||Helping launch the new rare diseases web site are (from l) OTT director Dr. Mark Rohrbaugh, OTT deputy director Dr. Bonny Harbinger and ORD director Dr. Stephen Groft.
“A lot of the work that goes on at NIH is related
to rare diseases and conditions, things that industry on its own initiative would probably not be researching,” said Dr. Bonny Harbinger,
OTT deputy director. “So we decided that it made sense to, in one spot, collect
all of the technologies
On the site, www.ott.nih.gov/rarediseases,
OTT and ORD have listed more than 500 technologies
related to more than 100 rare diseases and conditions, giving companies just one place to visit. “They can search by disease, or they can look at all of the technologies. And it’s easy,” Harbinger said. “That’s what’s really critical, to make things easy for industry.”
She explained that OTT has also invited nonprofits
outside of government—primarily academic
institutions—to list their technologies on the site in an effort to provide the largest possible database of technologies. And with these groups—the University of California system
alone expects to list numerous technologies—
the site should eventually include thousands
of technologies, said OTT director Dr. Mark Rohrbaugh.
Putting the current list together required the painstaking process of first finding all of the rare diseases—which number around 7,000 and are increasing daily—then matching the diseases to the technologies in the NIH/FDA portfolios and finally validating the selection of each technology. But Dr. Stephen Groft, director
of ORD, said the effort was well worth it. “It was something that was desperately needed,
because a good part of the pharmaceutical industry will not commit the resources to develop
products for rare diseases,” he said. “And we had these technologies, but we didn’t have a way of matching the new technologies with companies who might have an interest in developing
He commends OTT for the idea and for the speed in producing the site. “It’s a good example
of a collaborative effort when one NIH office develops a useful technology that can be utilized by another office of NIH that facilitates
meeting the goals of both offices,” he said. And he’s excited about the site’s potential,
primarily because of how it could eventually
lead to products used for the diagnosis
and treatment of a rare disease, but also because it will allow individual scientists to see what others have done and possibly find ways of collaborating. “They could develop the next generation of discoveries or interventions…
from linking their groups together.”
So far, feedback on the site has been positive, Harbinger said. She explained that what OTT really wants is to see companies interested in licensing technologies start their search with NIH, “for them to always think, let’s go to NIH first because they have so many things available,
and it’s organized so it’s easy to find the person you want to talk to.” She said companies
have often complained in the past about how many mouse clicks or phone calls it has taken to get this kind of information. Now, the site helps in reducing “the barrier for companies
to get access and move technologies forward,” Rohrbaugh said. OTT plans to produce
similar modules for other medical areas, starting by adding chronic diseases to the neglected diseases site.
OTT was created by NIH in 1989 to evaluate, protect, license and manage both NIH and FDA discoveries, inventions and other intellectual property. The office—among other responsibilities—
oversees patent prosecution, negotiates licensing agreements and develops technology
transfer policies. To date, about 25 products
that include technologies from NIH or FDA have received FDA approval, Rohrbaugh said. One of the most recent products to reach the market is Merck-produced Gardasil, the human papillomavirus vaccine used to protect against cervical cancer. The underlying technology for the vaccine originated at NCI (see sidebar).
To see a product like this reach the marketplace for a rare disease is the goal of the new module,
and both OTT and ORD are doing all they can to get the word out about it. But for now, the main thing they can do is sit back—like matchmakers—and watch for any connections to be made. “We just have to wait to see where the link-ups happen,” Groft said. “You have to watch and monitor, and perhaps contact the investigator a year or two down the road to see what the impact is and whether the program ever met with success.”
However long it takes for the benefits of the site to be seen, it’s great to know this kind of tool is readily available, Groft explained. “With the costs of product discovery and development increasing tremendously, many times rare diseases
don’t receive extensive focus,” he added. “Whatever we can do to facilitate the transfer of information about the availability of new technologies,
and perhaps generate the interest for a company to license them, it will always be useful.
With so many rare diseases that do not have treatments, there certainly is a need for new discoveries of diagnostics and interventions.”
|From Lab to Market: The HPV Vaccine
NIH director Dr. Elias Zerhouni (l) presents an honorary poster to Dr. Douglas Lowy (c) for his lecture, named for Dr. Philip S. Chen, Jr. (r).
Perhaps no other recent product on the market demonstrates successful health care technology transfer better than the human papillomavirus (HPV) vaccine, Gardasil, produced by Merck & Co. and approved by the FDA in June 2006. Based largely on technology developed at NIH, the vaccine works to prevent four types of the sexually
transmitted HPV that together cause 70 percent of all cervical cancer and 90 percent of genital warts.
Dr. Douglas Lowy, chief of NCI’s Laboratory
of Cellular Oncology, who, with his colleagues,
developed this underlying technology,
recently told the story of the HPV vaccine in the second annual Philip S. Chen, Jr. Distinguished Lecture on Innovation and Technology
In a lecture titled, “The Science, Technology and Promise of Preventive HPV Vaccines,” Lowy described, in detail, the more than 20 years of research he conducted on HPV with his colleague Dr. John Schiller.
It’s a “heroic” story about the effort to fight cervical cancer, the second most deadly cancer for women worldwide, said NIH director Dr. Elias Zerhouni in an introduction to the speech. He noted that he has talked about the vaccine’s creation to Congress and with the President on his recent visit to NIH. How researchers took the technology “from the lab to the marketplace is a journey we can learn from,” Zerhouni said.
Lowy explained that at the outset of their vaccine research, “it’s amazing how poor our qualifications
were” to work on this technology. He said they had no experience in vaccines, immunology, translational research or papillomavirus structural proteins and virus structure. But they had studied
papillomavirus biology, and that was the start.
Their key finding was that the outer coat protein of the virus, called L1, could “self-assemble” into non-infectious, virus-like particles (VLPs) that resemble the shell of the actual virus. They learned that exposure to VLPs causes the immune system to produce protective antibodies. The vaccine triggers these antibodies, so if an individual is exposed to the virus after having the vaccine, the antibodies bind to the L1 protein coat and prevent the virus from infecting cells.
Lowy discussed the high success rate of the vaccine and said it is most important to administer to girls between the ages of 11 and 13, prior to virus exposure. He stressed the fact that the vaccine is not effective against established cases of HPV or against some HPV strains, and therefore cervical cancer screening continues to be necessary.