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A shift in focus: Lorson moves basic research to drug development

By Roger Meissen & Erica Overfelt | Bond LSC

It takes a lot to move a discovery from lab bench to an application that can provide therapeutic benefits to those suffering from disease.

Bond LSC’s Chris Lorson is making moves to bridge that gap with the start of Shift Pharmaceuticals. With its formation in March 2017, Lorson adds co-founder and Chief Science Officer of the company to his list of titles that include Bond LSC investigator, professor of veterinary pathobiology and associate dean for research and graduate studies.

Shift Pharmaceuticals builds off of years of progress the Lorson Lab has made in understanding spinal muscular atrophy (SMA), which is the leading genetic cause of infant deaths. The disease causes neurons to die, leading to muscle failure, including those that affect walking, arm movement, and respiratory function. While SMA is technically a rare disease, it is remarkably common, affecting nearly 1/10,000 births.

“It’s a devastating disease for patients and families; while the primary defect in is nerves, this leads to problems in muscles, bones, and other vital systems,” Lorson said. “Historically, the majority of kids who develop SMA do not survive beyond 3-5 years.”

Lorson knew his research had potential for drug development, and MU’s Office of Technology Management & Industry Relations (OTMIR) pursued patent protection for the technology. This process helps safeguard the innovations resulting from the research and allows MU to better attract commercial interest to develop and market medical treatments originating from the technology.  But it took a partnership with co-founder Steve O’Connor to get the ball rolling. O’Connor is MU’s Entrepreneur in Residence and has significant experience in starting drug development businesses and now serves as CEO of Shift.

“I never knew how to start a company,” Lorson said. “I would argue most academics don’t – this isn’t part of our traditional training. I sat around not knowing what to do, realizing I had this thing that could do a lot, but the practical steps of setting up a biotech start-up were beyond me.”

“Steve thought this technology sounded really cool, so in late March he submitted paperwork and by the end of March we were a company. Shift’s first grant was submitted 3 days later.”

The goal of Shift Pharmaceuticals is to move their lead compound into the clinic for SMA.

“When you look at the disease, it’s not just one cell type and not just one clinical type of patient,” Lorson said. “It really is a disease that is complex and the idea is to bring more options to the fight.”

With the start of any new business, money is always a necessity. Funding from the advocacy group CURE SMA provided the initial funding for the discovery of this compound, while several other foundations including FightSMA, the Gwendolyn Strong Foundation, and Muscular Dystrophy Association have further contributed to the pre-clinical development. MU’s OTMIR negotiated an option agreement with Shift, giving them the exclusive rights to the technology, which helped them obtain a recent $2.73 million grant from the Department of Defense Congressionally Directed Medical Research Program (CDMRP). This will move the company toward the first phase of investigating a new drug.

The root of SMA

Lorson has spent most of his scientific career chasing the underlying causes of SMA.

That search focused in on a few key genes in those suffering from the disease. Two genes — named survival motor neuron-1 and -2 (SMN1 and SMN2, respectively) — are central to SMA development. In patients, SMN1 is mutated and doesn’t process enough of a key protein (SMN) that helps neurons function. While SMN2 acts as a backup gene for this function, a miniscule change in the SMN2 gene causes it to make less SMN protein than required by the body.

In 2016, the Lorson Lab at Bond LSC produced a compound that increases the lifespan of SMA mice . They targeted the back-up gene, SMN2, to produce more functional protein and discovered an increase of protein causing a significant lifespan extension in treated mice. This discovery showed promise for creating a cure for those with SMA.

Shift Pharmaceuticals will be working on developing a drug that builds off Lorson’s work and targets all forms of SMA.

Shift’s first two employees, Mizzou alumnus Paul Morcos and UMKC alumnae Diane Beatty, will help move toward that goal. With Morcos in research and development and Beatty negotiating regulatory affairs, the company hopes to move the drug toward FDA approval.

Thanks to the recent Department of Defense grant, the next step looks to testing in larger animals and at things Lorson did not consider before.

“The experiments we will be doing are not academic in nature, rather, they are focused on the singular goal of preparing our lead compound for an FDA submission. From a traditional academic lab perspective, these might sound rather boring,” Lorson said. “All of these things are not things you do in an academic setting, but that is exactly the point. This is drug development, not the quest for another paper.”

Within the Business Incubator, MU will provide space for the company as well for their research. This sort of partnership is just another part that may one day help translate vital basic research into future treatment.

“Almost everybody has the possibility of doing something that is translational, it is just envisioning it in a different way,” Lorson said.

Article originally published on Decoding Science.