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HD biomarkers in CSF
April 2013
by Lloyd Dunlap  |  Email the author
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EMERYVILLE, Calif.—KineMed Inc., Isis Pharmaceuticals Inc. and the CHDI Foundation Inc. are collaborating to utilize KineMed's translational biomarker platform with Isis' antisense therapeutic program for Huntington 's disease (HD). This collaboration, which builds on an earlier alliance between CHDI and KineMed to develop companion biomarkers of therapeutic response in HD, will provide Isis access to novel biomarkers for use in the development of an antisense drug to treat HD.
 
"There is a critical need to identify appropriate biomarkers to determine target engagement and predict early clinical efficacy for future Huntington's disease clinical trials," says Dr. Jonathan Bard, director of molecular pharmacology at the CHDI Foundation. "Combining Isis' knowledge of antisense therapies with KineMed's expertise in developing unique pharmacodynamic biomarkers creates a collaboration with great potential for discovering such tools for HD."  
 
The companion biomarker partnership represents "personalized medicine in action," says Dr. Patrizia Fanara, vice president of neuroscience at KineMed. "Our neurodegeneration-specific biomarkers, which provide dynamic measures of axonal transport deficits in degenerating brain cells, predict therapeutic response in neurological and neuromuscular disorders. This is a translational biomarker that could prove crucial to the advancement of disease-modifying treatments. With the Huntington's disease domain knowledge that CHDI brings and the therapeutic approach that Isis is pioneering, this partnership has the potential to develop biomarkers for specific therapeutics for HD and lead to personalized medicines for HD patients."  
 
"There is a critical need to identify appropriate biomarkers to determine target engagement and predict early clinical efficacy for future Huntington's disease clinical trials," says Dr. Jonathan Bard, director of molecular pharmacology at the CHDI Foundation. "Combining Isis' knowledge of antisense therapies with KineMed's expertise in developing unique pharmacodynamic biomarkers creates a collaboration with great potential for discovering such tools for HD."  
 
Dr. Marc Hellerstein, chief scientific officer at KineMed, notes that until now, there has been no way to determine when HD actually starts, or why symptoms so often show up in individuals at about age 40.   "HD is a single-gene Mendelian disorder, but what are the causal pathways?" he says. "Linking simple genetics to biology, which is not simple, has been the challenge."  
 
KineMed's neurodegeneration biomarker of axonal transport deficit has been recently published in the Journal of Clinical Investigation and validated in patients with other neurological disorders. The KineMed-led research identified a class of cerebrospinal fluid-based (CSF-based) kinetic biomarkers that reflect altered neuronal transport of protein cargo, a common feature of neurodegeneration. The research data indicated that CSF kinetic biomarkers of axonal transport provide direct in-vivo metrics of neurodegeneration that are translatable in humans.  
 
According to Fanara, disease onset and progression are both highly variable. "We can now monitor changes at the cellular level based on the relationship between changes in the brain and CSF, and correlate these changes to clinical symptoms," she says.  
 
KineMed CEO David Fineman says connecting biology to genetics will ultimately lead to the "missing link"—connecting biology to therapeutics.  
 
Huntington's disease is an inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. The defect causes a DNA sequence called a CAG repeat to occur many more times than normal. Each child of a parent with a mutation in the huntingtin gene has a 50 percent chance of inheriting the mutation. As a result of carrying the mutation, an individual's brain cells degenerate, leading to behavioral, cognitive and motor impairments that, over the course of the disease, significantly reduce the individual's quality of life and ultimately cause death within 15 to 25 years of symptom onset. There are currently no therapeutics approved that slow the progression of Huntington's disease.  
 
"A pharmacodynamic biomarker could be an important contribution to our clinical development efforts, and we look forward to working with KineMed and CHDI to evaluate KineMed's biomarker platform in our HD program," says Dr. Frank Bennett, senior vice president of research at Isis.  
 
Isis declined to provide further details on its therapeutic program.
 
 
 
 
Code: E041318

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