November 16, 2012
By Andy Hyland
|Scott Weir, PharmD, Ph.D.|
More evidence of the University of Kansas Medical Center's rising national attention arrived recently when Scott Weir, PharmD., Ph.D., director of KUMC's Institute for Advancing Medical Innovation (IAMI), was appointed by Health and Human Services Secretary Kathleen Sebelius to serve on two important national review boards.
Weir will serve on the National Center for Advancing Translational Sciences (NCATS) Advisory Council and the Cures Acceleration Network (CAN) Review Board of the National Institutes of Health.
A new center within the National Institutes of Health, NCATS' mission is to catalyze research that enhances the development, testing and implementation of new diagnostics and treatments for all kinds of human diseases.
Weir recently sat down to explain a bit about how those new groups in Washington are helping people all over the country.
Q: Why does NCATS matter?
DR. WEIR: More than 30 million Americans suffer from a rare disease for which there is currently no treatment. Of the nearly identified 7,000 human diseases, we have treatments for only about 500. The majority of diseases for which we have no treatments are considered rare. Each of us has been touched by rare diseases in some fashion. As an example, my youngest son is a third-year medical student. He recently saw a pediatric patient here locally who had a genetic disease where bones don't form and they get fractures. This particular patient, as I understand, had more than 4,000 bone fractures.
These folks - patients, parents of patients, siblings of patients - are literally coming to the NIH for help. NCATS is trying to speed the delivery of new drugs and diagnostics to patients. When it works, you've got an effective new therapy to treat, prevent or control one of these diseases that addresses an unmet medical need.
Q: What is KU Medical Center's role in supporting the NCATS mission?
DR. WEIR: At IAMI, we assist researchers in transforming their laboratory-based and bedside-based discoveries into medical innovations. Using industry input all along the process, we select the best projects andadvance those projects through a series of commercially-focused go/no go decision points. We then partner with the KU Center for Technology Commercialization, industry and disease philanthropy organizations tobring these medical innovations to market. We've built a unique program here at KU. We like to say this is not your father's academic project. And, this approach is not a stretch. NCATS is providing national leadership in this regard.
A good example of addressing the needs of patients suffering from rare diseases is a project involving a drug called Auranofin for the treatment of chronic lymphocytic leukemia. We repurposed this drug, which was originally approved by the Food and Drug Administration to treat arthritis, by working with government and disease advocacy groups in a unique partnership called The Learning Collaborative. This model brings together the strengths of three different organizations: KU, the Leukemia and Lymphoma Society, and NCATS, to discover and develop new treatments for patients suffering from blood cancers.
We still don't know if Auranofin's going to prove to be effective in treating chronic lymphocytic leukemia. But, together we were able to open a clinical trial 11 months after reviewing the data and making the decision to move forward. Today, it's being studied in patients who have not been successfully treated with standard chemotherapy or whose cancer has now become resistant to standard chemotherapy. So we're giving these folks a chance. And to me, this is why we are here.
Q: What's the typical drug development process like?
DR. WEIR: It can take years to discover and develop a new drug. Within the pharmaceutical industry, it can take 12-17 years to bring a new drug to market, and cost in excess of $1.5 billion. And the likelihood of success is daunting. One out of every 5000 to 10,000 chemicals becomes an approved drug. To discover and develop drugs in a university setting is even more challenging. Projects might never make it because folks in the university don't have the money, internal expertise or access to expertiseWe're trying to get these drugs and treatments to patients faster, so we're always looking for new and creative ways to speed things along and get to the end of the path.
Q: Much of the NCATS budget supports programs called Clinical and Translational Science Awards (CTSAs), and we have one of those at KU. What does it do?
DR. WEIR: Frontiers: The Heartland Institute for Clinical and Translational Research is a member of the national CTSA consortium. Frontiers is one of 61 CTSA programs under NCATS. In 2011, KU Medical Center received a five-year, $20 million grant, which has really galvanized the regional stakeholder institutions around translational research. We have an opportunity to play a national leadership role in assisting NCATS in achieving its mission. Frontiers is working to bring promising drugs to the public more quickly and inform and educate patients about clinical trials.
Q: What is the Cures Acceleration Network?
DR. WEIR: The Cures Acceleration Network (CAN) will advance the development of high-need cures and reduce significant barriers between research discovery and clinical trials. CAN provides NCATS with flexibility in applying funds to support high-impact projects. Under the Cures Acceleration Network, NCATS can provide support to allow projects to be actively, aggressively managed. And the investments CAN makes will be guided by their board, so I'll have input into what those investments are.
Q: How will your participation help the university?
DR. WEIR: I think clearly having someone from KU, someone from the region - and it could be anybody - on the CAN Board and NCATS Advisory Council, gives us visibility. But perhaps more importantly, we're able to bring our experiences to this national effort. I'm pretty darned certain one of the reasons they asked me to serve is based on our success in working with industry, government, academia and disease philanthropy organizations to advance new treatments to patients in need. So I'm really excited and looking forward to assisting NCATS and helping to advance translational research at KU and in our region.