Living Drugs: What's Hot in 2023

Consensus on C&G technologies and data sharing, and a new generation of CAR therapeutics

Standardizing analytical methods required for cell and gene therapy processes and products will precede their widespread launch.

Worldwide the market of so-called living drugs could triple from roughly US$ 8 billion to US$ 24 billion by 2030. Apparently, the transfer of genetic and cellular properties can be shaped into products for treatments of common and rare diseases alike. For example, chimeric antigen receptor (CAR) T-cell therapies against blood cancers have already been approved for the clinic, mostly for lymphomas. But there is hope of expanding this concept to cure solid tumors as well as neurogenerative conditions like Alzheimer’s, Parkinson’s, and Multiple Sclerosis.

With all these novel modalities drug developers must review strategies in place. That includes pharmacological considerations of safety and efficacy. What has worked for small molecule or antibody-based candidates may not be easily transferable to autologous and allogeneic products. Before the industry can commercialize on this therapeutic potential an adapted set of bioanalytics and quality control metrics need to be considered, established, and standardized. Stakeholders are looking for consensus on technologies and data sharing management, on best practices and processes to follow, so that these types of living drugs can be reliably characterized, and their manufacturing be upscaled and controlled from early development to clinical trial needs.

--Christoph Eberle, PhD, Principal Scientist III, In Vitro Pharmacology/Oncology, Charles River

Future CAR-cell therapeutics may harness members of our innate immune system

By now we have all read stories about how CAR-T cell therapies are revolutionizing how we fight disease. The early chimeric antigen receptor or CARs reprogram a patient’s T cells – one weapon in our immune system—to fight cancers, specifically lymphomas and other blood cancers. Could solid tumors be the next battlefield for CAR-cell therapeutics? May researchers think so, but they are eyeing a different arm of the immune system as the way forward. A number of clinical trials are testing CARs that harness macrophages or natural killer cells, components of our innate immune system that are among the first of our immune cells to respond when you are sick or injured. Both natural killer cells and macrophages avoid some of the toxicity, manufacturing, trafficking and other issues that hinder CAR-T cell performance. For instance, one preclinical study finds that CAR-macrophages infiltrate efficiently into tumors and abundantly while another clinical trial showed the efficacy and tolerability of a CAR-NK therapy in the clinic. Both macrophages and NK cells have their own limitations, of course. No therapy will be the ultimate magic bullet. But look next year and beyond to significant interest and movement in developing CAR-NKs or CAR macrophage therapy for breast cancer and other solid tumors.

-- Ulrike Herbrand, PhD, Scientific Director, Global In Vitro Bioassays, Charles River

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