Pioneering Gene Therapy Unlocks Extended Survival in Glioma Trials

Promising advancements in brain tumor therapy are observed in human trials, where a pioneering gene therapy regimen exhibits both safety and efficacy.

Human trials of gene therapy for brain tumors demonstrate encouraging outcomes as a combination of cell-killing and immune-boosting drugs proves both safe and effective in extending survival for glioma patients.

Given gliomas' poor prognosis and limited response to treatments like chemotherapy and radiation, the team looked to using the adenoviral gene therapy.

Brain Tumor
Brain tumors are the abnormal growth of brain cells that may be benign or metastatic. Brain tumors account for about 2.4% of all the tumor diagnosesandare most challenging due to their critically delicate location.

‘Exciting progress emerges in the field of brain tumor treatment, as human trials reveal that a novel gene therapy combining cell-killing and immune-boosting drugs can safely and effectively prolong the survival of glioma patients. # BrainTumor, #GeneTherapy ’

The therapy proved not only safe but also improved survival, reveals the study published in the journal The Lancet Oncology."Being able to move a novel therapy from bench to bedside in such a streamlined fashion is exciting, and represents a tour-de-force in translational medicine," said Oren Sagher, Professor of neurosurgery at the University of Michigan.

In the phase 1 trial, the team focused on two types of genetic therapies in high-grade gliomas. The first was a combination of HSV-1-TK -- a protein -- and Valtrex -- a drug used to treat viral infections like cold sores and chickenpox.

HSV-1-TK turns Valtrex into a cytotoxic compound that kills actively dividing cancer cells. The second was Flt3L -- a protein that recruits essential immune cells to the brain. When used in combination, these therapies showed exciting early results, including improved survival.

Brain Tumor in Children
An abnormal growth of cells in the brain is called brain tumor. Most tumors arise within the brain (primary). They can be benign or cancerous.

Of the 18 patients enrolled in the trial, six survived more than two years, three survived more than three years, and one patient, who is still alive at the time of publication, survived up to five years.

Glioma Patients Rejoice

With the current standard-of-care, the life expectancy for a tumor of this kind is just over 14 months. Further, the study found that this treatment wasn't toxic to the patients, suggesting that the highest dose used in this trial could be used in future trials.

Though the adenoviral gene therapy vectors were supposed to be active for up to a month, the team discovered that activity from the adenoviral vector expressing the HSV1-TK was active for up to 17 months.

Glioblastoma Multiforme
Glioblastomas are tumors that arise from astrocytes that make up the supportive tissue of the brain. Glioblastomas are usually highly malignant.

This discovery changes expectations of adenoviral gene therapy in the brain and extends the potential time during which the combination of HSV1-TK and Valtrex might be harnessed to combat tumor recurrence.

"This originated from a theoretical idea based on evolutionary hypotheses and was first tested in experimental models of the disease," said Pedro Lowenstein, Professor of Neurosurgery at U-M.

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"Finally, after many years, we're thrilled to report the results of testing this approach in human patients, obtaining results that will lead to better treatments for this group of brain tumor patients," said Maria Castro, Professor of neurosurgery at U-M.

Although more work is needed before this is brought to the clinic, the significance of long-term expression of HSV1-TK suggests implementation to improve treatment. The results of this study support the design of future phase 1b/2 clinical trials.

Source-IANS