PharmOptima Research Scientist Presents New Assay at 2013 International SMA Research Group Meeting

Monday, July 8, 2013

Portage, Mi (July 8, 2013) - "Spinal Muscular Atrophy (SMA) is a motor neuron disease. The motor neurons affect the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. It is a relatively common "rare disorder": approximately 1 in 6000 babies born are affected, and about 1 in 40 people are genetic carriers."ˡ 

Following the 2012 International SMA Research Group Meeting PharmOptima scientists came away believing there was a need for an assay capable of detecting SMN (Survival Motor Neuron) protein in plasma or CSF (cerebrospinal fluid).  PharmOptima's research scientists decided the development of a more sensitive and reliable assay would be a great way to contribute to SMA drug development.  "The ability to accurately measure SMN protein production is crucial in assessing the efficacy of therapeutic agents targeting Spinal Muscular Atrophy.  The lack of such an assay in easily accessible biological samples was hampering drug development efforts..." said Dr. Roger Poorman, CSO.  Doug Decker, Director of Biochemistry and Cell Biology added, "... many SMA patients are infants - so an important consideration as we developed this assay was keeping sample collection as non-invasive as possible and sample size as small as possible to maintain their comfort and safety..."

PharmOptima's Phil Zaworski, Director, Assay Development presented "Measurement of Spinal Motor Neuron Protein in Whole Blood" at the 2013 International SMA Research Group Meeting stating "...we have developed an electrochemiluminescence (ECL) based immunoassay which has successfully detected SMN in whole blood requiring as little as 1-2 µl sample thus making it possible to monitor SMN from a pin prick drop of blood..."

A. Christian Schauer, CEO of PharmOptima said "We are very proud of what our team accomplished; other researchers were unable to assay SMN in whole blood using standard methods, but this team's experience and ability to innovate produced an assay using whole blood that is already generating significant interest in the SMA research community."

About PharmOptima LLC

PharmOptima is a 10 year old preclinical CRO founded and operated by scientists with extensive experience from major pharmaceutical companies. PharmOptima is recognized as the premier CRO conducting Spinal Muscular Atrophy research.  The company’s in vivo research services include delivery, efficacy, tolerance, ADME and pharmacokinetics. Their in vitro expertise includes biochemistry, recombinant proteins: cloning and expression, cell-based assays, immunosorbent assays (electrochemiluminescence MSD formats  and traditional ELISA and) and biomarker research. Being owned and operated by scientists with extensive industry experience, they have first hand experience on both sides of the sponsor/CRO relationship and understand the difficulties and pressures sponsors face and work with their sponsors to move projects forward as efficiently as possible while also being mindful of budget constraints. 


PharmOptima LLC

Lance Martin,  Director Business Development

Phone: 269.492.3876


Click here for more information about Spinal Muscular Atrophy