Given a Death Sentence, ALS Patients Wait for Potential Disease Cure

On Aug. 5, 2014, Scott Hayes of Wilson, N.C. received some of the worst news imaginable. He was diagnosed with amyotrophic lateral sclerosis (ALS). He was given three to five years to live by his doctors.

Hayes had been a vigorous and hearty man, but the disease wore his body down. His legs were no longer able to support him and he was soon confined to a wheelchair. His body continued to deteriorate and he lost the strength in his arms, and began to experience a wide variety of difficulties as the disease ran roughshod over his body. Sonja Hayes, Scott’s wife of many years, carried him to countless doctor’s appointments as they vainly tried to find something that would stop the progression of the disease. Nothing worked to stave off the effects of the disease, which is also commonly called Lou Gehrig’s disease. Ultimately the disease won, and Scott lost his fight with the disease on July 3, nearly four years after his initial diagnosis. He was 53 years old when he died.

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects neurons in the brain and the spinal cord. The disease is usually fatal within two to five years of diagnosis. Patients with ALS lose the ability to control muscle movement, which eventually leads to total paralysis and then death. Currently, there is no known cure for the disease. The Centers for Disease Control and Prevention (CDC) estimates that approximately 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 diagnosed annually. 

While there is no cure for the disease, multiple companies are working on treatments, hoping to be the first to provide a viable option for patients like Scott Hayes. There are some treatments that may provide hope for patients.

Last year, Mitsubishi Tanabe and its new subsidiary, MT Pharma America, secured regulatory approval for Radicava, the first ALS drug approved in 22 years. Radicava is not a cure for ALS, but slows decline in the daily functioning of ALS patients. The drug was approved based on Phase III clinical trials that demonstrated patients taking the drug had a slower decline in physical function than those taking the placebo. Data demonstrated patients who received Radicava for six months experienced significantly less decline in physical function — by 33 percent.

Since its approval, Kevin O’Brien, MTPA’s vice president of market access, told BioSpace that the company has had some hurdles to overcome since approval of Radicava. Issues have included insurance hurdles and the establishment of infusion centers across the U.S.

In addition to Radicava, there is one other medication approved by the U.S. Food and Drug Administration (FDA) for ALS – Sanofi’s Rilutek (riluzole). It was the first drug approved by the FDA to treat ALS. Like Radicava, it’s no cure for ALS, but can slow progression of the disease.

One treatment currently in development that is generating a lot of buzz is BrainStorm’s NurOwn. Israel-based BrainStorm’s treatment has the potential to be the first ALS therapy to improve patient functioning as a regenerative medicine. Similar to CAR-T therapies, BrainStorm’s NurOwn uses a patient's own cells which have been engineered outside the body, to produce and secrete factors known to promote neuronal survival. BrainStorm’s experimental treatment was one of the drugs that was a driver in the passage of the federal Right-to-Try legislation. Although not yet approved, some ALS patients hoped the treatment would be made available to them outside of the clinical trial parameters as a potential treatment. That hope was based on Phase II trials that showed NurOwn slowed the progression of the disease following multiple dosings. The Phase III trial is expected to be completed in July 2019, according to the government’s clinical trials database.

Mitsubishi Tanabe, the maker of Radicava, teamed up with Amgen to back the launch of Cambridge, Mass.-based QurAlis, which is developing three therapeutics targeting subsets of ALS. The company is developing treatments that include: a transformative device to remove toxic proteins; a drug that mediates overactive neurons and prevents them from dying; and a drug that restores a dysfunctional waste clearance system in cells.

While some pipeline treatments show potential for patients, some experimental drugs have not lived up to hope. Case in point, last year South San Francisco-based Cytokinetics scrapped a late-stage ALS drug after it flunked a Phase III trial. Tirasemtiv failed in the Phase III VITALITY-ALS trial to differentiate itself from placebo in a statistically significant manner. Where tirasemtiv failed, Cytokinetics suggested last year that its fast skeletal muscle activator, CK-2127107 (reldesemtiv), could be a potential treatment for ALS. Phase II results are expected this year.   

As drug companies continue to develop potential treatments and one day, a hopeful cure, ALS diagnoses are expected to increase. The number of diagnoses is expected to grow globally more than 69 percent by 2040 to an expected 376,674 patients.