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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...

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New Gains in Frontline Non-Small Cell Lung Cancer

Each spring, those of us who watch the oncology space turn our attention to two big U.S. meetings for the latest research news: the American Association of Cancer Research (AACR) annual scientific meeting, held in April, and the American Society of Clinical Oncology (ASCO), which will take place in June. AACR previously focused primarily on...

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Alzheimer’s Disease: Continued Clinical Failure and a Potential New Development Pathway

Alzheimer’s disease continues to be a costly and frustrating minefield for drug developers. Most recently, Boehringer Ingelheim halted development of the phosphodiesterase type 9 (PDE9) inhibitor BI 409306 after it failed in phase 2. Merck also ended its trial of verubecestat, the company’s beta-secretase-1 (BACE1) inhibitor, in early stage Alzheimer’s disease patients for futility, a few...

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Celebrating Clinical Success in Depression

Depression is a difficult problem for drug developers. Current marketed antidepressants are effective for a relatively small percentage of patients (about 20 in 100) and most require weeks of treatment to show an effect. Patients often need to try several drugs to find one that works for them, and some patients do not respond to...

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A First Glimmer of Hope for Huntington’s Disease Patients

Huntington’s disease is a devastating and progressive inherited disorder that is caused by the expansion of the CAG trinucleotide sequence in the huntingtin (HTT) gene. This mutation results in abnormally long versions of the HTT protein, which progressively destroy neurons in the brain. Those affected experience a gradual loss of mental function and physical control...