Testimony for the Pennsylvania House Majority Policy Committee

Thomas Jefferson University

Representative Taylor, Distinguished Representatives, and Guests,

Good afternoon. I am Steven McKenzie, MD, PhD, the Vice President for Research for Thomas Jefferson University. I have been a physician-scientist throughout my career, practicing Hematology, the care of people with blood disorders, and directing an NIH-funded research laboratory. Beginning in July 2003, I served as Jefferson’s Vice President for Science Policy and Technology Transfer. Then in July 2005 I was asked by our President Robert Barchi, MD, PhD to serve as Vice President for Research. On behalf of Thomas Jefferson University, I thank you for the opportunity to appear before you today.

We are grateful that the Legislature enacted the unique partnership of health improvement and future economic development by way of enhanced research in the Pennsylvania Tobacco Settlement Act 77 in 2001. This established CURE, the Commonwealth Universal Research Enhancement program. CURE has been a godsend to us at Thomas Jefferson University. Jefferson is the 4^th largest recipient of CURE funds in the Commonwealth, after University of Pittsburgh, University of Pennsylvania, and Pennsylvania State University. In return, we have worked very hard to be outstanding stewards of these funds.

Our strategic focus has been excellence in Cancer, Cardiovascular disorders, and the Neurosciences. Our mission and vision is to bring our work from the lab bench to the patient bedside and on out to the community. We are very enthusiastic about the opportunities afforded by the new revised Jonas Salk Legacy Fund proposal, which I will refer to as the Salk proposal.

We support all of the three major aspects of the new Salk proposal: 1) infrastructure support for CURE recipients, 2) the ongoing support for the regional Life Sciences Greenhouses and, 3) the ongoing support for the Health Venture Investment Account. We very much appreciate the interest of the Pennsylvania House of Representatives and your committee in holding hearings throughout the commonwealth on this vital matter.

First and foremost, as an academic medical center, we at Jefferson look forward to applying for the infrastructure project support and starter kit project support identified in the Salk proposal. At this time when the federal government has held flat or actually cut its support for biomedical research for several fiscal years, we need to rejuvenate our research competitiveness. We do this by investing in new and renovated facilities in order to attract the best and the brightest investigators to Pennsylvania.

Let me give some examples of what we have done, and what we need to do:

In cancer research at Jefferson, by virtue of CURE support, we have been able to attract from Washington, D.C. and New York a new team for our Cancer Center, led by Richard Pestell, MD, PhD. This team is world-renowned in the hormone-responsive cancers, notably breast cancer and prostate cancer. We have leveraged this into a new multidisciplinary Breast Care Center. In addition, our work has led to the formation of a new company with new therapeutic approaches to these cancers.

We attracted from Johns Hopkins University in Baltimore our new Chief of Surgery, Charles Yeo, MD. He is one of the nation’s top experts in pancreatic cancer, a deadly disease unless removed surgically.

Dr. Scott Waldman at Jefferson has taken a basic observation in colon cancer and developed it with CURE support into a major diagnostic test to identify which colon cancers need chemotherapy after surgery. He leveraged this work into a multimillion dollar clinical trial from the National Cancer Institute. Now his team is developing new targeted therapy based on his fundamental observations. All of this progress has been in partnership with a Pennsylvania startup biotechnology firm.

In Cardiovascular research at Jefferson, CURE support enabled us to bring Dr. Arthur Feldman to Jefferson, leading a top team in heart failure research. Together with Drs. Koch and Whellan, attracted from Duke University, they have identified new therapies and launched a consortium of medical centers, mostly throughout Pennsylvania, to improve heart failure care. Dr. Feldman’s diagnostic and therapeutic intellectual property is with a Pennsylvania startup biotechnology firm.

CURE support then enabled Jefferson to attract Dr. Thomas Force from Harvard and Tufts in Boston. Dr. Force led a team which published recently the major newsworthy finding of cardiac disease in some people associated with some newer targeted cancer therapies, such as Gleevec for chronic leukemia. This work will enable clinicians to prevent people from defeating their cancer only to succumb to heart disease. It also opens up unexpected new knowledge into the workings of the heart.

CURE support has enabled Jefferson’s Dr. Bonita Falkner to tackle the cardiovascular problems that follow from our epidemic of obesity. She has spent over 20 years reaching out into the African American and Hispanic American communities to understand and prevent high blood pressure and type II diabetes from wreaking havoc on the blood vessels and heart. In this project, CURE has enabled new community partnerships and interventions on the front lines now, where they’re needed.

In Neurosciences research at Jefferson, new understanding of ALS, also know as Lou Gehrig’s disease, has been undertaken by Dr. Davide Trotti, just recruited from Harvard University. New community outreach to bring the optimal mix of pharmaceutical and non-pharmaceutical care to Alzheimer’s disease patients and their families in their homes has been undertaken by our Dr. Laura Gitlin. New therapeutic approaches to Parkinson’s disease have been undertaken by Drs. Lorraine Iacovitti and Jay Schneider. These new approaches provide the intellectual property underpinning for a new Pennsylvania startup biotechnology company.

In each case, our work moves from bench to bedside to community. In each case, we have rigorously pursued commercialization.

What do our cancer, cardiac and neurosciences doctors need to succeed in the coming 5 to 10 years?

1. They need new state-of-the-art laboratories. We need to undertake a substantial renovation of 1960s-era laboratories for modern molecular and genetic approaches. We need new laboratory animal facilities. Translating gene discoveries from a lab bench to a human clinical trial has a major intervening step, so-called preclinical work. This preclinical work, most often with genetically modified mice, enables our doctors to ensure that new treatments in patients are likely to be safe and effective. The Salk proposal is an ideal way for us to enable this work.

2. They need a clinical trials infrastructure that gets the most promising new treatments integrated into the care of Pennsylvania’s neediest patients. This means new facilities and new jobs to be filled by skilled professionals. We are among the top non-governmental employers in southeastern Pennsylvania. We are explicitly planning to include clinical trials infrastructure into our new ambulatory care center. The Salk proposal is an ideal way for us to enable this work.

3. They continually have new ideas for improved diagnostic tests and therapies. They need access to the financial and business support of the talented economic development folks, exemplified by the regional Life Sciences Greenhouses. We have regular, productive interactions with Barbara Schillberg of BioAdvance and her team. Together with the Science Center, the Ben Franklin Partnership, and the Keystone Innovation Zone, these dedicated folks at our life sciences greenhouse provide an invaluable link between discovery and commercialization. These first steps, out of the academic medical center and into industry, will never succeed without the availability of early venture capital managed by knowledgeable professionals. The wisdom of the Salk proposal in providing "evergreen" support for the greenhouses and venture capital is an ideal way to enable this work.

In summary, we cannot imagine doing our best at Jefferson to grow a robust, sustainable life sciences economic engine for Pennsylvania without the support of the bill you are considering.

Thank you for the opportunity to share my thoughts with you, and I am happy to answer questions.

Steven E. McKenzie, MD, PhD

Vice President for Research

Thomas Jefferson University

M41F Jefferson Alumni Hall

1020 Locust Street

Philadelphia, PA. 19107-6799

Phone: 215-955-3900 Fax: 215-955-4050

steven.mckenzie@jefferson.edu