Newswise — To be successful, an academic entrepreneur has "to have the big picture," said J. Marc Simard, MD, PhD.
Simard knows of what he speaks. The professor of neurosurgery, physiology, and pathology at the University of Maryland School of Medicine is the 2008 Entrepreneur of the Year.
The University has recognized Simard for discovering, testing, and patenting a novel channel in brain and nerve cells and for creating an opportunity for industry to develop his discovery into a wide range of new treatments.
Patients with, or at risk for, brain and nervous system conditions could one day benefit from the potential new treatments as a result of Simard's research and entrepreneurial creativity.
First, Simard and former graduate student Mingkui Chen, MD, PhD, discovered that an ion channel, previously unknown, appears to play a role in brain cell swelling. They then found that drugs commonly used to treat diabetes can block the new channel and that the blocking can limit swelling, the death of brain cells, and hemorrhaging from strokes.
"I came to recognize that this will relate to several diseases that are related to the brain and spinal cord head injuries, spinal cord injuries, germinal matrix hemorrhage [lesions in premature babies] and other nervous system conditions," Simard said.
The age range of potential patients is huge, he said, from premature babies to teens with spinal cord injuries, to military [combat] injuries, people with car accident injuries, to elderly at risk for strokes. Simard said one example may be children with cerebral palsy. "For those babies born with a high risk for brain injury--born premature and with weak lungs--giving them this drug may prevent hemorrhaging and then potentially hospitalization and much of the medical care thereafter."
The first product in development, called R-001, at Remedy Pharmaceuticals of New York, which was founded in 2004, is based on the patented work on the novel ion channel and potential stroke treatment by Simard and Chen, now a resident in pathology at Creighton University in Omaha, Neb.
Intuitively, from his knowledge of neurosurgery, he said, Simard filed patents on his breakthrough discovery through UMB even before he had gathered experimental data showing that it has potential to treat for conditions other than stroke.
"The hard part is to figure out if something is worthwhile. Then you have to figure out how to protect [the discovery] it. Then you have to get support for it to treatment phase. Many good ideas fall by the wayside. That's what's wrong with the process of drug discovery. You've got to figure this out very early in the process," said Simard.
Subsequently, investors from the New York-based Carrot Healthcare Ventures Inc. came to Baltimore looking for good technologies to back. "I remember that your Office of Research and Development showed us three or four patented new technologies. [Simard's] was head and shoulders above the rest in terms of the quality of the science," said Sven M. Jacobson, principal of Carrot and now CEO of Remedy. "It was well laid out and fantastically explored."
Jacobson notes that Simard approached the basic science from all different angles--molecular physiology, preclinical animal models—he even generated some human data, "then put all the pieces of the jigsaw puzzle together," he said.
As the story of Simard's discovery took a turn toward business, he put on the entrepreneur's hat. "To eventually help patients you have to have something commercially viable." Partnering with the pharmaceutical industry should be recognized as necessary, he added. "You have to have the big picture." Working with Carrot, Simard helped found Remedy Pharmaceuticals and he continues as a scientific advisor to the company.
Simard is not a Remedy employee, but he has been active in its fundraising by explaining to investors both the science and implications for patients. In fund raising, Simard's "entrepreneurial flair is self-evident in these lengthy discussions, which cover not only scientific and medical issues, but also touch on key business strategy too," said Jacobson.
Simard explained, "My enjoyment is to present to investors. To do this you need to talk to them in an efficient and knowledgeable way, to talk in a new way to very intelligent people in a field that is very different than mine."
Remedy intends to market R-001 globally. Said Jacobson, "There is a crying need." There are more than 700,000 strokes each year in the U.S. resulting in 280,000 deaths. Clinical trials for the drug will soon enter phase 2. Jacobson said there is "a long path ahead to complete difficult clinical trials. But there are many ways we and others think this will succeed."
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