Clinical Trials Prepare For Big Advances

Childhood cancer treatments becoming more successful, kinder

The most rewarding part of Ryan Hehir’s job is allowing young patients to benefit from the rapid advances being made in new, targeted cancer therapies. 

Ryan is the Study Coordinator of the early phase clinical trial program in the Children’s Cancer Centre at The Royal Children’s Hospital. The Children’s Cancer Foundation has funded his role for the past two years and has committed a further year of support.

Early phase clinical trials test new drugs or treatments with small groups of patients to evaluate their effectiveness and identify initial side effects. For children whose standard care treatments have failed, clinical trials can provide alternative treatment options and better outcomes. Early phase clinical trials are the best and the safest way to access innovative new drugs, which can be difficult in Australia, and provide hope to children with cancer and their families.

“I meet patients and their families to explain the purpose and requirements of the clinical trial, and walk them through the consenting process,” says Ryan. 

The most important part of Ryan’s role is helping the treating team to ensure that patients are following the clinical trial protocol to maintain the safety of patients receiving these new treatments. Ryan works with research nurses to collect samples and manage and report the data from the trial which we hope will help us better understand how these treatments work on the patient’s disease. 

Promise and progress for clinical trials

Early phase clinical trials that show promise may then progress to more extensive and detailed trials involving a much larger group of patients (called late phase clinical trials). 

The Children’s Cancer Foundation is committed to increasing access for children to clinical trials, investing $1.3 million since 2007 to fund study coordinators like Ryan, as well as data managers and clinical trial managers at both Monash Children’s Hospital and The Royal Children’s Hospital. The Foundation is currently funding four clinical trials staff at both hospitals.

Ryan’s job is to work with the doctors who identify which innovative research trials are the most appropriate for the children in their care. Since Ryan started in 2014, he has coordinated 11 new early phase trials, with another three to start in 2017. 

Results have shown dramatic benefits for some patients. 

One six-year-old girl had a rare disease called Langerhans cell histiocystosis that was resistant to multiple rounds of chemotherapy. This disease is known to have specific gene mutations which drive the development of this disease. The gene mutation is known to occur in many different cancers and recently a specific drug targeting this gene was developed and is now going through clinical trials.

Routine molecular testing for this gene was introduced at The Royal Children’s Hospital for several different cancer types. When this patient’s tumour was tested, it was found to have the specific gene mutation that could be targeted by the new drug and she was therefore eligible for entry on the early phase clinical trial. 

Prior to starting treatment, the patient had significant back pain, headaches and cerebellar signs [involving balance and coordination]. An immediate positive response was seen after starting the clinical trial treatment and this has continued 32 weeks after starting treatment. 

“Without access to clinical trials, patients like this would not benefit from the rapid advances being made in the development of new targeted cancer therapies, “ Ryan concluded.

Ryan Hehir, Study Coordinator