Lost in Translation: Overcoming Roadblocks in Translating New Therapeutics to the Clinic


Many hematologists/oncologists participate in the identification and development of new therapeutic approaches for hematological diseases, but they may lack knowledge or encounter barriers that impede successful clinical translation of promising technologies. In a format new to ASH, this session will be an actively moderated panel discussion using real-world examples to highlight key barriers in drug development. Two case studies will be presented, one focused on small molecule development and the other on cell therapy development, as a means of highlighting common roadblocks and challenging issues faced in the translation of experimental therapies into practice. A panel of experts drawn from academia, industry, business, and the U.S. Food and Drug Administration (FDA) will discuss these obstacles and how they might be overcome.

Dr. Andrew Roberts will discuss the development of venetoclax, a specific inhibitor of BCL2. From an academic perspective, the development of BCL2 inhibitors was complicated by both the failure in clinical trials of prior compounds putatively targeting BCL2 and by competition from big pharma. The case of venetoclax raises issues of deciding when a therapeutic target is worth pursing, how and when to partner with industry, and grappling with challenges in early clinical trials.

Dr. Colleen Delaney will discuss the development of non- human leukocyte antigen matched, ex vivo expanded cord blood cellular therapies. She will describe her path translating basic discovery to investigational new drug-enabling studies, chemistry, manufacturing, and control development and current good manufacturing practices in academia to support early clinical trials and the ultimate decision to spin this technology out of the Fred Hutchinson Cancer Research Center into a new start-up. This case study will examine lessons learned and the perceived and real hurdles faced on this path from academics to industry as well as address the decision-making that goes into beginning a biotech startup. She will examine issues related to the relationship between the academic institution and the startup and navigation of the 'valley of death' (financing in early stages when cash flow is a significant problem), among other issues faced by a biotech startup.

Dr. Gideon Bollag will provide the industry prospective on collaborating with academic institutions and highlight differences between drug development approaches in academia and industry.

Dr. Rosana Kapeller will comment on the cases presented by Drs. Delaney and Roberts from the point of view of the entrepreneur, explaining the role of the investment community in drug development and discussing what characteristics make a new therapeutic approach appealing or unattractive from the business prospective.

Dr. Ann Farrell will address the regulatory process for small molecules and cell-based therapeutics (e.g., obtaining an Investigational New Drug (IND), responding to FDA review, etc.) and will discuss common regulatory hurdles.


  • Rosana Kapeller
  • Gideon Bollag
  • Ann Farrell


Member Type Price
List Price $50.00
Active, International, Emeritus, and Honorary Members $35.00
Associate, International Associate, Student, and Resident Members $35.00
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