Glioblastoma is a type of brain tumor which is incredibly aggressive and is typically fatal within six to nine months of diagnosis. At present the only hope of extending this prognosis is to have invasive surgery, followed by extensive chemotherapy, but even then, the tumor typically regrows within months.
This type of cancer is challenging to treat in part because of the blood-brain barrier, which makes it difficult for chemotherapy drugs to reach the tumor. The tumours are also able to change their environment to one that prevents tumour fighting immune cells from reaching it. A team of researchers have used gene therapy in an attempt to prolong the survival of patients.
The gene therapy involves a ‘viral vector’ which encodes an immune-activator gene that produces interleukin 12 (IL-12). This is injected into the brain of patients following the removal of the tumour. The gene can be switched off and on using a medication called veledimex; taking the drug switches on the gene, stopping taking it switches off the gene. Because veledimex can cross the blood-brain barrier it is able to switch on the IL-12 in the brain, which in turn generates an immune response of tumour fighting immune cells.
Patients who received this gene therapy lived longer than those treated traditionally, some survived for as long as two years, which is a vast improvement on current survival times. Given the trial was small in size, further research and trials will be required, although it does offer hope that one day the prognosis for those diagnosed with glioblastoma will be improved.
Source: The Scientist
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