Featured Research

from universities, journals, and other organizations

Experimental targeted therapy shows early promise against medulloblastomas, type of childhood brain cancer

Date:
June 9, 2010
Source:
St. Jude Children's Research Hospital
Summary:
Researchers have presented findings of a pediatric brain tumor study using an experimental drug that targets the underlying genetic makeup of the tumor. The research focused on a new way to attack the tumors by blocking the Hedgehog pathway that is linked to approximately 20 percent of medulloblastomas.

Researchers from St. Jude Children's Research Hospital and the Pediatric Brain Tumor Consortium (PBTC) presented at the American Society of Clinical Oncology the findings of a pediatric brain tumor study using an experimental drug that targets the underlying genetic makeup of the tumor. The research focused on a new way to attack the tumors by blocking the Hedgehog pathway that is linked to approximately 20 percent of medulloblastomas.

The study is the first to report that the drug can be safely administered to children. The study also suggested that the drug is showing early signs of efficacy in this patient population, with some children still on treatment almost a year with no progression of disease. All the children on this study had medulloblastomas that persisted or returned despite standard treatment with radiation and chemotherapy. Recurrent medulloblastoma currently has a cure rate of less than 5 percent.

The researchers found that patients whose tumors had the Hedgehog molecular pathway activated appear to be some of the same patients who have responded to treatment in this trial, based on length of time on study. Investigators have observed tumor responses in similar young adult patients whose tumors had the Hedgehog molecular pathway activated. These early findings have given the green light for pediatric research to advance to a larger Phase II study scheduled to open later this year, and to increase the number of patients on the young adult study.

The Phase I study (PBTC -025) included 13 patients, 12 of them evaluable, ranging in age from 4 to 21 years. All received one of two different doses of GDC-0449 daily for a minimum of 28 days and continued on treatment for as long as their disease remained stable. In addition to determining the safety and dosing of this experimental drug for children, the trial also conducted extensive research into pathologic and genomic methods for better identifying tumors that have the Hedgehog pathway activated.

"Medulloblastomas are the most common malignant brain tumors in children," said Amar Gajjar, M.D., co-chair of the St. Jude Department of Oncology and principal investigator of the PBTC trial. "The trend in treating children with these cancers is toward targeted therapies like this one, which block key signaling pathways and disable the cancer's ability to function or reproduce. We know that this Hedgehog pathway is important in the growth of these especially hard-to-treat tumors."

The PBTC has an ongoing Phase II trial in recurrent medulloblastomas in young adults (age 22 and older), with this same agent, which has recently been expanded to include more patients. A Phase II trial of GDC-0449's effectiveness against recurrent medulloblastomas in children (up to age 21) will start later this year based on the results of today's reported Phase I trial.

All of the PBTC trials are being sponsored by the Division of Cancer Treatment and Diagnosis, National Cancer Institute (NCI) under a Cooperative Research and Development Agreement Letter of Intent between NCI and Genentech, Inc.

St. Jude is home to the nation's largest research-based pediatric brain tumor program. St. Jude investigators have played a pivotal role in advancing understanding of the molecular missteps that give rise to medulloblastomas as well as seeking new, more targeted therapies to combat the tumors. St. Jude investigators have published evidence that targeting the Hedgehog pathway eradicated medulloblastomas in laboratory models. During fetal development the Hedgehog pathway plays a central role in normal growth of the cerebellum. The cerebellum is located at the base of the skull and is the structure that helps coordinate movement and plays a role in mastering balance and other motor skills. Medulloblastomas begin in the cerebellum, and uncontrolled activity along this pathway is linked to several cancers, including basal cell skin cancer.

The Pediatric Brain Tumor Consortium (PBTC) is a federally-funded collaborative clinical trial group based at St. Jude Children's Research Hospital that also includes Children's Memorial Hospital, Chicago; Duke University Medical Center, Durham, N.C.; Texas Children's Cancer Center, Houston; Children's Hospital of Philadelphia; Children's Hospital of Pittsburgh; University of California at San Francisco; Children's National Medical Center, Washington D.C.; and the National Cancer Institute's Pediatric Oncology Branch. The consortium is dedicated to developing new treatments against the brain and central nervous system tumors that strike about 3,000 children and adolescents annually in the United States.


Story Source:

The above story is based on materials provided by St. Jude Children's Research Hospital. Note: Materials may be edited for content and length.


Cite This Page:

St. Jude Children's Research Hospital. "Experimental targeted therapy shows early promise against medulloblastomas, type of childhood brain cancer." ScienceDaily. ScienceDaily, 9 June 2010. <www.sciencedaily.com/releases/2010/06/100607101650.htm>.
St. Jude Children's Research Hospital. (2010, June 9). Experimental targeted therapy shows early promise against medulloblastomas, type of childhood brain cancer. ScienceDaily. Retrieved August 23, 2014 from www.sciencedaily.com/releases/2010/06/100607101650.htm
St. Jude Children's Research Hospital. "Experimental targeted therapy shows early promise against medulloblastomas, type of childhood brain cancer." ScienceDaily. www.sciencedaily.com/releases/2010/06/100607101650.htm (accessed August 23, 2014).

Share This




More Health & Medicine News

Saturday, August 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Drug Used To Treat 'Ebola's Cousin' Shows Promise

Drug Used To Treat 'Ebola's Cousin' Shows Promise

Newsy (Aug. 21, 2014) An experimental drug used to treat Marburg virus in rhesus monkeys could give new insight into a similar treatment for Ebola. Video provided by Newsy
Powered by NewsLook.com
Two US Ebola Patients Leave Hospital Free of the Disease

Two US Ebola Patients Leave Hospital Free of the Disease

AFP (Aug. 21, 2014) Two American missionaries who were sickened with Ebola while working in Liberia and were treated with an experimental drug are doing better and have left the hospital, doctors say on August 21, 2014. Duration: 01:05 Video provided by AFP
Powered by NewsLook.com
Cadavers, a Teen, and a Medical School Dream

Cadavers, a Teen, and a Medical School Dream

AP (Aug. 21, 2014) Contains graphic content. He's only 17. But Johntrell Bowles has wanted to be a doctor from a young age, despite the odds against him. He was recently the youngest participant in a cadaver program at the Indiana University NW medical school. (Aug. 21) Video provided by AP
Powered by NewsLook.com
American Ebola Patients Released: What Cured Them?

American Ebola Patients Released: What Cured Them?

Newsy (Aug. 21, 2014) It's unclear whether the American Ebola patients' recoveries can be attributed to an experimental drug or early detection and good medical care. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins