ALS Biomarkers in Drug Development

Amyotrophic Lateral Sclerosis (ALS) is difficult to diagnose, because it requires ruling out other diseases, which may take months to years. Nerve cells break down and reduce functionality in the muscles they supply. ALS gradually prohibits muscle control and results in the loss of the capacity to speak, swallow, walk, move and breathe. There is a strong need for research, understanding and potential treatments in ALS. This webinar will focus on ALS biomarkers.

The first part of the webinar will provide an overview of ALS biomarkers, both fluid-based (blood, urine and cerebrospinal fluid) and non-fluid based (electrophysiology and PET/imaging). From there, the panel will focus on individual types of ALS biomarkers, discussing which are most helpful in terms of the science of the disease, including identification and diagnosis. In addition, the group will talk about the application of ALS biomarkers in drug development, addressing which are most useful and best translate in clinical research and in assessing a drug’s effect on the disease.

Specifically, the fluid-based ALS biomarkers and topics to be individually covered include genetic markers, cellular markers of neurodegeneration (markers of axon injury, neurofilaments), markers of inflammation and oxidative stress, and exploratory miRNA markers. The non-fluid-based ALS biomarkers will include electrophysiology and PET/imaging.

Biomarkers derived from blood, urine and cerebrospinal fluid provide the necessary biological information for the diagnosis and can be used as an indicator of disease progress and severity. Use of the most appropriate biomarker, in relation to a drug’s mode of action, may well be a very useful tool in better defining a study’s patient population and following the drug’s effectiveness.

It is important to note that some of the biomarkers are currently more established, understood and useful than others. The panel will touch on areas of struggle where there is lack of clarity and where researchers are not certain which biomarkers are the leads at this time. There are questions moving forward about what needs to be done and discovered, and where the gaps exist in terms of ALS biomarkers.

Finally, the panel will make specific and even controversial recommendations regarding ALS biomarkers in clinical trials. Areas to be addressed include which are must-haves in trials, which are most costly, and which are operator-dependent, as well as the ramifications in clinical research.

Register for this webinar to hear about why ALS research and biomarkers should be strongly considered in every trial moving forward.

Speakers

Ignazio Di Giovanna, PhD, Vice-President, Europe, Atlantic Research Group

Dr. Ignazio Di Giovanna, ARG Vice-President of Europe, has deep experience in the clinical research domain, having worked in the CRO field since 1990 and the pharmaceutical industry as a whole since 1986. Previously, Ignazio owned and operated the European CRO CCA Clinical Research, from 1999 until its acquisition by ARG in 2018.

Ignazio has been an influence in clinical research through his board membership (and Chairmanship) of the Institute of Clinical Research (1999-2006), Presidency of the Clinical & Contract Research Association and participation in the Biotechnology & Pharmaceuticals Sector Advisory Group, advising and influencing the UKTI Department to improve the UK’s standing as a clinical research market. Ignazio was co-editor of “Principles in Clinical Research,” published in 2001, and was awarded an Honorary Fellowship by the Institute of Clinical Research in April 2004. Ignazio earned a PhD in Biophysics at Guys Hospital, based on the development of non-invasive cardiovascular diagnosis techniques. Ignazio has been involved in neurodegenerative disease drug development, with a focus on ALS, for over 10 years.

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Toby Ferguson, MD, PhD, Vice-President, Head Neuromuscular Unit, Biogen

Dr. Toby Ferguson is a neuromuscular neurologist and neuroscientist who joined Biogen in 2013. His professional experience has been focused on developing treatments for neuromuscular disease, with a focus on ALS. He is currently the Head of the Neuromuscular Development Unit at Biogen and leads the neuromuscular clinical development group. Toby plays a key role in developing ALS clinical trials, and in driving preclinical strategy within neurodegenerative and neuromuscular disease.

He is dedicated to the successful development of meaningful therapeutics for ALS and strongly believes that collaboration across industry, academia and advocacy organizations is crucial to providing people with ALS the therapies they need and deserve. Prior to Biogen, Toby had a clinical neuromuscular neurology practice and a lab focused on peripheral axon injury and regeneration at Shriners Research Center and Temple University in Philadelphia. Toby trained in neurology and neuromuscular disease at the University of Pennsylvania. He obtained an MD and PhD (Neuroscience) at the University of Florida and maintains an interest in axon regeneration and degeneration.

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Angela Genge, MD, Executive Director, Clinical Research Unit (CRU), Montreal Neurological Institute

Dr. Angela Genge has been the Director of the Clinical Research Unit at the Montreal Neurological Institute since 2004. Her career is notable for her groundbreaking research, prominent leadership positions, and significant distinctions particularly in neurodegenerative diseases. Angela’s research focuses on drug development in rare neurological diseases, and she is known for clinical and clinical trial design expertise as well as the introduction of innovative therapies. She led programs in the ALS, FTD, genetic forms of myopathies, and myasthenia gravis.

Angela completed her medical degree at the Memorial University of Newfoundland. She completed her Canadian and American certifications in Internal Medicine and Neurology prior to completing a fellowship in neuromuscular diseases. She joined the staff of the Montreal Neurological Hospital in 1994 and became Director of the ALS Clinic in 1998. Angela has distinguished herself in her career, receiving the Forbes Norris Award, the DIVA of Distinction Award, and the Governor General Diamond Jubilee Award.

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