The ALS Association, National Office
27001 Agoura Road, Suite 150
Calabasas Hills, CA 91301-5104
Phone: (818) 880-9007
Fax: (818) 880-9006
June 12, 2006
Trial of Ceftriaxone Begins Enrolling ALS Patients
Roberta Friedman, Ph.D., ALSA Research Department Information Coordinator
The ALS Association (ALSA) announced that a trial of ceftriaxone in ALS has begun enrolling patients. This multi-center clinical trial is the culmination of a drug screening program, a collaboration among ALSA, the National Institute of Neurological Disorders and Stroke (NINDS) and several other research organizations aimed at finding better treatment for diseases such as amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).
Ceftriaxone is an intravenously administered antibiotic approved for certain infections but has additional properties that give it promise for ALS. The drug screening effort found an unexpected action on glutamate, a messenger used by nerve cells. The Food and Drug Administration had required additional testing to support that ceftriaxone would be safe when used for longer periods of time, relevant to ALS treatment.
Compounds that show statistically significant activity in multiple lab tests called drug screens are especially promising candidates. Ceftriaxone was one such compound and could enter the nervous system as well. Jeffrey Rothstein, M.D., Ph.D., at Johns Hopkins and colleagues found that ceftriaxone increased levels of the glutamate transporter protein at concentrations known to reach the brain.
After the success in lab screening, the drug then showed promise in a mouse model of ALS in experiments co-funded by ALSA, NINDS, the Packard center, and Project A.L.S. (animal testing was performed at PsychoGenics). Ceftriaxone delayed loss of muscle strength and body weight when treatment began at disease onset at 12 weeks of age in the mice. Earlier treatment did not add to the effect.
The drug screening effort that discovered the possible benefit of ceftriaxone in ALS had been initiated as a joint project of ALSA, NINDS, the Hereditary Disease Foundation (HDF), and the Huntington's Disease Society of America (HDSA). Investigators from 26 laboratories took part in the initial 6-month, $1.3 million project, which tested 1,040 compounds using 29 different assays or tests.
Clinical Trial Plans - Questions and Answers
Q: Should I ask my doctor to prescribe ceftriaxone (Rocephin) or another similar antibiotic for me to take?
A: No, the researchers doing this study and other ALS clinical experts agree that it is premature for patients to take ceftriaxone for ALS. The clinical trial now enrolling is designed to answer the question of whether or not ceftriaxone is safe when used for long periods of time and has a positive effect for people with ALS. The information published and described in the ALSA release above is experimental.
Q: How can I learn more about the clinical trial and find out about enrolling?
A: The study will be conducted by the Northeast ALS consortium with centers in the United States and Canada. It is funded by the National Institutes for Neurological Diseases and Stroke (NINDS). Initially, the study will enroll 60 people with ALS at eight centers in the United States. This first study is designed to evaluate the safety of long term treatment with ceftriaxone and the optimal dosage. The study will then be expanded to include 600 people with ALS at approximately 40 ALS centers throughout the U.S. and Canada.
The principal investigator of the clinical trial grant from NINDS is Merit Cudkowicz, M.D., from Massachusetts General Hospital. Co-Principal investigators are Dr. Jeremy Shefner from SUNY at Upstate and Dr. Allitia Dibernardo at Massachusetts General Hospital. Project Manager for the study is Fran Murphy from Massachusetts General Hospital. For more information, Ms. Murphy can be reached at 617-643-3980 or by e-mail at fmurphy@partners.org .
For more information about ceftriaxone please see http://www.alsa.org/news/article.cfm?id=569 orhttp://www.clinicaltrials.gov/
The ALS Association, National Office
27001 Agoura Road, Suite 150
Calabasas Hills, CA 91301-5104
Phone: (818) 880-9007
Fax: (818) 880-9006
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Related:
Screening Effort Identifies Ceftriaxone as Potential ALS Therapeutic