Changing the Rare Cancer Landscape

Finn Sawyer was an energetic, happy and intelligent little boy who loved avocados and the outdoors and – like his namesakes Huck Finn and Tom Sawyer – craved adventure. In August of 2016, Finn was diagnosed with a tumor in his bladder/prostate; it was a rare and aggressive cancer called rhabdomyosarcoma. Doctors were hopeful that the standard of care protocol would effectively treat the tumor. But despite all efforts, which included 43 weeks of nine different chemotherapies, seven cumulative weeks of radiation, and three grueling full-day surgeries -- unprecedented for a boy Finn’s age -- Finn died on December 2, 2018.

Rare Isn’t Rare

Finn’s story is anything but rare. Research presented at the American Association of Cancer Research by SHEPHERD demonstrates that of 400 known distinct cancers, 380 are rare forms. That’s 95% of all forms of cancer, which means over 550,000 new rare cancer patients will be diagnosed this year alone. We also know that as of February 2019, a staggering 182 cancers lacked an FDA-approved targeted therapy — all but one of them rare cancers. This means that in 2019, over 245,000 new cancer patients faced a grim diagnosis with absolutely no FDA-approved?modern therapies.?Ask any patient or parent of a child who is faced with that reality what it feels like and they will tell you that there is simply no greater pain on this earth.

We Have a Data and Research Problem

Shocking? Absolutely – but it doesn’t stop there. Over 100 rare cancers have no publicly available data sets. Why does this matter? Because this causes a significant disparity in focus. Those rare cancer patients are almost seven times less likely to have an approved targeted therapy for their cancer compared to those whose cancers have at least one dataset.?

The logic here is clear: because so many research organizations and companies are operating in silos or in competition with each other, databases for many rare cancers are disparate and fragmented. Treating and curing cancer requires extremely precise information. The science cannot operate without the data; it must be collected in order for progress to be made. And while cancer does not discriminate, translational research does.?Rare cancer affects every population, however translational research and commercial drug development have traditionally neglected these patients.

In February, Andrew Kaczynski, CNN reporter and father to Francesca, who died of an aggressive brain tumor called atypical teratoid rhabdoid tumor on Christmas Eve 2020 at the age of 6 months, penned an opinion piece which laid out exactly what we as the rare cancer community must -- and are -- fighting for: increased funding for rare cancer research, increased data sharing and greater collaboration within the rare cancer community. And that is why we are taking this opportunity to highlight what can happen when the rare cancer community rallies together.

A Unified Voice is the Key

Two years ago, SHEPHERD, alongside G2G Consulting and an entire community of rare cancer organizations and advocates championed a new research program called the Rare Cancer Research Program, administered by the Congressionally Directed Medical Programs (CDMRP). The mission, while simple, is powerful: to elevate rare cancer research to enable clinically impactful discoveries for the benefit of service members, their families, Veterans and the American public.?

We are grateful that Congress heard our collective voices, and thanks to rare cancer champions in the Senate, including Senator Dick Durbin (D-IL) and Senator Richard Shelby (R-AL) and many others allies in both the Senate and the House, a new research program was established in the DOD appropriation under the Defense Health Agency (DHA) J9, Research and Development Directorate, specifically for rare cancer research. The program is designed to provide those cancers that don’t have any research tools an opportunity to potentially kickstart a whole new field of research and includes three categories: concept award, idea development award, and the Research Community Development Award (see more here). In FY20, we were collectively able to secure $7.5 million in federal funds, which saw an increase of $10 million for FY21. The response to the call for proposals far exceeded everyone’s greatest expectations with over 400 received, demonstrating the very real need and desire by the research community for this type of funding. In May, the first tangible outcomes were realized, with awards being made to almost 20 different research institutions across the country to conduct high impact and novel research on rare cancers. To build on this momentum, we are advocating for a continued increase in this funding.

Rejecting the Status Quo

It takes an entire community coming together to start seeing impactful changes, to reject the status quo and demand a better system. One that works for all patients, not just a select few. One that Finn and Francesca both deserved.

Here are a few ways you can help in this much-needed effort:

• If you are a researcher - please consider applying for one of these awards. You can find more information and links to submit a pre-application here.
• If you are an advocate in the rare or pediatric communities - please share their funding opportunity with your respective networks. Your help is critical for spreading the word widely about this important funding opportunity for rare cancers.
• If you are a patient, a parent, a caretaker, or anyone touched by rare cancer - your story matters. That’s why we have started a campaign to collect stories from around the country - with a purpose. Each chapter will be devoted to a US state and delivered to your state’s representatives on Capitol Hill. Help us show Congress and those in charge of creating policy that rare is in fact - not rare.

At the SHEPHERD Foundation, we are creating a #RareRevolution so that all patients may live. We are challenging the status quo, because survival should not be determined by skin color, zip code, income, or specific cancer diagnosis. We know that every single life is worth fighting for, and it’s time that we start holding the system to a new standard. Please join us at https://shepherd.foundation. Together, we can light the path to better outcomes.

Together - we can make a difference.