Seeking a cure: MUSC biotech spinoff wages its own small war on cancer

Posted by on Apr 30, 2013

CHARLESTON, SC – Just inside the entrance to the Medical University of South Carolina’s Hollings Cancer Center is a tall, bronze sculpture of a wing.

Painted on the wall beside it is a stanza from an Emily Dickinson poem.

Hope is a thing with feathers

That perches in the soul

And sings the tune without the words

And never stops at all.

It’s an uplifting message for a place where patients, doctors and researchers stare down grave diseases and seek to overcome them.

Dr. Jim Norris, whose cramped third-floor office features an enormous computer screen among piles of papers, golf paraphernalia and boxes of green tea, is one of those researchers, one who’s been at it longer than most.

While global cancer prevalence has increased even as treatments have progressed during his quarter-century at MUSC, Norris still has hope — hope that a molecule he discovered several years ago may, several years from now, become a commercially viable drug, part of the mythical cure.

His acid ceramidase inhibitor, the most promising of his company SphingoGene’s three molecules, is still in the beginning stages of development. But in a PowerPoint presentation to potential investors in Boston this month, Norris said if the preclinical results hold, then SPG105, as the inhibitor has been tagged, “has standard-of-care blockbuster drug potential in the radiation treatment industry.”

That is, if it works, by preventing cancer cell proliferation, the drug would allow for lower-dose, higher-efficacy radiation, improving patients’ quality of life while reducing relapse and death rates.

“When we inhibit the enzyme,” Norris explained, “we get a cure.”


Using the “C” word regarding cancer is tricky business. This month’s Time magazine cover claimed curing cancer is “now possible” thanks to “new cancer dream teams” and generated controversy in its wake.

Norris doesn’t buy the “dream team” idea. “The cure for cancer is going to come from little guys like me,” Norris said.

According to the plan, SphingoGene would start with prostate cancer, a common radiation target with almost a million cases treated annually worldwide, and then move on to applications for even more prevalent cancers such as those in the lung and breast.

According to the company’s commercialization plan, the total estimated worldwide market for SphingoGene’s products is projected to be between $1 billion and $5.2 billion per year, depending on market penetration, which would be on the high end if the drug eventually becomes standard of care. That’s a lot of lives extended and a lot of money for whoever’s selling the drug. But it comes with just as many “ifs.”

“Now, that’s completely hypothetical at the moment,” Norris, SphingoGene’s chairman and CEO, said last week. “But that’s the dream that we have.”

The problem is, some seven years and more than $2 million into a process that began with a National Cancer Institute grant, there’s still a long way to go and no money to get there.

Norris, who served as chairman of MUSC’s department of microbiology/immunology for a decade, said SphingoGene needs another $2 million just to get to the FDA’s doorstep to ask permission to do a clinical trial (i.e., test the drug on human patients).

“Five or six million dollars would really get us started toward knowing whether this is a good drug or not,” he said.

Ideal outcome

The SphingoGene team, including a veteran banker, have irons in the fire from a reformulated grant application Norris submitted to the National Institutes of Health this month to investment contacts around the country and beyond.

And last fall, a team of MUSC and College of Charleston students used SphingoGene’s portfolio to win the University of California-San Diego Rady School of Management’s Student Venture Open, a business plan competition.

But Norris can’t hold on much longer, lending his own money to the company. Paying for patent prosecution and license fees to MUSC “ain’t cheap,” he said, and the 69-year-old has his eye on retirement.

The ideal outcome, Norris said, is “develop this up through Phase II and then have Merck or Johnson & Johnson buy it, and we can go golfing, and they can develop it.”

But beggars, even with Ph.D. degrees and promising technology, can’t be choosers.

“I’m waiting for a grant or investor to come by,” he said. “I don’t care who does it. I just want to get it moving.”

To make that happen, he’ll need more than a wing and a prayer. But he’s not stopping yet.


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