Congratulations to Dr Samantha Ginn and collaborators for winning an award from Children’s Tumor Foundation (CTF) in the US to fund their Neurofibromatosis type 1 (NF1) research.

NF1 is a genetic disorder with an incidence of 1 in 3,000-4,000 newborns. The most common symptom that an individual with NF1 can develop are benign, often disfiguring, peripheral nerve-associated tumours. However, in about 10% of NF1 patients, these benign tumours may transform into malignant peripheral nerve sheath tumours, which are often fatal.

With generous funding from the US Children’s Tumor Foundation (CTF), Dr Ginn, along with co-investigators A/Prof Leszek Lisowski and Dr Matthieu Drouyer of CMRI, will characterise gene delivery systems for future clinical trial development for the treatment of NF1 and other disorders involving Schwann nerve cells.

Dr Samantha Ginn said, “We are grateful to the US CTF for their continued support of our research. This grant will allow us to validate the utility of our most promising gene delivery systems. In addition to NF1, this research will benefit other diseases including NF2, Schwannomatosis and Charcot-Marie-Tooth disease.”

Successful completion of this project will place them in a position to quickly transition these gene therapy delivery systems into human clinical trials where benefits for patients can start to be seen.