Australia Approves Genetically Modified Cell Therapy


In the past, patients with aggressive blood cancers who did not experience positive responses to conventional treatments would have to seek treatment overseas, generally in the United States. These lifesaving therapies were unavailable for many sufferers or gated behind huge traveling costs, but now the Australian government has stepped in.

The Therapeutic Goods Administration which “safeguards and enhances the health of the Australian community through effective and timely regulation of therapeutic goods”, similar to the FDA, has recently approved the use of Kymriah (Tisagenlecleucel, formerly CTL019). This is an immunotherapy drug designed for the treatment of pediatric and young adult patients with  B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant, or in second or later relapse; and for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma.


Kymriah is based on Chimeric Antigen Receptor T-cell therapies and created for each individual patient using their own cells. T cells are drawn from a patient’s blood and reprogrammed in the laboratory to create T cells that are genetically coded to recognize and fight the patient’s cancer cells and other B cells expressing a particular antigen.

We have seen a number of Australians travelling to the United States for this treatment and today’s announcement gives hope to patients and their families who are running out of treatment”, said Richard Vines, Founder and Chairman of Rare Cancers Australia.

Acute lymphoblastic leukemia (ALL) is a type of cancer that affects the blood and bone marrow. According to the press release, “ALL is the most common type of childhood leukaemia 3, and for patients who relapse from a standard of care therapies, the outlook is poor”. Even with multiple treatments, including chemotherapy, radiation, targeted therapy or stem cell transplant, chances of recovery are low.

Diffuse large B-cell lymphoma is an aggressive, complex and difficult to treat the form of non-Hodgkin lymphoma, accounting for up to 40 % of all cases globally. The disease has limited treatment options and again, survival rates are low.

Following the approval, Lauren Carey, General Manager Oncology of Novartis Australia, noted that: “The next step is to ensure public funding for Kymriah so it is accessible for all eligible patients and we are committed to working with the Australian Government to achieve this as soon as possible.”