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Science Daily (January 30, 2012)
Genetic Breakthrough for Brain Cancer in Children
ScienceDaily (Jan. 30, 2012) — An international research team led by the Research Institute of the McGill University Health Centre (RI MUHC) has made a major genetic breakthrough that could change the way pediatric cancers are treated in the future. The researchers identified two genetic mutations responsible for up to 40 per cent of glioblastomas in children -- a fatal cancer of the brain that is unresponsive to chemo and radiotherapy treatment. The mutations were found to be involved in DNA regulation, which could explain the resistance to traditional treatments, and may have significant implications on the treatment of other cancers.
The study was published this week in the journal Nature.
Using the knowledge and advanced technology of the team from the McGill University and Génome Québec Innovation Centre, the researchers identified two mutations in an important gene known as the histone H3.3. This gene, one of the guardians of our genetic heritage, is key in modulating the expression of our genes. "These mutations prevent the cells from differentiating normally and help protect the genetic information of the tumor, making it less sensitive to radiotherapy and chemotherapy," says Dr. Nada Jabado, hematologist-oncologist at The Montreal Children's Hospital of the McGill University Health Centre (MUHC) and principal investigator of the study.
"This research helps explain the ineffectiveness of conventional treatments against cancer in children and adolescents -- we've been failing to hit the right spot," says Dr. Jabado, who is also an Associate Professor of Pediatrics at McGill University. "It is clear now that glioblastoma in children is due to different molecular mechanisms than those in adults, and should not be treated in the same way. Importantly, we now know where to start focusing our efforts and treatments instead of working in the dark."
Inappropriate regulation of this gene has been observed in other cancers such as colon, pancreatic, lymphoma, leukemia and pancreatic neuroendocrine cancer, and future research could therefore reveal improved treatments for these diseases. "What is significant here is that for the first time in humans we have identified a mutation in one of the most important genes that regulates and protects our genetic information. This is the irrefutable proof that our genome, if modified, can lead to cancer and probably other diseases. What genomics has shown us today is only the beginning," says Dr. Jabado.
"Génome Québec is proud to have contributed to a project whose results will make a significant impact on the treatment of pediatric glioblastoma," underlines Marc Le Page, President and CEO of Génome Québec. "The outstanding contribution of experts in genomics and new sequencing technologies, made by the McGill University and Génome Québec Innovation Centre and as part of Dr. Jabado's project, is further proof that genomics has become essential for development and innovation in medical research. I wish to acknowledge the excellence of the teams involved in this study and the model of interdisciplinary collaboration that was implemented."
"Personalized medicine has amazing potential for many areas of health care, including infection, rare diseases and cancer. Researchers, like this team, play a vital role in translating discoveries into improved patient care," says Dr. Morag Park, Scientific Director of the CIHR Institute of Cancer Research. "Through research advancements like this, there is now greater emphasis on using genetic information to make medical decisions. We congratulate Dr. Jabado and her team on these results." Brain tumours are the primary cause of death for children with cancer in Europe and North America. The diagnosis of glioblastoma in a child or adolescent remains a death sentence and about 200 children in Canada die every year of this cancer. Most children will die within the two years of their diagnosis regardless of treatment. This work was supported by the Cole Foundation, and was funded in part by Genome Canada and the Canadian Institute for Health Research (CIHR) with co-funding from Genome BC, Génome Québec, CIHR-ICR (Institute for Cancer Research) and C17, through the Genome Canada/CIHR joint ATID Competition (project title: The Canadian Paediatric Cancer Genome Consortium: Translating next generation sequencing technologies into improved therapies for high-risk childhood cancer.
Research findings: http://www.sciencedaily.com/releases/2012/01/120130102522.htm
October 1, 2010
New Therapy Boosts Neuroblastoma Two-Year Cure Rate
Contact: Rachel Salis-Silverman, Department of Public Relations, 267-426-6063
Using immunotherapy—biologic agents that stimulate the body's immune system—pediatric oncologists have achieved the first substantial increase in over a decade in cure rates for the childhood cancer neuroblastoma. A newly released study shows that the new treatment improved two-year survival rates by 20 percentage points, compared to standard treatment for an aggressive form of neuroblastoma, a cancer of the nervous system.
The study appears in the Sept. 30 issue of the New England Journal of Medicine, along with a separate COG study on intermediate-risk neuroblastoma. The corresponding author of the immunotherapy study is Alice L. Yu, MD, PhD, of the University of California, San Diego.
Findings promise to set a new standard for treating neuroblastoma "We expect these findings will change clinical practice, setting a new gold standard of treatment for this often-deadly disease," said John M. Maris, MD, a co-author of the study and director of the Center for Childhood Cancer Research at The Children's Hospital of Philadelphia. Maris is the chair of the neuroblastoma committee of the Children’s Oncology Group (COG), the cooperative multicenter research organization that sponsored the study.
Neuroblastoma, a cancer of the peripheral nervous system, usually appears as a solid tumor in the chest or abdomen. It accounts for 7 percent of all childhood cancers, but because it frequently occurs in an aggressive form, it causes 15 percent of all childhood cancer deaths. While low-risk forms of neuroblastoma may spontaneously disappear, in high-risk forms, the cancer tends to return after initial treatment, usually with lethal results.
For more detail on how the study was conducted please visit the following link:
http://www.chop.edu/news/new-therapy-boosts-neuroblastoma-two-year-cure-rate.html
Gene That Causes Childhood Cancer Neuroblastoma Is Found
Children's Hospital of Philadelphia Researchers Lead the Way to an Amazing Discovery That May Lead to New Prevention and Treatment Strategies
PHILADELPHIA, Aug. 25 - Scientists have discovered gene mutations that are the main cause of the inherited version of the childhood cancer neuroblastoma. In addition, the researchers found that the same mutations play a significant role in high-risk forms of non-inherited neuroblastoma, the more common form of the disease.
"This discovery enables us to offer the first genetic tests to families affected by the inherited form of this disease," said pediatric oncologist Yael P. Mosse, M.D., of The Children's Hospital of Philadelphia, the first author of the study, published online Aug. 24 in the journal Nature. "Furthermore, because there already are drugs in development that target the same gene in adult cancers, we can soon begin testing those drugs in children with neuroblastoma."
Neuroblastoma is the most common solid cancer of early childhood. It accounts for 7 percent of all childhood cancers, but due to its often aggressive nature, causes 15 percent of all childhood cancer deaths. It arises in the developing nerves of a child, often appearing as a tumor in the chest or abdomen.
CureSearch Praises Passage of Landmark
"Conquer Childhood Cancer Act"
by Unanimous Consent in the United States Senate
Children with Cancer and Their Families to Benefit from Legislation
July 17, 2008 (Bethesda, MD) - CureSearch National Childhood Cancer Foundation salutes the United States Senate for its passage of the Conquer Childhood Cancer Act, which promises to significantly increase federal investment into childhood cancer research.
The bill, introduced in the Senate by Senators Jack Reed (D-RI) and Norm Coleman (R-MN), passed without amendment by unanimous consent, echoing the 416-0 vote June 12th in the U.S. House of Representatives. Senator Ron Wyden (D-OR) on behalf of Senator Reed and himself asked for unanimous consent to proceed with consideration of the bill on the Senate floor.
The bill authorizes $30 million annually over five years, providing funding for collaborative pediatric cancer clinical trials research, to create a population-based national childhood cancer database, and to further improve public awareness and communication regarding available treatment and research for children with cancer and their families.
"Too many young people's lives were cut short by cancer, but their hopes were not," said Reed. "We have made great advances in treating cancer, but there is still much more to be done. The Conquer Childhood Cancer Act will deliver much needed hope and support to children and families battling cancer and more resources for vital pediatric cancer research programs."
Senator Coleman, an original sponsor, noted that the legislation passed unanimously in both Houses of Congress, and lauded the overwhelming bi-partisan support of the measure, which addresses a critical national health issue that is finally receiving the attention it deserves.
"Passage of the Conquer Childhood Cancer Act in the Senate is a monumental step in the fight against childhood cancer," said Coleman. "I am proud that my colleagues were able to come together and pass legislation that will provide the resources to not only support children and families with childhood cancer, but also find a cure."
December 12, 2007, Bethesda, MD - A new report released by the Centers for Disease Control and Prevention (CDC) reports that the cancer death rate for children in the United States has declined sharply - down 20 percent from 1990 to 2004.
Dr. Gregory Reaman, Chair of the Children's Oncology Group (COG), the world's premier pediatric cancer research collaborative, notes that these vastly improved outcomes are a direct result of the collaborative research of the COG.
"The Children's Oncology Group pools the resources and talents of more than 5,000 medical experts at more than 200 children's and university hospitals. By working together and sharing results, clinical investigators can improve cure rates at a much faster pace than could any one individual or single institution working alone."
A total of 2,223 pediatric cancer deaths occurred in 2004 (the most recent year that data is available) compared to 2,457 in 1990. In fact, the death rate for pediatric cancer has declined an average of 1.7% percent per year throughout this 15-year period. The leading subgroups of cancer deaths continue to be those from leukemias (25.5%) closely followed by brain and other central nervous system neoplasms (25.0%).
However, in spite of this progress, cancer remains the leading cause of death by disease for U.S. children. Further gains will require a continuing commitment to research; only research cures cancer.
"While we have made remarkable breakthroughs in cancer treatments and improvement in survival rates, too many children continue to die," Reaman notes. "Cancer remains the leading cause of death from disease in children in the United States. In addition, the price of cure due to the long term side effects of therapy is costly for many childhood cancer survivors and families."
About the Children's Oncology Group
The Children's Oncology Group is the world's largest cooperative childhood cancer research organization, treating more than 90% of children with cancer in the United States. The Children's Oncology Group includes more than 5,000 medical experts in childhood cancer research and treatment, located at more then 200 leading healthcare institutions in North America. The Children's Oncology Group and its network of doctors, nurses and researchers have taken childhood cancer from a nearly incurable disease to one with an overall cure rate of 78%.
News From Congress
Pending Legislation
The following bills related to childhood cancer have been introduced in the 110th Congress and are summarized here along with their level of Congressional support.
S 911/HR 1553Conquer
Childhood Cancer Act of 2007
Sponsors: Senators Norm Coleman (R-MN) and Jack Reed (D-RI), Representatives Deborah Pryce (R-Ohio) and Chris Van Hollen (D-MD).
Bill Status: Introduced in the House on March 15, 2007. Referred to Energy and Commerce Committee. Introduced in the Senate.
Cosponsors: 29 (House)
Summary: The Conquer Childhood Cancer Act of 2007 proposes to authorize $150 million over 5 years to develop grants for Research Fellowships in Pediatric Cancer for clinical and translational investigators, to fund a population-based national childhood cancer database, and to award grants to childhood cancer organizations to raise public awareness, ensuring access to best available therapies for pediatric cancers.
CCCA Position: Increased investment in childhood cancer research is necessary to build on past success, improve current treatments and develop new, safer and more effective therapies. These research needs are among many that need additional federal funds in order to make progress in treating children with cancer.
Please visit www.childrenscause.org to find out how to become more involved in helping children with cancer.
NIH RESEARCH HAMPERED BY UNDERFUNDING
Researchers from medical and scientific institutions throughout the country testified before the Senate Appropriations Committee about the consequences of inadequate research funding for the National Institutes of Health (NIH). This year eight out of ten research grant applications are going unfunded, according to testimony.
In recent years, the NIH budget has barely increased-- two percent in 2005 and one-tenth of one percent last year. "We're giving up our ability to do clinical trials and change the science of medicine," said NIH Director Elias Zerhouni, MD. He told the committee that NIH research has lent to important progress in health for Americans, such as the decline of cancer deaths for the second year in a row. Zerhouni also mentioned the importance of lifting funding restrictions on human embryonic stem-cell research.
All those testifying agreed that the current funding situation, if prolonged, would threaten advancement in health research, including promising new molecularly-targeted therapies for cancer.
Senator Tom Harkin (D-Iowa) chair of the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and a critic of President Bush's proposed NIH budget, assured those testifying that he would put up a fight and not allow further cuts to be made. He and Senator Arlen Specter (R-Penn) introduced a budget resolution, which would restore the 4.5-percent biomedical inflation rate for fiscal years 2006-2008. The Senate passed the measure 52-47.
A report from a consortium of major academic medical institutions also cited the perils of inadequate funding. "Flat funding of the NIH, combined with inflation, is eroding research budgets. Scientists are being forced to downsize their laboratories and abandon their most innovative and promising work," according to the report. The "funding slowdown" comes at a time of "escalating threats to human health" and puts at risk a generation of younger researchers who may be discouraged to apply for funding through the NIH.
The report also claimed that more and more scientists would be turning to other funding sources including foreign funders. In the past several years, the Children's Oncology Group, the cooperative group that manages the majority of pediatric cancer clinical trials, has had to appeal to alternative funding sources to offset severe budget shortfalls.
The University of California, Columbia University, Harvard University, Johns Hopkins University, the University of Texas at Austin, Washington University in St. Louis, the University of Wisconsin at Madison, and Yale University contributed to the report, "Within Our Grasp -- or Slipping Away?" available online at http://hms.harvard.edu/public/news/nih_funding.pdf.
