Overcoming Resistance in High Risk Medulloblastoma

  • William Weiss, MD, PhD
  • Professor of Neurology
    University of California, San Francisco School of Medicine

Brain tumors are the leading cause of death from cancer in children, and medulloblastoma is the most common type of malignant brain cancer. Patients with high-risk medulloblastoma are particularly resistant to the treatments that currently exist. Researchers have identified mutations (changes) in genes not thought to cause cancer. These genes regulate the "epigenetic state" of the cell, and mutations in them inappropriately lead cells to express genes that should normally be silenced, or silence genes that should normally be expressed. The inappropriate expressing and silencing of these genes in high-risk medulloblastoma may cause the cancer to resist the treatments that are currently available.

A team of international researchers led by William Weiss, MD, PhD at the University of California, San Francisco, has been awarded a $1.88 million grant by CureSearch to investigate their hypothesis that drugs that reprogram (normalize) the epigenome, will block the inappropriate activation or silencing of DNA, thus normalizing gene expression. If this occurs, it will lead to improved outcomes for patients who do not respond to treatment.

To conduct their research, the team will first map the genetic changes (mutations) in genes regulating access to chromatin, which is a complex group of proteins that surround and compact the DNA. Then, based on their findings, they will begin testing medications on high-risk medulloblastoma specimens taken from patients during surgery, and in mice that have been genetically engineered to have this cancer. Their hope is that within three years, they can both identify and develop treatments that can be moved to a Phase I clinical trial in patients.

< Acceleration Initiative Grantees 


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