Research to lead to brain tumor therapies

Unique 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.”