From laboratory to Phase I/II cancer trials with recombinant biotherapeutics

Area of Science:

• Biotechnology
• Cancer Therapeutics
• Regulatory Compliance

Background:

• Academic research generates promising recombinant cancer medicines.
• Translating these into clinical trials requires adherence to Good Manufacturing Practice (GMP).
• Lack of GMP experience is a significant barrier for early-stage clinical translation.

Purpose of the Study:

• To provide a step-by-step guide on achieving GMP for recombinant protein production.
• To demonstrate that GMP standards can be met at a small scale within academic institutions.
• To facilitate the translation of experimental cancer therapies into clinical trials.

Main Methods:

• Illustrating GMP principles with a practical, step-by-step approach.
• Utilizing the yeast Pichia pastoris for expression of an antibody-based therapeutic.
• Purifying the recombinant protein to meet clinical trial standards.

Main Results:

• Successfully demonstrated that GMP can be achieved on a small scale in a research setting.
• Produced a purified antibody-based therapeutic compliant with GMP standards.
• The produced therapeutic was safely used in patients during Phase I/II trials.

Conclusions:

• GMP compliance for recombinant cancer therapies is achievable within academic research settings.
• This approach can overcome a major obstacle in translating experimental medicines to the clinic.
• The presented principles are broadly applicable to various recombinant proteins for early-phase cancer trials.