DISEASE CLASSIFICATION(S):
Amyotrophic Lateral Sclerosis(ALS)
NAME OF CLINICAL TRIAL/STUDY:
Biological Markers in Cerebrospinal Fluid
TRIAL RESULTS:
February 2006
Three proteins -- cystatin C, VGF (not VEGF), and a third identified only by its weight -- were lower in concentration in the ALS patients’ spinal fluid samples than in samples from unaffected study participants.
“Ultimately, we want to initiate treatments for ALS as early as possible,” said neurologist Merit Cudkowicz, a study author based at Harvard University and Massachusetts General Hospital in Boston. “Finding biomarkers that can assist physicians with diagnosis would be beneficial. Biomarkers are also important to help understand disease mechanisms and potential treatment pathway targets, and could also potentially help expedite clinical trials by providing ... outcome measures,” she said, adding that the results need to be replicated by others and in larger sample sizes.
The investigators published their results online Feb. 15 in the journal Neurology (see Publications, below).
TRIAL UPDATES:
11/2003:
The identification of an ALS-specific panel of biological markers present in the cerebrospinal fluid (liquid that circulates throughout the brain and around the spinal cord) of patients with the disease was announced Nov. 17, 2003, at the 11th annual meeting of the International Alliance of ALS/MND (Motor Neuron Disease) Associations and 14th International Symposium on ALS/MND.
The findings were announced by Robert Bowser, an associate professor of pathology and director of the ALS Tissue Bank at the University of Pittbsurgh School of Medicine, who was a co-investigator on the study with a Massachusetts General Hospital/ Harvard University team.
Bowser said the new markers, which are protein abnormalities, may allow physicians to more rapidly diagnose ALS and to more accurately monitor the effects of experimental treatments.
The study results are based on “protein profiling” of cerebrospinal fluid samples taken from 25 people with ALS and 35 people without ALS. Bowser and colleagues say they identified the ALS patients by their CSF protein profiles with nearly 100 percent accuracy.
They say they’ll confirm these results by examining a larger number of patients and will further evaluate the “signature pattern” of the CSF proteins during disease progression.
6/03:
Some findings already showing elevation of markers of DNA oxidative damage in cerebrospinal fluid, blood and urine of patients with ALS have been published.
PURPOSE AND RATIONALE:
The purpose of this study is to compare markers of cell injury in cerebrospinal fluid (CSF) of subjects with and without amyotrophic lateral sclerosis (ALS). The CSF will be obtained from ALS subjects who have volunteered to undergo a lumbar puncture.
STUDY DETAILS:
None
TARGET NUMBER OF PARTICIPANTS:
Not Determined
RECRUITMENT STATUS:
Open
ELIGIBILITY REQUIREMENTS:
- Clinical diagnosis of familial or sporadic ALS
- Men and women 18 - 75 years of age
- Willing and able to give consent
PUBLICATIONS:
< 29(7):652-658 2000; Medicine Biology Radical Free ME, Cudkowicz F, Beal H, O?Donnell D, Hayden W, Matson RH, Brown M, Bogdanov patients. ALS in DNA to damage oxidative>
Bogdanov M, Brown RH, Matson W, Hayden D, O’Donnell H, Beal F, Cudkowicz ME; Increased oxidative damage to DNA in ALS patients. Free Radical Biology Medicine 2000; 29(7):652-658
Pub Med Report
Pasinetti GM, Ungar LH, Lange DJ, Yemul S, Deng H, Yuan X, Brown RH, Cudkowicz ME, Newhall K, Peskind E, Marcus S, Ho L. Identification of potential CSF biomarkers in ALS. Neurology. 2006 Feb 15; [Epub ahead of print] Pub Med Report
