MTPA, Massachusetts General Launching Study on Biomarkers of Radicava Response

MTPA, Massachusetts General Launching Study on Biomarkers of Radicava Response

Mitsubishi Tanabe Pharma America (MTPA), in collaboration with the Massachusetts General Hospital (MGH), is conducting a study to identify biomarkers related to amyotrophic lateral sclerosis (ALS), which may be helpful to measure patient response to Radicava (edaravone).

The study will be sponsored by MTPA, Radicava’s seller in the U.S., and led by MGH Neurological Clinical Research Institute (NCRI).

They plan to enroll 200 patients at multiple centers across the U.S., who must be new to Radicava and complete a total of six cycles (24 weeks) of treatment. Clinical assessments and several potential ALS biomarkers will be obtained prior to and at the start of the treatment, as well as at specified points over the course of the study.

Patients will be tested for biomarkers addressing a range of ALS-associated parameters: oxidative stress (4-hydroxynonenal, 8-isoprostanes, 3-nitrotyrosine, 8-hydroxy-2′-deoxyguanosine, and urate), inflammation (matrix metalloproteinase-9), nerve cell injury and death (neurofilament heavy and light chain proteins, urinary neurotrophin receptor p75), and muscle injury (creatinine).

Biomarker and disease progression results will be compared with samples stored at biorepositories and progression models, respectively.

They anticipate enrolling the first patient late this spring, and early interim analyses are planned for the end of the year.

“ALS is a complex disorder with diverse pathophysiology, and we do not currently have validated biomarkers for diagnosing or following ALS progression,” James Berry, MD, a neurologist at MGH NCRI and the study’s primary investigator, said in a press release. “This study will broaden our understanding of numerous biomarkers that may be associated with ALS. It will increase our understanding of ALS and of the biological effects of [Radicava] in people with ALS undergoing therapy.”

Radicava is an ALS-specific treatment administered into the vein (intravenously). It was developed and is marketed by MTPA and, in 2017, became the first ALS therapy approved by the U.S. Food and Drug Administration in more than 20 years.

The medicine’s approval was primarily based on favorable efficacy and safety results from a pivotal Phase 3 trial (NCT01492686), which showed that a six-month treatment significantly slowed disability progression in ALS patients, compared with placebo.

“This biomarker trial has sparked exponential interest in the ALS community, and with the help of MGH, we hope to further our understanding of the potential role these measures may have in evaluating a treatment response,” said Stephen Apple, MD, MTPA’s director of medical affairs. “We are honored to be working with the team at MGH on our first clinical trial in the U.S.”

Ana Pena, PhD Author
Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.
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Ana Pena, PhD Author
Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.
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4 comments

  1. Dave Reckonin says:

    So…Launch the drug
    Make a shed-load of money….
    Then start a study of how effective it is.

    Radicava has been used for several years in the Far East with pALS.
    No data coming out of there ?

    Something is seriously wonky here.

  2. I have bulbar ALS diagnosed last December , I am already on Riluzol 50 md bid .I am going to start Radicava in one month . How long I have to take and what investigations have to be done during that ?

  3. Robert Spotswood says:

    Instead of concentrating on the things that are not working eg, muscles, why not concentrate on things that are still working and growing, such as my hair and nails which are still growing. Start looking in that direction on how they are still growing and incorporate that into looking at ways of getting the muscles working and growing.

  4. Mark Raskin says:

    Radicava came to us from Japan. Due to all of the trials done there it got fast-tracked by the FDA into the U.S. and instantly was marketed at a ludicrous exorbitant price. Still is. I’ve been on Radicava infusions for 6 months and unknown how truly effective it is has I have regressed quite a bit since I started. Dosage protocol is 10 days within a 14 day period, then 14 days off, then back on. It doesn’t end.

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