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Tuesday, October 4, 2016

How The 2016 Nobel Prize In Medicine Relates To Michael J. Fox (And You)

OCT 3, 2016

Today, Japanese biologist Yoshinori Ohsumi won the 2016 Nobel Prize in Physiology or Medicine for his groundbreaking work in what’s called “autophagy”. News reports from all over the world have already explained the science. Autophagy is a process — it’s how the cells inside your body destroy and recycle their own components.
It’s easy to be befuddled by Nobel Prize news even if you’re excited about it. When a prize goes to a discovery that can’t be seen or felt, how can you relate it to everyday life?
For this prize, that means talking about the illnesses that scientists think have a connection to autophagy. Think Type 2 diabetes, or Alzheimer’s disease, ailments that touch more and more families each year, perhaps even your own. Or think of Parkinson’s disease, which also is on the rise, and is where “Back to the Future” actor Michael J. Fox comes in.

378376 03: Actor Michael J. Fox testifies before the U.S. Senate subcommittee on Labor, Health, and Human Services, Education and Related Agencies September 14, 2000 on Capitol Hill in Washington during a hearing on stem cell research. Fox joined actress Mary Tyler Moore, Gina Gershon and producer Jennifer Estess to urge further funding for stem cell research to battle various medical conditions. (Photo by Alex Wong/Newsmakers)

Not long after Fox announced he’d been diagnosed with Parkinson’s disease, he launched the Michael J. Fox Foundation, which is dedicated to finding a cure for the condition.
It so happens that the foundation has provided funding to researchers working on autophagy. You can learn a lot just reading about what’s been accomplished with the money. Sure, you’ll learn about how today’s Nobel Prize may contribute to this disease, but you’ll also learn about the pace and the challenges of medical research.
First things first. What, exactly, does autophagy have to do with Parkinson’s disease? The short answer is that scientists are still working it out. But the longer answer goes something like this: Because of today’s Nobel laureate and others who followed up on his work, scientists now know that there are several kinds of autophagy at work in the body all the time, clearing out damaged proteins and components of cells, and providing materials for cells to rebuild. People with Parkinson’s disease have an accumulation of a protein called alpha-synuclein in the brain. It’s only one of many indicators of Parkinson’s. But the idea is that there could be a way to activate or enhance the autophagy cleaning mechanism to clear away cells’ accumulated protein gunk. If that were possible, then it might be possible to stop the progression of Parkinson’s.

The word autophagy comes from the Greek words for “self”, and “to eat”. It’s a process where the cell can recycle part of its own content. (Credit: Nobel Assembly)

We’re a long, long way from any medications. The foundation is starting with the basics. Here’s a grant to scientists developing more relevant animal tests for potential Parkinson’s medications, because it’s important that results scientists see early on in animals be somewhat predictive of what might happen in humans with Parkinson’s later.
And here’s a grant to a team which has developed better test-tube-type methods to tease out molecules that might kickstart autophagy’s cell cleanup process. It helps to have a decent magnifying glass when you’re searching for a needle in a haystack.
More recently, here’s a grant to researchers who report prototype Parkinson’s disease drugs that work by controlling autophagy — in other words, by cleaning up cellular clutter. (Scientific American calls molecules like this the Marie Kondo of the brain, after the mega-successful organization guru and author). Here’s what the scientists write about their prototype drugs.
“We propose that these drugs, after necessary modifications, could be utilized to eliminate the abnormal proteins that accumulate in the brain of [Parkinson’s disease] patients. We intend to use medicinal chemistry to further develop these drugs to increase their potency, improve their delivery to the brain and reduce any possible side effect toxicity.”
The team reported its first prototypes back in 2013, in the journal Nature Chemical Biology.
I keep saying “we’re a long way from a drug” here because it’s true. But when you look at how each grant seems to build on the ones that came before, and when you think about how much more scientists know about how Parkinson’s works as a result, that’s cause for excitement, on some level. And that’s the excitement I wish every news story about the Nobel Prize would convey to readers like you.
Back to today’s Prize itself. A major reason that Ohsumi won the prize solo is that there’s been an explosion of research on autophagy— including the Fox Foundation funded work— in the last 15 years or so. It’s a spike you can really chalk up to the groundbreaking work that he did. Just look at this chart from the Nobel Assembly. It outlines how many scientific papers on autophagy were published in a given year. Ohsumi started working in the field over 27 years ago, and his team published a major breakthrough in 1992.

In the 1990s, path-paving studies from 2016 Nobel Laureate Yoshinori Ohsumi generated a large amount of interest in autophagy, as the spike in publications shows. (Credit: Nobel Assembly)

Does that spike in papers equate to a cure for Parkinson’s disease or for another disease? No, it doesn’t. Not yet. “Even now we have more questions than when I started,” Ohsumi told Nobel Media in an interview. But the point of this prize is that Ohsumi is a giant in the field, whose work made it clearer how autophagy is relevant. Relevant enough that someplace like the Michael J. Fox Foundation thinks it’s worth looking into. And in medicine, you have to start somewhere.

http://www.forbes.com/sites/carmendrahl/2016/10/03/how-the-2016-nobel-prize-in-medicine-relates-to-michael-j-fox-and-you/#530ae895a505

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