FDA Public Workshops on Rare Childhood Diseases

Jennifer Inman-Cartwright emailed me today saying that she sat in on a conference call with the Children's Cancer Caucus - Advocacy (CCCA) where the topic was the FDA and orphan drug issues. She forward the slide presentations which you can view and download. The first presentation was by James E. Valentine, MHS, JD Candidate at…
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Early Life Factors and Risk of Childhood Rhabdomyosarcoma

By: Anshu Shrestha, Beate Ritz, Simona Ognjanovic, Christina A. Lombardi, Michelle Wilhelm and Julia E. Heck Frontiers in Public Health, 31 May 2013 - Although little is known about etiology of childhood rhabdomyosarcoma (RMS), early life factors are suspected in the etiology. We explored this hypothesis using linked data from the California Cancer Registry and…
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CytRx Reports Highly Statistically Significant Positive Results from its Global Phase 2b Clinical Trial

CytRx, a biopharmaceutical research and development company specializing in oncology, today announced highly positive top-line efficacy results from a multicenter, randomized, open-label global Phase 2b clinical trial. The trial investigated the efficacy and safety of aldoxorubicin compared with doxorubicin in subjects with first-line metastatic, locally advanced or unresectable soft tissue sarcomas (STS). Aldoxorubicin combines the…
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Gene Sequencing Project Finds Family of Drugs with Promise for Treating Childhood Tumor

(MEMPHIS, Tenn. – December 9, 2013) Drugs that enhance a process called oxidative stress were found to kill rhabdomyosarcoma tumor cells growing in the laboratory and possibly bolstered the effectiveness of chemotherapy against this aggressive tumor of muscle and other soft tissue. The findings are the latest from the St. Jude Children's Research Hospital–Washington University…
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Safety Study of Intratumoral Injection of Clostridium Novyi-NT Spores to Treat Patients With Solid Tumors That Have Not Responded to Standard Therapies

Reposted from Facebook via Mimi Olsson For more information on this study - According to the website, the trial is currently recruiting participants. On November 19, at MD Anderson Cancer Hospital in Houston, Texas I became the first human being in history to receive cancer treatment by injecting a bacteria (clostridium novi ) directly into…
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Important: Request from NIH/NCI for Public Input on Availability of Clinical Trial Results

Purpose With this Notice, the National Cancer Institute (NCI) announces and seeks public input on NCI’s plan to promote and ensure public availability of results from all commenced, NCI-supported clinical trials. Background NCI, a component of the National Institutes of Health (NIH), is dedicated to improving the health of Americans by conducting and funding biomedical…
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Tips for Interpreting Scientific Claims

Here's the link to an article that talks about ways to help non-scientists interrograte advisers and to grasp the limitations of evidence.  Published in the Nov 20, 2013 issue of Nature, the article suggests 20 concepts that should be part of the education of anyone who may have to interact with science or scientists.  Although…
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Loss of MEF2D Expression Inhibits Differentiation and Contributes to Oncogenesis in Rhabdomyosarcoma Cells

From Molecular Cancer: Rhabdomyosarcoma (RMS) is a highly malignant pediatric cancer that is the most common form of soft tissue tumors in children. RMS cells have many features of skeletal muscle cells, yet do not differentiate. Thus, our studies have focused on the defects present in these cells that block myogenesis. Methods: Protein and RNA analysis…
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Rb1 Loss Modifies but does not Initiate Alveolar Rhabdomyosarcoma

This article, published in Skeletal Muscle Journal by lead authors Ken Kikuchi , Eri Taniguchi and colleagues,  investigates the complementary role of Rb1 loss in aRMS tumor initiation and progression using conditional mouse models. From the article's abstract: Results Rb1 loss was not a necessary and sufficient mutational event for rhabdomyosarcomagenesis, nor a strong cooperative initiating mutation. Instead,…
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