Monash patients benefit from blood cancer drug for autoimmune disease

Publication authors Prof Stephen Opat,
Dr Sumita Ratnasingham and
Assoc Prof Jake Shortt at the
ASH Annual Meeting
3 December 2016

In a world-first, a Monash University study has shown an anti-myeloma drug to be an effective treatment against a range of autoimmune diseases affecting the blood.

Thrombotic thrombocytopenic purpura (TPP) is one such disease—a rare blood disorder characterised by clotting in small blood vessels. TPP causes microscopic clots to form throughout the body that can damage organs including the kidneys, heart and brain.

“At Monash, we were the first ever to use bortezomib (an anti-myeloma drug) in a patient with TPP,” said lead researcher and Monash Health haematologist Associate Professor Jake Shortt.

“Further to the patient’s full recovery and our New England Journal paper about this in 2013, we have successfully used bortezomib in a number of patients with a range of nasty autoimmune diseases affecting the blood.”

Presented as a case series, Associate Professor Shortt’s research was published this week in the inaugural issue of Blood Advances, a new peer-reviewed journal published by the American Society of Hematology and the first journal to join the Blood family in 70 years.

“If you consider that leukaemia and lymphoma are cancers of the immune system and that autoimmune disease is caused by an overactive immune system, you can rationalise that anti-lymphoma drugs could be good for autoimmune disease,” said Associate Professor Shortt, who is also Head of Haematology Research at the Monash Health Translation Precinct.

Associate Professor Shortt said that the subsequent use of bortezomib in ten Monash Health patients was carried out ‘off label’ through compassionate access—a real credit to Monash Health’s attitude towards drug availability for patients in need.

“Although our paper is a case series rather than a clinical trial, we have observed high response rates with minimal toxicity,” said Associate Professor Shortt.

Associate Professor Shortt presented an abstract of this work at last year’s American Society of Hematology annual meeting, and it was subsequently listed in the Cleveland Clinics top 10 most compelling 2015 benign haematology abstracts.

The article published in Blood Advances was also distributed to 27,000 delegates at the American Society of Hematology's Annual Meeting in San Diego last weekend.