Prof. Adrain Krainer's Distinguished Lecture Highlights Antisense Therapy Research for Brain Cancer

Prof. Adrian Kariner

I was honored to host Prof. Adrian Krainer from Cold Spring Harbor Laboratory last week, who presented a Distinguished Lecture entitled "Antisense Therapy for H3.3K27M-Related Diffuse Midline Glioma."

This prestigious lecture, held in honor of Dr. Martin Rodbell, brought together scientists across the National Institute of Environmental Health Sciences (NIEHS) to learn about cutting-edge antisense oligonucleotide research and its potential to treat brain cancers.

Prof. Krainer, an internationally recognized leader in RNA splicing and antisense drug development, shared his lab's groundbreaking work developing Spinraza, an FDA-approved antisense therapy that has proven revolutionary for treating spinal muscular atrophy. The simplicity of the antisense RNA strategy and its remarkable impact on patients' quality of life seems almost miraculous.

He then discussed his current research focused on diffuse midline gliomas, a devastating pediatric brain cancer lacking effective therapies. Prof. Krainer's preliminary findings using antisense oligos to target oncohistone mutations in gliomas using mouse models are encouraging. Antisense RNA has already dramatically altered disease treatment for neurodegeneration; given these positive early results, its potential utility in cancer merits further investigation.

NIEHS Rodbell Lecture Award

Prof. Krainer's presentation facilitated insightful dialogue around expanding antisense technologies towards previously untreatable conditions. Over lunch with NIEHS fellows and in meetings with investigators, he discussed critical next steps for translating fundamental research on antisense RNAs and RNA splicing dysfunction into clinical therapies that substantially improve human health.