Brain and Spinal Cord Tumor Research Results and Study Updates

Recent results from several small clinical trials have suggested it may be possible to develop an effective immunotherapy for glioblastoma. Among them are findings from a four-patient trial testing a unique type of mRNA cancer vaccine.

FDA has granted an accelerated approval to tovorafenib (Ojemda) for kids and teens who have low-grade glioma with changes in the BRAF gene. In a small clinical trial, the drug shrank or completely eliminated tumors in nearly 70% of patients.

The activity of 34 genes can accurately predict the aggressiveness of meningiomas, a new study shows. This gene expression signature may help oncologists select the best treatments for people with this common type of brain cancer than they can with current methods.

An NCI-supported study called OPTIMUM, part of the Cancer Moonshot, was launched to improve the care of people with brain tumors called low-grade glioma in part by bringing them into glioma-related research.

Treating craniopharyngioma often requires surgery, radiation therapy, or both. But results of a study suggest that, for many, combining the targeted therapies vemurafenib (Zelboraf) and cobimetinib (Cotellic) may substantially delay, or even eliminate, the need for these treatments.

In a large clinical trial, vorasidenib slowed the growth of low-grade gliomas that had mutations in the IDH1 or IDH2 genes. Vorasidenib is the first targeted drug developed specifically to treat brain tumors.

Researchers have found that the aggressive brain cancer glioblastoma can co-opt the formation of new synapses to fuel its own growth. This neural redirection also appears to play a role in the devastating cognitive decline seen in many people with glioblastoma.

Two companion studies have found different forms of some brain tumors, diffuse midline glioma and IDH-mutant glioma, become dependent for their survival on the production of chemicals called pyrimidines. Clinical trials are planned to test a drug that blocks pyrimidine synthesis in patients with gliomas.

The combination of dabrafenib (Tafinlar) and trametinib (Mekinist) shrank more brain tumors, kept the tumors at bay for longer, and caused fewer side effects than chemotherapy, trial results showed. The children all had glioma with a BRAF V600 mutation that could not be surgically removed or came back after surgery.

Two separate but complementary studies have identified a new way to classify meningioma, the most common type of brain tumor. The grouping system may help predict whether a patient’s tumor will grow back after treatment and identify new treatments.

A nanoparticle coating may help cancer drugs reach medulloblastoma tumors in the brain and make the treatment less toxic. Mice treated with nanoparticles containing palbociclib (Ibrance) and sapanisertib lived substantially longer than those treated with either drug alone.

A new test could potentially be used to identify children treated for medulloblastoma who are at high risk of their cancer returning. The test detects evidence of remaining cancer in DNA shed from medulloblastoma tumor cells into cerebrospinal fluid.

Standard radiation for medulloblastoma can cause long-term damage to a child’s developing brain. A new clinical trial suggests that the volume and dose of radiation could be safely tailored based on genetic features in the patient’s tumor.

In people with glioblastoma and other brain cancers, steroids appear to limit the effectiveness of immunotherapy drugs, a new study shows. The findings should influence how steroids are used to manage brain tumor symptoms, researchers said.

Results from two studies show that a liquid biopsy that analyzes DNA in blood accurately detected kidney cancer at early and more advanced stages and identified and classified different types of brain tumors.

A method that combines artificial intelligence with an advanced imaging technology can accurately diagnose brain tumors in fewer than 3 minutes during surgery, a new study shows. The approach can also accurately distinguish tumor from healthy tissue.

Glioblastoma cells sneak many copies of a key oncogene into circular pieces of DNA. In a new NCI-funded study, scientists found that the cells also slip several different genetic “on switches” into these DNA circles, helping to fuel the cancer’s growth.

Men and women with glioblastoma appear to respond differently to standard treatment. A new study identifies biological factors that might contribute to this sex difference.

A liquid biopsy blood test can detect DNA from brain tumors called diffuse midline gliomas, researchers have found. This minimally invasive test could be used to identify and follow molecular changes in children with these highly lethal brain tumors.

Despite continued efforts to develop new therapies for glioblastoma, none have been able to improve how long patients live appreciably. Despite some setbacks, researchers are hopeful that immunotherapy might be able to succeed where other therapies have not.