Live biotherapeutic products (LBPs) represent an innovative class of medicines that use living organisms for therapeutic purposes. These unique products stand apart from conventional pharmaceuticals because their active components are living microbes, primarily bacteria or yeasts. The development of LBPs marks a significant shift in how diseases are approached, moving towards interventions that interact with the body’s complex biological systems. This emerging field holds promise for addressing various health conditions by leveraging the body’s intricate microbial relationships.
What Are Live Biotherapeutic Products?
Live biotherapeutic products are defined as biological products containing live organisms, such as bacteria or yeasts, intended for the prevention, treatment, or cure of a disease or condition in human beings, and are not considered vaccines. Regulatory bodies like the U.S. Food and Drug Administration (FDA) classify them as medicinal products. Unlike common probiotics, which are dietary supplements, LBPs are developed under rigorous pharmaceutical standards with specific therapeutic claims and undergo comprehensive safety and efficacy testing. LBPs can consist of single or multiple microbial strains, and some are even genetically engineered to achieve specific therapeutic effects.
How Live Biotherapeutic Products Function
Live biotherapeutic products exert their effects through intricate interactions within the host’s body. Many LBPs primarily act by modulating the host’s native microbiota, influencing the balance and activity of microorganisms already present. This can involve inhibiting the growth of harmful pathogens, a process known as competitive exclusion.
Some LBPs produce beneficial metabolites, such as short-chain fatty acids, which can reduce inflammation. These products can also modulate the immune system, either locally within the gut or systemically, by interacting with immune cells and signaling pathways. Additionally, LBPs may enhance barrier functions, such as strengthening the intestinal lining.
Applications of Live Biotherapeutic Products
Live biotherapeutic products are being investigated across a broad spectrum of therapeutic areas. One prominent application is in treating recurrent Clostridioides difficile infection (CDI), a serious gastrointestinal condition often exacerbated by antibiotic use. Two microbiome-based therapeutics have recently received FDA approval for preventing recurrent CDI.
Beyond infectious diseases, LBPs are also explored for gastrointestinal disorders like inflammatory bowel disease (IBD) and irritable bowel syndrome (IBS), where they aim to restore microbial balance and reduce inflammation. Research is also underway in metabolic disorders such as obesity and type 2 diabetes, where LBPs could potentially regulate metabolic functions and restore gut microbiota balance. Furthermore, there is growing interest in their application in oncology, with some LBPs showing promise in modulating the gut microbiome to enhance the efficacy of immune checkpoint inhibitors in cancer treatment.
Developing and Regulating Live Biotherapeutic Products
Bringing live biotherapeutic products to market involves a rigorous development pathway. Manufacturing LBPs requires specialized expertise in identifying, growing, and maintaining live microorganisms under controlled conditions. Quality control is important to ensure the consistency, purity, and potency of these living drug substances.
The regulatory landscape for LBPs is continuously evolving, with agencies like the FDA providing guidance for investigational new drug (IND) applications. Sponsors must submit comprehensive chemistry, manufacturing, and control (CMC) information, adhering to specific regulations for biological products. This stringent regulatory oversight, including extensive preclinical and clinical trials, ensures the safety and efficacy of LBPs before patient use.