Could a dose of an enzyme, administered through food by the use of “sprinkles,” help reduce the symptoms of autism?
That possibility is being investigated in a clinical trial at two New Jersey locations. Both are looking for children ages 3 to 8 to be study participants.
The drug, called blüm, is based on research that showed children with autism often have a shortage of certain enzymes that result in the inability to digest protein.
That in turn affects the availability of amino acids, which are essential for brain function.
“There’s a growing body of research on the links between the gut and the brain, so there seems to be some connections there,” said Mark Mintz, the pediatrician running the study site at the Clinical Research Center of New Jersey, in Voorhees.
A second study site is in Toms River, at Children’s Specialized Hospital research center.
The goal of the treatment is to reduce irritability, agitation, and hyperactivity among the children who participate. A secondary effect might be to produce some improvement in social interaction, Mintz said.
The substance under study is a biologic, an enzyme instead of a drug. That means it’s comparable to Lactaid, the commercial product containing the lactase enzyme that people with lactose intolerance are lacking.
The study is a Phase III Food and Drug Administration clinical trial, which means the medicine has already proved to be safe.
This newest round of research is one in which some of the children will receive the medicine, while others will receive a placebo, or inert substance. Neither the researchers nor the parents will know which children got the drug until the study is concluded.
The biologic will be administered in the form of “sprinkles” that are put atop food. Parents will have to keep logs of a child’s behavior, and bring in a stool sample once a month.
The study lasts for 14 weeks and participation is free. Participants can also get reimbursed for travel costs as well.
Children will have an initial behavioral assessment to determine if they qualify. Mintz said in general the study is looking for children who have moderate-to-severe autism who are either non-verbal or minimally verbal, or who are already receiving services for their diagnosis.
The study, now beginning at 25 sites nationally, is being run by the biologic’s developer, Curemark, a drug research and development company.
At this time, it is limited to children ages 3 through 8.
“If you’re going to make a change in autism, it’s better to start earlier,” Mintz said.
Kathleen O’Brien may be reached at email@example.com. Follow her on Twitter @OBrienLedger. Find NJ.com on Facebook.