Neuroblastoma tyrosine kinase signaling networks involve FYN and LYN in endosomes and lipid rafts.

Protein phosphorylation plays a central role in creating a highly dynamic network of interacting proteins that reads and responds to signals from growth factors in the cellular microenvironment. Cells of the neural crest employ multiple signaling mechanisms to control migration and differentiation during development. It is known that defects in these mechanisms cause neuroblastoma, but how multiple signaling pathways interact to govern cell behavior is unknown. In a phosphoproteomic study of neuroblastoma cell lines and cell fractions, including endosomes and detergent-resistant membranes, 1622 phosphorylated proteins were detected, including more than half of the receptor tyrosine kinases in the human genome. Data were analyzed using a combination of graph theory and pattern recognition techniques that resolve data structure into networks that incorporate statistical relationships and protein-protein interaction data. Clusters of proteins in these networks are indicative of functional signaling pathways. The analysis indicates that receptor tyrosine kinases are functionally compartmentalized into distinct collaborative groups distinguished by activation and intracellular localization of SRC-family kinases, especially FYN and LYN. Changes in intracellular localization of activated FYN and LYN were observed in response to stimulation of the receptor tyrosine kinases, ALK and KIT. The results suggest a mechanism to distinguish signaling responses to activation of different receptors, or combinations of receptors, that govern the behavior of the neural crest, which gives rise to neuroblastoma.

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Cancer and non-cancer mortality among French uranium cycle workers: the TRACY cohort.

The health effects of internal contamination by radionuclides, and notably by uranium, are poorly characterised. New cohorts of uranium workers are needed to better examine these effects. This paper analyses for the first time the mortality profile of the French cohort of uranium cycle workers. It considers mortality from cancer and non-cancer causes.
The cohort includes workers employed at least 6 months between 1958 and 2006 in French companies involved in the production of nuclear fuel. Vital status and causes of death were collected from French national registries. Workers were followed-up from 1 January 1968 to 31 December 2008. Standardised mortality ratios (SMRs) were computed based on mortality rates for the French general population.
The cohort includes 12 649 workers (88% men). The average length of follow-up is 27 years and the mean age at the end of the study is 60 years. Large mortality deficits are observed for non-cancer causes of death such as non-cancer respiratory diseases (SMR=0.51 (0.41 to 0.63)) and circulatory diseases (SMR=0.68 (0.62 to 0.74)). A mortality deficit of lower magnitude is also observed for all cancers combined (SMR (95% CI): 0.76 (0.71 to 0.81)). Pleural mesothelioma is elevated (SMR=2.04 (1.19 to 3.27)).
A healthy worker effect is observed in this new cohort of workers involved in the uranium cycle. Collection of individual information on internal uranium exposure as well as other risk factors is underway, to allow for the investigation of uranium-related risks.
Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to View Source

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8-K – Current report

On April 7, 2016 Heat Biologics, Inc. ("Heat") (Nasdaq: HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported that the company is implementing cost-saving measures and a focused corporate strategy to achieve, with its current cash on-hand, data readout in the fourth quarter of 2016 for its lead Phase 2 program evaluating HS-410 for the treatment of non-muscle invasive bladder cancer (Filing, 8-K, Heat Biologics, APR 7, 2016, View Source [SID:1234510529]).

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These cost-saving measures include a workforce reduction of approximately 22% of the company’s headcount and a reduction in compensation for the remaining leadership team. Heat also announced the voluntary resignation of two members from its Board of Directors. In addition, Heat will further decrease operating expenses by advancing only the eight patients currently enrolled in its Phase 1b clinical trial evaluating HS-110 for the treatment of non-small cell lung cancer instead of enrolling the eighteen patients initially planned. Heat intends to focus its resources to achieve the important near-term milestones for these two product candidates, both of which are expecting topline data in the fourth quarter of 2016.

"We remain committed to advancing our lead clinical program for HS-410 and the focused reductions announced today extend our cash runway to reach significant inflection points expected later this year," said Jeff Wolf, Heat’s Founder and CEO. "While difficult, these cost-saving measures are necessary. We are truly grateful for the meaningful contributions of the talented individuals impacted."

CHMP Issues Positive Opinion to Include New Data in European Label for XTANDI(R) (Enzalutamide)

On April 7, 2016 Medivation, Inc. (NASDAQ: MDVN) and Astellas Pharma Inc. (TSE: 4503) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending approval of a type II variation to include data from the head-to-head TERRAIN trial of enzalutamide versus bicalutamide in the European label for XTANDI (enzalutamide) capsules (Press release, Medivation, APR 7, 2016, View Source [SID:1234510502]).

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The positive CHMP opinion is based on results from the TERRAIN study, which enrolled 375 patients in North America and Europe with metastatic prostate cancer whose disease progressed despite treatment with a luteinizing hormone-releasing hormone (LHRH) analogue therapy or following surgical castration. The trial showed statistically significant progression free survival (PFS) improvement with enzalutamide versus bicalutamide [median PFS 15.7 months vs 5.8 months, HR = 0.44 (95% CI: 0.34, 0.57), p < 0.0001].The median time on treatment in the TERRAIN trial was 11.7 months in the enzalutamide group versus 5.8 months in the bicalutamide group. Grade 3 or higher cardiac adverse events were reported in 5.5% of enzalutamide-treated patients versus 2.1% of bicalutamide-treated patients; the higher rate in the enzalutamide group may be partly due to the twice longer duration of treatment in that group. Two seizures were reported in the enzalutamide group and one in the bicalutamide group. The most common side effects occurring during treatment and more common in the enzalutamide-treated versus bicalutamide-treated patients included fatigue, hot flush, hypertension, diarrhea, weight decreased and pain in extremity. These results were published in Lancet Oncology in January 2016.

"We are very pleased with the CHMP’s decision to update the XTANDI label to include these data," said David Hung, M.D., founder, president & chief executive officer of Medivation. "TERRAIN was the first and largest head-to-head trial comparing enzalutamide against bicalutamide in the treatment of patients with metastatic CRPC."

"We are pleased that the CHMP has recommended inclusion of the TERRAIN data in the European label for XTANDI," said Claire Thom, Pharm D., senior vice president and oncology therapeutic head, Astellas.

The EMA is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the 28 countries of the European Union. The CHMP’s positive recommendation does not change indications or contradictions, meaning that no European Commission decision is needed for this variation before implementation of the update to the Summary of Product Characteristics. The label update will take effect immediately and will be applicable in all of the European Union.

XTANDI is approved in the European Union for the treatment of adult men with metastatic castration-resistant prostate cancer who are asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated and for the treatment of adult men with metastatic castration-resistant prostate cancer whose disease has progressed on or after docetaxel therapy.

About the TERRAIN trial
The TERRAIN trial enrolled 375 patients in North America and Europe. The trial enrolled patients with metastatic prostate cancer whose disease progressed despite treatment with a LHRH analogue therapy or following surgical castration. The primary endpoint of the trial was PFS, defined as time from randomization to centrally confirmed radiographic progression, skeletal-related event, initiation of new anti-neoplastic therapy or death, whichever occurred first. The trial was designed to evaluate enzalutamide at a dose of 160 mg taken orally once daily versus bicalutamide at a dose of 50 mg taken once daily, the approved dose in combination with an LHRH analogue.

About XTANDI
XTANDI (enzalutamide) capsules is an androgen receptor inhibitor that blocks multiple steps in the androgen receptor signaling pathway within the tumor cell. In preclinical studies, enzalutamide has been shown to competitively inhibit androgen binding to androgen receptors, and inhibit androgen receptor nuclear translocation and interaction with DNA. The clinical significance of this MOA is unknown.
XTANDI is approved by the U.S. Food and Drug Administration for the treatment of patients with metastatic castration-resistant prostate cancer (CRPC).

Important Safety Information
Contraindications XTANDI is not indicated for women and is contraindicated in women who are or may become pregnant. XTANDI can cause fetal harm when administered to a pregnant woman.

Warnings and Precautions
Seizure In Study 1, conducted in patients with metastatic castration-resistant prostate cancer (CRPC) who previously received docetaxel, seizure occurred in 0.9% of XTANDI patients and 0% of placebo patients. In Study 2, conducted in patients with chemotherapy-naive metastatic CRPC, seizure occurred in 0.1% of XTANDI patients and 0.1% of placebo patients. There is no clinical trial experience re-administering XTANDI to patients who experienced a seizure, and limited safety data are available in patients with predisposing factors for seizure. Study 1 excluded the use of concomitant medications that may lower threshold; Study 2 permitted the use of these medications. Because of the risk of seizure associated with XTANDI use, patients should be advised of the risk of engaging in any activity during which sudden loss of consciousness could cause serious harm to themselves or others. Permanently discontinue XTANDI in patients who develop a seizure during treatment.

Posterior Reversible Encephalopathy Syndrome (PRES) In post approval use, there have been reports of PRES in patients receiving XTANDI. PRES is a neurological disorder which can present with rapidly evolving symptoms including seizure, headache, lethargy, confusion, blindness, and other visual and neurological disturbances, with or without associated hypertension. A diagnosis of PRES requires confirmation by brain imaging, preferably MRI. Discontinue XTANDI in patients who develop PRES.

Adverse Reactions
The most common adverse reactions (≥ 10%) reported from two combined clinical studies that occurred more commonly (≥ 2% over placebo) in XTANDI patients were asthenia/fatigue, back pain, decreased appetite, constipation, arthralgia, diarrhea, hot flush, upper respiratory tract infection, peripheral edema, dyspnea, musculoskeletal pain, weight decreased, headache, hypertension, and dizziness/vertigo.

In Study 1, Grade 3 and higher adverse reactions were reported among 47% of XTANDI patients and 53% of placebo patients. Discontinuations due to adverse events were reported for 16% of XTANDI patients and 18% of placebo patients. In Study 2, Grade 3-4 adverse reactions were reported in 44% of XTANDI patients and 37% of placebo patients. Discontinuations due to adverse events were reported for 6% of both study groups.

Lab Abnormalities: Grade 1-4 neutropenia occurred in 15% of XTANDI patients (1% Grade 3-4) and 6% of placebo patients (0.5% Grade 3-4). Grade 1-4 thrombocytopenia occurred in 6% of XTANDI patients (0.3% Grade 3-4) and 5% of placebo patients (0.5% Grade 3-4). Grade 1-4 elevations in ALT occurred in 10% of XTANDI patients (0.2% Grade 3-4) and 16% of placebo patients (0.2% Grade 3-4). Grade 1-4 elevations in bilirubin occurred in 3% of XTANDI patients (0.1% Grade 3-4) and 2% of placebo patients (no Grade 3-4).
Infections: In Study 1, 1% of XTANDI patients compared to 0.3% of placebo patients died from infections or sepsis. In Study 2, 1 patient in each treatment group (0.1%) had an infection resulting in death.
Falls (including fall-related injuries), occurred in 9% of XTANDI patients and 4% of placebo patients. Falls were not associated with loss of consciousness or seizure. Fall-related injuries were more severe in XTANDI patients, and included non-pathologic fractures, joint injuries, and hematomas.
Hypertension occurred in 11% of XTANDI patients and 4% of placebo patients. No patients experienced hypertensive crisis. Medical history of hypertension was balanced between arms. Hypertension led to study discontinuation in < 1% of all patients.
Drug Interactions

Effect of Other Drugs on XTANDI Avoid strong CYP2C8 inhibitors, as they can increase the plasma exposure to XTANDI. If co-administration is necessary, reduce the dose of XTANDI.
Avoid strong CYP3A4 inducers as they can decrease the plasma exposure to XTANDI. If co-administration is necessary, increase the dose of XTANDI.

Effect of XTANDI on Other Drugs Avoid CYP3A4, CYP2C9, and CYP2C19 substrates with a narrow therapeutic index, as XTANDI may decrease the plasma exposures of these drugs. If XTANDI is co-administered with warfarin (CYP2C9 substrate), conduct additional INR monitoring.

For Full Prescribing Information for XTANDI (enzalutamide) capsules, please visit View Source

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Scientists identify possible double whammy to attack leukaemia

On April 7, 2016 Cancer Research UK scientists reported that they have identified a potential double drug combination against B-cell acute lymphoblastic leukaemia, according to a study published in the journal Cell Death and Disease today (Press release, Cancer Research UK, APR 7, 2016, View Source [SID:1234510501]).

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"Cancer cells often outwit us by rewiring themselves, but this early research offers a promising idea to get ahead of them." – Professor Richard Marais
The scientists, from the Cancer Research UK Manchester Institute at the University of Manchester, studied how two drugs called trametinib and ABT-263 work in cancer cells and mice.

Trametinib blocks the MEK/ERK signalling pathway to stop cancer cells from growing out of control. However, when the scientists studied B-cell acute lymphoblastic leukaemia cells in the laboratory trametinib did not work as well as expected and did not stop cells growing.

The scientists found that this was because these cancer cells had high levels of some proteins that help the cells to survive and overcome the effects of the drug.

They therefore decided to test if another drug known as ABT-263 – which targets the survival proteins – could work alongside trametinib to counteract this problem.

Their laboratory experiments in cell lines and mice showed that when they combined these drugs and blocked both signalling pathways, the cells could not escape the effects of trametinib and died.

There are 820 new cases of acute lymphoblastic leukaemia each year in the UK and B-cell acute lymphoblastic leukaemia is the most common type of the disease. More than half of these cases are diagnosed in children.

Professor Richard Marais, lead author and director of the Cancer Research UK Manchester Institute, said: "Cancer cells often outwit us by rewiring themselves, but this early research offers a promising idea to get ahead of them. We’ll still need to do further research to prove that this is the case beyond cancer cells in the laboratory and it may take many years before we see it in the clinic, but it’s the first step to finding a new effective drug combination for B-cell acute lymphoblastic leukaemia."

Dr Áine McCarthy, Cancer Research UK’s science information officer, said: "This research uses our knowledge of the routes cancer cells use to stay alive to find a way to stop them in their tracks. These early steps are crucial for finding new ways to treat a cancer, but there is still much more work needed before we know for certain whether this will help patients."

This work also received funding from the Kay Kendall Leukaemia Fund.