Fibrolamellar Cancer Foundation President on What's Next For Rare Cancers


John Hopper, MBA, highlights advances made in the fight against rare cancers and discusses what’s next for the community.

At the National Organization of Rare Diseases’ annual Rare Diseases & Orphan Products Breakthrough Summit (NORD Summit), held in Washington DC, Rare Disease Report® sat down with John Hopper, MBA, the president of the Fibrolamellar Cancer Foundation and moderator on a breakout session, which explored what’s next for the rare cancer community.

In our interview, Hopper discussed key highlights from the session and explained how the NORD meeting serves as a meeting place for providers, patients, and pharmaceutical companies to catch-up on the latest advances being made in the rare disease space, advances that could not have been made without communication and collaboration across the board.

RareDR®: Can you tell us about the breakout session you participated in on what’s next for the rare cancer community?

John Hopper, MBA: This is the first panel for the Rare Cancer Coalition at NORD and we’re very excited about this. The first panel that we’re having is to discuss not only what the Rare Cancer Coalition is about, but also, what’s in the future for rare cancers?

About 20% of our members here at NORD are involved with rare cancers, but what we have found is that most of us don’t know each other. Because we’re all involved with rare diseases, we know we need to be networking with one another in areas of research and patient advocacy, and in areas of what the government can be doing and what industry can be doing.

Today’s meeting is the first major panel discussion we’re having; it includes government in terms of the National Cancer Institute, it includes industry, it includes patient advocacy groups, it includes patient survivors and their inspirational stories. But again, the real goal of this is to inspire all of those coming in here that together we can create a voice that’s impactful in all areas of cancer research.

What are some advances being made in the fight against rare cancers?

This really covers a broad spectrum, but first I’m going to start off with diagnostics. The idea of genetic diagnosis, looking for what we call checkpoint inhibitors, looking at what might be precision medicine, is all coming into the area of rare cancers.

What we find is that many of the rare cancers have genetic mutations, and once we discover that, it helps us to target therapies around that. So, what does that mean? What that means is by those discoveries coming through—which is fairly new—we can work with drug companies to develop whatever drugs might be targeting that. Repurposing drugs is a key one for rare cancers in an essence because there may be drugs that exist out there right now for the larger cancers, such as ovarian cancer, pancreatic cancer, etc. But by finding the certain genetic mutations that rare cancers have that might be common to some of those larger cancers, we can look at screening thousands and thousands of drugs that might be out there to see whether or not that can target a specific cancer. There’s a lot of hope from that perspective, in terms of what’s in the future of drug discovery?

The other part is, there’s a whole new slew of microbiology/biotechnology companies coming through because they’re discovering molecules now that may affect rare diseases, where before, it was the larger drug companies. Some might be working side by side with Merck in the areas of immunotherapy or checkpoint inhibitors, but they might be working with a small company called Puma or Blueprint Medicines— there are dozens of those—who may be having just a small molecule out there that might be a kinase inhibitor, let’s say, which might be what helps to stall a cancer and allows somebody to go into remission, etc.

What’s happening is, we’re seeing that work being done from the larger drug companies, from the smaller drug companies, and hopefully, the patient advocacy groups that NORD brings together, can help bring the knowledge for all of us to say, ‘Hey, what about our cancer?’

Are there new treatments for rare cancers coming down the pike that patients and providers should be aware of?

The answer is always, yes. There are new discoveries every day and the most important recommendation I can make for the community here is to be proactive about going to the large pharmaceutical companies as well as the microbiologists and the biotechnology companies, to find out if they have discoveries that pertain to you.

What’s we’re finding here with NORD, with the Rare Cancer Coalition, is that we’re hoping that by networking together we can [share cancer discoveries, notice any similarities, and discuss drug companies].

I will tell you my own personal experience when I was at NORD 4 years ago, I happened to be sitting next to somebody who worked for Sanofi Genzyme and then just moved over to this company called Blueprint Medicines, and we were talking about hepatocellular carcinoma, which is the larger liver cancer. I said, ‘Well, why aren’t you researching fibrolamellar, which is an adolescent/young adult liver cancer but different from the larger cancer. He goes, ‘Well, actually, we are. We just started that discovery.’ And so, that put us in connection with them, where we helped them with studies, we helped them with patient advocacy.

I would encourage everyone to be networking as much as possible with the large companies and the smaller ones because you never know if you’re cancer might be the one [for which new treatment is being worked on]. Again, it’s everything from immunotherapies, checkpoint inhibitors, kinase inhibitors, and HER2 inhibitors. Every cancer is slightly different, but really, I would encourage everyone to open up their network and make sure they talk to as many companies as they can about that.

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