July 14, 2006

Research Update — from ALSA’s National Office

Enrollment Begins in The ALS Association’s TREAT ALS Combination Trial

Roberta Friedman, Ph.D., ALSA Research Department Information Coordinator

Patients with ALS can now volunteer to participate in a trial of combined, investigational therapies funded through the new initiative of The ALS Association TREAT ALS (Translational Research Advancing Therapy for ALS). Twenty centers are participating in this Phase II trial which seeks which combination might be effective in amyotrophic lateral sclerosis (ALS, also called Lou Gehrig’s disease).

TREAT ALS is a drug discovery program and clinical trials process that accelerates discovery and testing of clinical candidates.

Each of the combinations to be tested has prolonged survival in rodent models of the disease to a greater degree than any of the compounds used alone. If neither combination turns out to provide beneficial effects in ALS, the study investigators will be able to decide this by enrolling just 120 patients. This streamlined trial should therefore serve as a model for future studies to speed decisions about clinical candidates for ALS.

If one or both should produce a slower decline in function compared to a historic control group, further testing will proceed under an expanded, Phase III protocol with a placebo control.

“This study provides an excellent example of how TREAT ALS seeks to hasten the pace of clinical discovery in ALS,” said Lucie Bruijn, Ph.D., The Association’s science director and vice president.

Minocycline plus creatine and celecoxib plus creatine are the only two drug combinations so far shown by published reports to have additive effects in the mouse model of the disease. Since each of these is an already marketed compound, safety in people as well as beneficial effects in animal models of ALS is already known.

Despite the lack of definitive cause for most cases of ALS, multiple processes have been shown to promote nerve cell death. These drugs to be tested affect different aspects of the processes that may lead to the demise of motor neurons, including the inflammation that appears during the disease.  Click here for more information on inflammation.

Markers of disease progression that the investigators will use include the ALS Functional Rating Scale and a measure of respiratory function. These surrogate markers will assess how patients progress with the treatments. Comparison to historic controls from prior trials will also streamline and accelerate the study—all patients volunteering will receive drugs and not a placebo. The only other drug patients are allowed to use while on the trial is riluzole, the sole approved treatment for ALS.  Trial participants will have had ALS onset within the past 5 years. Breathing capacity must be at a certain level to participate: Forced Vital Capacity (FVC) must be above 60%.

There is no cure for ALS. It is only through well-designed clinical trials that more effective treatments will be found. Clinical trials are entirely dependent upon the participation of patients with ALS.

For more information about the trial contact:

Carolyn Doorish, Project Coordinator

E-mail: cd2141@columbia.edu

Phone: (212) 305-2027

Study centers are listed below.

For further details about clinical trials, click here.

 

Combination Trial Questions & Answers

1.      How can I learn more about the clinical trial and find out about enrolling?

The Principal Investigator of the clinical grant trial is Dr. Paul Gordon at Eleanor and Lou Gehrig MDA/ALS Research Center at Columbia University Medical Center.  For more information, contact Carolyn Doorish, the Project Coordinator at 212.305.2027 or cd2141@columbia.edu.

2.      Where is the study being conducted?

There are 20 centers nationally that are involved in the study which are listed below.

3.      What is the length of the study?

The study will last between six to seven months.

4.      Should I ask my doctor to prescribe one of these combinations for me?

No, the clinical trail is being conducted to answer the question of whether or not the combination drugs have a positive effect for people with ALS.  The information published and described in The ALS Association’s update shown above is experimental.

5.      How many people will be enrolled in this phase of the study?

The number of enrollees is 120 for this phase.

6.      Will I be able to be assured that I will receive one of the combinations of the two drugs if I am enrolled in the study?

During Phase II of the study, all enrollees will receive one or the other of the drug combinations, which will be either minocycline plus creatine and celecoxib plus creatine.  Patients will be randomly selected to receive one of these combinations.

7.      When will enrollment begin?

Patients can now volunteer to participate in the study.

8.      Do I have to go to the study site to be enrolled in the study? 

Yes, site visits are required.

Study centers include:

FACILITY	CONTACT
Beth Israel Medical Center, NY, NY	Theresa Imperato	imperato@als-ny.org 212-844-8490
Columbia University Medical Center, NY, NY	Carolyn Doorish	Cd2141@columbia.edu 212-305-2027
California Pacific Medical Center, San Francisco, CA	Terence Santos	santost@sutterhealth.org 415-600-3758
Duke University Medical Center, Durham, NC	Karen Grace	karen.grace@duke.edu 919-668-2844
Mayo Clinic Jacksonville, Jacksonville, FL	Anne Evans	Evans.anne@mayo.edu 904-953-7720
Mayo Clinic Rochester, Rochester, MN	Cindy Stoppel	stoppel.cynthia@mayo.edu 507-266-0761
Medical College of Georgia, Augusta, GA	Meledy Kise	mkise@mail.mcg.edu 706-721-2681
Oregon Health and Science University School of Medicine, Portland, OR	Sarah Johnson	johnssar@ohsu.edu 503-494-5236
Phoenix Neurological Associates, Phoenix, AZ	Lynne Flynn	lynneflynn@pnal.net 602-258-2863
University of California at Los Angeles, Los Angeles, CA	Rebecca Alvarez	RRALvarez@mednet.ucla.edu 310-825-9816
UMDNJ-Robert Wood Johnson Medical School, New Brunswick, NJ	Barbara Belsh	belshba@umdnj.edu 732-235-7340
University of California at Irvine, Orange, CA	Veronica Martin	vero@uci.edu 714-456-2332
University of Illinois at Chicago, Chicago, IL	Judy Richman	judyg@uic.edu 312-413-8605
University of Kansas Medical Center, Kansas City, KS	Maureen Walsh	mwalsh@kumc.edu 913-588-0645
University of New Mexico, Albuquerque, NM	Jeannie Boyle	jboyle@salud.unm.edu 505-272-3342
University of Pennsylvania, Philadelphia., PA	Mary Kelley	kelleym@pahosp.com 215-829-3053
University of Vermont College of Medicine, Burlington, VT	Dario Cutura	Dario.cutura@med.uvm.edu 802-656-3876
University of Texas Southwestern Medical Center, Dallas, TX	Barbi Estes	Barbi.Estes@UTSouthwestern.edu 214-648-7976
Washington University School of Medicine, St. Louis, MO	Barbara Abrams	abramsb@neuro.wustl.edu 314-747-8288