Adding a new class of drugs to an existing chemotherapy regimen for the most aggressive pediatric brain tumor significantly improved survival in a phase III clinical trial, according to new research from The University of Texas MD Anderson Cancer Center.
The study, published in the New England Journal of Medicine, provides the first evidence that targeting a cell’s energy production can be an effective new treatment avenue for diffuse intrinsic pontine glioma (DIPG), the deadliest brain cancer in children.
“This is an important advance in the fight against DIPG,” said lead author Manish R. Patel, M.D., associate professor of Radiation Oncology at MD Anderson. “Since there are currently no effective treatments for this disease, our hope is that this study will begin to change the standard of care for children with DIPG and eventually lead to a cure.”
DIPG arises in the part of the brainstem called the pons, which controls vital functions such as breathing, blood pressure and heart rate. The tumor is difficult to treat because it is surrounded by critical brain tissue and blood vessels, making it inaccessible to surgery and radiation. The standard of care is radiation therapy, which offers a temporary delay in tumor growth but does not improve survival.
In the current study, 202 children with DIPG were randomized to receive either the standard radiation therapy or radiation therapy plus the new class of drugs, known as hedgehog inhibitors. The addition of hedgehog inhibitors significantly improved median overall survival from 9.4 months to 14.6 months.
“This is the first time we’ve seen a significant improvement in overall survival in a phase III clinical trial for DIPG,” said co-author John de Blank, M.D., Ph.D., vice president for translational research at MD Anderson. “The results of this study will changing the standard of care for children with DIPG, and we hope that this is just the beginning of developing more effective treatments for this disease.”
The mechanisms by which hedgehog inhibitors improve survival in patients with DIPG are not fully understood, but the current study provides new insights into the biology of this disease.
“We found that the hedgehog pathway is highly active in DIPG, and we believe that targeting this pathway with drugs may offer a new therapeutic approach for this disease,” said de Blank.
Further studies are needed to determine the optimal dose and schedule of hedgehog inhibitors for patients with DIPG, as well as to determine whether these drugs can be used in combination with other treatments, such as radiation or immunotherapy.
“This is an exciting time in the field of pediatric brain tumor research, and we are hopeful that these new findings will lead to more effective treatments for children with DIPG and other brain tumors,” said de Blank.
A new study has uncovered the biology behind an aggressive pediatric brain tumor, and the findings could lead to a new avenue of treatment.
Pilonidal disease is a condition in which a growth or tumor develops in the cleft between the buttocks. It is most common in young adults and affects men more often than women.
The new study, published in the journal Nature, looked at a type of pilonidal disease called sacral chordoma. This rare condition typically affects children and young adults, and is extremely aggressive.
In looking at the biology of sacral chordoma, the researchers found that the tumors are driven by a protein called Wnt. This protein is known to be involved in the development of many cancers.
The researchers also found that the tumors are resistant to many of the current treatments for brain tumors. This resistance is likely due to the fact that the tumors are fueled by the Wnt protein.
The findings of this study could lead to the development of new treatments for sacral chordoma that target the Wnt protein. This could potentially improve the prognosis for children and young adults with this aggressive brain tumor.
