Mitochondrial apoptosis mediator?
MDA’s 2017 Scientific Conference got off to a quick start Monday morning, with platform presentations covering a variety of topics delivered by the world’s preeminent neuromuscular disease researchers. They spoke about targeting long non-coding RNA, deep phenotyping using motion sequencing and advanced oligonucleotide therapeutics for neuromuscular disease.
Most of us don’t have the first clue what these scientific terms mean, or what their importance to neuromuscular disease research may be. But for the more than 500 scientists, clinicians, industry partners, pharma and biotech representatives, and other stakeholders in attendance, MDA’s Scientific Conference is an opportunity to come together and share ideas and updates, gain insight into others’ research methods and results, and find ways to collaborate and build off others’ successes as they work to unlock the secrets of neuromuscular diseases and develop therapies to treat them. It’s just one of the many different ways MDA is pushing for progress as we work to find treatments and cures for all the diseases in our program.
So, now you’re probably wondering how a conference could really be that important.
Well, you may have read or heard before that the power in MDA’s research program lies in its big-picture approach, which leverages information and insights gained from research in one disease area to inform and advance discoveries and breakthroughs in others — that is, research across diseases. But what does that mean? Well, to start with, breakthroughs aren’t made in silos. Eureka moments don’t happen in a vacuum. In many cases science is a process of accumulation — results forged from earlier work that itself is built upon the results from even earlier studies. In other cases, science can be a process in which complete lines of thought may be overturned and abandoned in light of new information and refined technology.
Either way, for these different processes to work and for advances to be made, people — scientists, researchers — need to get together and share what they’ve learned. And when MDA sets the stage and the world’s top researchers in muscular dystrophy, motor neuron diseases, mitochondrial diseases and myopathies — and other fields such as epigenetics and behavioral science — show up, that’s when you can see research across diseases in action. Such as when:
- Matthew Wood, Ph.D., from the University of Oxford in England, talks about oligonucleotide therapies (short strands of DNA or RNA that are used to change the way cells read genetic information and manufacture proteins) that may have application in Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), myotonic dystrophy (DM), ALS (amyotrophic lateral sclerosis) and related diseases.
- Peter Jones, Ph.D., from the University of Nevada in Reno, talks about the potential for therapies based on epigenetics (changing gene activity, rather than changing the DNA itself) — therapies that could be developed for facioscapulohumeral dystrophy (FSHD), Friedreich’s ataxia(FA) or SMA.
- Sanjay Bidichandani, MBBS, Ph.D., from the University of Oklahoma Health Sciences Center, Robert Brown, M.D., Ph.D., from the University of Massachusetts Medical School, Lindsay Wallace Ph.D., from Nationwide Children’s Hospital in Boston, Andrew Berglund, Ph.D., from the University of Florida, and others talk about different therapeutic approaches to treat diseases caused by repeat expansions (expanded sections of DNA), including FA, some forms of ALS, FSHD and DM.
I overheard one presenter suggest to another that they ought to get together and talk about how certain difficult experiments may be designed. Another remarked on how MDA’s Scientific Conference and other such gatherings are valuable in a number of ways — including the fact that they can be the catalyst for the successful therapy development for one neuromuscular disease leading to the later application of the strategy to other diseases that have similar underlying mechanisms or outward effects.
So even though we may envision scientists locked away in their labs, hunched over a microscope and studying complicated-looking tables and graphs as they search for treatments and cures (which actually isn’t that accurate a picture anyway), the truth is that attending meetings like the Scientific Conference plays as vital a role in advancing science and making breakthroughs as any of the other activities that make up a researcher’s repertoire.
And with 500 of the sharpest minds in research working — together, today — to find solutions, there’s good reason for all of us to be excited.
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