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Advances in Brain Tumor Clinical Care and Research

, by Brittany Cordeiro, NCI-CONNECT Program Manager

Dr. Jing Wu talking about brain MRI scans

Dr. Jing Wu


The NCI Center for Cancer Research's Neuro-Oncology Branch is advancing care and treatment for people with brain tumors through collaborative scientific workshops, research discoveries, and successful clinical trials.

At the NCI Center for Cancer Research's Neuro-Oncology Branch (NOB), our neuro-oncology providers care for people with brain tumors. They also work with other specialists to improve treatments to help patients live longer and with a better quality of life.

Our neuro-oncology scientists work behind-the-scenes in labs to understand the biology of brain tumors. Their discoveries often lead to studies in people, or clinical trials. And a successful clinical trial can set a new treatment standard.

Together, our health care providers and scientists have made significant progress in clinical care and research for people with brain tumors. Read about our most recent accomplishments.

Collaborative Scientific Workshops and Meetings

NCI-CONNECT scientific workshops bring together brain cancer experts from around the world and across specialties—neuro-oncology, neuropathology, neurosurgery, and scientific research—with patient advocates to discuss the biology of a specific disease and find ways to collaborate to improve therapies and develop new clinical trials.

In 2018 and 2019, we successfully brought together health care providers, scientists, and advocates to discuss histone mutated (midline) gliomas, oligodendrogliomas, medulloblastomas, and ependymomas—and hosted a Clinical Outcomes Assessment Meeting. We also co-hosted the Fourth Biennial World Summit of Brain Tumor Patient Advocates.

Experts at the Histone Mutated Glioma Workshop identified the need for diagnostic and treatment standards, disease biology and biological targets, and disease-specific clinical trial designs. Their recommendations and action plan were published in Neuro-Oncology Advances in January 2020.

Participants at the Oligodendroglioma Workshop recognized the need to combine data, resources, and samples to develop better treatments for patients with oligodendrogliomas. Their recommendations and action plans were published in Neuro-Oncology Advances in December 2019 and include a multifaceted and interrelated approach that covers: biology and preclinical models, data sharing, advanced molecular diagnosis and imaging, clinical trial design, and patient outreach and engagement.

At the Medulloblastoma Workshop, participants reviewed advances in research and shared insights related to scientific and clinical challenges in studying and treating patients. Working groups identified unmet needs in clinical trial design, tissue acquisition and testing, tumor modeling, and measurement of clinical outcomes. Participants also identified opportunities for collaboration; discussed plans to create a working group of clinicians, researchers, and patient advocates; and developed specific action items and next steps to expedite progress in adult medulloblastoma.

The Fourth Biennial Ependymoma Consensus determined areas of research for ependymoma, including grading and diagnosing ependymoma, molecular and genetic testing, and clinical care and treatments to make progress in patient outcomes. With these goals and strategies, participants will collaborate to more effectively address these issues.

Participants at the Clinical Outcomes Assessment Meeting discussed the importance of incorporating—in both clinical care and clinical research—how a person with a brain tumor feels and functions. People with brain and spine tumors often struggle with many functional limitations and experience symptoms that negatively impact their daily lives. This group, representing clinical care experts, advocates, and clinical trial experts, developed core symptoms and functions that should be assessed. Input from the meeting was used to publish a paper in Lancet Oncology.

The Fourth Biennial World Summit of Brain Tumour Patient Advocates was co-hosted with the International Brain Tumour Alliance. It brought together 96 patient advocates from 25 countries around the world and leaders in neuro-oncology for three days. It was focused on the most important topics for the patient advocacy community, including treatment advances, quality of life measures, support and education resources, and more.

The success of our scientific workshops shows the importance of community collaborations. It encourages and motivates the development of partnerships and networks for the care of people with brain tumors.

Discoveries in Tumor Biology

In brain tumor research, NOB Investigators Orieta Celiku, Ph.D., Mark Gilbert, M.D. (NOB chief), and Orit Lavi, Ph.D., studied how glioblastoma cells can evolve over time through exploratory adaptation. This concept explains how organisms, including cancer cells, adapt and survive in stressful or new conditions. Being able to predict how these changes occur is a crucial step to increasing patient survival. The findings were published in Nature Communications in December 2019. This is an important step to understanding various parts of the tumor and how cells change or regrow after surgery, which is the first and best treatment option to increase patient survival in glioblastoma.

Another NOB investigator, Chunzhang Yang, Ph.D., discovered a new strategy for treating brain tumors with mutations in metabolic enzymes. He found that cancers with mutations in key metabolic enzymes, called isocitrate dehydrogenases (IDHs), disrupt normal oxygen metabolism. This leads to a buildup of a byproduct known as reactive oxygen species.

Using a preclinical model, Dr. Yang showed that a small molecule could inhibit the action of a protein that allows mutated cancer cells to survive. His findings were published in the Journal of the National Cancer Institute in February 2019 and introduce conceptual advances in the understanding of IDH-related malignancies.   

Progress in Clinical Care and Outcomes

NOB Deputy Chief Terri Armstrong, Ph.D., studies how patients feel and function during clinical trials. This includes what symptoms they experience, as well as when and how these symptoms impact their daily activities.

In a new study published in Lancet Oncology in February 2020, Dr. Armstrong showed how including specific data on how patients function and their symptoms in clinical trials can help determine the benefit of a trial. The study combined input from patients and their families, literature, and clinical groups to develop a standard list of recommended outcomes. The next steps are to formally establish these outcomes in all clinical trials.

Another remarkable feat was led by NOB Investigator Jing Wu, M.D., Ph.D. In December 2019, the U.S. Food and Drug Administration granted orphan drug status to zotiraciclib for use in patients with glioma. Gliomas comprise about 30 percent of all brain and central nervous system (CNS) tumors and 80 percent of all cancerous brain tumors. The types of gliomas include astrocytoma, ependymoma, glioblastoma, and oligodendroglioma.

This designation is based on results from Dr. Wu’s phase 1 clinical trial to evaluate zotiraciclib plus temozolomide for the treatment of recurrent anaplastic astrocytoma and glioblastoma. Dr. Wu’s team is now working with Dr. Gilbert to open a phase 2 study of zotiraciclib plus temozolomide versus temozolomide alone in recurrent high grade glioma patients.

Importantly, Dr. Armstrong, Dr. Gilbert and NOB Neuro-Oncologist Marta Penas-Prado, M.D., reviewed the recent progress in the molecular understanding and therapeutic options of select rare CNS tumors. The paper, published in Current Opinions in Neurology in December 2019, was designed to present an overview of the current state of the science related to patient care and tumor treatment. They concluded there is a need for dedicated clinical trials with a strong correlative component in patients of all ages with rare CNS tumors. Critical research questions include relevance of the selected target for specific tumor types, persistence of the actionable biomarker at recurrence, blood-brain barrier penetration, and analysis of mechanisms of primary and acquired resistance.

Improving Ependymoma Treatment Guidelines

Ependymoma is a rare tumor for which the role of chemotherapy has not been established—either for newly diagnosed or recurrent disease. Dr. Gilbert led the first prospective adult clinical trial of chemotherapy for recurrent ependymoma. The trial treated 50 adult patients with temozolomide and lapatinib.  

Results from this study, published in Neuro-Oncology in November 2018, demonstrated evidence of clinical activity, including objective responses and a nearly 40 percent stable disease rate at one year. Importantly, even in those with stable disease, patients undergoing this treatment had improvement in disease-related symptoms and stable-to-improved functional status. This combination regimen was so well tolerated that it has been endorsed by the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology as a standard treatment for adult patients with recurrent ependymoma. "This is a major step to providing hope for ependymoma and other rare brain and spine tumors," says Dr. Gilbert.

Our scientific and clinical care progress exemplifies the impact doctors, nurses, and researchers across the world can make when they come together. Brain tumors are challenging to understand and treat, but with focused collaborations we can continue to improve care and treatment.

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