Tested and refined over the past decade by SAHMRI’s Professor Tim Hughes, asciminib, known by the brand name Scemblix, is produced by Swiss multinational pharmaceutical corporation, Novartis.

It works by blocking the action of an abnormal protein that signals cancer cells to multiply and was designed to be both highly potent and specific, minimising side effects and toxicity. 

Mother of two, Janet Anderson, was one of the original patients to take the drug, participating in the world-first clinical trial in 2020/2021. The Adelaide Symphony Orchestra violinist has been on asciminib ever since and says it has changed everything for the better.

ASO violinist Janet Anderson

“Before switching to this medication my mouth was full of ulcers, I was really lethargic, often feeling completely out of it and wasn’t able to be there for the with the kids,” Anderson said.

“It has worked incredibly well for me. I feel great and have been able to keep living my life to the full! I am literally living the dream for cancer treatment.”

The drug has recently passed the ultimate test, proving its standout quality in a phase III global randomised control study.

The study involved 405 men and women with a median age of 52, who had been recently diagnosed with chronic myeloid leukaemia (CML). They were treated with either asciminib or an older tyrosine kinase inhibitor (TKI) therapy, to compare treatment efficacy and safety.

Presenting the findings at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Hughes, who has played an integral part in the drug’s development, said the latest trial results showed asciminib is an overall superior option for people with new cases of CML compared to current standard treatments.

“We’re excited, because this trial demonstrates that asciminib achieves statistically superior efficacy and, importantly, safety and tolerability were also excellent,” he said.

“Asciminib could soon become the treatment of choice for many newly diagnosed CML patients.

“That’ll mean people can stay at work, they can stay productive, and we’ll see significantly more achieving treatment free remission.”

Treatment of chronic phase CML requires the long-term use of TKIs to prevent the disease from progressing to a more aggressive phase. However, around half of all patients will need to switch TKIs if the CML stops responding to treatment or if the treatment cannot be tolerated due to side effects.

Between 10 and 20 per cent of patients have significant intolerance to TKI therapy, which may not improve after switching to a different medication.

To measure how well treatment worked, researchers assessed each individual’s major molecular response (MMR).

Patients who have an MMR are more likely to have CML that is well controlled, and reaching MMR early is also a predictor of better long-term outcomes.

“After 48 weeks, 68 per cent of participants on asciminib had achieved an MMR compared to 49 per cent on other TKIs,” Prof Hughes said.

“A deep molecular response was also observed in 39 per cent of those taking asciminib in comparison to 21 per cent in the control group. People who have a deep molecular response may eventually be considered in remission and could stop treatment.

“Additionally, asciminib has shown lower rates of discontinuation, dose adjustment and treatment interruptions, indicating better tolerance.

“It also all but eliminated the threat of blood clots, a severe side effect of TKIs, present in only 1 per cent of participants who took asciminib.”

Researchers will continue to follow participants to understand the long-term safety of asciminib and determine if reaching an earlier MMR continues to be a way to predict better outcomes for patients.

Future studies will measure overall survival, progression-free survival and whether treatment-free remission is reached.

Hughes is confident Anderson is on a trajectory to achieve treatment-free remission in the coming years.   

“I will be forever grateful for all the decades of CML medical research, this amazing drug trial and for all the incredible and never-ending support from my family and friends,” Anderson said.

Celebrating her clean bill of health, Anderson has signed up for the Noosa Triathlon in November to raise money for CML research at SAHMRI. You can support her campaign here.