Genentech, a member of the Roche group, has initiated a Phase I clinical study evaluating GDC-0134, an oral investigational medicine designed to block dual leucine zipper kinase (DLK), for the potential treatment of ALS. The trial is enrolling now in sites across the United States.

DLK is an enzyme found in nerve cells of the brain and spinal cord. Activation of DLK may cause nerve cells to die when they come under stress. Blocking DLK may be able to prevent motor neurons from dying in people with ALS. Preclinical data shows that blocking or removing DLK is beneficial in a variety of animal models, including a superoxide dismutase 1 (SOD1) mouse model. Mutations in the SOD1 gene are the second most common cause of inherited ALS.

Genentech is conducting a double-blind, randomized, placebo-controlled dose escalation Phase 1 clinical study that will explore the safety and tolerability of multiple-ascending-doses of GDC-0134 in people with ALS. Trial participates will receive GDC-0134 or placebo once daily for 28 days. Genentech is aiming to enroll approximately 70 participants across 9 study centers in the United States (see below).

“We are enthusiastic that Genentech is dedicated to finding a treatment for ALS.” -Dr. Lucie Bruijn, Chief Scientist, The ALS Association

Genentech is working to discover and develop transformative medicines for people with major diseases of the nervous system, such as ALS and Alzheimer’s disease and DLK may play an important role in both.

To learn more about DLK, please see, “Square One: Understanding Neurodegeneration.”

To learn more about the trial, please visit the Northeast ALS Consortium clinical trial page and the clinicaltrials.gov study page.

For questions, please contact Genentech’s Clinical Trial Information Support Line at (888) 662-6728.

The following clinical trial sites are located in the United States:

California: Forbes Norris ALS Research Center, San Francisco

Florida: Mayo Clinic Hospital, Jacksonville; University of Miami Miller School of Medicine, Miami; Compass Research, Orlando

Georgia: The Emory ALS Clinic, Atlanta

Maryland: Johns Hopkins University School of Medicine, Baltimore

Massachusetts: Massachusetts General Hospital, Boston

North Carolina: Wake Research Associates, Raleigh

Tennessee: New Orleans Center for Clinical Research, Knoxville