An international research collaboration between the US and Australia has revealed a new drug for the treatment of patients affected with high-risk leukemia.

Biotechnology firm OncoTartis and Children’s Cancer Institute of Australia have jointly published two research manuscripts in onco-haematological journal Leukemia, both on the OncoTartis’ clinical drug candidate OT-82.

A notable share of blood cancers in both adults and children are often fatal and resistant to treatments.

Studies conducted by the international research collaboration in Buffalo, US and Sydney, Australia, identified a new treatment for patients who have incurable blood cancers with the introduction of a new drug, which has shown to be very effective in preclinical models.

OncoTartis chief scientific officer Andrei Gudkov said: “The main principle of the research strategy that led us to OT-82 was the identification of a pharmacological agent specifically toxic for malignant cells of blood origin.

“We were surprised when the compound that came out of an unbiased search appeared to be a NAMPT inhibitor, an enzyme that has been considered as a target for cancer treatment but whose association with malignancies of blood origin was unknown.”

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By GlobalData

The first paper on OT-82 describes the discovery, mechanism of action, toxicological profile and preclinical efficacy of the drug, which was developed by OncoTartis to treat refractory leukaemias and lymphomas.

Meanwhile, the second paper details a study carried out at Children’s Cancer Institute (Sydney). The Institute has a wide collection of refractory children’s leukemias grown in laboratory mice for new drug testing.

This collection was used to compare the safety and efficacy of OT-82 with current treatments in children with leukaemia.

Children’s Cancer Institute Sydney executive director and professor Michelle Haber said: “For children with particularly aggressive leukaemia, the options for effective yet safe treatments are extremely limited and therefore novel therapeutic options are urgently needed.

“If OT-82 is found to be safe and effective in adults, we are very hopeful that it may provide an exciting new treatment approach for the worst cases of pediatric leukaemia, with the added potential benefit of allowing reductions in doses of chemotherapy and hence diminished adverse treatment side-effects for these children.”

Under the joint research, OT82 showed remarkable efficacy when used alone. It also demonstrated to be even more effective when used in combination with conventional treatments in children.

OT-82 is currently being tested in a clinical trial on adults with refractory lymphoma. After the completion of this trial, the drug is expected to proceed to a clinical trial in children suffering from high-risk acute lymphoblastic leukaemia.