SAN FRANCISCO—Aimed at becoming a leader in the growing pack of global diagnostics companies, Amarantus BioScience Holdings announced July 29 that its most significant study had been accepted for publication in the Elsevier journal Gene. Entitled “MSPrecise: A molecular diagnostic test for multiple sclerosis using next generation sequencing,” the manuscript describes how MSPrecise has diagnosed multiple sclerosis (MS) patients with an accuracy of 84 percent.
“This is the most important study published by Amarantus’ diagnostics division,” says Dr. Benjamin M. Greenberg, director of the Neurosciences Clinical Research Center at the Children’s Medical Center of University of Texas Southwestern Medical Center in Dallas. “It ranks extremely high with respect to MS patients and lays the foundation for the publication of our clinical validation study.”
Another study “has already been conducted with over 100 subjects, and we are in the long-term follow-up phase of that study,” he tells DDNews.
Diagnosing MS is particularly difficult because the condition “presents like Lyme disease and several other immune or autoimmune disorders,” Greenberg said. “The symptoms are indecipherable, especially early on.”
MS is characterized by an autoimmune attack on myelin, the substance that insulates nerve cells, allowing them to conduct impulses and communicate. Early identification could aid in treatments and possibly in prevention of myelin degeneration, hopefully slowing or even halting MS progression.
“Our short-term goal is to commercialize this test through the CLIA pathway,” according to Greenberg. “Scientifically, our goal is to diagnose patients at the initial onset of symptoms, or perhaps pre-symptomatically in predisposed populations.”
Amarantus’ long-term goal is “to make MSPrecise the standard of care in terms of the diagnosis of MS,” he adds. “We are also building an Alzheimer’s portfolio in parallel to provide a fuller offering to the neurology community.”
Colin Bier, chief development officer of Amarantus Diagnostics, stated in a news release that, “Given the early misdiagnoses that often occur with multiple sclerosis, physicians have an increasing need for new methods to accurately diagnose their patient.”
MSPrecise’s scoring in this study, completed in 2013, “is a powerful approach to aid clinicians in this task,” he added.
Yet two years later, the accuracy of Amarantus’ diagnoses is even better.
“In early 2015, Amarantus reported data from a later study that showed a significant increase in the accuracy of MSPrecise when adding oligoclonal banding (OCB) to the algorithm,” Bier stated. “We intend to publish those data, combining MSPrecise and OCB, in the near future.”
Researchers in the approved study state: “We have previously demonstrated that cerebrospinal fluid-derived B cells from early relapsing-remitting multiple sclerosis (RRMS) patients that express a VH4 gene accumulate specific replacement mutations. These mutations can be quantified as a score that identifies such patients as having or likely to convert to RRMS.”
Furthermore, “we showed that next-generation sequencing is an efficient method for obtaining the sequencing information required by this mutation scoring tool, originally developed using the less clinically viable single-cell Sanger sequencing,” the researchers added.
The diagnostic test showed a sensitivity of 75 percent on the RRMS cohort and a specificity of 88 percent on the “other neurological disease” cohort. The accuracy of the test in identifying RRMS patients or patients that will develop RRMS is 84 percent.
The study’s conclusion: “MSPrecise exhibits good performance in identifying patients with RRMS, irrespective of time with RRMS.”
MSPrecise augments the current standard of care for the diagnosis of multiple sclerosis by providing a more accurate assessment of a patient’s immune response to a challenge within the central nervous system, according to Amarantus.
Approximately 2.5 million MS patients worldwide are currently being treated with over $14 billion in drugs, Amarantus states. Misdiagnosis rates of more than 50 percent have been routinely reported as the cost of each false-positive diagnosis has grown to an estimated $100,000 to $250,000 per patient. The worldwide market is estimated at more than $2 billion, growing along with the cost of MS therapy.
“The MSPrecise CSF assay, along with our LymPro Test blood diagnostic for Alzheimer’s disease, are the two key assets in our diagnostics division,” Gerald E. Commissiong, president and CEO of Amarantus, has frequently stated. “Together, MSPrecise and LymPro will allow Amarantus Diagnostics to very rapidly achieve critical mass as one of the premier neurodiagnostic testing companies in the world.”
Speaking of LymPro, in other Amarantus news, on July 22 the paper, “Amarantus Diagnostics Meets Primary and Secondary Endpoints in Blinded, Multi-Center LP-002 Clinical Study for LymPro Blood Diagnostic for Alzheimer’s Disease and Confirms LymPro’s Fit-For-Purpose Use in Clinical Trials” was presented before the 2015 Alzheimer’s Association International Conference in Washington, D.C.
Amarantus Diagnostics presented positive results on the accuracy of the LymPro Test, its blood-based diagnostic assay for Alzheimer’s disease (AD), to discriminate between subjects with clinically diagnosed AD versus healthy controls.
The data presented at the conference “solidifies LymPro’s importance in the investigational Alzheimer’s space for identification and disease screening,” Commissiong said. “The LymPro blood-based diagnostic panel is a robust and reproducible measure of a key pathology of AD. Further, the findings corroborate previously published LymPro data.”
Results of this study reportedly validate LymPro’s utility for use as a diagnostic assessment tool in therapeutic Alzheimer’s clinical trials. Amarantus Diagnostics provides the pharmaceutical industry with LymPro biomarker services for investigational use only in clinical development programs.
“This additional validation demonstrated for LymPro raises the importance for its potential use in biomarker-supported selection of Alzheimer’s disease patients for clinical trials,” stated Dr. Paula T. Trzepacz, clinical professor of psychiatry at Indiana University School of Medicine and member of Amarantus’ clinical advisory board. “It has the convenience of being a blood test, while reflecting the same type of cell cycle reentry dysfunction found in the brains of these patients.”
“LymPro has the potential to become one of the fundamental diagnostic tools in Alzheimer’s drug development, and we look forward to adding FDA-approved PET imaging agents to further validate LymPro vis-à-vis diagnostic gold standards,” Trzepacz added.
The Alzheimer’s Association estimates that over 5.4 million people in the U.S. suffer from Alzheimer’s disease. Over 500,000 patients are diagnosed annually, with nearly one in eight older Americans affected by the disease.
Alzheimer’s disease is the third-leading cause of death in the U.S., the association states. The cost of unpaid care in the U.S. is estimated at over $210 billion annually. Total payments for care are estimated at over $200 billion annually, including $140 billion in costs to Medicare and Medicaid.
There is no cure or effective treatment for Alzheimer’s disease. Worldwide, about 35.6 million individuals have the disease and, according to the World Health Organization, that number will double every 20 years to 115.4 million people with Alzheimer’s by 2050.
