate symptoms, that are manageable for most patients," he said.
Carter Grodi, a high school junior who participated in the clinical trial, used to avoid peanuts entirely. After participating in the trial, Grodi no longer has to live in fear of encountering unexpected traces of peanut protein in his meals.
"To me, a peanut was like a weapon, like you have a gun to your head and you could have the trigger pulled any time," Grodi said. But, with the medication "[t]here's not really that gun anymore. I have that protection, that shield for the amount I can tolerate."
Daniel Adelman, Aimmune CMO and lead author of the study, said the company will submit a license application to FDA by the end of 2018. The drug would go through an accelerated process for FDA approval, which means it could be on the market by the end of 2019, according to a spokesperson.
Demand for the drug is expected to be high, according to the Wall Street Journal.
Carolee Grodi, Carter's mother, said the new treatment's possible approval is "pretty exciting," for children and the parents of children with peanut allergies. "It's not eradication of the allergy, it's just protection against an accidental exposure and that's huge," she said. "People don't realize it, but peanuts are everywhere" (Loftus, Wall Street Journal, 11/18; Caryn Rabin, New York Times, 11/8).
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