Clinical trial starts for prospective ALS treatment co-developed at Scripps Research Experimental drug 3K3A-APC has shown broad brain-protecting effects in animal studies.
December 17, 2021
LA JOLLA, CA An experimental treatment called 3K3A-APC, which was co-developed at Scripps Research and has shown promise against amyotrophic lateral sclerosis (ALS) in preclinical studies, is now being tested in patients with the disease.
The clinical trial, at Macquarie University in Sydney, Australia, will enroll a total of 16 ALS patients, and aims to provide evidence of the experimental treatment's safety and effectiveness against ALS a progressive neurodegenerative disease that is usually fatal and has no cure.
Often called Lou Gehrig's disease (after the famous baseball player who died of it in 1941), ALS is estimated to afflict up to 20,000 people in the United States at any one time. The disorder features the progressive deaths of muscle-controlling neurons, causing weakness and paralysis in muscle groups and ultimately leading to respiratory failure and death. Despite decades of intensive research, scientists still have not identified the precise causes of ALS in its most common, non-inherited form, and, largely for that reason, there is no Food and Drug Administration (FDA)-approved treatment that markedly improves this condition.
I'm very hopeful that 3K3A-APC will benefit these patients in preclinical tests it has displayed remarkable anti-inflammatory, anti-programmed-cell-death, and other cell-stabilizing activities, says John Griffin, PhD, a professor in the Department of Molecular Medicine at Scripps Research whose lab invented 3K3A-APC.
Griffin initially developed and studied 3K3A-APC with University of Southern California (USC) researcher Berislav Zlokovic, MD, PhD. Zlokovic co-founded Houston-based ZZ Biotech, which has taken 3K3A-APC through advanced preclinical and early clinical development and which provides it for this ALS trial.
The 3K3A-APC drug is a modified version of a natural human protein, activated protein C (APC), which works as a powerful anticoagulant and also has neuroprotective properties. To minimize its chances of causing brain bleeding when used as a treatment, Griffin's lab altered APC in a way that removes 90 percent of its anticoagulant effect without removing its other neuroprotective properties.
Preclinical studies by the researchers and their colleagues have found that 3K3A-APC slows disease progression and extends survival in a commonly used mouse model of ALS. The protein appears to have this effect by delaying the activation of inflammation-promoting immune cells in the brain and spinal cord. The molecule also restores the integrity of the special blood-vessel-lining cells that protect the brain and spinal cord from immune cells and other potentially harmful factors in the blood.
The new clinical trial is being led by neurology Professor Dominic Rowe, MD, PhD, at the Macquarie University Centre for Motor Neuron Disease Research, one of the leading ALS research institutions in Australia. The trial is a Phase 2 open label trial (NCT05039268) that will assign 16 ALS patients to one of two doses of the treatment. The principal goal is to demonstrate the safety and tolerability of 3K3A-APC in ALS patients, although the results may also provide preliminary evidence that the treatment slows the ALS disease process.
Translational Medicine Griffin, John
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Clinical trial starts for prospective ALS treatment co-developed at Scripps Research Experimental drug 3K3A-APC has shown broad brain-protecting effects in animal studies. 
