Finding a cure

October 19, 2020
Dr. Jezabel Rodriguez Blanco


Dr. Jezabel R. Blanco, one of MUSC's researchers, has made it her mission to find a cure for medulloblastoma, the most common of pediatric malignant brain tumors. Recently, Dr. Blanco shared with us a bit about her research and a prestigious award she received to study pediatric brain cancer.

You joined the MUSC team in January of this year; can you tell us what brought you to MUSC?

One of the main reasons I decided to join MUSC was the fact that it has not only an NCI-designated cancer center (MUSC Hollings Cancer Center), but also one of the only 15 centers in the United States dedicated to pediatric research (Darby Children’s Research Institute). It is not so easy to find a combination like this, and I needed it to develop my program in pediatric neuro-oncology.

Also, MUSC happens to pay special attention to junior faculty like me, providing us with mentoring, funding, and protected time for research. Moreover, all laboratories at MUSC are well-equipped and researchers have access to common equipment, standard resources, and more than 35 state-of-the-art shared research cores and facilities physically housed in campus. A proof of the quality of the research at MUSC is the $284 million in research grants that scientists bring annually to the institution. Moreover, MUSC and its pediatric research institute also promote the collaboration among pediatric scientists and physicians, which results in a more efficient and productive approach to pediatric research.

Altogether, MUSC, Hollings Cancer Center and Darby Children’s Research Institute offer a brilliant and energetic scientific environment. I feel honored to be part of this team, and I am confident that the exceptional environment at MUSC will guarantee the success of my science.

Can you explain a bit about the work you are doing and how it will benefit children with cancer?

My research program is focused on pediatric brain tumors and aimed at identifying novel therapeutics for this underserved group of patients. Due to the significant morbidity that current multimodal therapy (surgery, radiation, and chemotherapy) has in developing children, one of my major research lines is focused on finding targeted therapeutics for these tumors. Toxicity associated to actual treatments goes from mutism to mental retardation. These novel therapeutics are specifically intended for the treatment of medulloblastoma, the most common malignant brain tumor in children. I am not only looking for novel approaches to treat these tumors, I am also making sure that the toxicity associated to their use will not affect kids suffering of medulloblastoma for the rest of their lives.

Another part of my program is focused on finding novel therapeutics for some of the most lethal forms of pediatric brain tumors, such as those medulloblastomas harboring mutations in P53 or amplifications in MYC, or the diffuse intrinsic pontine glioma. Currently all these very high-risk pediatric brain tumors are virtually incurable. Thus, existing treatments only reduce the symptoms, but do not expand the survival of these children. I am trying to understand the drivers of the growth and propagation of these tumors, paying special attention to those cells responsible for tumor relapse: medulloblastoma stem cells. We need to make sure that next time a kid gets a diagnosis for one of these very high-risk tumors, the doctor will have a specific treatment available. We do not want more palliative care for these kids, we want to find a cure. This cannot wait.

What inspired you to do this type of research?

I got my Ph.D. in neurosciences, while my postdoctoral research was in a lab working in development and cancer signaling. As it happens that a developmental signaling pathway named Sonic Hedgehog plays key roles in controlling the growth of a subset of brain tumors, I could take advantage of my neurosciences background and my knowledge of cancer signaling to develop a program in Sonic Hedgehog-driven pediatric brain tumors. Everything kind of matches. I am glad I decided to focus my career on pediatric cancer research. In my opinion, saying the word “cancer” is always scary, but when you add the word “pediatric” before it the story totally changes. The kind of pain felt by the families going through a battle against pediatric cancer leaves me just wordless. They never recover from this. We need to end pediatric cancer, and we need to make sure morbidity associated to treatments is not going to compromise these kids for life. This should be a priority in science.

You were one of only two recipients nationwide to receive a grant from the Rally Foundation; can you share what the grant is and how it has/will help you advance your research?

My Rally award is focused on finding novel interventions for very-high risk patients with medulloblastoma, in particular, those harboring mutations in the tumor suppressor P53. I am trying to understand the drivers of the growth and relapse of these tumors, with the intention of finding novel druggable components. Identification and further validation of compounds targeting such druggable proteins could be game changing for the treatment of this subset of kids. If I am able to find a drug that will help just one of these kids and families, my research will be worth it.