According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis (ALS). The new research grants have a total value of over $2 million – an impressive gesture from the non-profit that was a partial celebration of the upcoming ALS Awareness Month that comes every May.

About Amyotrophic Lateral Sclerosis

This disease is known by a few different names: ALS, MND (motor neuron disease), the less-formal “Lou Gehrig’s disease.” However, all of these describe the same group of rare neurological conditions that affect the nerve cells responsible for transmitting signals controlling voluntary movement.

The course of ALS is progressive, meaning individuals will likely at first experience mild symptoms that later advance into more serious stages of the disease. Early symptoms can include muscle cramping, slurred speech, and difficulty chewing, among other things. Over time, patients will progressively lose motor function until things like walking, standing, and eventually breathing, become impossible when unassisted.

Though roughly 10% of ALS cases are familial (genetically inherited), the other 90% are “sporadic,” appearing in individuals with no family history of the disease. There is little consensus about what forces drive these sporadic cases, but genetic mutations, exposure to toxic or infectious agents, and even diet might play contributing roles in the development of ALS.

About the Grants

The eight grants went out to groups of researchers approaching the study and treatment of ALS in vastly different ways:

1. Jonathan Glass, MD – $151,592 – Glass represented the NEALS Consortium – a group of over 100 research institutions that collaborate on ALS research. The grant is meant to fund the group’s repository of sample tissues and fluids, as well as their annual conference.
2. Csaba Konrad, PhD – $210,000 – Konrad’s grant will go towards the study of improved biomarkers that allow the identification of ALS and predict its likely progression.
3. Ze’ev Melamed, PhD – $210,000 – Melamed’s grant will aid research determining if stathmin-2 protein therapy has potential in treating ALS. Recently this protein was found to be in reduced levels in the neurons of sporadic ALS patients. Melamed’s research hopes to determine if countering this deficiency could counter progression of the disease.
4. Tania Gendron, PhD – $285,000 – Gendron’s grant will be used in research aiming to find a reliable biomarker for cognitive impairment in the blood and spinal fluid of ALS patients. Since only some people with ALS develop cognitive impairments, being able to identify and treat them early could lead to significant improvement in their outcome.
5. Junjie Guo, PhD – $297,678 – Guo’s grant will fund research to determine the nature of potentially toxic clumps of RNA called “foci,” as well as any potential links to foci and the development of ALS.
6. John Landers, PhD – $300,000 – Landers’ grant will go towards RNA research that hopefully identifies targets for potential pharmaceutical treatment.
7. Thomas Gaj, PhD – $300,000 – A mutation to the gene SOD1 is thought to cause about 2% of all cases of ALS. With his grant, Gaj and his team will study if gene editing techniques prove effective in treating cases of ALS linked to the mutation.
8. Marka Van Blitterswijk, MD, PhD and Mark Ebbert, PhD – $300,000 – The duo’s grant money will fund research into the C9ORF72 gene mutation associated with many genetic instances of ALS. The researchers hope to determine if the length of the mutation in an individual could assist in predicting survival time and possible cognitive changes.

Do you think events like ALS Awareness Month are important for generating research? Will you be participating this year? Patient Worthy wants to hear from you!