To maintain our leadership we need to invest in what makes us unique, which means investing in young people, thought leaders, and a new way of doing business with core facilities and collaborative projects. A focus on the big science questions with an emphasis on applying the product of that science to helping people, coupled with an innovative structure, will create a new model for how to conduct early stage R&D. This is a problem that neither academia nor industry has yet solved. Specifically, HSCI will build on the platform we have created to date by investing in the following areas going forward:

Seed Grants

Investing in early stage, innovative science and taking reasonable bets on risky science is a hallmark of HSCI's strategy. Doing so is also a vehicle for HSCI to enable junior scientists to launch their careers while pursuing important science that can be a springboard to larger projects.

Disease programs

Our approach is to combine faculty across labs and institutions with a bias toward engaging junior faculty in leadership roles to help them launch their careers as well as move exciting science forward.

Existing programs – we are expanding the scope of each program to include the relevant seed grants, seminars, events, "think tanks", and other community building activities in addition to the individual program projects.

New programs – we will start new programs when there is a confluence of a critical mass of investigators and exciting new work in the field.

Core Facilities

The Therapeutic Screening Center has proved the value of using stem cells as tools to find drugs that could be therapeutic compounds themselves or that could stimulate endogenous stem cell replication. We will expand this facility and link its operations with the new iPS Core to analyze specific diseases. We expect that this combination of novel biology with novel disease models will help us identify new drugs with a higher success rate. The ability to develop pluripotent cells from specific population phenotypes is an important advance in understanding human disease and developing new therapeutics. Through the iPS Core Facility, we will continue to invest aggressively in this new technology because it offers great promise for novel approaches in regenerative medicine, including human cell-based assays for target validation, lead development screens, efficacy and toxicity testing, functional pharmacogenomics and personalized medicine.

Clinical studies

This past year HSCI funded its first pre-clinical trial of a cell-based therapy – the creation of an autologous tendon for repair of neonatal diaphragmatic hernia. This is an example of several aspects of HSCI's operations:

• the importance of the connection between the clinic and the basic research as driven by a real clinical need,
• the need for a non-profit organization to respond to unmet medical needs in markets that are too small for companies to address until a solution is available,
• the increased readiness of selected cell therapies for the clinic,
• the use of Harvard's Center for Human Cell Therapy as the coordinating mechanism and leveraged resource for HSCI when entering the clinic, negotiating with the FDA, and planning trials.

Education

Because of the important connection between "bench" and "bedside" clinician scientists are critical to the future of the field. For several years, HSCI has supported the last two years of a MD/PhD student. We recently expanded this program to provide grant support for newly minted MD/PhDs and for Instructors a few years into their careers to help create and build their independent labs. Similarly, educating new citizens and new scientists is critical to the success of the field. HSCI's Undergraduate Intern Program (HIP) continues to be one of our most successful programs in support of this aspect of mission. Both students and faculty have described this program as exciting and, in some cases, even life-changing.

In summary, we will:

• Build on existing disease programs
• Enhance junior faculty leadership opportunities
• Expand unique centers of excellence
  • iPS Core
  • Therapeutic Screening Center
• Accelerate pre-clinical and clinical work
• Expand relationships with the commercial sector
• Leverage intellectual property creation
• Attract new faculty and develop university resources through the Department of Stem Cell and Regenerative Biology