GBM-Vax Study

Clinical development

During the last years, in Europe as well as in the US, legislations regulating modern high-tech treatment concepts were introduced. One of the most important laws is the Advanced Therapy Medicinal Product (ATMP) guideline, which states that somatic cell therapy represents a drug treatment. Cancer immune therapy with Dendritic Cells (DC) belongs to the group of ATMPs and is therefore a drug therapy. This brings significant advantages as it clearly regulates how clinical development and regulatory approval needs to be conducted.

Glioblastoma multiforme (GBM)

Glioblastoma multiforme is the most frequent and most aggressive primary brain cancer. It occurs in 5 out of 100.000 people and represents 1.4% of all cancer cases worldwide. The average survival is little more than a year.

Further information about Glioblastoma multiforme (GBM)

GBM-Vax

First line standard therapy of glioblastoma multiforme with or without add-on treatment with Activartis’ cancer vaccine, an anti-tumour immune therapy based on tumour-lysate charged dendritic cells
EudraCT: 2009-015979-27;
ClinicalTrials.gov Identifier: NCT01213407

The GBM-Vax study is designed as a randomised phase II clinical trial. Recruitment of about 60 patients should be complete in the late summer of 2011. First results will be available in 2012.

Treatment groups

Patients with newly diagnosed histologically confirmed glioblastoma multiforme (GBM).

Treatment schedule

The participating patients will be divided into two groups in a 1:1 ratio. Patients randomly assigned to the control group will receive the current first line standard therapy comprised of surgery, radiotherapy and chemotherapy with temozolomide (Temodal^®). Patients randomly assigned to the treatment group will, in addition to surgery, radiotherapy and chemotherapy with temozolomide, receive  Activartis’ novel cancer immune therapy based on dendritic cells.

Activartis’ DC cancer vaccine will be applied as an add-on to the standard first line therapy of glioblastoma. Initially, the patients undergo surgery during which a piece of tumour tissue is removed and used to extract tumour antigens. Approximately 7-10 days after surgery, a leukocyte apheresis is performed in order to collect a sufficient amount of monocytes. Subsequently, the patients receive radiotherapy for six weeks, combined with a chemotherapy with temozolomide (Temodal^®). The first four vaccines are applied in the four-week intermission – one vaccine a week – after chemo- and radiotherapy. After that, a maintenance therapy with temozolomide starts. At the same time, the patients receive additional treatments with Activartis’ cancer vaccine every four weeks.

Study substance

Activartis‘ cancer immune therapy is a cancer vaccine based on dendritic cells that is derived individually from each patient. It has the capacity to present components of the tumour cells, the tumour antigens, to killer cells of the immune system. This enables the killer cells to recognise and destroy cancer cells. Activartis’ cancer vaccine differs from previous utilised dendritic cell cancer vaccine technologies in its ability to release a specific signalling substance, Interleukin-12, which supports killer cell activation.

In previous studies it was observed that an optimal anti-cancer immune response was initiated when the cancer vaccine was applied in a phase of stabilisation or in slowly growing disease. The objective of the GBM-Vax trial is to apply Activartis’ DC cancer vaccine during a phase of disease stabilisation accomplished by chemo- and radiotherapy in order to trigger a potent tumour antigen-specific immune response.

Sponsor: Activartis Biotech Gmbh

Study objectives

The primary objective is progression-free survival; the secondary objective is overall survival.

The GBM-Vax trial is also open for paediatric GBM patients, since glioblastoma may occur at every age, including childhood, adolescence and young adulthood. This is due to Activartis’ history as a spin-off of the St. Anna Children’s Cancer Research Institute. We aim at making the newest biotech drugs, particularly cancer immune therapy with dendritic cells, available to paediatric cancer patients as well.

Participating treatment centres

• Landesnervenklinik Wagner-Jauregg, Linz
• Medical University Vienna
• Medical University Graz
• Medical University Innsbruck
• Rudolfspital, Wien
• Kaiser Franz-Josef Spital, Wien
• Donauspital, Wien
• Landeskrankenhaus Feldkirch
• Salzburger Landesklinikum