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Spotlight on MND


Explainer: Platform trials for MND

Less than 1 in 20 new treatments that are tested in clinical trials make it through to the regulatory approval stage1.
In addition, these trials to ensure a treatment for MND is safe, and actually works2 can take several years. Years that so many people living with MND simply don’t have.
But what if the search for an effective MND treatment could be sped up? Can clinical trials be designed differently to help save time, money, effort and, crucially, people’s lives?
One of the most exciting and innovative developments in MND research does just that: platform trials.
We took a look at two major new MND platform trials being run in the US and Europe, including how they work, what makes them different, Australia’s role in helping to increase patient participation, and why they’re important.

What are Platform Trials for MND and how do they work?

A platform trial is a flexible, ‘adaptive’ way3 of running a randomised control trial (RCT), also known a clinical trial.
Platform trials are able to test multiple treatments for MND, at the same time1.
Like a standard clinical trial, a platform trial for MND randomly assigns participants into treatment and control/placebo groups, as well as applying the usual strict scientific approaches to ensure accurate and objective outcomes. A platform trial has a number of key differences, however, and they tend to be:
  • testing multiple treatments simultaneously
  • use of ‘shared’ control groups
  • the ability to expand to incorporate promising new treatments as they become available, even while the trial is underway1.
  • use of broader inclusion criteria.
With broader inclusion criteria and multiple treatment groups, more people are able to participate in platform trials3.

Where are MND platform trials being run?

There are three major platform trials for MND currently in development. The trials are being held in the US, Europe and in Scotland.
The first ever platform trial is underway in the US at the HEALEY Centre, Massachusetts General Hospital.
The second trial, TRICALS, is a European initiative being held in multiple locations. Sixteen countries are involved in the TRICALS project, including Australia, enabling access for Australian patients.
Another trial with a similar design to that of the HEALEY Centre has also recently commenced in Scotland, the MND SMART trial. Unfortunately, this has currently been paused due to COVID-19. 

Why use platform trials for MND?

One of the major reasons that platform trials are being used for MND is that they are more efficient by reducing the cost and time required1.
Platform trials can identify which treatments are most promising or not, and focus more time and resources on treatments that are more likely to benefit people living with MND.
Another significant reason is that with more people living with MND able to participate in platform trials, it is possible to improve the quality of the research. With more participants, it’s easier to determine whether any benefits from a treatment are experienced by many people, and are not just a random effect in only a few people.
Platform trials have been used effectively for other life shortening and neurodegenerative diseases such as breast cancer4 and Alzheimer’s disease5.
Platforms trials for MND are seen as an important option to explore given there is still no cure for the disease, and treatment and care options are limited. Finding funding for research can be challenging and innovative approaches like platform trials help secure support to improve our chances of developing new treatments.

What do HEALEY and TRICALS hope to achieve?

The HEALEY ALS platform trial and TRICALS are both testing multiple new treatments for ALS/MND in parallel using flexible and adaptive techniques, but in different ways.
TRICALS have based their trial design on extensive data collection from patients with ALS/MND from across the world, drawing on the research of Project MinE. A better understanding of the variability in different patient’s disease enables them to design highly specific and efficient trials that also ensure maximum flexibility of patient eligibility and better targeting of the different treatment options to individual patients.
The HEALEY ALS platform trial, like similar studies in cancer drug development, is testing multiple drugs at the same time, using specialized statistical tools. There is only one placebo group, however, meaning that 80% of patients receive a treatment rather than a placebo. New drugs are to be added to the trial as they become available. It’s hoped that a number of promising drugs can be efficiently tested, and with increased access for people with ALS/MND.

COVID-19 and MND Research

Researchers in Australia and across the world are closely monitoring the impact of COVID-19 on all MND studies, including MND platform trials.
The conduct of MND research in Australia, and how it responds to the daily changes and impacts resulting from COVID-19, is guided by various university and Australian government policies, legislation and recommendations, including those of the Department of Health, NHMRC and Therapeutic Goods Administration (TGA). All efforts are being made to help continue clinical trials and other research into MND during this challenging time.
If you are concerned about participating in clinical trials for MND due to issues with COVID-19, please contact the centre running the relevant trial. All trial centres are well-aware of the potential risk and are taking all precautions to minimise exposure risk.
This article was reviewed by the following expert: Dr David Schultz, Head of Neurology and Stroke, Flinders Medical Centre, South Australia.
Read more about platform trials
Ground-breaking MND drug trial launched: Clinical drug trial will test multiple new treatments for MND in Scotland
Global collaboration key to ending MND: Report on 30th International Symposium on ALS/MND
The Platform Trial: An Efficient Strategy for Evaluating Multiple Treatments

As a footnote, we would like to acknowledge the passing of Sean M Healey on May 27 2020 from ALS. Mr. Healey was a former President and CEO of AMG where he oversaw its growth into a $600 billion company. In November 2018, with support from AMG, friends, and colleagues, he established The Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital (MGH) and has raised more than $40 million to date. The Healey Center is the largest hospital-based ALS research program in the world and supports a broad range of early-stage trials of promising ALS treatments, including the first-ever ALS platform trial described above.

1. Eijk, Ruben P. A. van, and Angela Genge. 2020. “The Rise of Innovative Clinical Trial Designs: What’s in It for Amyotrophic Lateral  Sclerosis?” Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration 21 (1–2): 3–4.
2. Jaiswal, Manoj Kumar. 2019. “Riluzole and Edaravone: A Tale of Two Amyotrophic Lateral Sclerosis Drugs.” Medicinal Research Reviews 39 (2): 733–48.
3. Berry, Scott M., Jason T. Connor, and Roger J. Lewis. 2015. “The Platform Trial: An Efficient Strategy for Evaluating Multiple Treatments.” JAMA 313 (16): 1619–20.
4. Chien, A. Jo, Debasish Tripathy, Kathy S. Albain, W. Fraser Symmans, Hope S. Rugo, Michelle E. Melisko, Anne M. Wallace, et al. 2019. “MK-2206 and Standard Neoadjuvant Chemotherapy Improves Response in Patients With  Human Epidermal Growth Factor Receptor 2-Positive and/or Hormone Receptor-Negative  Breast Cancers in the I-SPY 2 Trial.” Journal of Clinical Oncology : Official Journal of the American Society of Clinical  Oncology, February, JCO1901027.
5. Bateman, Randall J., Tammie L. Benzinger, Scott Berry, David B. Clifford, Cynthia Duggan, Anne M. Fagan, Kathleen Fanning, et al. 2017. “The DIAN-TU Next Generation Alzheimer’s Prevention Trial: Adaptive Design and  Disease Progression Model.” Alzheimer’s & Dementia : The Journal of the Alzheimer’s Association 13 (1): 8–19.


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