Medication prescribed for a certain type of epilepsy may offer a new method for treating malignant infantile brain tumors. A specific mTOR inhibitor has the ability to cross the blood-brain barrier to both reach and attack the tumor at source. This has been demonstrated by researchers from Uppsala University, in collaboration with US and UK colleagues, whose research has now been published in the scientific journal Cell Stem Cell.

Approximately 100 children suffer infantile brain tumors in Sweden each year. The most common type of malignant brain tumor in infants and children is medulloblastoma. Radiation therapy is part of the standard treatment for medulloblastomas and modern radiation therapy has saved the lives of many children suffering from these often aggressive cancers; however, as it often comes with serious side effects for healthy brain tissue, it is seldom prescribed for infants. Although a presumably better solution would be to give more targeted treatment, in order to establish such a therapy it would naturally need to be proven to be both more effective and come with fewer side effects than current treatments.

Many infantile medulloblastomas are amplified by MYCN, an oncogene that drives tumor growth and metastasis to the spinal column, leading to a very poor prognosis. In the study in question, the researchers cultivated a particular type of neural stem cell and were able to demonstrate that MYCN was quickly able to turn these into cancer cells. This suggests that these cells are likely to be the origin of infantile medulloblastomas.

The FDA is helping to speed up the process of researching and approving psilocybin, a hallucinogenic substance in magic mushrooms, to treat major depressive disorder (MDD).

For the second time in a year, the U.S. Food and Drug Administration (FDA) has designated psilocybin therapy — currently being tested in clinical trials — as “breakthrough therapy,” an action that is meant to accelerate the typically sluggish process of drug development and review. It is typically requested by a drug company and granted only when preliminary evidence suggests the drug may be an enormous improvement over already available therapy, according to the FDA.

It turns out the gut is full of surprises. And one of those surprises may have offered up a key for unlocking a new way of treating multiple sclerosis (MS). Investigators from Brigham and Women’s Hospital have discovered a microRNA—a small RNA molecule—that increases during peak disease in a mouse model of MS and in untreated MS patients. When a synthetic version of the microRNA was orally given to the mice, it prevented disease. While several steps remain before these insights can be translated into therapy for patients, the researchers describe their results as both exciting and unexpected. Their findings are published in Cell Host & Microbe.

“We’ve discovered a new mechanism to regulate the microbiome and treat human disease that hadn’t been known before,” said senior author Howard Weiner, MD, co-director of the Ann Romney Center for Neurologic Diseases at the Brigham. “The gut microbiome is known to play an important role in MS and other diseases. Our findings, which show that a microRNA can be used to target and influence the microbiome with precision, may have applicability for MS and many other diseases, including diabetes, ALS, obesity and cancer.”

Weiner, lead author Shirong Liu, MD, Ph.D., an instructor in the Weiner laboratory, and their colleagues investigated how the altered gut microbiome affects the course of MS. To do so, they studied the microbiome and microRNAs found in the experimental autoimmune encephalomyelitis (EAE) model of MS. Unexpectedly, they found that when they transferred fecal matter from EAE mice at peak disease, it protected the mice who received the transfer. The team found that a specific microRNA, known as miR-30d, rather than live bacteria, was responsible for preventing disease. The investigators found that miR-30d is enriched in untreated, relapsing-remitting MS patients as well.