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Anti-angiogenesis treatment improves hearing in some NF2 patients
July 09, 2009First successful medical treatment for tumor-inducing genetic disorder
Treatment with the angiogenesis inhibitor bevacizumab improved hearing and alleviated other symptoms in patients with neurofibromatosis type 2 (NF2). In a paper to appear in the July 23 New England Journal of Medicine, which is receiving early online release, researchers from Massachusetts General Hospital (MGH) report that bevacizumab treatment successfully shrank characteristic tumors in a small group of NF2 patients, the first reported successful NF2 treatment not involving surgery or radiation.
"This kind of treatment response is unprecedented," says Scott Plotkin, MD, PhD, of the Pappas Center for Neuro-Oncology in the MGH Cancer Center, lead author of the NEJM paper. "Our study is the first to provide evidence that a drug can shrink vestibular schwannomas - benign tumors on the balance and hearing nerves - and the first to show that patients' hearing can be improved."
NF2 is an inherited genetic disorder in which benign tumors develop throughout the nervous system. Vestibular schwannomas are the most common NF2-associated tumors, and although they grow slowly, they usually cause patients to lose all or most of their hearing by young adulthood or middle age. The tumors can be removed surgically or treated with radiation, but in patients with vestibular schwannomas on both sides, which is typical in NF2, such treatment usually leads to complete hearing loss. Growing vestibular schwannomas can also press on the brainstem, leading to headaches, difficulty swallowing and other serious neurologic symptoms.
Since vestibular schwannomas are benign tumors, it was believed that they did not stimulate formation of new blood vessels as malignant tumors do. However, when the researchers studied tissue samples from NF2-related schwannomas, sporadic tumors not caused by NF2 and normal spinal nerves, they found evidence of excess blood vessel development and increased expression of angiogenesis-related molecules in both NF2-associated and sporadic vestibular schwannomas. With this suggestion that angiogenesis was involved in these tumors, members of the research team offered treatment with bevacizumab (Avastin), which is FDA-approved for treatment of several forms of cancer, to NF2 patients in danger of complete hearing loss or other significant neurological damage.
Among the first ten NF2 patients to receive bevacizumab, treatment led to tumor shrinkage in nine, and six had 20 percent or greater reduction in tumor size. In those six patients, tumor shrinkage lasted from 11 to 16 months, longer than the four months typically seen in bevacizumab treatment of malignant brain tumors. Of seven patients who had started to lose their hearing before treatment, four experienced some hearing restoration - two returning to work or school as a result - improvement that has also lasted for up to 16 months. In one patient without significant tumor shrinkage or hearing improvement (he had lost all hearing prior to treatment), treatment alleviated headaches and nausea caused by brainstem compression, allowing him also to return to school.
"This study has opened a new approach to research and understanding of these tumors," says Emmanuelle di Tomaso, PhD, the study's senior author, formerly with the Steele Laboratory of Tumor Biology in the MGH Department of Radiation Oncology. "There had been a dogma that these tumors do not produce edema and are not angiogenic, concepts that now need to be reevaluated." She adds that the study also suggests that VEGF - the angiogenesis factor blocked by bevacizumab - may have a role in nerve physiology beyond the stimulation of blood vessel growth.
Plotkin notes, "Based on the results of this study, we have just opened the first formal clinical trial of a drug treatment for NF2. We are testing an exciting new, oral VEGF inhibitor that will be easier for patients to take - bevacizumab is administered intravenously - and may have fewer side effects."
Massachusetts General Hospital
NF2 is an inherited genetic disorder in which benign tumors develop throughout the nervous system. Vestibular schwannomas are the most common NF2-associated tumors, and although they grow slowly, they usually cause patients to lose all or most of their hearing by young adulthood or middle age. The tumors can be removed surgically or treated with radiation, but in patients with vestibular schwannomas on both sides, which is typical in NF2, such treatment usually leads to complete hearing loss. Growing vestibular schwannomas can also press on the brainstem, leading to headaches, difficulty swallowing and other serious neurologic symptoms.
Since vestibular schwannomas are benign tumors, it was believed that they did not stimulate formation of new blood vessels as malignant tumors do. However, when the researchers studied tissue samples from NF2-related schwannomas, sporadic tumors not caused by NF2 and normal spinal nerves, they found evidence of excess blood vessel development and increased expression of angiogenesis-related molecules in both NF2-associated and sporadic vestibular schwannomas. With this suggestion that angiogenesis was involved in these tumors, members of the research team offered treatment with bevacizumab (Avastin), which is FDA-approved for treatment of several forms of cancer, to NF2 patients in danger of complete hearing loss or other significant neurological damage.
Among the first ten NF2 patients to receive bevacizumab, treatment led to tumor shrinkage in nine, and six had 20 percent or greater reduction in tumor size. In those six patients, tumor shrinkage lasted from 11 to 16 months, longer than the four months typically seen in bevacizumab treatment of malignant brain tumors. Of seven patients who had started to lose their hearing before treatment, four experienced some hearing restoration - two returning to work or school as a result - improvement that has also lasted for up to 16 months. In one patient without significant tumor shrinkage or hearing improvement (he had lost all hearing prior to treatment), treatment alleviated headaches and nausea caused by brainstem compression, allowing him also to return to school.
"This study has opened a new approach to research and understanding of these tumors," says Emmanuelle di Tomaso, PhD, the study's senior author, formerly with the Steele Laboratory of Tumor Biology in the MGH Department of Radiation Oncology. "There had been a dogma that these tumors do not produce edema and are not angiogenic, concepts that now need to be reevaluated." She adds that the study also suggests that VEGF - the angiogenesis factor blocked by bevacizumab - may have a role in nerve physiology beyond the stimulation of blood vessel growth.
Plotkin notes, "Based on the results of this study, we have just opened the first formal clinical trial of a drug treatment for NF2. We are testing an exciting new, oral VEGF inhibitor that will be easier for patients to take - bevacizumab is administered intravenously - and may have fewer side effects."
Massachusetts General Hospital
Neurofibromatosis Current Events and Neurofibromatosis News RSSLoss of tumor supressor gene essential to transforming benign nerve tumors into cancers
Researchers at UCLA's Jonsson Comprehensive Cancer Center showed for the first time that the loss or decreased expression of the tumor suppressor gene PTEN plays a central role in the malignant transformation of benign nerve tumors called neurofibromas into a malignant and extremely deadly form of sarcoma.
New research strategy for understanding drug resistance in leukemia
UCSF researchers have developed a new approach to identify specific genes that influence how cancer cells respond to drugs and how they become resistant. This strategy, which involves producing diverse genetic mutations that result in leukemia and associating specific mutations with treatment outcomes, will enable researchers to better understand how drug resistance occurs in leukemia and other cancers, and has important long-term implications for the development of more effective therapies.
Chromosomal problems affect nearly all human embryos
For the first time, scientists have shown that chromosomal abnormalities are present in more than 90% of IVF embryos, even those produced by young, fertile couples.
Rice University study finds possible clues to epilepsy, autism
Rice University researchers have found a potential clue to the roots of epilepsy, autism, schizophrenia and other neurological disorders.
Mapping a clan of mobile selfish genes
Much of human DNA is the genetic equivalent of e-mail spam: short repeated sequences that have no obvious function other than making more of themselves.
Pediatric study finds alternatives for radiation of low-grade brain tumors
A multi-institutional study led by researchers at The University of Texas M. D. Anderson Cancer Center has found that using chemotherapy alone and delaying or avoiding cranial radiation altogether can be effective in treating pediatric patients with unresectable or progressive low-grade glioma.
Anti-cancer drug prevents, reverses cardiovascular damage in mouse model of premature aging disorder
An experimental anti-cancer drug can prevent -- and even reverse -- potentially fatal cardiovascular damage in a mouse model of progeria, a rare genetic disorder that causes the most dramatic form of human premature aging, National Institutes of Health (NIH) researchers reported today.
Gene's newly explained effect on height may change tumor disorder treatment
A mutation that causes a childhood tumor syndrome also impairs growth hormone secretion, researchers at Washington University School of Medicine in St. Louis have found.
Protein key to neuro-regeneration
Researchers at the Peninsula Medical School in the South West of England, University College London, the San Raffaele Scientific Institute in Milan and Cancer Research UK, have for the first time identified a protein that is key to the regeneration of damage in the peripheral nervous system and which could with further research lead to understanding diseases of our peripheral nervous systems and provide clues to methods of repairing damage in the central nervous system.
Research to lead to brain tumor therapies
Unique human in vitro model (cell culture) research currently underway at the Peninsula Medical School in the South West of England is set to identify and develop therapies for the treatment of multiple tumours in the brain.
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