Rare Tumor Patient Engagement Network: Getting Patients Involved in Cancer Research


Karlyne Reilly, PhD, discusses the Rare Tumor Patient Engagement Network and the importance of getting patients with rare conditions involved in cancer research.

In an effort to meet the Blue Ribbon Panel recommendation of establishing a network to facilitate direct patient involvement, the National Cancer Institute (NCI)’s Center for Cancer Research has launched the Rare Tumor Patient Engagement Network.

Funded by Cancer Moonshot, the overall aim of the project is to build a national network to evaluate rare pediatric and adult tumors and create a network of clinical trials. The project has 2 central arms: the NCI-CONNECT arm, which aims to advance understanding of central nervous system cancers, and the MyPART arm, which will focus on pediatric, adolescent, and adult rare solid tumors.

In an exclusive interview at the National Organization of Rare Diseases’ Rare Diseases & Orphan Products Breakthrough Summit (NORD Summit), Rare Disease Report® sat down with Karlyne Reilly, PhD, director of the NCI’s Rare Tumor Initiative, and head of the MyPART arm of the network, to discuss the project and the importance of patient involvement in cancer research.

RareDR®: What is the Rare Tumor Patient Engagement Network?

Karlyne Reilly, PhD: The Blue Ribbon panel of the Beau Biden Cancer Moonshot recommended as one of its first recommendations that patients be more engaged in research, that they have better access to their own data, that they have a better understanding of what it means to be involved in cancer research, and that they feel more like a team player.

I heard a patient say, ‘He doesn’t feel like a guinea pig; he feels like a test pilot,’ and that’s what we really want. We want patients to understand how critical they are in the process. Because at the Center for Cancer Research, where I work, we feel we have an opportunity in rare tumors that may be difficult to address in other parts of the country, we purposed a Rare Tumor Patient Engagement Network.

The idea behind this is to set up a patient portal where rare tumor patients can come, have their own little piece of portal, which has a place where they can provide data for research, they can keep track of their informed consents, and they can then get results back—either their own individual results or finding out what’s going on with a study, what are we learning about the kind of cancer they have. And so, the idea is to really make the patients feel like they’re an important part of the process and this is their little place as part of it.

The Rare Tumor Patient Engagement Network, right now, has 2 major arms; one is for neurooncology because all brain cancers are rare, and the other is a pediatric and young adult branch because all pediatric tumors are also rare. And so, that’s where we’re starting, but we’re hoping that we’ll build infrastructure that can be used across all rare cancers.

The one that I’m working on most closely is this Pediatric and Young Adult Rare Solid Tumor Network.

Can you tell us more about the My Pediatric and Adult Rare Tumors (MyPART) Network?

MyPART is one of the arms of the Rare Tumor Patient Engagement Network and it is a network that is focused on patients who are pediatric adolescents or young adults—about 30 years or less—and have solid tumors. There’s a big focus on sarcomas and we’ve got a lot of neuroendocrine tumors that we’re looking at.

The idea is that young adults and adolescents have some unique challenges that they face when they receive a cancer diagnosis that’s sort of compounded by having a rare cancer diagnosis. There are feelings of isolation—not knowing anybody else like them with this kind of a disease. And so, there’s also a component to all of this, of trying to provide support, trying to learn how to support adolescents and young adults better, and pediatric patients and their families better.

We’ve also got a psychosocial aspect to the research that we’re doing, which I’m very excited about. And so, it’s sort of what I was saying, we’re having the patients come in, provide information about their experience, and provide samples. And then, try to compile all of that together and we’re hoping that we’ll find some patterns that haven’t been seen before that might lead to new therapeutics.

And in all of this, we’re going to be looking at whether these patients are also candidates for intervention trials because we do want to get these patients into trials that might provide a treatment as well. But this allows us a blanket protocol that anybody can join—even if they’re not eligible for a clinical trial, they can still participate in this.

When it comes to the development of new therapies for rare tumors, how is patient involvement so important?

We’re trying to work very closely with the advocacy organizations and NORD is a great example of that, of course. But in some cases, the patient may not have an advocacy organization for their particular kind of tumor; it may be so rare that there aren’t enough people to form a group.

And so, we’re looking into ways of taking advantage of social media—Twitter, Facebook—and Rare Disease Day and things like that where we can try and reach out and get the patients more involved without necessarily having to go through that advocacy group.

But I think the ways in which advocates and patients really help out researchers is participating in clinical trials. I think that there are ideas that don’t get tested because there aren’t enough people to test them in the trial and that’s a really sad thing, that there might be a therapy out there that never gets tested rigorously.

And so, participating in clinical trials is very huge and we want people to understand what that really means so that it’s not such a scary thing necessarily, and so that they understand the value that they’re providing us.

Another way that patients really help us is when they provide samples of their tumors so that we could study them. In many cases, we can try and use those tumor samples to make model systems that are able to be perpetuated so that we can test drugs on them over and over and over again. And so, that’s a huge opportunity.

And little things like going and talking to your congressman, going and talking to your government representatives about how even though rare tumors individually only affect a small number of people, as a group, they’re affecting a lot of people, and they’re an important thing to be funded and to be supported. One-quarter of all cancer deaths are people with rare cancers, so it’s an important issue and patients are the best spokesmen.

I also think just coming and being honest with us, contacting us and telling us what’s important. We get focused on what we think is the most important thing but it’s very, very important for the patients to come to us and say, ‘These are the issues that really affect my quality of life when I have this disease.’

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