(Bloomberg) — Thousands of ice bucket dumps later, funds from the ALS Association’s blockbuster charity effort that raised $115 million last summer are going to a drug in late-stage trials for the first time.
Cytokinetics Inc. said it received $1.5 million to help finance testing of an experimental drug to improve lung function in patients with the devastating neurodegenerative disorder. The South San Francisco biotechnology company is the first with a drug at such an advanced stage to get money from the viral fundraising effort, which gathered millions of social media views as it gained supporters ranging from actor Matt Damon to billionaire Bill Gates dumping a bucket of ice on their heads as a charity stunt.
Cytokinetics is starting a phase 3 trial for its compound, called tirasemtiv. Although a mid-stage trial of the drug failed to show overall benefit on what is known as the ALS Functional Rating Score, it showed improvement in maintaining patients’ lung function, said Cytokinetics’ Chief Executive Officer Robert Blum. The current study is designed to confirm this effect and test if tirasemtiv can help delay the need for mechanically assisted ventilation.
The grant from the ALS Association is small compared with the estimated trial costs of about $40 million, said Blum. However, it will enable collection of blood plasma samples from patients to be studied by the Northeastern ALS Consortium Repository, which is looking for biological indicators that can be used to measure disease progression and better understand the underlying causes of the disease.
“There haven’t been large international trials like ours from which to bank samples for academic research,” said Blum. He said the grant is “a symbol of interest and hope from the patient community.”
Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells in the brain and the spinal cord. Patients in later stages may become paralyzed. About 30,000 Americans have the disease, according to the ALS Association.
The ALS Association, a nonprofit organization, estimates that about $77 million of the Ice Bucket Challenge funds raised last year will be used for researching drugs, according to the website. So far, grants have been given to various research initiatives including Project MinE, a gene sequencing effort. Two mid-stage trials, run by the California Pacific Medical Center and the University of Washington Medical Center, have also received funding.
The Cytokinetic study “if successful could have significant impact on patient quality of life,” said Lucie Bruijn, chief scientist of the ALS Association, in an e-mail. The blood plasma sample collections from hundreds of patients in the trial will also benefit many future ALS trials, she said.