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New Treatment for Phenylketonuria (PKU) Is in the Works

Too much phenylalanine in the body can cause brain damage, seizures, tremors, and behavioral or emotional problems, so those with PKU must eliminate phenylalanine from their diet. Unfortunately, this diet isn’t easy. Foods like milk, dairy products, meat, fish, chicken, eggs beans, nuts, aspartame-containing foods, breads, and pasta must be completely avoided in order to prevent against phenylalanine build up in the body.

Luckily, scientists have been trying to develop a new treatment, SYNB1618, that would help reduce the negative effects of protein-containing foods in those with PKU. If the FDA approves it, SYNB1618 will be one of the first medications to treat this rare disease.

What is the new PKU treatment?

Syn1618 is made of bioengineered E. coli bacteria and is designed to work in the gastrointestinal tract. The bacteria would break down phenylalanine before it builds up, allowing those with PKU to eat protein-containing foods.

While the treatment may not be available for some years, the manufacturer, Synlogic, is recruiting patients for a clinical trial that will help determine a safe dose range for Syn1618 in those with PKU.

Are there any other ways to treat PKU?

At present, a PKU-specific diet is the main way to control symptoms. Fortunately, there are options out there to help patients who are on this diet. One company, VitafriendsPKU, makes medical foods to help those with PKU get the nutrients they need while on a protein-free diet.

To date, only two other medications have been approved to treat PKU—Palynziq, which was just approved in May, and Kuvan. Both of these treatments can be expensive and have serious side effects. Additionally, Kuvan only works for a specific subset of PKU patients.

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