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Nuclear waste will remain a deadly threat for hundreds of thousands of years. Despite having decades of hazardous waste in temporary storage, the world is only now finalising plans for long-term containment

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Do persistent stress granules point the way to a treatment for ALS?

Aquinnah Pharmaceuticals’ latest research offers new hope to ALS, Alzheimer’s, and other neurodegenerative disease patients

ALS – the neurodegenerative disorder better known in the US as Lou Gehrig’s disease – has seen a surge in international awareness thanks to the ice bucket challenge. But there remains no effective treatment. Aquinnah Pharmaceuticals is investigating one exciting new possibility. President and CEO Glenn Larsen talks about the prognosis for ALS patients today, and Chief Scientific Officer Ben Wolozin explains what stress granules are supposed to do – but how when they stick around, they can start causing disease. Together they discuss the compounds Aquinnah has created to tackle this pathology, and their hopes to push forward the field of neurodegenerative diseases with an effective treatment for ALS and Alzheimer’s.

The New Economy: ALS – the neurodegenerative disorder better known in the US as Lou Gehrig’s disease – has seen a surge in international awareness thanks to the ice bucket challenge. But there remains no effective treatment. Aquinnah Pharmaceuticals is investigating one exciting new possibility. Glenn Larsen and Ben Wolozin join me now.

Glenn, what happens today when a patient is diagnosed with ALS or another neurodegenerative disease, like Alzheimer’s?

Glenn Larsen: A diagnosis for ALS or Alzheimer’s leaves little hope for patients today. There aren’t any approved drugs that actually are effective to prevent disease progression: to reverse it, or to even slow it down, at this point.

ALS is a progressive motor-neurone deficiency. And what that does to the body is actually prevent the normal bodily functions from occurring, because of motor-neurone degeneration. And this affects your ability to walk, to talk, to swallow, and eventually to breathe.

Right now with a diagnosis of ALS, 50 percent of patients die within three years. So you can see there’s a significant need for improved therapy.

The New Economy: Ben, your research is based on the discovery of persistent stress granules in neurodegenerative patients, so tell me about this.

Ben Wolozin: So it all begins with the body’s response to stress. If you hit your head, the body goes, ‘Oh my god, I’ve got to fix this!’ You have these stress granules form, and what they do is, they orient the body’s response so it can repair itself.

It’s supposed to be a very short response, that lasts – let’s say, 10 or 15 minutes. But imagine you have a mutation, that’s a genetic change in some of those genes; or you have a chronic disease that lasts years. Those stress granules become either stickier, or persistent. And as they become persistent, they become pathological, and start causing disease.

The key thing to understand about the brain – what’s really unusual about it – is what determines the type of disease, how you feel it as a patient – is actually where in your head the stress granules build up. So if the stress granules build up in your spinal chord, well that controls movement. Guess what? You have a movement disorder, that we call ALS. But if the stress granules build up in your cortex or your hippocampus, which controls memory, you have what we would consider dementia.

The New Economy: So this really is a very important discovery.

Glenn Larsen: Absolutely. We’re very excited about it, we think it’s a great breakthrough.

What Ben did actually in his lab was create something called an assay, which gave us the ability to test 75,000 different compounds. And out of that entire screen, we actually identified a small number of compounds that were highly attractive to prevent stress granule formation.

The New Economy: Talk to me about your partnership in Aquinnah. How are you going to be driving this research forward?

Ben Wolozin: I have been in the field studying neurodegenerative diseases for over 30 years, having a research laboratory that investigates ALS, Alzheimer’s disease and Parkinson’s disease. And it’s really part of the fabric of what makes me tick.

Glenn Larsen: Ben has actually received numerous awards; the prestigious Zenith Award, in addition to the AE Bennett award. He found the first marker used to identify tangles in Alzheimer’s disease. He was the first to actually discover the role of persistent stress granule formations.

Myself, I have lots of pharmaceutical drug industry experience. I personally have been involved in bringing 15 new chemical entities to clinical development; five of those which were approved as commercial products, with sales over $10bn. So I have a lot of experience knowing what’s important to focus on, and to do the right experiments at the right time. Which is clearly important to navigate the complicated path of drug discovery and development.

The New Economy: As you say it is a complicated path; a long path as well. What are your hopes, moving forward?

Ben Wolozin: Glenn and I are both very, very optimistic about the future of treatment for neurodegenerative diseases, and what Aquinnah itself can bring. I think that actually the entire field of neurodegenerative diseases is poised for such a leap forward, and that the future of the field actually is incredibly bright. And I think Aquinnah Pharmaceuticals is going to be an important part of that future.

Glenn Larsen: Right now we are testing our best candidates; we actually have worked very hard to come up with molecules that are appropriate for clinical development. They’re potent, you can take them as a pill, they pass the blood-brain barrier, which is a critical element for neurodegenerative diseases. So we know our molecules will actually get into the brain and into the spinal chord.

Currently we’re in model testing for these compounds. And what we plan on doing once we have success in those models is to move forward into clinical development, which would be in about two to three years for ALS.

Assuming success for ALS – which can be done in a relatively short period of time, because the disease progresses relatively fast, so you can conduct a clinical trial in one year – we will then move on to clinical studies for Alzheimer’s.

The New Economy: Glenn, Ben, thank you both so much.

Glenn Larsen, Ben Wolozin: Thank you.