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Natural History Study for Rare Brain and Spine Tumors Enrolls One Thousand Participants

, by Raleigh McElvery, Neuro-Oncology Branch Scientific Communications Editor

Dr. Armstrong conducts a cognitive assessment with a patient

Dr. Terri Armstrong's team leads the Natural History Study, which is an observational study that collects data from patients to track their disease over time.


In the six years since it began, the Natural History Study has amassed an enormous database of information that is helping to improve treatment and quality of life for patients with rare central nervous system cancers.

The NCI Center for Cancer Research’s Neuro-Oncology Branch (NOB) recently reached a momentous milestone: enrolling 1,000 participants in its Natural History Study.

A natural history study is an observational study that collects information and data from patients to track their disease over time. Unlike clinical trials, it does not test new treatments. Instead, researchers monitor patients’ clinical trajectory through the course of their illness using patient-reported data, clinical assessments, and biological samples. The ultimate goal is to learn about a disease by studying the people living with it. 

The NOB’s Natural History Study is open to any adult (age 18 and older) with a brain or spine tumor—whether they are at risk of developing these cancers, recently diagnosed, on active treatment, or undergoing imaging surveillance. The researchers gather information about the patient’s clinical course, including: the treatments they receive, their intercurrent illnesses, and the results of any imaging studies conducted to monitor their tumor. The researchers also collect and analyze tissue and blood samples, self-reported patient and family health history, past exposures, and socioeconomic factors—in addition to patient-reported outcomes designed to assess current health status, mood disturbances, and perceived cognitive symptoms. Using this information, the researchers aim to better understand these rare tumors and uncover areas for further research that could improve treatment and quality of life for cancer patients. 

The NCI-CONNECT program regularly utilizes data from the Natural History Study to design clinical trials to treat select rare brain and spine tumors. NCI-CONNECT’s ongoing phase 2 trial examining the efficacy of a drug called nivolumab is one such example. NCI-CONNECT researchers also leverage Natural History Study data to explore other research questions, including how to assess patients’ cognitive function, how to define the characteristics of different brain tumors, and how the COVID-19 pandemic has affected symptom burden. 

“It’s extremely exciting that the Natural History Study has enrolled 1,000 patients,” says Terri Armstrong, Ph.D., the NOB’s deputy director and co-leader of NCI-CONNECT. “The data have allowed us to better understand the experiences of patients diagnosed with these tumors—and the impact of our treatment on not just the tumor, but the person as well. The study has also helped us to identify areas of need for treatment, education, and supportive care interventional trials.” 

When the study was launched in 2016, Dr. Armstrong’s team quickly got to work to ensure the data collected would capture a comprehensive overview of the patient experience. 

Alvina Acquaye is a psychosocial behavioral specialist for NCI-CONNECT and has been a member of Dr. Armstrong’s patient outcomes research team since joining the NOB with Dr. Armstrong in 2016. She has helped extensively with the Natural History Study, and previously managed sending web-based surveys to patients and monitoring completion status. She now serves as the program manager for Dr. Armstrong’s Patient Outcomes Research Program and has enjoyed working as a team to ensure patient participation remains high. 

“Having such a dedicated team and so many patients willing to contribute allows us to collect an enormous amount of data that will help us understand our patients and their needs,” she explains. “It will also help us develop interventions to help them cope with their disease.”

To date, there have been more than a dozen published manuscripts and over 50 abstracts presented at national meetings using data from the Natural History Study data. Dr. Armstrong’s lab alone has made landmark discoveries providing insight into post-radiation fatigue and hypersomnolence, exploring lived experience for those surviving longer than five years, identifying symptom patterns across ethnoracial groups, analyzing the role depression and anxiety play in disease progression, and more. The Natural History Study data have also been used to evaluate molecular and imaging changes associated with treatment, as well as discover new tumor entities—including MYCn amplified ependymoma, which was included in the World Health Organization’s 2021 classification of brain tumors.

Having such a dedicated team and so many patients willing to contribute allows us to collect an enormous amount of data that will help us understand our patients and their needs.

Alvina Acquaye, Outcomes Section Program Manager

Data collection, storage, and analysis is overseen by Mathematical Statistician Elizabeth Vera, who has worked with Dr. Armstrong for 15 years. Brain and spine tumors are so rare, Vera says, that a dataset of one or two hundred is considered exceptional. “The fact that our Natural History Study has over 1,000 patients enrolled is outstanding. That’s more participation than we could have hoped for.”

Research groups often contact Vera asking whether the Natural History Study could incorporate other variables to help answer additional research questions. For example, Vera once added specific questions to the patient questionaries to help a group of radiation oncologists understand how cancer patients perceive themselves after radiation treatment. She also helped gather data for a team of psychologists interested in the meaning of life.

“I get excited when I hear other people talk about our data,” Vera says. “We like to collaborate with investigators, and if we can support their special research interests through the Natural History Study, we’re happy to do that.”

Anna Choi, NCI-CONNECT’s clinical research coordinator, has been assisting with the Natural History Study since 2019. She screens patients, prepares reports, mentors and supervises trainees, and conducts one-year follow ups with patients, among other key duties. She remembers when—not too long ago—the study had only a few hundred patients enrolled. 

“I am happy to watch the study grow and to be part of the study team,” she says. “Communicating with the patients and collecting data to better understand rare brain and spine tumors is my favorite part.”

While Dr. Armstrong and her team of staff and trainees are thrilled about enrolling 1,000 patients, they are most excited about how the data have helped them understand how tumors and treatment approaches affect all the patients who come to the NOB. 

“I don’t see an end in sight,” Vera says. “The study will go on for as long as there are researchers and patients who are excited to participate, and we will continue to learn from them to improve the care we provide.”

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