Novel, stem cell-based therapy could slip past blood-brain barrier
DUARTE, Calif. — Despite continual advances in the treatment of brain tumors, infants and children diagnosed with medulloblastoma — a fast-growing tumor in the cerebellum portion of the brain, near where the spinal cord joins the brain — still face significant challenges overcoming this disease. One particularly tricky obstacle is the blood-brain barrier, which prevents cancer drugs from passing into the brain and attacking the tumor.
Margarita Gutova, M.D., an assistant research professor in the laboratory of Karen Aboody, M.D., professor in the Department of Neurosciences and the Division of Neurosurgery, in collaboration with Robert Wechsler-Reya
, Ph.D., director of the tumor initiation and maintenance program at the Sanford Burnham Medical Research Institute
, may have found a way to get through that barrier — using neural stem cells.
Neural stem cells can generate the different cell types in the brain. In their stem cell state, these cells can cross the blood-brain barrier and migrate selectively to invading tumor cells in the brain. Used as a delivery vehicle, neural stem cells can be engineered to target and deliver anti-cancer agents specifically to brain tumor sites. This method results in concentrated therapy at the tumor sites, while minimizing harm to surrounding normal tissue.
Five foundations — Alex’s Lemonade Stand Foundation, Pediatric Cancer Research Foundation (PCRF), The Matthew Larson Foundation for Pediatric Brain Tumors — and two anonymous foundations have announced grants totaling $450,000 in support of Gutova’s work, allowing her to continue her translational research of this novel treatment method. If additional results proves promising, Gutova said she will proceed with development towards patient trials.
These specific neural stem cells have already demonstrated safety when administered into the brain in adult patients with recurrent glioma. Dr. Gutova is studying a way to deliver the therapeutic neural stem cells intranasally. This novel delivery method, if proven effective, is much less invasive, and could reduce the number of complicated procedures — and their associated risks — that these young patients must often endure.
She is especially hopeful that the neural stem cells can give children a better option than surgery and chemotherapy. These standard therapies can be “damaging to brain and skeletal development, especially for pediatric patients’ still-growing bodies,” Gutova said.
“When the traditional treatment protocol failed for my daughter Alex, clinical trials became our best and only option for combating her cancer,” said Jay Scott, co-executive director of Alex’s Lemonade Stand Foundation. We know firsthand how important these trials are to bettering the lives of childhood cancer patients, and we are dedicated to bringing promising research from the lab to the clinic. We see promise in Dr. Gutova’s brain tumor research and are glad to be able to support her efforts. “
“We are proud to support City of Hope and Dr. Gutova’s research. City of Hope is a leader in making a difference every day in the laboratory, clinics and the lives or our young patients,” said Jeri Wilson, executive director of PCRF. “I know Dr. Gutova and her colleagues will strive every day to ensure their research delivers the best possible outcomes to families who so richly deserve a cure.”
“Dr. Gutova’s work in neural stem cells is an exciting development in pediatric brain tumors and we’re delighted to help make that research a reality,” says Kelly Larson, president of The Matthew Larson Foundation.
Medulloblastomas are the most common type of malignant brain tumors among children and infants, with approximately 2,600 new diagnoses in the United States each year. According to the American Brain Tumor Association, prognoses for medulloblastoma vary widely, depending on age and stage of diagnosis. For infants diagnosed with localized disease, the five-year survival rate is 30 to 50 percent.
About Alex’s Lemonade Stand Foundation
Alex's Lemonade Stand Foundation (ALSF) emerged from the front yard lemonade stand of cancer patient Alexandra “Alex” Scott (1996-2004). In 2000, 4-year-old Alex announced that she wanted to hold a lemonade stand to raise money to help find a cure for all children with cancer. Since Alex held that first stand, the Foundation bearing her name has evolved into a national fundraising movement, complete with thousands of supporters across the country carrying on her legacy of hope. To date, Alex’s Lemonade Stand Foundation, a registered 501(c)3 charity, has raised more than $75 million toward fulfilling Alex’s dream of finding a cure, funding over 375 pediatric cancer research projects nationally. For more information on Alex’s Lemonade Stand Foundation, visit http://www.AlexsLemonade.org
About Pediatric Cancer Research Foundation
The Pediatric Cancer Research Foundation (PCRF) is an independent, nonprofit organization, wholly dedicated to funding leading-edge pediatric cancer research nationwide. Since its founding in 1982, PCRF has raised over $32 million toward the ultimate goal of ending pediatric cancers so children and their families can refocus on the joys of childhood. This commitment has translated into immediate treatment for children with cancer by developing innovative treatment protocols; improving patients’ long-term quality of life; and supporting those scientists working on the precipice of important medical breakthroughs. For more information, please visit www.PCRF-kids.org
About The Matthew Larson Foundation for Pediatric Brain Tumors
The mission of The Matthew Larson Foundation for Pediatric Brain Tumors is to raise awareness and funds necessary to overcome pediatric brain tumors and to help affected children and families. The Foundation hopes to ease the financial burdens on the families who have a child battling this horrible disease with family assistance grants, while also funding medical and scientific research to find a cure. It is the Foundation’s hope that one day no child will have to endure what Matthew and the thousands of other newly diagnosed children face every year. The Foundation has assembled a team of distinguished medical professionals and a committed group of hard working board members and volunteers to help us carry out its mission.
About City of Hope
City of Hope is a leading research and treatment center for cancer, diabetes and other life-threatening diseases. Designated as a comprehensive cancer center, the highest recognition bestowed by the National Cancer Institute, City of Hope is also a founding member of the National Comprehensive Cancer Network, with research and treatment protocols that advance care throughout the nation. City of Hope’s main hospital is located in Duarte, Calif., just northeast of Los Angeles, with clinics in Antelope Valley and South Pasadena. It is ranked as one of "America's Best Hospitals" in cancer by U.S.News & World Report. Founded in 1913, City of Hope is a pioneer in the fields of bone marrow transplantation and genetics.