After being diagnosed with ALS, many people wonder: “Why did this happen to me?” There is no simple answer to this question because the exact cause of ALS is largely unknown. However, a number of factors, including genetics, environmental exposures, and lifestyle/occupational choices, have been linked to an increased risk of developing the disease.
Biomarkers (short for “biological markers”) are genes, proteins, or other substances or characteristics health care professionals can test for or measure. They can be found in blood, cerebrospinal fluid (CSF), other body fluids, or tissues and are extremely important for diagnosing and managing a wide range of diseases.
Clinical studies funded by the Association in the TREAT ALS™ research program include both clinical management projects that improve people’s lives in the present and clinical trials that hold promise in the future – for people living with ALS, caregivers and loved ones. Both are extremely important in the fight against ALS.
Stem cells have the ability to divide for indefinite periods in culture and give rise to multiple specialized cell types. They can develop into blood, neurons, bone, muscle, skin and other cell types. They have emerged as a major tool for research into the causes of ALS, and in the search of new treatments.
Amyotrophic lateral sclerosis (ALS) attacks motor neurons (a type of nerve cell) in the brain and spinal cord. There are two types of motor neurons: upper motor neurons (UMNs) that send nerve fibers down the from the motor cortex in the brain (part of the brain that controls movement) to the spinal cord; lower motor neurons (LMNs) send nerve fibers from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed.
The drug development process takes years to complete its many steps and is costly – $1-3 billion dollars – to move a basic research idea all the way through clinical trials and FDA approval. Collaboration is essential. Researchers from all over the world in academia, government, non-profit and industry work together to move along the pathway of drug development as rapidly and efficiently as possible.
Research has shown that about two out of three of people with familial ALS and about one out of 10 people with sporadic ALS have a mutation (or change) in at least one of the more than 40 genes linked to the disease.
The ALS Association is pleased to announce the continuation of its national collaboration with Mitsubishi Tanabe Pharma America, Inc. (MTPA), which has resulted in significant support of the Walk to Defeat ALS program, various national conferences and events, and an extensive care grant distributed to The ALS Association chapter network.
Cytokinetics, Incorporated (Nasdaq:CYTK) and The ALS Association today announced the continuation of their partnership in the fight against ALS. Cytokinetics is renewing its Gold Level Sponsorship of the National Walks to Defeat ALS® and Premier Level National ALS Advocacy Conference Sponsorship as well as Platinum Level Sponsorship for initiatives led by The ALS Association Golden West Chapter, including grant funding for care services for people with ALS in the Bay Area.
An independent research organization reported that donations from the 2014 ALS Ice Bucket Challenge enabled The ALS Association to increase its annual funding for research around the world by 187 percent. During this time, ALS researchers made scientific advances, care for people living with ALS expanded and investment in disease research from the federal government grew.