Glioblastoma, one of the most lethal brain cancers, remains a challenge to treat despite advancements in conventional therapies. Oncolytic virus therapy, which can selectively target and kill tumor cells while stimulating the immune system, has shown promise in clinical trials.
Hemispherian AS has received IND clearance for GLIX-1 for the treatment of glioblastoma from the U.S. FDA. GLIX-1 targets DNA repair mechanisms, specifically the enzyme TET2, selectively in tumor cells while sparing healthy tissues.
Malignant gliomas, which include glioblastomas, are the most frequent primary brain cancer and are associated with extremely poor prognosis. They nearly always recur after treatment, rapidly leading to death.
Diffuse midline gliomas (DMGs) are pediatric brain tumors with a very dismal prognosis since less than 5% of patients survive 2 years after diagnosis. Radiotherapy remains the standard treatment for DMG, and several combination chemotherapies and single-agent target therapies are currently being explored.
Glioblastoma is the most frequent and aggressive primary brain cancer in adults, and patients can expect to live shorter than 2 years, regardless of therapy. The cancer can be treated with CAR T cells, but many patients develop resistance because tumors mutate or delete the antigens recognized by the T cells, while the tumor microenvironment suppresses T-cell activity.
Loss-of-function variants in the ELP1 gene are the most prevalent predisposing genetic factors in childhood medulloblastoma, accounting for about 30% of sonic hedgehog medulloblastoma (SHH-3 subtype).
Myosin Therapeutics Inc. has obtained IND clearance by the FDA for MT-125. A phase I study will evaluate MT-125 in combination with standard-of-care radiation in patients with newly diagnosed IDH wild type, MGMT unmethylated glioblastoma.
The EMA’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending European orphan drug designation for Hemispherian AS’s GLIX-1 for the treatment of glioma.
Glioblastoma is the most common and aggressive malignant brain tumor in adults. Although microtubule-targeting agents (MTAs) are among the most widely used first-line therapies in cancer, their efficacy in glioblastoma is limited by poor penetration of the blood–brain barrier.
