Trade Group Study: Hundreds of Rare Disease Drugs in Development

According to a new report released last month by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical pipeline is innovative and robust, with a high percentage of potential first-in-class medicines (meaning a new treatment where nothing currently exists) targeting diseases with limited treatment options. In addition to identifying medicines in development for conditions and diseases such as septic shock, ovarian cancer, sickle cell disease, and Lou Gehrig’s disease (amyotrophic lateral sclerosis), which haven’t had any new product approvals in the last ten years, the report offers positive news for the rare disease community: one third of the products currently in clinical development have a rare disease designation by the U.S. Food and Drug Administration (FDA).


New ALS Model is Based on Human Cells from Autopsied Tissue

By isolating cells from patients’ spinal tissue within a few days after death, researchers funded by the National Institutes of Health have developed a new model of the paralyzing disease amyotrophic lateral sclerosis (ALS). They found that during the disease, cells called astrocytes become toxic to nerve cells — a result previously found in animal models but not in humans. The new model could be used to investigate many more questions about ALS, also known as Lou Gehrig’s disease.


Biomarker Bulletin: February 7, 2011

Biomarker Bulletin is an occasionally recurring update of news focused on biomarkers aggregated at Biomarkers are physical, functional or biochemical indicators of normal physiological or disease processes. The individualization of disease management — personalized medicine — is dependent on developing biomarkers that promote specific clinical domains, including early detection, risk, diagnosis, prognosis and predicted response to therapy.

Biomarker Commons
  • Prize4Life Awards $1 Million Prize for Discovery of ALS Biomarker

    Prize4Life, a non-profit organization dedicated to accelerating the discovery of a cure for Amyotrophic Lateral Sclerosis (ALS) by offering incentives to drive innovation, today announced that Dr. Seward Rutkove, Chief of the Division of Neuromuscular Disease at Beth Israel Deaconess Medical Center and Associate Professor of Neurology at Harvard Medical School, has received the $1 million dollar Prize4Life award for the discovery of a new ALS biomarker.

  • Rosetta Genomics Signs Agreements to Advance Development of Body Fluids-Based Diagnostic Tests

    Rosetta Genomics has announced that it has signed two new agreements for the development and validation of microRNA-based diagnostics for various indications related to its Gen 3 products. Rosetta Genomics Gen 3 tests focus on cardiovascular indications, neurodegenerative diseases, women’s health and early detection of certain cancers, and are designed to leverage microRNA biomarkers extracted from body fluids.

  • Genomic Health Announces Results of Biomarker Discovery Program Using Next-Generation Sequencing

    Last week, Genomic Health, Inc. announced that its scientists had successfully used Illumina next-generation sequencing technology to survey expression of the whole human transcriptome and test hypotheses for biomarker discovery in archived tumor and normal breast tissue samples.

  • Spinal Muscular Atrophy Foundation and Rules-Based Medicine Collaborate on SMA Biomarker Panel

    The Spinal Muscular Atrophy (SMA) Foundation and Rules-based Medicine (RBM) have reached the first milestone in a program to develop a panel of plasma protein biomarkers for SMA using RBM’s Multi-Analyte Profiling (MAP) technology platform.

  • Computable Genomix Secures Investment for the Development of Genetic Biomarker Tests

    Memphis-based Computable Genomix announced this week that it has secured an investment from venture capital firm Innova Memphis to pilot a novel process for developing genetic biomarker tests. Leveraging its next-generation computational discovery capability, the company is developing highly targeted genetic biomarker tests for clinical researchers.