DEPARTMENT OF DEFENSE - CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAMS

Treatment Spotlight: Q&A with ALSRP Investigator Dr. Sytske Moolenaar

Posted May 9, 2018
Dr. Sytske Moolenaar, Treeway

In 2016, Dr. Sytske Moolenaar, a scientist at the pharmaceutical company Treeway, received a Therapeutic Development Award (TDA) from the Amyotrophic Lateral Sclerosis Research Program (ALSRP) to develop a new formulation of the drug Edaravone for the treatment of ALS. We caught up with Dr. Moolenaar recently and asked her a few questions.

Q: Tell us about drugs used to treat ALS today. Why is Edaravone important?
A: Aside medications to treat symptoms like cramps, spasms, and fatigue, there are only two drugs approved by the U.S. Food and Drug Administration for treating ALS. For a long time, riluzole (sold as Rilutek® and Teglutik®) was the only drug approved for treating ALS. Riluzole is given orally, but only prolongs the life of people with ALS by a few months and does not prevent progression of debilitating symptoms. Edaravone (sold as Radicava®) was approved for treating ALS in 2017. It has been shown to delay the decline in functioning in people with ALS in a Japanese clinical trial.

Q: How does Edaravone work? Can it cure ALS?
A: Unfortunately there is no cure for ALS yet. Edaravone can only delay and reduce ALS symptoms. Edaravone readily crosses the blood-brain barrier and is thought to be a free-radical scavenger, but the exact mechanism by which it exerts its therapeutic effect in patients with ALS is unknown. Studies have shown that Edaravone delays the progression of debilitating symptoms of ALS. In one Japanese study, the decline in physical function in people with ALS was slowed down by 33% after 6 months of treatment.

Q: How is Edaravone administered in clinical practice?
A: Edaravone is currently available only as an intravenous medicine called Radicava and marketed by Mitsubishi Tanabe. It is administered through a needle in a vein under the supervision of a doctor at a medical facility. Treatment roughly follows a cycle of 2 weeks of daily IV infusions at the facility followed by 2 weeks of no treatment, and the cycle continues indefinitely.

Q: Can you describe Treeway’s development of an oral formulation of Edaravone?
A: As Treeway was founded by two ALS patients, we always have been in close contact with patient organizations and neurologists in the field of ALS. They have indicated that an oral version of Edaravone would be beneficial for ALS patients. Therefore, we are developing Edaravone as an oral formulation, which has been given the codename “TW001.” We aim to give patients this formulation on a daily basis, which they can take at home.

Q: Can you tell us how your ALSRP-funded project to develop TW001 is going?
A: We have done studies using our prototype oral TW001 formulation and have shown that oral Edaravone is safe and well tolerated by healthy volunteers and ALS patients. We have shown that this oral formulation gives rise to effective levels of the drug in the bloodstream. Currently, we are performing development activities on the TW001 product needed to start larger clinical trials, which are funded by the TDA. Once these activities are finalized, Treeway can start additional ALS trials.

Q: What are your next steps?
A: We are determined to make TW001 available to the patients as quickly as possible so ALS patients all over the world can take this drug and thereby delay the disease’s progress.

Treeway research team
Treeway Research Team: Dr. Moolenaar seated middle row on the left.

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Last updated Wednesday, May 9, 2018