LOS ANGELES, Feb. 4, 2004 (PRIMEZONE) -- A new study to find a more rapid and accurate diagnostic test for amyotrophic lateral sclerosis (ALS) using "biomarkers" found in cerebrospinal fluid and blood is being funded by The ALS Association.
The study, "Identification of Diagnostic Biomarkers and Therapeutic Targets for ALS," brings together researchers from academic institutions and biotechnology sectors in a collaborative project that utilizes two distinct, cutting-edge technologies.
Investigators from Massachusetts General Hospital, the University of Pittsburgh School of Medicine and Metabalon, Inc. in North Carolina will work together on the project, which is being funded for one year with the potential for renewal after presentation of the initial data to The ALS Association.
Currently, people are diagnosed with ALS primarily by a process of ruling out other diseases in a lengthy and expensive process that is often fraught with discomfort and uncertainty.
"There is an urgent need to find a faster and more reliable diagnostic process that will enable earlier treatment and improve chances that therapy will alter the course of ALS," said Dr. Lucie Bruijn, science director and vice president of The ALS Association.
The research also may yield new methods to determine the progress of the disease by comparing the amounts of biomarkers -- small molecules associated with ALS -- at early and late stages. This type of test may also make it possible to measure the effectiveness of different drug treatments in clinical trials.
In the new study, investigators will analyze blood and cerebrospinal fluid from a control group and from ALS patients by using two different technologies that have the potential to strengthen and validate the findings.
Researchers believe that biomarkers for ALS are more likely to be detected in the cerebrospinal fluid that is contained in the central nervous system and bathes motor neurons in the spinal cord and brain. This fluid is in direct contact with cells that are dying in the ALS disease progression.
However, easier diagnostic testing in patients could be achieved if the same markers are found in the blood.
An initial study in 2003 by Robert Bowser, Ph.D., of the University of Pittsburgh School of Medicine, found several small proteins in the cerebrospinal fluid of ALS patients that are not present in the same fluid from control patients and were sensitive diagnostic markers for ALS.
In the new study, the Ciphergen Protein Chip mass spectrometry proteomic system will be used to confirm these initial findings in a larger sample pool of cerebrospinal fluid and from blood taken from ALS patients and control patients. Samples obtained from Massachusetts General Hospital and the University of Pittsburgh will include a variety of other neurological disorders and Alzheimer's disease. The diversity of control samples is critical to ensure that markers identified are specific to ALS.
The same set of samples will be tested at Metabalon, where the company's metabolomics platform will search for signatures of ALS by accurately measuring the spectrum of biochemical changes and mapping these changes to metabolic pathways. Metabalon is a leader in the application of metabalomics, a powerful and new scientific approach for the discovery and development of drugs and the early diagnosis of disease states.
Dr. Bowser, of the University of Pittsburgh School of Medicine, Merit Cudkowicz, M.D., Massachusetts General Hospital, Robert H. Brown, Jr., Ph.D., M.D., Massachusetts General Hospital, and Rima Kaddurah-Daouk, Ph.D., Metabalon, Inc., are the principal researchers in the new study.
To provide samples for this study, patients and physicians should contact Merit Cudkowicz at 617-726-0563, Kristyn Newhall at 617-726-9122 or Robert Bowser at 412-383-7819. Investigators are seeking to analyze samples from familial ALS, sporadic ALS, PLS and pure lower motor neuron disease.
The ALS Association is the only national not-for-profit voluntary healthcare association dedicated solely to the fight against ALS. For information, visit ALSA's website at www.alsa.org or contact ALSA at 818-880-9007.
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