Pharmacy team working towards pediatric brain cancer research breakthrough

UNC Eshelman School of Pharmacy professor David Drewry and Carolina students are using open science to help find a cure for a rare pediatric brain cancer.

The team has been working to revolutionize the discovery and development of affordable medicines for diffuse intrinsic pontine glioma, an aggressive cancer that primarily affects young children. Limited treatment options are available, and life expectancy upon diagnosis is about a year.

Conducting their work through open science will make new medicines more affordable for families. Open science is a framework in which researchers share knowledge and research openly so others in the field can contribute to the project.

Project meetings are available on YouTube, and all research is done publicly, so researchers can choose to donate their time, ideas and resources to the project.

“We happen to be at one of the best universities for pharmaceutical sciences,” said Ikeer Mancera-Ortiz, a doctoral candidate in the pharmacy school’s chemical biology and medicinal chemistry division who is working on the research. “Research projects at ESOP are innovative and the faculty are at the top of their fields.”

This open science research has been done through the Structural Genomics Consortium at the UNC Eshelman School of Pharmacy. The SGC is a not-for-profit, public-private partnership that performs basic science of relevance to drug discovery. All material and intellectual output of the SGC is placed in the public domain for use without restriction.

“What’s so nice about the school of pharmacy is you have colleagues here with pharmaceutical industry experience, who know about the science of drug discovery, we have great lab facilities, and everyone is open to collaboration,” said Drewry.

Drewry is leading the medicinal chemistry portion of this research at Carolina with his group of students. A father of four, he feels committed to helping families experiencing this disease.

“One of my daughter’s classmates in school from probably 25 years ago now had a very rare cancer and sadly died from it. That got me interested in these diseases where the number of people studying them is much smaller than the more common cancers,” said Drewry. “It made me realize we need more people to think about these rare cancers and be able to run experiments to try to help find a cure. That was my initial realization: that these rare, understudied cancers need more people thinking about how to slow these cancers down or knock them out if possible.”

Mancera-Ortiz is mentoring a group of undergraduate students with this research. He also found inspiration to work on the project from his personal life.

“I have a little brother that I care about deeply, and the study of rare cancers is important. When Professor Drewry offered me this, I immediately jumped on it to help,” said Mancera-Ortiz.

Mutations in the kinase, ALK2, are linked to 25% of DIPG cases, making the kinase an attractive target for the Drewry lab that specializes in targeting this family of proteins. The team is working in partnership with M4K Pharma, a virtual biotech company focused on transforming how medicines for childhood diseases are developed. The goal of M4K Pharma is to advance an ALK2 inhibitor into clinical trials for DIPG.

The medicines created would ideally slow down the progression of the cancer and give children a longer lifespan. Due to most cancer medications not being efficacious on their own, combination therapy may be a viable solution for an aggressive cancer such as DIPG. An ALK2 inhibitor may be an important component of a successful therapy.

The M4K team has chosen a clinical candidate and is currently acquiring needed data to present a package to the Food and Drug Administration. When all elements have been collected, the team will work to advance their chosen candidate into a Phase 1 clinical trial.