CBL137 Single Agent Clinical Trials

CBL137 is a novel drug developed for adult cancer in USA. It had not been tested for childhood cancer until the Children's Cancer Institute looked at its qualities to target neuroblastoma tumours; specifically, those types of neuroblastoma that are linked to the MYCN gene which produces a protein in high volumes that is proven to create the metabolic environment for this cancer to grow aggressively.

Early results in laboratory models show the effectiveness of CBL137 in reducing aggressive neuroblastoma tumours. Not only does this research indicate the drug is very effective at reducing the cancer when used in combination with chemotherapy, but also that CBL137 does so with few damaging side effects because it does not damage DNA. It is DNA damage that is responsible for these side effects and if CBL137 can mean the cancer can be treated with lower dosages of toxic chemotherapy then this will not only improve cure rates but also reduce long term damage to the children treated.

The research project involved identifying whether this drug could be effective in treating high-risk neuroblastoma in laboratory models as a single agent, at the next stage this single agent will go to a phase one clinical trial to confirm the acceptable dosage of CBL137 to treat this aggressive cancer in children.

CBL137 is in clinical trial for adults in USA and Russia, these trials will establish the optimal dosage required to kill the cancer without serious side effects to patients, currently the trials are continuing due to the success of the drug and the lack of side effects suffered. As soon as the adult clinical trial is completed the dosage for children will be established as 80% of that identified as optimal for adults.

The next phase of this project will be a clinical trial of CBL137 for children at leading children’s cancer centres in the United States and at Sydney Children’s Hospital, Randwick, conducted through the US-based Children’s Oncology Group (COG), the largest children’s cancer study group in the world. This is the first time that a COG trial of this sort would be made available to Australian children. The clinical trial in both countries will be run in the hospitals by Dr David Ziegler, a senior researcher at CCI and Head of Clinical trials at the Kids Cancer Centre, Sydney Children’s Hospital (Randwick).