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Multiple Myeloma: The View from ASH 2018

Multiple myeloma (MM) is a deadly bone marrow cancer. In recent years, the treatment landscape for this disease has improved considerably. However, most patients still relapse and the need for new and better therapeutic options endures.   Encouraging news about several approaches were reported at the American Society of Hematology (ASH) in December. Results from...

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Neuromuscular Drug Development Summit

At the end of October, we attended the inaugural Neuromuscular Drug Development Summit. This new meeting brought together thought leaders from industry, academia, and the patient advocacy community to discuss scientific progress and issues affecting companies developing drugs for these mostly rare diseases.   Neuromuscular diseases — such as muscular dystrophies, motor neuron diseases, mitochondrial...

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Report on ESMO 2018 – Part Two

We recently returned from the European Society for Medical Oncology (ESMO) meeting where conference news highlighted the increased movement of targeted therapies and immunotherapies into adjuvant and early-stage cancer settings. Read our earlier report on combination therapy trials in melanoma, colon cancer and breast cancer, including triple-negative breast cancer. Here, we cover other targeted therapies....

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Report on ESMO 2018 – Part One

We recently returned from the European Society for Medical Oncology (ESMO) meeting where conference news highlighted the increased movement of targeted therapies and immunotherapies into adjuvant and early-stage cancer settings, plus plenty of positive data from a variety of combination therapy trials in breast cancer, including triple-negative breast cancer.   (Neo)Adjuvant Treatment Gets a Boost  ...

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FDA Issues Set of Guidelines on Gene Therapy

Back in December, the U.S. Food and Drug Administration (FDA) granted its first approval for a gene therapy product, Spark Therapeutics’ Luxturna. According to the Alliance for Regenerative Medicine, as many as 50 more such treatments — addressing a diverse range of health problems from genetic, autoimmune, and heart diseases, to cancer and HIV/AIDS —...

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Steps Toward the Use of Real World Evidence

“Real-world evidence” (RWE) is data on the use and accrued long-term benefits/risks of a drug or device that is gathered outside of a clinical trial — generally after the product has been approved. Clinical trials don’t often reflect the breadth and diversity of patient experience with a particular treatment that can be seen once the...

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Positive Results Reported in Alzheimer’s Disease — But Questions Remain

In early July, Biogen and Eisai announced that analysis of their 18-month Phase 2 trial of BAN2401 revealed encouraging results, showing an effect on both lowering of amyloid levels and slowing of cognitive decline in patients treated with the highest dose of the drug. This spurred some analysts to hail the news as encouraging support...

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New Options for Diabetes Therapy on the Horizon

Research presented in June at the American Diabetes Association (ADA) meeting highlighted potential advances for diabetes patients who use drugs in a class called incretin mimetics –specifically GLP-1 (glucagon-like peptide 1) agonists — to help control their blood sugar. Such drugs work by increasing the secretion of insulin from the pancreas, inhibiting glucagon (a hormone...

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Precision Medicine and NGS Cancer Panels — ASCO Round-Up Part Two

As we wrote in our last post, precision medicine in oncology was the big theme of this year’s ASCO meeting. One of the key advances that has made possible this focus on treating cancer based on particular biomarkers expressed by a patient’s tumors, has been the advent of Next-Generation Sequencing (NGS)-based analysis of tumor DNA....

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First Up For The New FDA RMAT Designation: Gene Therapeutics for Hemophilia

We recently wrote about the U.S. Food and Drug Administration’s (FDA) creation of a Regenerative Medicine Advanced Therapy (RMAT) designation in an effort to speed up the approval process for certain types of treatments, including gene therapies. The agency has specifically chosen gene therapy for hemophilia as its first focus, due to the considerable effort...