Clinical trial of cancer and NF therapies recruiting
The University of Minnesota serves as 1 of 15 sites participating in a 4-part phase I - IIa trial evaluating the safety and efficacy of trametinib in patients with cancer or plexiform neurofibromas and of trametinib and dabrafenib in patients with cancers harboring BRAF V600 mutations. Patients, including those with neurofibromatosis 1 (NF1), are being recruited, and the study is expected to be completed by October 15, 2020. The study seeks to establish safe, pharmacologically relevant doses of the drug therapies in infants, children, and adolescents. Investigators also seek to determine preliminary activity of trametinib monotherapy and trametinib with dabrafenib in selected recurrent, refractory or unresectable childhood tumors. (NCT02124772)
Swine model of NF1 aids in preclinical drug testing
Pigs genetically engineered to carry mutations for NF1 are providing researchers insights into potentially effective therapies. The pigs have been used to test characteristics of drugs being considered for future trials in humans with NF1 and are planned to play a role in continuing research into new therapies. The modified pigs show characteristics of NF1, including café au lait spots, one of the diagnostic criteria for the disease. Their size, longer lifespan, and similar physiology to humans make them better suited as experimental animals than smaller animals. David Largaespada, PhD, and Christopher Moertel, MD, co-lead 1 of the Children’s Tumor Foundation consortia that helped developed the swine model in cooperation with Recombinetics.
New head of neurosurgery
Clark C. Chen, MD, PhD has joined the University of Minnesota Medical School faculty as the Lyle French Chair in Neurosurgery and the Head of the Department of Neurosurgery. He also serves as a University of Minnesota Health neurosurgeon. Chen is a nationally recognized surgeon and brain tumor researcher. Among his interests are understanding how glioblastomas become resistant to radiation and chemotherapy. He also brings an active NIH-funded research program focused on developing novel diagnostic and therapeutic approaches for brain tumor patients. He has received numerous awards for his work, and in 2015 received Taiwan’s Presidential Award of Achievement, the country’s highest award.
Glioma therapy clinical trial to open
A clinical trial investigating a genetically engineered virus used to target gliomas will soon be open to participants at University of Minnesota Health locations. The phase III clinical trial will involve 2 steps. Participating patients will first be injected with the engineered virus, named Toca511, at the time of surgery. Designed to infect only the tumor cells and not normal brain tissue, the virus, once inside the cancer cell, produces the enzyme cytosine deaminase (CD). Following the procedure, the patient will receive Toca FC in pill form, a compound that remains inert until it comes in contact with a cancer cell producing CD. CD converts Toca FC into a potent chemotherapy, which kills the cancer cell. In addition to the direct destruction of the cancer cells, the virus also boosts the patient’s immune system to better allow the body to fight off cancer.
In a phase I study, impressive results were observed with this therapy. Glioma patients who suffer recurrence after standard-of-care therapy generally have a survival rate of fewer than 9 months. In patients treated with the combination therapy Toca511 and Toca FC, 20% survived beyond 3 years. The impressive clinical outcome has earned the therapy an FDA breakthrough designation in the U.S. and the designation of European Medicines Agency Priority Medicines.
Gliomas, a broad category of spinal cord and brain tumors, are among the cancers that NF1 patients are prone to develop.
NF patients are at increased risk of additional tumors and cancers. Clinical and research collaborations, novel drugs, and MRI-guided procedures aim to improve outcomes for these patients.Continue reading
A young patient with neurofibromatosis 1 seeks a second evaluation of a deep brain lesion. An MRI-guided biopsy enables laser ablation of the tumor. The patient recovers with no neurological deficit.Continue reading