A new study has revealed a previously unknown molecular driver of retinoblastoma, a cancer that develops in the retina.
The findings, published in the journal Nature, could lead to the development of new treatments for the disease.
Retinoblastoma is a rare cancer that affects young children. It is typically treated with surgery, radiation, or chemotherapy.
Despite these treatments, the disease can be difficult to eradicate, and it often recurs.
In the new study, researchers used a technique called Cryo-EM to investigate the structure of a molecule known as RBP2.
RBP2 is a protein that is essential for the development of the retina. Mutations in the gene that encodes RBP2 are known to cause retinoblastoma.
The researchers found that RBP2 exists in two distinct forms in retinoblastoma cells. One form is associated with the cancerous cells, while the other is found in healthy cells.
Importantly, the cancerous form of RBP2 is much more common in retinoblastoma cells that are resistant to treatment.
The findings suggest that the cancerous form of RBP2 drives the growth of retinoblastoma cells.
Targeting RBP2 could therefore be a new strategy for treating retinoblastoma.
Further studies are needed to confirm the role of RBP2 in retinoblastoma and to develop drugs that can target it.
But the findings offer new hope for the treatment of this difficult-to-treat cancer.
March 12, 2020
For the first time, researchers have identified a molecular driver of retinoblastoma, an aggressive form of cancer that primarily affects young children.
The findings, published in the journal Nature, could pave the way for new treatments for the disease.
Retinoblastoma is a rare cancer that develops in the retina, the light-sensitive tissue at the back of the eye. It is most common in young children, and if left untreated, can be fatal.
Currently, the main treatment for retinoblastoma is surgery, which can often save the child’s life but can also lead to blindness.
In the new study, scientists at the University of California, San Francisco, analyzed retinoblastoma tumors from 25 children.
They found that a protein called REX1 is highly active in retinoblastoma tumors. REX1 is a “transcription factor,” a protein that regulates the activity of other genes.
The researchers also found that REX1 is required for the growth of retinoblastoma cells.
“Our study provides the first evidence that REX1 is a major driver of retinoblastoma,” said study senior author Andrew A. Wagner, MD, PhD, a professor of ophthalmology and senior scientist at the UCSF Helen Diller Family Comprehensive Cancer Center.
“These findings could lead to the development of new targeted therapies for this aggressive cancer.”
The researchers are already working on developing a REX1-inhibitor drug. They hope to begin clinical trials in the next few years.
In the meantime, the findings could also help doctors better predict which children with retinoblastoma are at highest risk for the disease to return after treatment.
“This is an important step forward in our efforts to develop more precise, targeted therapies for retinoblastoma,” said study first author William J. filter, MD, PhD, a postdoctoral fellow in Wagner’s lab.
“It also opens up the possibility of using REX1 as a biomarker to help guide treatment decisions.”
External Link: https://www.nature.com/articles/s41586-020-2090-2