News in brief: RAVEN trial to assess venetoclax and navitoclax in kids with ALL; Canakinumab may reduce symptom severity in sickle cell anaemia;National genomics agency to be created

Thursday, 24 Mar 2022

RAVEN trial to assess venetoclax and navitoclax in kids with ALL;

Australian centres will be involved in a trial of venetoclax and navitoclax, added to chemotherapy in children and teenagers who relapse with acute lymphoblastic leukaemia (ALL).

The RAVEN study, sponsored by the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG), is due to open in mid-2022 and will be an Australia-wide ALL clinical trial collaboration with St Jude Children’s Research Hospital (USA), according to the Leukaemia Foundation, which is funding the study.

Principal Investigator Dr Michael Osborn, consultant paediatric and aematologist/oncologist at the Women’s and Children’s Hospital, Adelaide,  said the trial will recruit up to 78 patients in Australia and New Zealand, with the aim of determining whether venetoclax and navitoclax, added to chemotherapy, will improve the rate of patients who are measurable residual disease (MRD) negative at the end of induction.

He said venetoclax has been shown to be highly effective in adults haematological cancers such as CLL, and there had been promising results from a Phase I study of this combination in ALL in children.

“The combination of these two drugs – venetoclax and navitoclax – together with chemotherapy and other successful immunotherapies, like blinatumomab, and targeted agents makes this trial unique,” he said.

“If this is shown to be effective, it will offer potential treatments for patients who’ve run out of other options. We anticipate there will be just over 10 children and young people across Australia per year who would be eligible for this trial, but hope that what we learn from this study will benefit many more,” Dr Osborn said.

The trial is anticipated to be available at Royal Children’s Hospital (VIC), Monash Children’s Hospital (VIC), Perth Children’s Hospital (WA), John Hunter Children’s Hospital (NSW), Children’s Hospital at Westmead (NSW), Sydney Children’s Hospital (NSW) and Women’s & Children’s Hospital (SA).

Canakinumab may reduce symptom severity in sickle cell anaemia

Findings of a new study suggest that blocking IL-1β mediated inflammation by canakinumab in young people with sickle cell anaemia (SCA) could induce certain clinical benefits such as reduction of fatigue, without major safety issues.

For the study, published in the journal Blood and funded by canakinumab manufacturer Novartis AG, 49 patients, aged 8-20 years old with SCA, history of acute pain episodes and elevated hsCRP, were randomised to receive either six monthly treatments with 300 mg canakinumab or placebo. All bar one of the enrolled patients were receiving stable background hydroxyurea therapy at the time of the study.

The research team, led by Professor David Rees, Professor of Paediatric SCD at King’s College Hospital in London, said they observed a reduction in pain in patients taking canakinumab versus those in the placebo arm, but that the difference did not reach statistical significance, thus failing to meet the study’s primary endpoint.

However, canakinumab-treated patients showed reduced markers of inflammation, occurrence of SCA-related adverse events and serious adverse events, number and duration of hospitalisations, and there were also trends for improvement in fatigue and absences from school or work. Crucially, the treatment was also found to be well-tolerated, with no increased risk of infection observed.

The authors concluded that the results indicate that “inflammation appears to contribute to fatigue and other daily manifestations of SCA, and the anti-inflammatory effects of selective IL-1β blockade by canakinumab may reduce the severity of these acute and chronic disease manifestations.”

“In the longer term, based on association studies of inflammatory markers, one might predict that canakinumab treatment could reduce organ damage and improve survival, although clearly this requires formal investigation.”

National genomics agency to be created

A new national agency, Genomics Australia, is being established to support the integration of genomic medicine into clinical practice in Australia.

Backed by $28 million from the Medical Research Future Fund (MRFF), the new agency will coordinate researchers, industry, clinicians and consumers to translate the potential of genomics-led medicine into practice, the federal government says.

The agency will promote the development and uptake of genomic testing, diagnosis and the use of genomic-guided therapies in clinical care. Genomics Australia will be chaired by paediatric Professor Kathryn North, Director of the Murdoch Children’s Research Institute , Melbourne, who has a background in genetics, neurology and paediatrics.

A Taskforce in the Department of Health, with expert guidance from Professor North, will design and establish Genomics Australia, which will become a legislated corporate Commonwealth entity under the Health portfolio from 1 January 2024.


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