Drug Discovery

In recent years, academic scientists have become increasingly involved in guiding their own projects through the crucial early stages of drug development, where deliverables from basic and translational research are first tested for their potential to become novel clinical products.

Several initiatives now under way are enabling the University’s scientists to take active roles in therapeutic discovery while maintaining control of their inventions. There are two main reasons to the keep projects in this so-called “pre-competitive space.”

• Academic scientists, with their great breadth of fundamental knowledge, have demonstrated greater success in identifying productive drug targets than more narrowly focused corporate R&D operations.
• Pharmaceutical manufacturers, biotech companies, and venture capitalists now require academic labs to demonstrate that their findings have the potential to generate safe, effective new medicines before they will invest in intellectual property.

Rockefeller now has several programs in place to help its faculty members take an active role in developing biological therapeutic agents and small molecule drugs.

• The University awards proof-of-concept grants in the $50,000 to $300,000 range for early stage efforts aimed at identifying and validating therapeutic targets. Current projects focus on such conditions as Alzheimer’s disease, inflammatory bowel disease, and hearing loss, to name a few.
• Clinical development grants of $1 million or more are available for studies that have advanced to a stage that calls for the manufacture of provisional drugs and/or clinical trials. Radical new approaches to treating HIV/AIDS, cancer, and autoimmune diseases are being funded in this way.
• A separate, inter-institutional drug development initiative is helping investigators to realize their goals by working with medicinal chemists and other experts affiliated with industry partners. This innovative program is proving beneficial to all participants.

Grants are awarded competitively through a process that includes presentations to external review boards comprised of leaders in industry and life sciences investment. The application process is providing important educational experiences for Rockefeller investigators and is helping them to develop the entrepreneurial skills that are essential in today’s changing biomedical field.

Generous benefactors have provided support to launch these initiatives, but new philanthropic partners are needed to sustain and grow them. Gifts to support drug discovery—either unrestricted or focused on specific disease areas—will help to ensure that promising discoveries made in Rockefeller labs have a strong chance of becoming new medicine.