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Committed to Discovery: A Researcher’s Tale

Dr. Mariano Viapiano was a talented, young researcher. He had a promising idea for a potential new treatment for brain cancer. But what he didn’t have was funding to do the necessary studies to make the idea a reality that would benefit patients.

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Dr. Mariano Viapiano

“It all started with NBTS,” says Dr. Viapiano. “I can trace back each element of this story to things we started to do with NBTS’ funding.”

Dr. Viapiano’s story is one of opportunity, promise, and perseverance. It’s also one that almost didn’t happen.

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In 2007, after having completed a post-doctoral fellowship at the prestigious Yale University in applied neuroscience, Dr. Viapiano accepted his first faculty position as Assistant Professor of Neurosurgery at Ohio State University. With this appointment came the first opportunity for Dr. Viapiano to oversee his very own laboratory.

His lab quickly made a very promising discovery – a protein that was present in glioma (brain cancer), but not healthy brain tissue.

This initial finding was made with internal funding provided to the laboratory through the university itself. But in order to do the type of more intense, impactful research needed to uncover the meaning and implication of the new protein, they would need larger, external grants.

“So we knew [the protein] was specific to gliomas – and that was important – but the point really was, does it do something critical to tumor growth? And can we do something about it?” says Dr. Viapiano.

So Dr. Viapiano and his laboratory team began furiously sending out grant applications to get the necessary funding to study this protein at a level where the initial discovery could advance from an interesting piece of knowledge to an actionable target for new treatments.

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Researchers at work studying brain tumors

“I had started applying for funding in 2007; and in 2007 and 2008 we only had a little bit of help from the University…we were submitting for grants pretty much every month.”

But for two years they were stalled, as application after application went unanswered. It got to the point where Dr. Viapiano says of the grants they had mailed off, “we would just kind of forget about them.”

And so it was when, one day in 2009, after three frustrating years, the phone rang.

“I get a call from [National Brain Tumor Society’s former Chief Scientific Officer] and he sounded very excited,” says Dr. Viapiano. “And he said, ‘Mariano, I want to give you great news that we’re awarding you a grant.’

And he called me on a day that, like I said, I was completely oblivious to what was going on, so I didn’t know how to react. All I could say is, “Who is this?!'”

Yet despite the unceremonious start to the relationship, it would prove to be an enduring and fruitful partnership. Dr. Viapiano and his lab went on to use the funding from the National Brain Tumor Society to establish that the protein they had previously identified was actually regulating mechanisms of tumor growth in gliomas that had never been seen before, making it an even more attractive target to continue researching.

And that’s exactly what Dr. Viapiano did. A year after receiving the grant from the National Brain Tumor Society, he was able to produce enough data on the importance of the protein to apply for a highly sought after and competitive grant from the National Institutes of Health (NIH). As the largest funders of biomedical research in the country, NIH grants are the lifeblood for researchers looking to make major advances in disease like brain tumors. And this time his grant application to further investigate the protein as a therapeutic target was readily accepted by the NIH.

“That was literally a one-to-one, direct consequence of us having received the NBTS funds,” Dr. Viapiano says of his successful NIH grant application.

“If we hadn’t had the money from NBTS to do our preliminary research to submit to NIH, we never would have received the additional funding to continue our work.”

Two years later in 2012, Dr. Viapiano and his lab published a major article on his complete findings on the protein in the esteemed journal, Cancer Research. At the same time he was recruited to move from Ohio State to Boston for a joint appointment at the highly regarded Brigham and Women’s Hospital and Harvard Medical School, where he now serves as Assistant Professor of Neurosurgery, as well as Director of the Harvey Cushing Neuro-Oncology Laboratories.

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A member of Dr. Viapiano’s lab works at a culture station

After arriving in Boston, Dr. Viapiano and his team continued to work on the protein, though they made the conscious decision to pivot their lab’s main focus from primarily a basic biochemistry laboratory, toward a completely translational approach. This meant beginning to move from studying just how the protein acted in gliomas, to actually trying to create treatments that would target the protein and improve patient’s lives.

To date, Dr. Viapiano and his lab have created both a monoclonal antibody and a small fragment of that antibody to test as potential new treatments, which work by blocking the protein’s mechanism for driving the tumor’s growth. Early testing in preclinical models has shown encouraging results for the potential new treatments, and Dr. Viapiano is now seeking to patent his work and forge partnerships in the biopharmaceutical industry to help commercialize the treatment and move it through the clinical trial and FDA approval process on an ambitious timetable.

“We have gone full-out from being a basic science laboratory that just discovered a new protein in brain cancer, all the way to being a full-fledged neuro-oncology lab that is developing creations against this protein, holding IP with the creations, and very interested in looking for commercial partnerships – in other words, Industry – that could help support the clinical application of these creations,” says Dr. Viapiano. “In fact, what we are really very focused on is taking these creations all the way to FDA approval within two years.

“I think that is probably the most concrete example of how the initial money from NBTS helped create a full-fledged research program, and now even a specific treatment.”

Dr. Viapiano cautions that his potential new treatment is not a cure-all, but – if the promising results continue and the FDA eventually approves the treatment – would be a significant improvement for brain tumor patients, who desperately need more and better treatments.

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In any event, the efforts of Dr. Viapiano’s lab demonstrate the potential that can occur when you combine persistence with critical outside philanthropic funding from organizations like the National Brain Tumor Society.

“I’m personally convinced on this target we’ve been studying for many years,” says Dr. Viapiano. “And we decided to never let it go. Many other groups might study a protein and say, ‘Oh, great this is a glioma protein,’ and then move onto the next protein that they could study. But we decided to really bind on here and not let it go…But [NBTS] knows better than I, the amount of money and resources needed to take even the best ever creation coming out of a laboratory to the clinic. It’s something that very rarely can be handled in academia alone.”

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Inside the Harvey Cushing Neuro-Oncology Laboratories at Harvard Institute of Medicine

In other words, we all have a role to play: researchers operating in academia and academic-based medical centers, biopharmaceutical companies, patient advocacy organization…and YOU, who provide the generous support necessary to allow NBTS to play this vital role in the brain tumor drug discovery and development process.

In addition to funding research to help advance brain tumor research, the National Brain Tumor Society helps move treatments through the rest of the drug development pipeline by working with all these other stakeholders involved in the process (researchers, biopharmaceutical companies, government agencies, regulators like the FDA, and clinicians). We aim to improve the system in which potential brain tumor treatments are developed, evaluated, and approved.

If you’re interested in contributing to the advancement of brain tumor research that can ultimately help improve the lives of brain tumor patients and eventually cures, please visit here.

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