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Researching Medulloblastoma with Care and Knowledge

, by Brittany Cordeiro, NCI-CONNECT Program Manager

Dr. Marta Penas-Prado


Dr. Marta Penas-Prado discusses her role on the NCI-CONNECT team and her interest in improving the lives of patients with medulloblastoma.

Medulloblastoma is a rare primary central nervous system (CNS) tumor in adults. Only an estimated 140 adults are diagnosed each year in the United States, making it challenging to develop clinical trials and gather enough knowledge to treat it. Neuro-oncologist Marta Penas-Prado, M.D., is ready to tackle this challenge to improve the lives of patients with medulloblastoma.

Dr. Penas-Prado recently joined the NCI Center for Cancer Research's Neuro-Oncology Branch as an associate research physician for a program called NCI-CONNECT. The program is dedicated to improving clinical care and research for patients with adult rare CNS tumors, including medulloblastoma, an area of expertise and focus for Dr. Penas-Prado.

Dr. Penas-Prado has worked in neuro-oncology for the past 13 years. Her interest in studying the brain and spinal cord started during her neurology residency in Spain. She had several mentors and colleagues who were passionate about neuro-oncology and that energy transmitted to her. When she finished her residency, she did a neuro-oncology fellowship in the United States. “I felt the need to contribute to the development of this relatively new field and to search for better therapies for patients with CNS tumors,” says Dr. Penas-Prado.

She also learned there are many ways to help a patient beyond prescribing a treatment. “We should not underestimate the power of helping patients understand their diagnosis by listening to their questions and concerns and answering them to the best of our knowledge,” says Dr. Penas-Prado.

A cancer diagnosis is life-changing for patients and their families and we have the ability to help guide them.

Dr. Penas-Prado

Her patients have taught her to live one day at a time and make the best of every day.

At NIH, Dr. Penas-Prado works with the neuro-oncology team to manage the care and treatment of patients with rare brain and spine tumors and leads translational and clinical research studies, which she hopes will help her learn more about the molecular characteristics of medulloblastomas in adults.

Understanding the Molecular Profile of Medulloblastomas 

Medulloblastomas are cancerous and fast-growing tumors. They form in the cerebellum, the bottom part of the brain located at the back of the skull. Dr. Penas-Prado has a special interest in understanding the natural history and the molecular high-risk factors of adult patients with medulloblastoma and developing translational studies to investigate new therapies. This information is key to improving care and treatment and, ultimately, patient outcomes.

“In the last decade, researchers have discovered that medulloblastoma is no longer a single disease and can be classified into molecular subgroups,” explains Dr. Penas-Prado. “Research also shows potential actionable molecular targets for some molecular subtypes, like Sonic Hedgehog activated tumors.”

Knowing the molecular profile or characteristics of a disease is a critical step to develop drugs to target and kill cancer cells. Developing and testing drugs in research labs is the first step to ensure the drugs are safe for a clinical trial or testing in patients and more clinical trials are needed for adult patients with medulloblastoma. 

“Clinicians have debated for decades whether or not chemotherapy should be used in combination with radiotherapy as the first treatment for all adult patients with medulloblastoma,” says Dr. Penas-Prado. “Due to the lack of trials, this question remains without a definitive answer. Investigating the molecular factors to determine more aggressive tumor behavior is critical to finding out which patients need more intense therapy after they are diagnosed.”

Collaborating to Find Better Therapies

With Dr. Penas-Prado’s expertise, NCI-CONNECT plans to investigate the molecular risk factors for adult medulloblastoma to understand the differences in patient outcomes. “By gathering a better understanding of the clinical course of these patients (or how the disease behaves over time) and the molecular features of their tumors, we aim to adjust the intensity of therapy to what each patient needs, improving survival and decreasing the long-term side effects from treatment,” says Dr. Penas-Prado.

Dr. Penas-Prado also wants to help patients in every possible way, including physically and emotionally, even when there is not a cure. She believes NCI-CONNECT will help crystalize the efforts of health care providers, researchers, advocacy organizations, and patients to work together on finding better therapies and further, educating and supporting patients and their families.

The first step is for patients to visit NIH for an evaluation. “We can help counsel patients on the best course of action to treat their tumors and they can help us gather the clinical and molecular information we need to learn more about their tumors and improve care,” says Dr. Penas-Prado. Together, NCI-CONNECT and its collaborators hope to make a difference for patients.

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