BrandiLee’s Story

FY 2021 Written Testimony for the Record Prepared by BrandiLee Schafran, Director of Patient Advocacy, SHEPHERD Foundation

Thank you for the opportunity to provide my testimony to the House Appropriations Subcommittee on Labor, Health and Human Services, Education and Related agencies. I am here to discuss the $10 million funding request for joint NCI/NCATS Rare Cancer Therapeutic Research and Development Program within NIH. I want to thank Chairwoman DeLauro and Ranking Member Cole for their strong support addressing diseases neglected in the current marketplace. By calling attention to rare cancer, you can help millions of people.

My name is BrandiLee Schafran and I am the Director of Patient Advocacy for SHEPHERD Foundation, exclusively focused on addressing rare cancer. I came into this role when my youngest of three boys died tragically over a year ago from a 2.5 year battle with an aggressive rare cancer that lacked targeted therapy. Through the devastation of losing my child to cancer, I am deeply passionate about sharing Finn’s story and what I have learned about the lack of funding and treatment options. His journey and suffering have given me a desire to see that patients and their families have a voice and platform to make a difference for rare cancer patients.

Finn Sawyer was an avocado-loving, energetic, and intelligent little boy who loved playing with friends, reading books, and doing all the other childhood things that little boys do. Like his namesakes Huck Finn & Tom Sawyer he craved adventure, laughed often, and loved the outdoors. In August of 2016, Finn was diagnosed with a tumor in his bladder/prostate, which was a very aggressive rare cancer called rhabdomyosarcoma. There are only about 350 diagnoses a year in the United States, most of which are pediatric cases. At this point, we were not offered molecular diagnostics which would have given us critical information about clinically relevant biomarkers and genomic alterations to help match Finn’s tumor to approved targeted therapies, immunotherapies, and/or clinical trials. Although rare and aggressive, doctors were “hopeful” that the standard of care protocol would effectively treat the tumor. After 43 weeks of chemotherapy, coupled with 28 days of radiation, Finn finished his primary treatment which left a small residual mass we hoped was scar tissue. Unfortunately, a mere three weeks after the end of treatment, Finn’s initial symptoms came back. Subsequent tests showed that Finn’s treatment was not successful, and the residual mass was growing rapidly.

In September of 2017 at the age of 2.5, Finn underwent a major surgery to remove his bladder and prostate. Though a successful surgery, Finn still had roughly an 85% chance of relapse if an additional chemotherapy regimen was not taken. In November of 2017 Finn began a standard 35 week-long relapse chemotherapy regimen aimed at targeting any cellular-level cancer cells left over in his body after surgery. Tragically, again, in February of 2018 while still on chemotherapy, a routine scan revealed a new tumor in his abdomen. We now know that even though his medical team was trying to treat his cancer with established and occasionally effective chemotherapies, they were not the right chemotherapies for his specific tumor mutations. In fact, based on molecular diagnostics at the time of relapse, there were no approved therapies for his mutations.

In March of 2017 we brought Finn to the renowned Mayo Clinic in Minnesota where a world-class team of surgeons and doctors performed two all-day surgeries that were afterward described as heroic and nearly unprecedented for a child of Finn’s age. After removing Finn’s rectum, sections of pelvic bone, lymph nodes, part of a nerve and muscle, and any other abdominal soft-tissue that could host future tumors, Finn began the recovery process. Finn then began a chemotherapy regimen that was by and far the most intense he had ever experienced. With the exception of another major emergency surgery in May to fix a closed loop bowel obstruction caused by his three major abdominal surgeries, things progressed relatively smoothly until Finn’s body could no longer take the intensity of the chemotherapy.

In July of 2018 Finn had his first post-treatment scan and all came back clear. A mere six weeks later, however, Finn’s next scan showed two tumors growing in his pelvic cavity. We went back to the Mayo Clinic where the same team prepped for another heroic surgery — this time with the hopes of buying time to study the tumors and find a more targeted treatment based on his current tumor genetics. I would like to note that like us, most patients and families do not know about this option to have molecular diagnostics conducted, however, this should be done for every cancer patient at the time of diagnosis and we wish we were given that option for Finn. This is the most critical lesson I have learned after navigating Finn’s tragic cancer journey. After a week of proton radiation, the day of surgery came and surgeons found that the cancer had spread throughout the Finn’s entire abdomen. A surgical approach was no longer an option. While Finn began his recovery process, tumor samples were sent to the Mayo Lab as well as St. Jude and another private lab — time was now in shorter supply than ever before.

As Finn struggled to recover and fight the cancer that kept growing with devastating effects on his body, we brought him home on hospice care and were desperate for results from the scientists who were frantically working on his behalf. While keeping Finn comfortable at home, we received news that St. Jude and the private lab had come up empty. As Finn continued to deteriorate, the Mayo lab was able to discover a long-shot combination of two oral chemotherapies. We immediately started these drugs and saw some progress, but in the end, it proved to be too little too late as we helplessly watched our beloved son slip further and further away.

In the early morning hours of Sunday, December 2, 2018, Finn finished his race and went home to Heaven. He ran the race that was set before him, and he ran it with all his might. The impact that such a small boy had and continues to have on such a big world is astounding. The impact he had on MY world is priceless, and the emptiness of his absence is indescribable.

We know Finn’s story isn’t as rare. We know that out of 400 forms of cancer, 380 forms are rare. That is using the most conservative estimate of what constitutes a rare cancer, the American Cancer Society’s metric of fewer than 6 new diagnoses per 100,000 people per year. That’s 95 percent of all forms of cancer and means that over 550,000 new rare cancer patients will be diagnosed this year. That is not a small number.

It often surprises people to hear that all pediatric and brain cancers are considered rare. However, many may not realize that women and minorities as well as service members, veterans and their families are disproportionately faced with rare cancer diagnoses. Those who have served our country are disproportionately affected by over 60 forms of cancer. Almost 70 percent of those cancers are rare, and only 25 of them have an FDA approved targeted therapy.

Clinical trials are one of the best opportunities for cancer patients, however, rare cancer patients face extreme challenges here as well. Analysis shows that of all clinical trials between 2012 and 2016 showed that 74.89 percent of all trials did not include even one rare cancer by name. Only around 13 percent of all rare cancers were specifically named as a focus of a phase 3 clinical trial in those five years. I can’t help but see the direct correlation between that and the lack of new treatments for rare cancer patients. Some 80 percent of all new cancer patients lack even one FDA-approved targeted therapy for their cancer is a rare cancer patient. As of February 2019, 182 cancers lacked a FDA-approved targeted therapy — 181 of them were rare cancers. That means that in 2019 almost 200,000 new rare cancer patients were diagnosed without a modern treatment.

It is critically important that the federal government invest not only in the research that is needed but also in the translational medicine that turns this research into treatments and on clinical trials. I want to thank this subcommittee for initiating the joint NCATS-NCI rare cancer translational medicine initiative that was encouraged through the FY2020 appropriations package. It encourages public-private partnerships to study novel systems for identifying how rare cancers function, examining the commonalities across these rare forms of cancer to develop platform treatments for rare cancers, and supporting the development of life-saving therapeutics faster for patients who often have no other options can address these problems.

Further, I want to thank the Agriculture and FDA Subcommittee for including $5 million for the Rare Cancer Program within the Oncology Center of Excellence in FY2020. It is set to address gaps in the current system, streamline resources and hire rare cancer experts that can use their unique expertise throughout the Center. It will also investigate and implement novel small patient population clinical trial strategies, conduct natural history studies, accelerate the development of rare cancer therapies, and advance the field of research overall. We are working with both of these government efforts to address the gaps in rare cancer research and development.

Patients and families around the country know that these investments and policy changes will go a long way to help so many people. Please stand with these patients and families to increase federal spending on rare cancers, to encourage and reimburse frontline molecular diagnostics for all newly diagnosed patients, and to prioritize research, data sharing, and translational development for cancers that lack an FDA approved targeted therapy. Thank you once again for letting me share my story today.

SHEPHERD is on a mission to build a movement to revolutionize the rare cancer system and in our efforts to shine a light on the major gaps in the system, one that fails most rare cancer patients, we are launching our stories collection.

Stories are a way for us to connect as a community. A way for those who have suffered to share their pain. A way for us to begin to collectively heal. We invite you to share your stories with us, so that we can start to make real change. Share your story with us here:

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