Re-Purposing FDA-Approved Inflammation Drug for Sanfilippo

February 6, 2020

Cure Sanfilippo Foundation is sponsoring an “Open-label Study of Anakinra in MPS III” clinical trial in collaboration with The Lundquist Institute (formerly LABiomed), and Sobi (Swedish Oprhan Biovitrium AB).

This study is an open-label, single-arm, no placebo or control group trial of the IL-1 antagonist drug Kineret. The goal of the study is to determine if a number of disease symptoms can be improved by treatment with Kineret, thereby improving patients’ quality of life.

This drug is already FDA approved for several indications, one of which is a pediatric disorder. Testing an existing drug for a new disease, as in this trial, is called “drug repositioning” or “drug repurposing.”

While Kineret is approved for other diseases, it is not currently approved for Sanfilippo syndrome. This means that it is not covered by insurance programs and due to high cost, not accessible to patients “off-label”.

Pre-clinical research has shown that reducing inflammation can impact symptoms of the disease. However, unlike steriods which are often used to combat inflammation, Kineret does not carry the same side effects of long-term steriod use.

Read the trial’s full listing on

Study Description

Inflammation has been connected with disease pathogenesis in the MPS disorders. Therapies aimed at decreasing inflammation are currently being studied in many MPS disorders and showing benefits.

IL-1 is a cytokine molecule involved in the inflammatory cascade. Research into the animal model of MPS III has identified IL-1 as a major target for addressing inflammation in the body and brain of affected animals. Blocking the ability to respond to IL-1 has shown benefits in the progression of brain disease and behavior in MPS III animals. (Read more about this research at Thus, the use the IL-1 receptor blocker, anakinra (Kineret), holds significant potential to improve behavioral and other problems in children with MPS III.

This study will be assessing for beneficial changes in a number of disease related symptoms. Some of these include, but are not limited to, improved behavior, sleep, stooling, communication, mood, and mobility; as well as decreased seizure frequency, disordered movement, and fatigue as they may apply to each individual.

Administration of Kineret does not impact the primary disease enzyme deficiency of Sanfilippo syndrome. However, due to the cascade of detrimental effects from the primary disease, evidence would suggest that addressing this target in the inflammatory cascade could help slow or improve some of the secondary disease effects.

This study is open for any Type of Sanfilippo (A, B, C, D).

Study Contact Information

In December 2019, the trial was listed as “Enrolling” on

Study contact: Adolfo Morales, The Lundquist Institute, 310-357-9023,

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