Healey Center at Mass General Launches First ALS Platform Trial with Five Candidate Treatments

Healey Center at Mass General Launches First ALS Platform Trial with Five Candidate Treatments

The Sean M. Healey & AMG Center at Mass General has chosen five “promising” candidate treatments from among 30 applications to test in its first platform trial for amyotrophic lateral sclerosis (ALS).

The platform trial — a type of a clinical study that tests the efficacy of multiple therapies simultaneously — is designed to advance effective and innovative treatments for people living with ALS. Newly developed clinical treatments can be added into the trial as it progresses to reduce the time from identification of a promising new therapy to its testing.

“The Healey Center brought together top leaders from around the world to boldly re-think how we design ALS trials, to create a groundbreaking platform trial program, and deliver compassionate care,” Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Mass General, said in a press release.

This clinical trial model has shown success in finding treatments for certain types of cancer. The unique trial design helps advance development of treatments by letting researchers assess more medicines, increase patient access to clinical trials, and decrease costs by rapidly determining the effectiveness of numerous treatments.

“This approach cuts the time to find an effective treatment in half, decreases costs by a third or more, and is supported by our patients, the FDA, ALS clinicians and scientists and our pharma colleagues,” Cudkowicz said.

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Further, because separate treatments are being tested within the same infrastructure and a similar master protocol, results from all subjects in all placebo groups can be combined together — leading to significantly higher statistical power for each study and therefore, more robust data.

In addition to testing different therapies, the HEALEY ALS Platform Trial also will include several promising biomarkers and outcome measures aimed at developing new tools to evaluate the effectiveness of ALS therapies.

“We are thrilled to be in a position to test several treatments for ALS and, at the same time, facilitate the development of breakthrough biomarkers that can more quickly predict therapeutic success,” said Sabrina Paganoni, MD, PhD, a faculty member at the Healey Center.

Prior to initiating this clinical trial, the Healey Center announced it was looking for the best ALS treatments preparing to enter clinical trial stages. The center received approximately 30 applications from 10 countries.

A group of leading ALS scientists and members of the Healey Center Science Advisory Committee picked what they considered to be the top five most promising treatments. Initially, the platform trial will be launched with three therapies, and two more will be added shortly after.

The experimental treatments include Ra PharmaceuticalsZilucoplan, Biohaven PharmaceuticalsVerdiperstat, Clene Nanomedicine’s Bioenergetic Nanocatalysis (CNM-Au8, nanocrystalline gold), Prilenia‘s Pridopidine, and Implicit Bioscience‘s IC14 immunotherapy.

Partners of these five chosen companies are collaborating with the Healey Center Trial Design Team, as well as the  Northeast ALS Consortium (NEALS), to design the HEALEY ALS Platform Trial.

NEALS is an international non-profit group composed of 128 research sites working together to conduct clinical research in ALS and other motor neuron diseases.

“The investigators in NEALS are enthusiastic about this approach,” says Timothy Miller, MD, PhD, co-chair of NEALS, and a professor at Washington University School of Medicine. “The ability to test multiple drugs at one time will move us more quickly toward the goal of finding a treatment for ALS.”

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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6 comments

  1. Pauline Milan says:

    My husband has ALS. He was diagnosed in Feb 2017. I would like to know more about getting him involved in the ALS Platform Trial with 5 candidate treatments to be held at the Healy Center at Mass General.
    Many thanks
    Pauline Milan

  2. Asha shettigar says:

    My mom was diagnosed with ALS in June 2018. She is currently in India. Can she be admitted in the ALS platform trial?

    Thanks,

    Asha

  3. Ronald Gruneberg says:

    My husband was diagnosed with ALS in April 2019 with undiagnosed previous symptoms. He would be very interested in registering for the platform trial.

  4. James C. Metz says:

    I was diagnosed in 2016, confirmed in January of 2017
    I went through a clinical trial at Cleveland Clinic, unsuccessful but still a piece of Hope. I went on Radicava in January of 2018, in March of this year I was hospitalized with a bleeding ulcer (not because of the drug) However I’m home bound and cannot get it anym

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