Gap funds could propel discovery for brain tumors
Posted on: 05/19/2003
Gap funds could propel discovery for brain tumors
By Scott Shepard
Memphis Business Journal
May. 19 — Duane Miller and Eldon Geisert Jr. have filed a patent on a drug that appears to disrupt a particularly nasty form of brain tumor that is otherwise fatal within a year.
If it works in humans as well as in lab rats in fighting glioma cancer cells, the drug would save 50 lives a day. But just like dozens of potential medical miracles in Memphis, and hundreds in the region, this drug may languish in obscurity for a lack of early stage funding.
"We call it 155; it's the 155th molecule that we tested, and it worked," says Miller, a professor and associate dean at the College of Pharmacy at the University of Tennessee Health Science Center. "High grade glioma is essentially a death sentence even after radiation and surgery. It's scary how fast these grow, and there's been no major advance in chemotherapy in 30 years.
"If we have a compound that blocks that, it's an important discovery," Miller says.
Miller and Geisert will share the patent with UT. Whoever commercializes 155 would pay licensing fees to the patent owners.
155 came about through an accident that sounds like something from a movie script. Geisert is a professor of ophthalmology and of anatomy and neurobiology, and was working with other researchers on a way to interfere with a protein on the surface of Hepatitis C cells. Glioma cells, which lack that protein, were also treated for comparison.
"A graduate student thought it didn't work because nothing happened to the Hepatitis C," Miller says. "Someone noticed that the glioma cells were being affected. The next question was if it works in rats."
A post-doctoral fellow who has since returned to South Korea, Suk Kang, induced glioma brain cancer in numerous rats, achieving astounding results when treating them with 155.
"Consistently we put the drug in and were able to block the activity of these cells," Miller says. "If ever there was a case where we needed to transfer technology quickly, this is probably the case."
The next phase would be proof of concept, followed by clinical trials in humans. Miller and Geisert hope to get orphan drug status from the FDA, which would allow faster development of the drug and get it into the market sooner. Orphan drugs are those that treat people with relatively rare and often devastating ailments who lack viable alternatives.
This fast-track status could get 155 into clinical trials for less than $2 million, Geisert says.
"Duane and Eldon want to start a company and do something with this drug, but to find the gap funding to carry it from discovery to the patient is pretty significant," says Robert Palmer, director of technology transfer for UT-Memphis. "It's not like a piece of software or a mechanical device where you can work out the bugs in the lab in a year. This is human medicine, so the gap is a major issue."
Palmer's been shopping 155 around to investors, foundations and even big pharmaceutical companies, but so far has found no takers. He's got four other medical products with scientists anxious to start companies with their technology, but lack the gap funds. He believes there are plenty more who would like to try.
"If the environment were right, and the gap funding were available, we'd see a lot more faculty start-ups than we have," Palmer says. "These are people who'd say they don't want to license their technology but want to do something with it themselves."
The environment is emerging, Palmer says, with the Memphis Biotech Foundation's planned incubator space leased from the Regional Medical Center at Memphis. Also, UT's Tri-Star will open eight new on-campus incubator labs in July. Half of those are already leased by local start-ups.
The missing element is the cash it takes to continue the research.
"When you're a full-time professor and you want to start a company, it all needs to be there," Palmer says. "You don't have the time or ability to go beat out the resources you need."
The situation in Memphis is not unique. Bob Acuff is director of government relations for the College of Medicine at East Tennessee State University, and watched a very promising piece of medical technology leave for lack of development capital.
Scientists with a Johnson City anesthesiology group found a way to shine a light against the back of the eye and measure blood sugar.
"If you look at the number of diabetics who stick themselves numerous times each day, this has great potential," Acuff says.
They sought in vain to find development money, and eventually took the technology elsewhere.
"If they can't find the dollars here, they'll take the technology out of the state and their business with it," he says. "It's disheartening because to drive economic development, it's going to be these types of emerging companies that grow out of these ideas."
Investors are always in the hunt for solid, proven ideas, says Frank Montgomery, a venture capitalist in Jackson, Miss., who specializes in medical technology. But VC money cannot help early stage concepts such as the 155 drug. It's too soon to see a clear profit potential, and most VC investors don't operate in the long time frames it takes to develop biotech ideas.
"Most venture capitalists invest after the proof of concept, or certain milestones are met and there's some visibility that this could be a business," Montgomery says. "Most venture capitalists don't have a seven-year time horizon in looking for a return on their investment. Typically, when a VC makes an investment he hopes to see some form of exit within four years."
Acuff and Montgomery both hold out great hope for another local start-up, the Technology Resource Foundation. It grew out of last year's Governor's Task Force on Biotechnology, where the common lament was a lack of gap funding.
TRF is a non-profit that will award grants to help scientists reach the stage that a VC would find their ideas attractive. As a non-profit, TRF would not seek a return. Instead, plans are to seek regional economic development funds from federal agencies and other government sources.
Montgomery has agreed to be the executive director while Acuff is serving as second vice president and head of government relations.
"TRF is the ideal vehicle to help researchers because it will get involved in the early stage investing," Montgomery says. "TRF will evaluate the technology, do the necessary market research and any other due diligence to make a judgment to whether this has a chance to be something of therapeutic benefit."
The foundation will also provide mentoring, legal and business planning and other services to help companies become viable.
Miller and Geisert face an odd dilemma with 155; they know the science and it's business potential. As with many drugs, the mechanism of 155 is a mystery. By keeping the R&D local, this compound could be modified to treat any number of similar cancers with the economic growth remaining in Memphis.
They also know that 36,000 people die each year from glioma tumors, and so ethically they would license the drug to another company if that would make it available sooner.
"When you see something that looks this good this early, you don't want to let it go," Geisert says. "The bottom line is if it's going to help people, we have to get it into clinical trials, and if it fails, it fails. But if it works, you save 50 people a day."
Copyright 2003 American City Business Journals Inc.
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