Mallinckrodt is permanently halting its Phase 2b PENNANT Study (MNK14042068) investigating the safety and efficacy of a hormone injection for the treatment of ALS. Acthar Gel is a highly purified preparation of Adrenocorticotropic hormone (ACTH) injected either beneath the skin or into the muscle. It is approved by the FDA for the treatment of 19 indications including acute exacerbations of multiple sclerosis in adults.

I call ALS “the nice guys’ disease.” For, as indiscriminate as it appears, ALS certainly has a way of finding the best and most courageous people to attack. Watching people endure the wasting of their muscle control and seeing them defiant in the face of the sure and steady loss of communication and independence, makes me defiant too. People with ALS fight the disease with great courage. Seeing the fire and determination of my patients inspires me every day. Our team puts that inspiration to work in the clinic and in my lab, where we are on a promising path to finding effective therapies to slow ALS.

Furthering our understanding of disease and the creation of effective therapies won’t happen in a vacuum. My peers and colleagues in the medical research community know first-hand the necessity of global collaborations that bring diverse specialties into the process of understanding complex medical mysteries such as ALS.

Watching my mother struggle to interact with the world as her ALS progressed was extremely difficult for our family, and for me personally. Her struggle inspired me to find solutions that could help people like my mother better interact with computers and their environment, even after they have lost almost all of their ability to move. I had the motivation to advance technology in a way that would bring a new quality of life to people living with ALS under Steve Saling's motto that until medicine proves otherwise, technology is the cure.

In 2016, my colleagues and I published some remarkable results of a new drug called CuATSM in a mouse model of ALS. Moving CuATSM from mice to humans is a long and difficult road that involves the cooperation and scrutiny of many people. The checks and balances are critical to balance the risks with the benefits of any new therapy.

There was a lot of skepticism in the research community when we proposed development of designer DNA drugs as a way to treat ALS. This therapy is an approach that uses DNA-based designer drugs to "silence" genes that are known to cause a particular disease. The ALS Association was the first funding organization to invest in designer DNA technology research and development for therapy in neurodegenerative disease.

Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows.

Biogen has initiated a phase 3 clinical trial evaluating tofersen (previously called BIIB067), an antisense oligonucleotide (ASO), a type of antisense drug, targeting superoxide dismutase (SOD1), for the potential treatment of ALS. The trial is now enrolling and aims to enroll approximately 60 people with SOD1 ALS.

The ALS Association is proud to be a longtime supporter of the Airlie House ALS Clinical Trials Consensus Guidelines, which have been revised and published in Neurology, the most widely read and highly cited peer-reviewed neurology journal.

Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows. The awards were founded in memory of Mr. Safenowitz by the Safenowitz family – through The ALS Association Greater New York Chapter. The program encourages young scientists to enter and, importantly, to remain in the ALS field.

Next week at the American Academy of Neurology Meeting (AAN) in Philadelphia, Biogen will present promising results of the phase 1/2 study of its newly named investigational therapy tofersen (previously BIIB067), which is now enrolling in a phase 3 trial. Tofersen is an antisense oligonucleotide (ASO), a type of designer DNA drug, targeting SOD1.

Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows. The awards were founded in memory of Mr. Safenowitz by the Safenowitz family – through The ALS Association Greater New York Chapter. The program encourages young scientists to enter and, importantly, to remain in the ALS field.

Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows. The awards were founded in memory of Mr. Safenowitz by the Safenowitz family – through The ALS Association Greater New York Chapter. The program encourages young scientists to enter and, importantly, to remain in the ALS field.

Every two years, hundreds of ALS health care professionals come together to discuss new trends, share best practices and guidelines, exchange ideas and successes, and talk about challenges and potential solutions. To capture the collaborative power of the 2018 event in Fort Worth, Texas – our largest-ever Clinical Conference, with more than 500 attendees – we put together a short video highlight reel.

As assistant professor of Neuroscience at the Mayo Clinic in Jacksonville, Fla., I fight ALS by working every day in the lab to find a cure for this devastating disease. My work focuses on optimizing ALS biomarkers to track and better understand the most common genetic mutation in inherited ALS, called C9orf72.

In 2018, many new research discoveries and collaborations accelerated the momentum toward finding treatments and a cure for ALS. We helped lead the way by awarding new grants to top scientists and clinicians all over the world.

The ALS Association is proud to be the first investor in antisense technology, dating back to 2004 when antisense was just an idea in Dr. Don Cleveland’s lab at University of California San Diego (UCSD). Fast forward to 2018 and we are seeing promising results in antisense drugs targeting the two most common causes of inherited ALS, mutations in the SOD1 and C9orf72 genes.

Dr. Brian Wainger from the Massachusetts General Hospital (MGH) presented initial top-level results from a recently completed phase II clinical trial of ezogabine (retigabine) on motor neuron excitability (NCT02450552). The study, supported by The ALS Association, met its main goal of quantifying a reduction in motor neuron excitability in people with ALS following treatment. Results were presented during the 29th International Symposium on ALS/MND in Glasgow, Scotland, last week.

In a promising new study by Drs. Robert Brown and Christian Mueller at the University of Massachusetts Medical School report that a type of viral gene therapy using synthetic microRNAs (miRNAs) targeting the ALS SOD1 gene is safe and effective in nonhuman primate macaques (monkeys). The ALS Association provided $1.7 million in funding for this study, which demonstrated an efficient reduction of the SOD1 protein without side effects. This paves the way forward for further development of this potential therapy.

Mitsubishi Tanabe Pharma America (MTPA) will present initial data on efforts to create an oral version of edaravone, a key drug in the treatment of ALS that is currently only available intravenously, during the International Symposium on ALS/MND in Glasgow, Scotland. The symposium will be held Dec. 7-9. MTPA is expected to present results that demonstrate that oral edaravone is processed in the body in the same manner as the infused formulation.