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Archive - Jan 2, 2019

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Stem Cell Medicine Ltd Licenses Exosome Technology (Exosomes Derived from Adult Stem Cells) for Intranasal Treatment of Autism Spectrum Disorder (ASD) from Tel Aviv University; Pre-Clinical Testing Shows Improvement in Several Behavioral Phenotypes

On January 2, 2019, Stem Cell Medicine Ltd. (SCM), a biotechnology company developing new therapies for neurological indications, including mesenchymal stem cells (MSCs) and anti-BMP (bone morphogenic protein) molecules for the treatment of multiple sclerosis, and gene therapy for the treatment of neuropathic pain, announced that it has licensed an innovative exosomes-based technology for the treatment of neurodegenerative and neuropsychiatric indications, specifically, autism spectrum disorder (ASD). The treatment is based on exosomes, derived from adult stem cells (MSC-exo), that are administered intranasally. The technology was developed by Professor Dani Offen, Sackler School of Medicine, Sagol School of Neurosciences, Tel Aviv University, and was licensed from Ramot, the Business Engagement Center at Tel Aviv University. The first indication to be developed with the exosome technology is ASD, which is a group of neurodevelopmental disorders characterized by three core symptoms: severe impairment of social interactions and communication skills, increased repetitive behaviors, and cognitive inflexibility. The prevalence of ASD has been steadily increasing in children over the past several years, with no effective treatment, hence, it represents a growing unmet medical need. More than 3.5 million Americans live with ASD. According to the US Department of Health and Human Services (CDC), the prevalence of autism in US children in 2018 is estimated to be 1 in 59 children and has increased from 1 in 110 children in 2010, making it the fastest-growing developmental disability with currently no FDA-approved drug. Given the size of the patient population and lack of treatments, the market opportunity is compelling.

Sex Differences in Glioblastoma Brain Tumors Revealed by Analysis of Patient Imaging, Transcriptome, and Survival Data—“We Should Definitely Develop and Evaluate Sex-Specific Treatment Regimens for Glioblastoma,” Research Leader Says

For decades, scientists have recognized that more males get cancer and die of the disease than females. This is true for many types of cancer, including the deadly brain tumor glioblastoma (GBM). Now, a team of researchers led by scientists at the Washington University School of Medicine in St. Louis has identified distinct molecular signatures of glioblastoma in men and women that help explain such underlying disparities in patients’ response to treatment and survival. The research suggests that tailoring treatments to men and women with glioblastoma based on the molecular subtypes of their tumors may improve survival for all patients. The findings were published January 2, 2019 in Science Translational Medicine. The open-access article is titled “Sex Differences in GBM Revealed by Analysis of Patient Imaging, Transcriptome, and Survival Data.” “It is our expectation that this study could have an immediate impact on the care of patients with glioblastoma and further research, as the findings indicate we should be stratifying male and female glioblastoma into risk groups and evaluating the effectiveness of treatment in a sex-specific manner,” said Joshua B. Rubin, MD, PhD, a Washington University Professor of Pediatrics and of Neuroscience and the study’s co-senior author. “The biology of sex differences and its applications in medicine are highly relevant, but almost always ignored aspects of personalized treatments.” Glioblastoma is the most common malignant brain tumor and kills about half of patients within 14 months of diagnosis. It is diagnosed nearly twice as often in males, compared with females. The tumor is most often diagnosed in people over age 50, and standard treatment is aggressive -- surgery, followed by chemotherapy and radiation.

New Findings on Genes Driving Male-Female Brain Differences & Puberty Timing; Scientists ID Genetic Pathway to Sexual Maturation in C. elegans That May Serve Same Function in Humans; Sex Differences in Men’s & Women’s Brains May Be Hard-Wired

Researchers have identified a group of genes that induces differences in the developing brains of male and female roundworms and triggers the initiation of puberty, a genetic pathway that may have the same function in controlling the timing of sexual maturation in humans. The study, led by Columbia University scientists, offers new evidence for direct genetic effects in sex-based differences in neural development and provides a foundation to attempt to understand how men's and women's brains are wired and how they work. The research was published January 1, 2019 in eLife, an open-access journal founded by the Howard Hughes Medical Institute (HHMI), the Max Planck Society, and the Wellcome Trust. The article is titled “Timing Mechanism of Sexually Dimorphic Nervous System Differentiation.” Scientists have long known that puberty is accompanied by substantial changes in the brain characterized by the activation of neurons that produce hormonal signals. But what causes the brain to start releasing the hormones that switch on puberty has remained elusive. "In this paper we show that a pathway of regulatory genes acts within specific neurons to induce anatomical and functional differences in the male versus female brain," said lead study author Oliver Hobert, PhD, Professor in Columbia's Department of Biological Sciences and a HHMI investigator. "Remarkably, we found that each member of this pathway is conserved between worms and humans, indicating that we have perhaps uncovered a general principle for how sexual brain differences in the brain are genetically encoded." For their study, the researchers worked with the transparent roundworm C. elegans, the first multicellular organism to have its genome sequenced.