Puzzle Pieces Are Taking Shape in ALS Research

by Jane Larkindale, D.Phil. on Wed, 2013-05-15 05:00

MDA Vice President of Research Jane Larkindale

Speaking as a scientist with an interest in drug discovery, ALS is hard. Well, that’s probably pretty obvious, as if it were easy, we’d have a cure by now. When I first came into the field and whenever I thought about ALS, I felt stupid — I am missing something here! I just don’t get it! As time passed, I realized that it wasn’t just me — no one really understands ALS. At the time, no one even seemed particularly close. That was only five years ago. It’s amazing how much has changed!

I’m not saying we all have a clear understanding of all ALS now (I wish I could say that). However, when I go to meetings and talk to scientists, I now have a strong feeling that the puzzle pieces that used to be just a jumbled pile, are beginning to take shape. I feel like the overall picture is tantalizingly close, even if I can’t quite grasp it yet. We are beginning to see connections between the fantastic work in lab A and the equally good work in lab B — which in the past seemed completely unconnected, possibly even contradictory. We are not there yet, but we are beginning to have some good leads.

So where does that put us with therapy development? Well, without a solid understanding of the disease, it is difficult to develop therapies — essentially each try is a shot in the dark. So far, we still don’t have a big picture view of all of ALS, but we are beginning to understand small parts — and where we understand, we work fast. Take the discovery of a new form of familial ALS, caused by flaws in a region of the genome called C9ORF72. We didn’t know about that target until October 2011. And 18 months later, we already have people talking about clinical trials of a therapy that hits that target. We’re not there yet, but we are pretty close. As we discover more about these diseases, and the big picture comes clearer, I think we will see that more and more, one subset of ALS after the next will have rational therapies designed against it, until we can treat all ALS. The challenge is how fast we can do it.

About the Author

MDA Vice President of Research Jane Larkindale spends her days overseeing MDA’s Research department, which includes basic, translational and clinical research programs. She determines MDA’s research strategy — how to use donated funds to have the greatest impact on therapy development. In doing this, she works closely with drug development companies, clinicians and academics, often bringing stakeholders together for meetings to share ideas. Jane’s focus at MDA is currently on moving promising drug development targets and novel therapeutics out of academia and into clinical development, which is one of the major bottlenecks in developing new therapies.

Jane brings a broad background in science to MDA. She completed her D.Phil. (Ph.D.) at Oxford University in the United Kingdom in 2001 (which she attended on a Rhodes Scholarship), and has done research in areas as diverse as molecular biology, biochemistry, genomics, plant science, medical physics, marine biology and industrial chemistry.

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