Biomarkers to Accelerate ALS Therapy Development

In this video, Merit Cudkowicz, MD, MSc, chief of Neurology Service, director of the Sean M. Healey & AMG Center for ALS, director of the Amyotrophic Lateral Sclerosis (ALS) Clinic and the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital and Partners HealthCare, discusses biomarkers that can help to diagnose patients quicker and target individualized treatments to patients.

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This is Part 3 of a three-part series on ALS treatment.

• Part 1
• Part 2

Transcript

People are now talking a lot of about personalized medicine and I think this is really important in ALS. ALS is likely not one illness and therefore, one drug for everybody is probably not going to be the solution.

What biomarkers can do is help us identify what biology is happening in an individual person and then target treatments much better to that person as well as in a different phase of the illness, the technology to devote. Biomarkers exist now. It really didn’t before, which is why they don’t have any but now between imaging and ways to look at stems cells from people and look at their blood proteins, we really are starting to develop these signatures to be able to better tailor our treatments.

All this requires funding and philanthropy is so important, so I’m speaking up for the whole process.

Biomarkers are important for a couple of things. One is that they can help diagnose people quicker. Right now it takes about 12 months for the first symptom to when someone’s diagnosed with ALS and that’s way too long. We want to get that down to three months, two months so that we can start caring for people much sooner.

I think that we’ll have much better success and really being able to slow that illness if we can diagnose people sooner. So, it’s really important to develop those type of biomarkers whether that’s going to be an imaging test or a blood test. There’s a lot of progress in that and ALS and people working all over the world and developing those type of biomarkers.

The other way biomarkers are really helpful are to help us screen for treatments much faster. Every disease that has developed a biomarker of drug effect has seen an exponential growth in treatments coming up to the market for their patients, so there’s a huge effort to develop those type of biomarkers—they’re called surrogate biomarkers for ALS.

So here at MGH, we have a huge program to develop those types of biomarkers using advanced technologies and imaging as well as biomarkers in fluids such as blood or urine or spinal fluid. Patients participate in those studies are enormously important so that they’re helping push that field forward faster.

One of the ideas we’d like to do with biomarkers is called a platform trial. This is where you have, let’s say, five or six food ideas of treatments that are ready to bring to patients and you can test them all in the same trial using biomarkers to tell early within eight weeks, 12 weeks is your drug going to work or not so then you can keep bringing only the best treatments forward for your patients.

Learn about the ALS Multidisciplinary Clinic at Mass General

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