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Archive - Nov 6, 2015

MGH/Harvard-Led Team Reports First-Ever Successful Systemic Treatment of Recurrent, BRAF-Mutant Brain Tumor (Craniopharyngioma) with Monoclonal Antibody BRAF Inhibitor Dabrafinib

A team led by Massachusetts General Hospital (MGH) investigators has reported the first successful use of a targeted therapy drug to treat a patient with a debilitating, recurrent brain tumor. In a paper published online on October 23, 2015 in the Journal of the National Cancer Institute (JNCI), the researchers report that treatment with the BRAF inhibitor dabrafinib led to shrinkage of a BRAF-mutant craniopharyngioma that had recurred even after four surgical procedures. More than a year after dabrafinib treatment, which was followed by surgery and radiation therapy, the patient's tumor has not recurred. The article is titled “Dramatic Response of BRAF V600E Mutant Papillary Craniopharyngioma to Targeted Therapy.” "This is the first time that a systemic therapy has shown efficacy against this type of tumor," says Priscilla Brastianos (photo), M.D., co-lead author of the JNCI report. Dr. Brastianos is Director, Central Nervous System Metastasis Program. Massachusetts General Hospital. Harvard Medical School. She is also Instructor of Medicine, Harvard Medical School. "This has the potential of completely changing the management of papillary craniopharyngiomas, which can cause lifelong problems for patients - including visual defects, impaired intellectual function, and pituitary and other hormonal dysfunction." Craniopharyngiomas are pituitary tumors that, while technically benign, can cause serious problems because of their location near critical structures, such as optic and other cranial nerves and the hypothalamus. Not only does the growing tumor compromise neurological and hormonal functions by impinging on these structures, but treatment by surgical removal or radiation therapy can produce the same symptoms by damaging adjacent tissues.

Capricor Therapeutics to Present 6-Month Safety & Exploratory Efficiency Results from Ongoing Phase I DYNAMIC Clinical Trial of Cardiosphere-Derived-Cell (CDC) Therapy (CAP-1002) for Advanced Heart Failure on Monday, November 9

Capricor Therapeutics, Inc., (NASDAQ: CAPR), a biotechnology company focused on the discovery, development, and commercialization of first-in-class therapeutics, announced, on Friday, November 6, 2015, that results will be presented from its ongoing Phase I DYNAMIC (Dilated Cardiomyopathy Intervention with Allogeneic Myocardially Regenerative Cells) clinical trial evaluating CAP-1002 in patients with advanced heart failure. The data will be presented in a poster to be delivered at the 2015 American Heart Association (AHA) Scientific Sessions taking place November 7-11, 2015 in Orlando, Florida. The poster is titled “Dilated Cardiomyopathy Intervention with Allogeneic Myocardially Regenerative Cells (DYNAMIC): Six Month Safety and Exploratory Efficacy Results,” and it will be presented on Monday, November 9, 2015, 5:30 - 6:45 pm ET in Poster Hall, A2, BS, in the “APS.06.02. Stem/Progenitor Cells II” session (Poster Number: M 104). CAP-1002 is Capricor's lead investigational allogeneic, cardiosphere-derived-cell (CDC) therapy. The Phase I DYNAMIC trial is evaluating CDCs (CAP-1002) ( in patients with advanced heart failure. The trial enrolled 14 patients with either ischemic or non-ischemic dilated cardiomyopathy with left ventricular ejection fraction (LVEF) of 35% or below and New York Heart Association (NYHA) Class III or Ambulatory Class IV heart failure. Suitable patients underwent sequential intracoronary infusion of CAP-1002 in up to three coronary territories. The primary safety endpoints were assessed at the 1-month visit. Other safety and exploratory efficacy endpoints, including ejection fraction, ventricular volumes, and a six-minute walk test were evaluated at 6 months and will be evaluated at a 12-month follow-up.

Worrisome Dung Beetle Decline in Mediterranean; Eco-Important Beetle’s Olfactory, Locomotor, & Reproductive Capacities Damaged by Low Doses of Ivermectin, Anti-Parasitic Drug Commonly Adminstered to Livestock

Research led by the University of Alicante's Ibero-American Centre for Biodiversity (WHERE) in Spain shows the adverse effects of an anti-parasitic drug (ivermectin), commonly administered prophylactically to livestock, on populations of a key dung beetle in Mediterranean ecosystems. A multidisciplinary research team, made up of researchers from the Spanish universities of Alicante, Jaen and Granada, the French Universities of Montpellier and Paul-Valéry Montpellier, the CSIC's National Museum of Natural Sciences, and the IUCN Centre for Mediterranean Cooperation, has analyzed the impact of ivermectin on Scarabaeus cicatricosus populations in the Mediterranean region. Led by José R. Verdú, Ph.D., from the Ibero-American Centre for Biodiversity (CIBIO) at the Universidad de Alicante, this research shows that arthropods that ingest this substance, even in low doses, become unable to interact with their surroundings because the drug affects both their olfactory and locomotor capacity. This fact may explain the population decline observed for this dung beetle. The open-access article reporting this finding, “Low Doses of Ivermectin Cause Sensory and Musculoskeletal Disorders in Dung Beetles,” was published online on September 9, 2015 in Scientific Reports. Ivermectin is a very effective anti-parasitic drug that has been used as a preventative in livestock since its discovery in 1981. Since then, the use of ivermectin use has increased exponentially to become a standard drug in the treatment and prevention of common parasites, including in human beings. The drug is considered by the World Health Organization (WHO) to be as an essential medication. Dr. Verdú points out that, although this drug has proven very effective, its widespread use comes at a price.

Exosome Diagnostics to Present New Data on Plasma EGFR Mutation Detection Using a Combined Exosomal RNA and Circulating Tumor DNA Approach & on ID of Novel Exosome mRNA Signature of Ipilimumab Response in Metastastic Melanoma Patients

Exosome Diagnostics, Inc., a developer of revolutionary, biofluid-based molecular diagnostics, announced on November 6, 2015 that the company will present two posters at the 26th AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, taking place November 5-9 at the Hynes Convention Center in Boston, Massachusetts. The first poster contains new data on the company’s exosomal RNA (exoRNA) plus cell-free (cfDNA) liquid biopsy panel for solid tumors. According to Exosome Diagnostics, this data demonstrates superior detection of activating and acquired resistance mutations to EGFR therapy in patients with non-small cell lung cancer (NSCLC) versus a cfDNA-only approach. The title of this poster is “Plasma EGFR Mutation Detection Using a Combined Exosomal RNA and Circulating Tumor DNA Approach in Patients with Acquired Resistance to First-Generation EGFR-TKIs” (#15-A-456-AACR). This poster will be presented on Saturday, November 7, from 12:30 to 3:30 p.m. ET. The second Exosome Diagnostics poster demonstrates the ability of the company’s proprietary exoRNA technology to enrich cancer-specific exosomes in order to more precisely determine tumor-specific gene changes in response to immunotherapy treatment. The title of this second poster is “Early Exosome mRNA Changes Are Associated with Improved Progression-Free Survival of Metastatic Melanoma patients on Ipilimumab: Identification of a Novel Exosome mRNA Signature of Ipilimumab Response” (#LB-C05). This poster will be presented on Sunday, November 8, from 12:30 to 3:30 p.m. ET. The image shows Johan Skog, Ph.D., Chief Scientific Officer and Founding Scientist of Exosome Diagnostics.

Characterization of Gene (Nup170) Involved in Nucleus-Cytoplasm Connection Will Allow Diagnosis of Patients & Carriers of Genetic Disease Causing Infertility & Lack of Puberty in Women; Prenatal Diagnosis & Disease Prevention Also Enabled

In the open-access cover story of the November issue of the Journal of Clinical Investigation (first published online on October 20, 2015), scientists at the Hebrew University of Jerusalem Faculty of Medicine, together with colleagues at other institutions in Israel, announced a discovery that will allow doctors to diagnose a disease causing infertility and lack of puberty in women, and potentially to develop new treatment options. The characterization of the nucleoporin 107 (Nup107) gene and discovering its function in ovarian development will provide a greater understanding of how signaling between the cytoplasm and the nucleus is uniquely involved in ovarian development. On a medical level, it will allow the diagnosis of both patients and carriers of the disease, as well as prenatal diagnosis and prevention of this difficult morbidity of lack of puberty and infertility. Potentially, it could also lead to a method of treatment for problems of infertility and premature ovarian failure. The research paper was accompanied by a special commentary written by two world-renowned experts in the fields of Drosophila and human reproductive systems. The research article is titled “A Mutation in the Nucleoporin-107 Gene Causes XX Gonadal Dysgenesis.” The commentary is titled “Poreless Eggshells.” The mammalian ovary functions, not only as the reproductive organ that contains the germ cells (oocytes) responsible for creating the next generation, but also as a hormonal gonad (reproductive gland) regulating many aspects of female physiology and development. Very little is known about the processes involved in the development of the ovary and the egg. To date, only a few genes have been identified as important in ovarian development, and any findings in this area are of great importance with regard to the fields of infertility and fertility.

Tapeworm in Immune-Compromised Columbian Man Developed Cancer-Like Cells That Spread to Infected Man and Killed Him, CDC Study Finds; First-Ever Such Case Is Described in New England Journal of Medicine

Scientists at the Centers for Disease Control and Prevention (CDC) have discovered cancer cells originating in a common tapeworm may take root in people with weakened immune systems, causing cancer-like tumors. It is the first known case of a person becoming ill from cancer cells that arose in a parasite – in this case, Hymenolepis nana, the so-called “dwarf tapeworm” that is the most common tapeworm found in humans. The report, published in an open-access article in the November 5, 2015 issue of the New England Journal of Medicine, raises concern that other similar cases, if they occur, may be misdiagnosed as human cancer – especially in less developed countries where this tapeworm and immune-system-suppressing illnesses like HIV are widespread. The article is titled “Malignant Tranformation of Hymenolepis nana in a Human Host.” (Please see links to the open-access NEJM article and to numerous popular press articles at end of this summary).“We were amazed when we found this new type of disease – tapeworms growing inside a person essentially getting cancer that spreads to the person, causing tumors,” said Atis Muehlenbachs, M.D., Ph.D., Staff Pathologist in the CDC’s Infectious Diseases Pathology Branch (IDPB) and lead author of the study. “We think this type of event is rare. However, this tapeworm is found worldwide and millions of people globally suffer from conditions like HIV that weaken their immune system. So there may be more cases that are unrecognized. It’s definitely an area that deserves more study.” In 2013, doctors in Colombia asked the CDC to help diagnose bizarre biopsies from lung tumors and lymph nodes of a 41-year-old man who was HIV-positive. The tumors looked similar to a human cancer, but initial CDC lab studies revealed the cancer-like cells were not human.