Advancements in science and precision medicine mean new hope for patients
Monday, July 13, 2015
Washington, D.C. — America’s biopharmaceutical research companies are currently developing 420 medicines for patients suffering from neurological disorders, including epilepsy, Alzheimer’s disease, multiple sclerosis (MS) and Parkinson’s disease. As highlighted in a new report from the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Epilepsy Foundation, scientists around the globe are collaborating to find new or more effective treatments for patients with these complex disorders that attack the nervous system.
“Researchers have made tremendous advances in understanding how the nervous system works at the molecular and genetic levels which in turn has translated into the development of more effective treatments for neurological disorders,” said John J. Castellani, president and chief executive officer, PhRMA. “These developments mean new options and new hope for the millions of patients suffering from these devastating disorders. As an industry, we remain steadfast in our efforts to unlock the mysteries of these diseases and reduce their burden for generations to come.”
Neurological disorders consist of more than 600 conditions, impacting an estimated 50 million Americans every year. Although some are well known, many are rare disorders that affect a small number of patients who currently have access to few – if any – effective treatments.
The new report, “Medicines in Development for Neurological Disorders,” demonstrates how the newest advancements seek to treat the underlying mechanisms of neurological disorders. Examples of treatments currently in development include:
- A monoclonal antibody that inhibits the activity of calcitonin gene-related peptide (CGRP). Research suggests that CGRP pathways may be involved in the development of migraines and by inhibiting CGRP activity, anti-CGRP antibodies help inhibit the transmission of pain signals associated with migraines.
- A medicine for Huntington’s disease that targets an enzyme, PDE10A, which is present in the neurons most damaged in this inherited disorder that causes a progressive breakdown of nerve cells in the brain.
- A monoclonal antibody for amyotrophic lateral sclerosis (ALS), which is an inhibitor of the protein Nogo-A that may help keep the motor neurons and muscle fibers connected in hopes that it will lead to a slowing or stopping of disease progression.
- A monoclonal antibody for relapsing MS, which targets a protein involved in the development of myelin, a protective sheath covering the nerve fibers. This medicine could help restore nerve communication in MS patients.
“As a parent of a child living with epilepsy, I know just how important innovation is to improving the quality of life for individuals living with seizures,” said Philip M. Gattone, president and CEO of the Epilepsy Foundation. “Innovative new therapies are particularly important for the one-third of people with epilepsy who don’t have their seizures under control, as well as the millions of other Americans living with chronic conditions that cannot be managed by current treatment options.”
With 420 medicines in human clinical trials or under review by the Food and Drug Administration (FDA), these new treatments represent a growing understanding of neurological disorders, fueled by both successes and setbacks in research, which will ultimately provide patients and their families with better options.
The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives. Since 2000, PhRMA member companies have invested more than $600 billion in the search for new treatments and cures, including an estimated $51.2 billion in 2014 alone.