Target ALS has recently launched a new research initiative, in which they will dedicate $15 million to amyotrophic lateral sclerosis (ALS) research, specifically the discovery of biomarkers. These millions will be allocated towards grants and resources that will help to find new biomarkers, as they could aid in diagnosis and monitoring disease progression.
About ALS
Amyotrophic lateral sclerosis (ALS) is a progressive, neurological disease in which nerve cells in the brain stem, brain, and spinal cord deteriorate. Due to this deterioration, muscles weaken and people lose control of them and their voluntary movement. In the late stages of ALS, the muscles necessary for breathing weaken, resulting in death. There are two forms of this disease: sporadic and familial. The former is the most common, with 90-95% of cases falling into this category. In the familial form of the disease, it is known that a mutated gene is inherited from parents, but it is still not fully understood and only accounts for 5-10% of cases. Otherwise, the cause of ALS is unknown. Researchers believe that there is a connection between frontotemporal dementia and ALS. Another theory is that exposure to certain substances or toxins leads to the development of ALS.
Symptoms of ALS vary between individuals. They also worsen as the disease progresses. Symptoms begin with difficulty with small movements and everyday things like walking. At the onset of the disease, people may trip and feel weakness in their arms, hands, and legs. As it progresses, people experience difficulties with speaking and swallowing, slowed and slurred speech, twitches and cramps in the muscles, and difficulty holding good posture. In the later stages people will be unable to move their muscles gradually, which affects the entire body. This inability affects movements like blinking. While people with ALS experience loss of muscle function, they do not lose any of their cognitive abilities.
Target ALS
Target ALS Diagnosis Initiative is the first effort of its kind, created with input from medical professionals, patients, stakeholders, and caregivers. Its aim is to create better diagnostic methods for ALS, along with improvements in treatment. In addition to this $15 million effort, they are also running a five-year natural history study and constructing an integrated Biosamples and Genomics Core.
The $15M Effort
This money will go towards the discovery of biomarkers in ALS. At least 30 researchers coming from nonprofit, pharmaceutical, and academic backgrounds will work to find, validate, and develop biomarkers. Their research will build on previous work, as Target ALS funded biomarker research back in 2017.
Some of this money will also go towards competitive research. Target ALS will accept grant proposals, with the only condition being collaboration between an academic and the pharmaceutical or biotechnology fields.
In order to ensure that these researchers can do their best work, some of the $15 million will go towards longitudinal fluid samples. These samples are often necessary for ALS research but are often inaccessible. To combat this problem, Target ALS will provide researchers with samples. They will do the same with other resources.
The Biosamples and Genomics Core is an extension of the resources offered. Six core facilities will be established, all of which will provide resources to researchers. Examples include human-derived stem cells, datasets, and viral vectors.
Hopefully this initiative will discover ALS biomarkers, as it would greatly improve the diagnostic and treatment processes for patients.
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