Life Science Newsletter – 5/1/2018

In this issue: What about itching?, Mayo on migraines, putting ‘person’ in personalized medicine, editing out blood disease, mystery of rare bone disorder, FDA on caffeine in supplements, practicing generosity, shedding light on cancer drug shortages, getting the heart to sleep, polyps to cancer-which ones?, blood test for Alzheimer’s risk, mysteries of the senses, butterflies of the soul, and more…


FDA takes new steps to advance the development of innovative products for treating opioid use disorder

The U.S. Food and Drug Administration announced today the latest action to encourage and support the development of treatment options for people with opioid use disorder (OUD).

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UCLA study reports nearly 1 in 3 California kids have a sugary drink daily

Increase since 2009 alarms public health professionals

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Editing Out Blood Disease

Gene therapy successful in 22 patients with severe form of the blood disorder beta-thalassemia

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UroGen Pharma Announces Presentation of Results from Interim Analysis of Pivotal Phase 3 OLYMPUS Trial of UGN-101 (MitoGel™) for Non-Surgical Treatment of Upper Tract Urothelial Cancer (UTUC)

UroGen Pharma Ltd. (URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, with a focus on uro-oncology, today announced a new data presentation from an interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel™), an investigational mitomycin formulation for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).

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New clues point to relief for chronic itching

Targeting an opioid receptor calms itch

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Putting the person in personalised medicine

Dr Sam Dick, our Science Information and Policy Officer, considers the importance of assessing a patient’s views about their quality of life for the future of personalised medicine.

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Motif Bio Initiates NDA Rolling Submission for Iclaprim and Provides Business Update

Motif Bio plc (AIM:MTFB) (NASDAQ:MTFB), a clinical-stage biopharmaceutical company specialising in developing novel antibiotics, today announced the initiation of a rolling submission of a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for iclaprim, a targeted, Gram-positive investigational antibiotic, for the treatment of acute bacterial skin and skin structure infections (ABSSSI). The NDA submission is expected to be completed during the second quarter of 2018.

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Transplanted livers help body defend against organ rejection, Mayo Clinic study finds

Transplanted livers change the profile of blood cells in the recipients, reducing the potential for organ rejection, Mayo Clinic research shows. The findings are published in Kidney International.

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Avenue Therapeutics Receives Notices of Allowance for Patent Applications Covering Methods of
Administration for Intravenous Tramadol

Avenue Therapeutics, Inc. (NASDAQ: ATXI) (“Avenue”), a company focused on the development and commercialization of intravenous (IV) tramadol, today announced receipt of Notices of Allowance from the U.S. Patent and Trademark Office (USPTO) for two patent applications covering methods of administration for its lead…

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Early Skin Cancer is More Accurately Diagnosed by a Dermatologist Than Other Providers, New Research Shows

Physician assistants (PAs) are increasingly used in dermatology practices to cut costs and improve access to care, but are more likely than dermatologists to perform unnecessary skin biopsies to check for cancer, while being less likely to accurately diagnose early stage skin cancers, according to new research conducted by the University of Pittsburgh School of Medicine.

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Southampton scientists discover sweet spot of activity in immune system key to fighting cancer

Scientists at the University of Southampton have shown how stimulating a specific location on the surface of immune cells can be targeted with antibodies to help in their fight against cancer.

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Researchers find five sub-types of the brain cancer glioma

There are five distinct molecular sub-types of the brain cancer glioma, according to findings from the largest ever genetic study of its kind of the cancer.

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Statement from FDA Commissioner Scott Gottlieb, M.D., on new enforcement actions and a Youth Tobacco Prevention Plan to stop youth use of, and access to, JUUL and other e-cigarettes

Protecting our nation’s youth from the dangers of tobacco products is among the most important responsibilities of the U.S. Food and Drug Administration – and it’s an obligation I take personally. We recognize that if the FDA is to end the tragic cycle of successive generations of nicotine and tobacco addiction, we must take every opportunity to disrupt that process where it starts: youth access to and use of tobacco products.

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Getting a better look at living cellsNanoscale-level imaging of living cells has become a reality in the past few years using transmission electron microscopy and sealed sample holders that keep cells alive in a liquid environment. But do the high-resolution images obtained using these tools truly reflect the structures and functions of cells, or do they show cells damaged by the high-intensity electron beam used in transmission electron microscopy?

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Macular degeneration linked to aging immune cells

Older cells promote inflammation, abnormal blood vessel growth that can lead to blindness

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Research offers clues for improved influenza vaccine design

Influenza vaccines that better target the influenza surface protein called neuraminidase (NA) could offer broad protection against various influenza virus strains and lessen the severity of illness, according to new research published in Cell.

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FDA issues new guidance on highly concentrated caffeine in dietary supplements

The U.S. Food and Drug Administration has issued a new guidance document for industry addressing safety concerns related to highly concentrated caffeine in dietary supplements.

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Public awareness of cancer drug shortages is lacking, but most would want to know about drug substitutions if they were patients, study finds

Relatively few Americans are aware of ongoing shortages of many drugs used in cancer treatment, but they widely agree that patients with cancer should be informed about any substitute drugs used in their care, a nationwide survey conducted by investigators at Dana-Farber Cancer Institute and Brigham and Women’s Hospital shows.

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   New definition of Alzheimer’s changes how disease is researched

When you think of dementia, most people automatically think of Alzheimer’s disease, too. But, under a new definition of Alzheimer’s, the two terms no longer will be considered interchangeable.

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NIH researchers crack mystery behind rare bone disorder

Study finds gene mutations that cause “dripping candle wax” bone disease.

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Practicing generosity

Alumna Jane Hardesty Poole learned from her physician-father the importance of giving. Today, she continues to support the university in honor of his lifetime of service.

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People with Type 2 diabetes who eat breakfast later, more likely to have a higher BMIBeing an “evening person” is linked to higher body mass indices among people with Type 2 diabetes, and having breakfast later in the day seems to be what drives this association, according to a new paper in the journal Diabetic Medicine.

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Six in seven women at high risk of breast cancer shun tamoxifen as a preventative measure

Six in seven women with a family history of breast cancer opt out of taking tamoxifen as a preventative measure, according to a study funded by Cancer Research UK and published (link is external)in Breast Cancer Research and Treatment *.

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Genetic markers may predict which melanoma patients benefit from immunotherapy drug

Cancer immunotherapy drugs known as “checkpoint inhibitors” are powerful new tools for treating metastatic melanoma, but physicians struggle to predict which patients will benefit from a given drug and which may suffer unnecessary toxicity. Scientists at Dana-Farber Cancer Institute, however, have discovered genetic markers for resistance to ipilumumab, the first checkpoint inhibitor approved by the Food & Drug Administration for the disease, according to a new report in the Journal Cell.

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Mayo study shows easy-to-use, noninvasive stimulation device can help prevent migraine attacks

A migraine is much more than just a bad headache. Migraine symptoms, which can be debilitating for many people, are the sixth leading cause of disability, according to the World Health Organization.

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For women with kidney cancer, belly fat matters

Abdominal fat linked to shorter survival

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Science Surgery: ‘Do we all have potentially cancerous cells in our bodies?’

A recipe for trouble
There’s a crucial set of ingredients needed for a cell to turn cancerous, which we’ve previously explored. A cell that’s capable of growing uncontrollably collects mistakes in its DNA code, which cause a gene or set of genes to go awry. If the cell also loses certain safety mechanisms, this pushes it towards cancer. It’s like cutting the brakes on a speeding car.

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Global Cancer Trial Sets New Standard for Post-Surgery Chemotherapy

Some stage III colon cancer patients can cut in half the number of chemotherapy treatments they receive after surgery, significantly reducing the costs, treatment time, and long-term toxic effects of chemotherapy, according to results of a unique global clinical trial collaboration published for the first time in the New England Journal of Medicine.

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Blood test indicates risk of developing Alzheimer’s disease

A newly developed blood test may indicate Alzheimer’s disease on average eight years before the clinical diagnosis. This was demonstrated by scientists from the Ruhr University Bochum (RUB), the German Cancer Research Center (DKFZ) and the Saarland Cancer Registry with a large population-based cohort study from the Saarland.
Joint press release of Ruhr University Bochum and German Cancer Research Center

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Colon cancer chemo could be halved after surgery

Chemotherapy courses after surgery could be cut in half for some colon cancer patients, according to a new global study.

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Mysteries of the Senses

Scientists will discuss latest research, new gene therapies at international sensory biology conference

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Science Saturday: Identifying characteristics of colon polyps most likely to progress to cancer

Researchers at Mayo Clinic are closer to answering the question, why does one colon polyp transform to cancer while another seemingly identical polyp does not? A colon polyp is a small clump of cells that forms on the lining of the colon. While most colon polyps are harmless some can develop into colon cancer, which can be fatal if found too late.

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A new class of antibiotics to combat drug resistance

Researchers from the University of Illinois at Chicago and Nosopharm, a biotechnology company based in Lyon, France, are part of an international team reporting on the discovery of a new class of antibiotics.

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Three-quarters of COPD cases are linked to childhood risk factors that are exacerbated in adulthood

Three-quarters of chronic obstructive pulmonary disease (COPD) cases have their origins in poor lung function pathways beginning in childhood. These pathways are associated with exposures in childhood, and amplified by factors in adulthood, according to a cohort study published in The Lancet Respiratory Medicine journal.

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Butterflies of the Soul

New study sheds light on the developmental origins of interneurons

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Science Saturday: Getting to the Heart of Sleep

Growing up in South Africa, Virend Somers and his two cousins would lie on a big bed with their grandfather as he told them stories. “We really enjoyed the stories. But what we also looked forward to — I say this with a bit of guilt now — is when he’d fall asleep.

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Dana-Farber Cancer Institute, Novartis and Harvard team up to develop next generation biomaterial systems to deliver immunotherapies
Scientists from Dana-Farber Cancer Institute and Wyss Institute for Biologically Inspired Engineering at Harvard University are teaming up to develop biomaterial systems for Novartis’ portfolio of immuno-oncology therapies.

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Soft drinks sugar tax to tackle obesity ‘has already had an effect’

The Soft Drinks Industry Levy (link is external) charges manufacturers based on the amount of sugar added to beverages. Campaigners hope it will reduce obesity levels and improve health.

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