In late May, physicians at OHSU injected the first Pacific Northwest patient with an experimental drug called RTL1000.

The patient, Anne Foster of Montesano, Wash., so far has reported no adverse reaction to the drug – a positive development because early trials are intended to look for possible side effects.

RTL1000 was developed by scientists working at OHSU and the Portland Veterans Affairs Medical Center. In experiments involving laboratory mice, “it was the best thing I’d seen in 35 years,” said Arthur Vandenbark, OHSU neurology professor, Veterans Affairs Medical Center scientist and head of the research team that discovered and developed RTL1000.

MS affects more than 2.5 million people worldwide, 400,000 in the United States, and is projected to be a $76 billion market. Because it’s such a potentially lucrative field, institutions such as OHSU have begun to emphasize more than traditional basic research. In the medical field, it’s called “translational research” – breakthroughs that translate into patents and moneymaking therapies.

RTL1000 is a product of such translational research. And Vandenbark’s opinion of its potential is especially noteworthy, since he is credited with inventing another drug, NeuroVax, that also is viewed as a possible MS breakthrough therapy.

While Vandenbark stands to collect royalties from NeuroVax should it ever come to market, he said the lessons he has learned from his experience developing the drug and selling its patent have influenced the way he has gone about handling RTL1000.

MS, which usually first strikes adults between the ages of 20 and 40, is caused by white blood cells, called T cells, that attack myelin, the sheath that insulates nerve fibers. The nerve fibers lose the ability to conduct impulses.

In multiple sclerosis-infected mice, Vandenbark said, RTL1000 not only halted the damage done to myelin and nerve fibers, but it appeared to reverse the damage, essentially reversing the effect of MS. It works by binding to the T cells and rendering them inactive.

For all its promise, RTL1000 is years away from coming to market as a therapy for MS. Eleven years in development, it is just now entering the first of three phases of clinical trials on humans that eventually could lead to approval by the federal Food and Drug Administration.

The trials process itself could last up to 10 years and cost up to $125 million, according to Vandenbark.

Scientists temper excitement

Vandenbark, who has been studying T cell receptors since the mid-1980s, is a perfect example of the new expectations facing researchers at medical centers and universities. For the first decade of his career he was looking at T cell receptors, he said, but without any real sense of what tangible applications might result.

“It took me 10 years of my career to learn how to get there,” Vandenbark said. “I’m a basic scientist at heart, but I realize if you’re going to make an impact you have to be a translational scientist. You have to get up from the bench and animals and start treating people.”

Dennis Bourdette, chairman of neurology in the OHSU School of Medicine and director of the Multiple Sclerosis Center of Oregon, is both excited about RTL1000’s potential and sobered by the obstacles the therapy must overcome.

“It does have the potential to be a breakthrough drug for the treatment of MS,” Bourdette said. But, he added, the current optimism is based on the success RTL1000 has had in treating MS in animal models. That, Bourdette said, doesn’t always translate into the same effects in human trials.

“Human patients are much more complex than the very controlled animal models that (scientists) are working with,” Bourdette said. “You take these mice that are genetically identical, you get to control everything. Because you’re dealing with this homogenous model you can get excited if you give four mice something and they get better. As a clinician, you have the breadth of experience to know you don’t get excited about one person or two or three people responding.”

But it is that excitement that helps bring in investors and allows a new therapy such as RTL1000 to get off the ground, Vandenbark has learned.

Investors bring on the cash

Just as Vandenbark and his team of scientists have worked to devise a molecule to counter the renegade MS T cells, OHSU’s technology transfer office has had to devise a model to secure patents and find funding to carry the drug into clinical trials.

It has done that by licensing RTL1000 to a Tigard-based biotechnology company called Artielle ImmunoTherapeutics Inc., set up to oversee the drug’s future.

Artielle has found venture capitalists willing to put up an initial $11 million to get RTL1000 through Phase One clinical trials, which is when the drug is first tested on humans and which are designed to discover possible side effects.

Vandenbark, for one, is not surprised that funding has been found for his new invention. “There’s so much money out there for investment,” he said of the biotechnology field. “If you’ve got something out there that works, there’s plenty of money. The investment houses are fat right now.”

While RTL1000 begins Phase One clinical trials, NeuroVax is a step ahead, in Phase Two trials, designed to measure a new drug’s effectiveness.

And the two drugs – both of which Vandenbark had a large hand in developing – will be competing against each other. NeuroVax’s approach is to boost the activity of T cells that regulate MS rather than rendering renegade T cells inactive.

Joseph O’Neill, president and chief executive officer of the Carlsbad, Calif.-based Orchestra Therapeutics, which owns the rights to NeuroVax, said that competition could benefit both companies and MS sufferers.

“I like our product because it’s elegant, and we’re in Phase Two so we’re further along,” O’Neill said. “Ultimately competition is going to be sorted out by which one works best with the fewest side effects. I guess you could say there’s a competition. But competition brings out the best in people.”

peterkorn@portlandtribune.com

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