CSO Perspectives: Looking Ahead to Having a Bigger Impact and Greater Success

By Bryan Wolf, MD, PhD, Chief Scientific Officer and Director of the Research Institute

I always look toward the new year with great optimism and hope, to the promise and potential it brings, and to the experiences that will shape us individually, professionally, and organizationally.

When I reflect upon the past year at the CHOP Research Institute and look at our plans moving forward, I will say we have a lot to be excited about. We recently completed the tremendous amount of work that came with our strategic planning process. That involved taking a close look at how we function as an Institute, at our various programs and organizational strengths, and making honest assessments of what we can improve upon, where we want to go, and how to get there. S

o what will guide us in the coming year and beyond? Where are we going as an institution, and what are the driving factors and goals that will move us forward?

It comes down to two things: impact and success.

On the surface, that may seem simple and obvious. After all, our mission has always been one that focuses on making an impact on the health and well-being of children, and we have historically used that as a marker of our success.  But when I refer to impact and success, I am talking about something deeper — a concentrated effort to support research programs and initiatives that have both a high impact on the lives of children as well as a high likelihood of success.

We can look to our experiences with our gene therapy, genomics, and cancer programs as examples of those with a high impact and — as demonstrated over time — a high likelihood of success.

Looking ahead, we have identified four big areas of research that have the potential to have a similar trajectory in terms of impact and success. Those four areas aim to:

• Understand the causes and develop cures for rare and complex diseases
• Advance CHOP’s leadership in novel therapeutics and device development
• Innovate in research along the fetal-adult continuum, also known as lifespan research
• Develop breakthrough precision health research

Our effort with respect to rare and complex diseases will focus on developing a basic science recruitment program for the next several years in the targeted areas of computational and quantitative biology, epigenetics/epigenomics, bioenergetics, metabolomics, and developmental and cell biology. We will also fortify our partnership with Penn’s basic science departments and invest in our “big data” and quantitative capabilities.

Novel therapeutics presents several exciting opportunities for the CHOP Research Institute. The most promising area of potential therapies to emerge from CHOP rests with biologic therapeutics, and our future success will focus on more basic science capabilities. We will also place heavy emphasis on entrepreneurship and the development of pediatric drugs and devices. CHOP is positioned to become the international leader in pediatric devices and can serve as the place for first-in-pediatrics drug trials.

Our lifespan research initiative will have a multidisciplinary research approach that links pediatric and adult groups through clinical partnerships. We will work with various institutions on this program, which will have designated approaches to developing and integrating various diverse datasets that span the continuum from genetic to healthcare utilization. Our aim with this program is to prevent adult diseases through interventions in children and younger adolescents before symptoms emerge. In addition, our lifespan research will aim to provide better long-term outcomes for children living with disease.

The fourth aim, on precision health, focuses on getting the right preventive or therapeutic intervention to the right person at the right place and time. To achieve this, the CHOP Research Institute will partner with economists, engineers, public health researchers, and mathematicians to join our growing enterprise. We will also develop a pediatric knowledge network to support lifespan and precision health studies and trials, and establish new core programs — like community-based research and mHealth/digital health research — to support precision health research.

When we look at these ambitious goals, it is clear we are ushering in a new era of research, one that facilitates research and innovation breakthroughs while enabling CHOP to function as a high-performing Research Institute. These are exciting times, and I hope you share my enthusiasm for where we are headed. With your continued support and dedication, the CHOP Research Institute will help solve the most challenging problems in child health — and what tremendous hope and excitement that brings!

I wish you and your families a happy and healthy 2017!