Update on planned polyamine inhibition trial
Update 6th August 2024
Following very promising pre-clinical research which was presented by Prof Michelle Haber at our Neuroblastoma Parent Global Symposium in 2020, SKCUK had hoped to fund further clinical development of the combination of AMXT1501/DFMO (polyamine inhibition) currently being studied in adults. Recently, we have been made aware of the possibility of developing severe cardiac toxicity resulting in cardiac arrest. Consequently, the decision has been made to pause development of the planned clinical trial of AMXT1501/DFMO in children with high-risk neuroblastoma and DIPG (diffuse intrinsic pontine glioma) until further information is available.
While AMXT1501/DFMO remains a potentially effective treatment approach, trialling this drug combination in children must be done safely and with all data to hand. CCI will therefore be carrying out extensive laboratory testing to explore a potential link between this drug combination and cardiac toxicity. CCI are collaborating with cardiac experts to understand the mechanism of toxicity, and to investigate if any such toxicity can be avoided by changes in drugs, dosages or scheduling. We also await the results of the AMXT1501/DFMO Phase 1 clinical trial currently underway in adult cancer patients.
Solving Kids’ Cancer UK will continue to monitor progress of developments in our regular communication with Professor Haber and remain hopeful that a solution may be found. For further information, see here the link to a statement issued by Children’s Cancer Institute in Australia.
If any families affected by high-risk neuroblastoma have queries about this treatment combination, please reach out directly to our family support team at support@solvingkidscancer.org.uk.