| Email Article | Print |
Research to lead to brain tumor therapies
March 24, 2008Unique human in vitro model research currently underway at the Peninsula Medical School in the southwest of England is set to identify and develop therapies for the treatment of multiple tumors in the brain
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.
The tumours are caused by mutations affecting a protein called merlin, which in turn cause cancers in a range of cell types including Schwann cells in the brain. Schwann cells produce the sheaths that surround and insulate neurons.
Although the tumours are benign, they are frequent, can be inherited and come in numbers. The sheer number of them can overwhelm a patient, often leading to deafness and eventually death. Patients can suffer from 20 to 30 tumours at any one time, and the condition typically affects older children and adults.
No therapy, other than invasive surgery aiming at a single tumour and which may not eradicate the full extent of the tumours, exists.
The condition of multiple tumours is known as neurofibromatosis type two (NF2) and affects one in every 2,500 people worldwide. It can affect any family, regardless of past history, through gene mutation and currently there is no cure.
Research at the Peninsula Medical School is led by Professor Oliver Hanemann. By working with human cells in vitro, Professor Hanemann and his team are able to find new therapeutic targets for NF2. They have secured initial success by re-profiling an existing drug, sorafenib, and because they are using the human in cell culture model and re-profile new cancer drugs they do not need to carry out huge toxicity studies - which means they now can go straight to clinical trials and introduce therapies to patients sooner rather than later using sorafenib or similar drugs.
Said Professor Hanemann: "Ours is a unique model and a unique approach to the issue. We are on the verge of working with inpatient clinics to trial our latest breakthrough, and we are investigating other therapeutic targets using other drugs."
He added: "Using human in vitro cell culture, which is the unique aspect of our work, allows us to move seamlessly and relatively quickly from lab-based biochemistry to drug therapies, clinical trials and hopefully successful outcomes."
Maria Toman, chair, the Neurofibromatosis Association, commented: "The Neurofibromatosis Association (www.nfauk.org), as the only charity working in the UK with patients and families affected by neurofibromatosis, welcomes this breakthrough heralding the possibility of new, non invasive treatment for NF2 with the potential to transform management of this debilitating and long term condition, and will be watching progress of the trials with close interest. Our members have benefited hugely from Professor Hanemann's clinical work since his arrival at the Peninsula Medical School and by his establishment of the first specialist neurofibromatosis clinic in the South West of England. We hope that similar approaches in neurofibromatosis type one, one of the commonest genetic diseases, will bring treatment trials in the near future."
Dr. Kim Hunter-Schaedle, chief scientific officer of the Children's Tumor Foundation said: "Dr. Hanemann's work is very exciting. There is currently no effective treatment for neurofibromatosis. As a physician and researcher he should be able to take positive results quickly from the bench to the patient, getting promising neurofibromatosis drug therapies as quickly as possible into clinical trials. " The Children's Tumour Foundation has provided seed funding to Professor Hanemann and to an international consortium of research establishments of which the Peninsula Medical School is one. It will fund over $3 million for neurofibromatosis research programmes in 2008. More information is available by logging on at http://www.ctf.org.
More information is available by logging on at http://www.pms.ac.uk.
CASE STUDY
Tom Wakenshaw, 21, from Gunnislake in Cornwall, first became aware that there was a problem when he started to suffer from very bad headaches at age 15.
He was a pupil at Tavistock College at the time and in the middle of his GCSEs. He was referred for an MRI which identified a tumour on his brain, and was immediately rushed to Bristol for surgery to remove it.
Said Tom: "I had the operation, and then they told me afterwards that I had neurofibromatosis. When you're young you just take these things on board, which is what I did at the time. But it has had a huge effect on my life since."
Tom lives with his mother Val, and is currently being helped by the local Job Centre to try to find a job that can accommodate the effects of the neurofibromatosis. The condition causes him to get very tired and affects his balance. The operations he has had over the past years to remove more tumours have resulted in numb fingertips and feet.
He said: "I had a job as a window cleaner once, but a had an accident where a nail slipped between my toes. The worrying thing was that, even though it did not go into me, I couldn't feel it passing by my toes."
Tom is currently working with his local Job Centre which his finding him placements - his next is a six week spell working in the kitchen at the Bedford Hotel in Tavistock.
Tom is the youngest of four children, and some of his family live close by. Said Tom: "My Mum, Dad and brother live locally and see what it's like on a day-to-day basis. It's good to have them close by."
Tom attends the clinics run by Professor Oliver Hanemann at Derriford Hospital in Plymouth. "They're really useful, and a good opportunity to talk about how I'm doing," said Tom. "Professor Hanemann gives us good ideas and suggestions to help cope with the condition, and just talking about it is a great help."
The Peninsula College of Medicine and Dentistry
Although the tumours are benign, they are frequent, can be inherited and come in numbers. The sheer number of them can overwhelm a patient, often leading to deafness and eventually death. Patients can suffer from 20 to 30 tumours at any one time, and the condition typically affects older children and adults.
No therapy, other than invasive surgery aiming at a single tumour and which may not eradicate the full extent of the tumours, exists.
The condition of multiple tumours is known as neurofibromatosis type two (NF2) and affects one in every 2,500 people worldwide. It can affect any family, regardless of past history, through gene mutation and currently there is no cure.
Research at the Peninsula Medical School is led by Professor Oliver Hanemann. By working with human cells in vitro, Professor Hanemann and his team are able to find new therapeutic targets for NF2. They have secured initial success by re-profiling an existing drug, sorafenib, and because they are using the human in cell culture model and re-profile new cancer drugs they do not need to carry out huge toxicity studies - which means they now can go straight to clinical trials and introduce therapies to patients sooner rather than later using sorafenib or similar drugs.
Said Professor Hanemann: "Ours is a unique model and a unique approach to the issue. We are on the verge of working with inpatient clinics to trial our latest breakthrough, and we are investigating other therapeutic targets using other drugs."
He added: "Using human in vitro cell culture, which is the unique aspect of our work, allows us to move seamlessly and relatively quickly from lab-based biochemistry to drug therapies, clinical trials and hopefully successful outcomes."
Maria Toman, chair, the Neurofibromatosis Association, commented: "The Neurofibromatosis Association (www.nfauk.org), as the only charity working in the UK with patients and families affected by neurofibromatosis, welcomes this breakthrough heralding the possibility of new, non invasive treatment for NF2 with the potential to transform management of this debilitating and long term condition, and will be watching progress of the trials with close interest. Our members have benefited hugely from Professor Hanemann's clinical work since his arrival at the Peninsula Medical School and by his establishment of the first specialist neurofibromatosis clinic in the South West of England. We hope that similar approaches in neurofibromatosis type one, one of the commonest genetic diseases, will bring treatment trials in the near future."
Dr. Kim Hunter-Schaedle, chief scientific officer of the Children's Tumor Foundation said: "Dr. Hanemann's work is very exciting. There is currently no effective treatment for neurofibromatosis. As a physician and researcher he should be able to take positive results quickly from the bench to the patient, getting promising neurofibromatosis drug therapies as quickly as possible into clinical trials. " The Children's Tumour Foundation has provided seed funding to Professor Hanemann and to an international consortium of research establishments of which the Peninsula Medical School is one. It will fund over $3 million for neurofibromatosis research programmes in 2008. More information is available by logging on at http://www.ctf.org.
More information is available by logging on at http://www.pms.ac.uk.
CASE STUDY
Tom Wakenshaw, 21, from Gunnislake in Cornwall, first became aware that there was a problem when he started to suffer from very bad headaches at age 15.
He was a pupil at Tavistock College at the time and in the middle of his GCSEs. He was referred for an MRI which identified a tumour on his brain, and was immediately rushed to Bristol for surgery to remove it.
Said Tom: "I had the operation, and then they told me afterwards that I had neurofibromatosis. When you're young you just take these things on board, which is what I did at the time. But it has had a huge effect on my life since."
Tom lives with his mother Val, and is currently being helped by the local Job Centre to try to find a job that can accommodate the effects of the neurofibromatosis. The condition causes him to get very tired and affects his balance. The operations he has had over the past years to remove more tumours have resulted in numb fingertips and feet.
He said: "I had a job as a window cleaner once, but a had an accident where a nail slipped between my toes. The worrying thing was that, even though it did not go into me, I couldn't feel it passing by my toes."
Tom is currently working with his local Job Centre which his finding him placements - his next is a six week spell working in the kitchen at the Bedford Hotel in Tavistock.
Tom is the youngest of four children, and some of his family live close by. Said Tom: "My Mum, Dad and brother live locally and see what it's like on a day-to-day basis. It's good to have them close by."
Tom attends the clinics run by Professor Oliver Hanemann at Derriford Hospital in Plymouth. "They're really useful, and a good opportunity to talk about how I'm doing," said Tom. "Professor Hanemann gives us good ideas and suggestions to help cope with the condition, and just talking about it is a great help."
The Peninsula College of Medicine and Dentistry
Neurofibromatosis News and Current Neurofibromatosis Events RSSMouse model tightly matches pediatric tumor syndrome, will speed drug hunt
Frustrated by the slow pace of new drug development for a condition that causes pediatric brain tumors, a neurologist at Washington University School of Medicine in St. Louis decided to try to fine-tune the animal models used to test new drugs.
Cancer gene drives pivotal decision in early brain development
A gene linked to pediatric brain tumors is an essential driver of early brain development, researchers at Washington University School of Medicine in St. Louis have found.
Scientists find new agent to fight genetic disorders -- Zorro-Locked Nucleic Acid
A study to appear in the June 2007 issue of The FASEB Journal describes a new agent, called "Zorro-LNA," which has the potential to stop genetic disorders in their tracks.
Tracing the pathways of neurofibromatosis
Michael Stern's latest research into the formation of neurofibromatosis tumors reads something like a federal racketeering indictment, except that Stern's tracing proteins instead of laundered money, and he's looking not at offshore accounts but at biochemical paths of cause and effect.
For kids with high blood pressure, surgery can help when medicines fail
High blood pressure may seem like something that only adults get, after years of smoking, overeating and little exercise. But children can develop it too - and just as in adults, uncontrolled high blood pressure can pose serious risks to children's hearts, brains and lives.
Protein plays broader role than originally thought in neurofibromatosis
Neurofibromatosis type I is a common genetic disorder in which tumors grow along certain types of nerves and can also affect other tissues such as bone, heart, and skin.
Mouse to man: The story of chromosomes
The complete sequencing of human chromosome 17 and mouse chromosome 11 offers unique insights into the evolution of the genome of higher mammals.
A commonly prescribed drug reverses learning and attention deficits in a mouse model of the genetic disorder Neurofibromatosis type I
This week, researchers report evidence that a statin drug already shown to be safe for use in humans has proven effective at correcting cell-cell communication and curing learning disfunction in a mouse model of Neurofibromatosis type I, a human genetic disorder that causes learning disabilities in millions of people worldwide.
UCSF surgeon develops new spinal surgery technique
Called a lateral paramedian transpedicular approach, the technique uses advances in spinal instrumentation and reconstructive strategies to provide a direct approach to the removal of cervical spinal tumors with minimal, or no, neural manipulation.
New therapeutic target identified in inherited brain tumor disorder
Researchers studying a mouse model of neurofibromatosis 1 (NF1), a genetic condition that causes childhood brain tumors, have found their second new drug target in a year, a protein called methionine aminopeptidase-2 (MetAP2).
More Neurofibromatosis News Articles
| © 2008 BrightSurf.com |