Stem-cell-derived immunotherapy for glioblastoma

A form of immunotherapy derived from induced pluripotent stem-cells has been shown to be effective in treating brain cancer in mice.

Glioblastoma is an aggressive brain cancer with a poor prognosis. Developments in treatments such as surgery, chemotherapy and immunotherapy have not resulted in the extension in life-expectancy seen for many other cancers. This resistance to treatment is partly due to the cancer's ability to evade and suppress the body's natural immune response. Researchers have now genetically-engineered immune cells derived from stem cells, and designed them to target and dismantle the immune suppressive tactics employed by the tumour, meaning they can eliminate the cancer cells.

'Our preclinical studies showed these immune cells to be particularly remarkable in targeting and completely eliminating the growth of the tumours,' Dr Sandro Matosevic, associate professor at Purdue University, Indiana, and lead researcher said: 'We found that we can engineer these cells at doses suitable for clinical use in humans.

'This is significant because one of the major hurdles to clinical translation of cell-based therapies to humans has been the poor expansion and lack of potency of cells that were sourced directly from patients.'

In their study published in Nature Communications, researchers at Purdue University used 'off-the-shelf' stem cells to generate natural killer cells, a type of immune cell known for their ability to identify and attack cancer cells.

Unlike traditional 'personalised' stem cell therapies that require cell collection from each patient, this approach used 'off-the-shelf' stem cells. These are lab-grown from the blood or skin cells of healthy donors, making them readily available and eliminating the need for additional collection procedures. This is a significant advantage, considering the time-sensitive nature of brain cancer treatment.

Researchers discovered that expansion of these stem cells was higher than that normally seen in stem cells collected from patients, though the reasons why were not explored.

The researchers administered the engineered natural killer cell immunotherapy to mice bearing human brain cancer tumours with promising outcomes, significantly improving survival times and reducing tumour size.  

'Our ultimate goal is to bring this therapy to patients with brain tumours,' Dr Matosevic said. 'These patients urgently deserve better and more effective treatment options. We believe there is true potential for this therapy, and we have the motivation and capacity to bring it to the clinic'.