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There are hundreds of FACT and JACIE accredited clinical centers worldwide where thousands of dedicated technologists and their peer mates toil day-in, day-out in cell processing facilities supportive of hospital-based cell therapy programs.  To this hospital-based workforce an ever-expanding commercial cell manufacturing CDMO (contract development and manufacturing organization) ecosystem is added as well as mushrooming clinical-stage cell therapy pharmaceutical developers that require the same talent pool to deliver hands-on technical skill to advance cell therapeutics to the clinic.

Indeed, ever since the inception of FACT more than a quarter century ago, the technical landscape of cell processing and manufacturing has evolved from the handling of minimally-manipulated marrow and leukapheresis collected hematopoietic stem cells to a wide range of more-than-minimally-manipulated (MTMM) bespoke investigational cell therapies manufactured at place of care, including CAR-T, TILs (tumor infiltrating lymphocytes), VSTs (Virus Specific T-cells) and MSCs (Mesenchymal Stromal Cells) to name a few.  In addition, cell therapy platforms including ES (embryonic stem cell) and iPSC-derived off-the-shelf products are being developed at commercial scale.  All of these MTMM investigational therapies are regulated by FDA as drugs and require INDs (Investigational New Drug) licenses in USA and the equivalent in other national regulatory jurisdictions.  The hospital-centered, place-of-care cell processing has evolved from artisanal practice of medicine to GMP-grade processes now widely adopted by healthcare enterprises and hub-centric commercial developers.  This rapid evolution creates a huge demand for upscaling of skill sets for cell processing technologists and their QA (quality assurance) and regulatory teammates part of multidisciplinary manufacturing/clinical teams engaged in IND-sponsored clinical trials and later deployment of commercially approved cell therapies.

The ISCT is unique amongst its peer organizations for its historic focus on the practicalities of distributed clinical-grade cell processing, training, quality controls and regulatory compliance.  This theme informs in good part the content of ISCT regional meeting offerings in Australia, Asia, US and Europe with the goal of accessible and affordable workforce training for technologists, quality assurance and regulatory specialists in sharing of best practices and knowledge transfer.  This was reflected in the content of the August 2023 ANZ ISCT regional meeting in Perth, Australia co-chaired by Emily Blyth and Zlatibor Velickovic and most recently in the successful North American ISCT regional meeting held in Houston September 2023 co-chaired by Patrick Hanley and Jaap-Jan Boelens.

Focusing on the Houston event, more than 419 delegates attended, mostly (91%) from the US with representation from an additional 14 member countries. Lab/Manufacturing/Quality/Regulatory expertise accounted for 56% of attendees evenly split from academia and industry.  More than 65% of delegates were new to ISCT, speaking to the expanding interest in this type of offering. 

The impact of staff engagement is hard to measure but is unmistakable.  From my own PACT (Program for Advanced Cell Therapy) group in Wisconsin, Jessica Murphy is one of our Process Development and Manufacturing Scientist and has an ISCT laboratory membership.  She did not make it to the ISCT Paris 2023 meeting and pivoted to the Houston meeting, capitalizing on the convenience and cost effectiveness of domestic travel and housing.  She had a poster and gave an oral presentation and was able to network professionally and scientifically with peers in a manner that is simply not accessible in other meeting formats for those with pragmatic interests in clinical cell processing.  Her feedback on the meeting mirrors that obtained from post meeting surveys where meeting size is appealing for networking and discussions and the high level of interaction within sessions and 96% respondents would recommend ISCT Regional Meetings to their colleagues/peers.. Reflective of ISCT’s mixed academic/industry membership, industry participation is unique and adds value to the experience had by technologists from both ecosystems.  Specific sessions or topics that attendees found particularly valuable were lab operations/regulatory/QC as well as Point of Care sessions and topics.   

The themes of Point of Care and distributed manufacturing are clearly trending hot, both from a research and pragmatic perspective for commercial developers and hospital-based cell manufacturing.  Indeed, the top two plenary sessions, as measured by attendance were “State of off-the-shelf cellular therapies in the clinic” and “The Current Reality and Future of Point of Care Manufacturing”.

I’ll dwell on the latter theme of “point of care” manufacturing.  The matter has recently been reviewed by Magdi Elsallab and Marcela V. Maus in a comprehensive manner worth the read[i].  In essence, there are two convergent trends in the autologous or bespoke (one patient/one product) cell therapy space.  Commercial cell therapy developers are exploring alternative cell manufacturing operations from the traditional centralized “hub” model, to novel distributed regional point-of-care adjacent sub-hubs, in an effort to address the particular logistic requirements of bespoke living cell therapies.  To this commercial distributed trend, an intersect is emerging with the grass roots hospital-based manufacturing model built upon the foundation of FACT/JACIE accredited place-of-care cell processing laboratories.

This evolution in cell pharmaceutical manufacture and deployment were discussed from the hospital place-of-care (POC) perspective in a lively plenary chaired by Sarah Nikiforov and yours truly where Kim Schultz (FDA), Nirav Shah (Medical College of Wisconsin) and Yongping Wang (UPenn) gave their respective perspectives on feasibility and sustainability of GMP-compliant CAR-T POC manufacture.

The next step-change for this POC deployment scheme is whether it is compatible with FDA marketing approval and latter margin friendly reimbursement from payors  Interestingly, the FDA recently granted the first ever BLA for POC manufactured Landitra[ii] which is an allogeneic islet cell therapy for brittle diabetes.  The sponsor is CellTrans (https://www.celltransinc.com/) which partnered with University of Illinois in Chicago where cell processing takes place.  This precedent may provide a marketing approval roadmap within the current regulatory FDA framework for other POC, hospital deployed CGTs and also creates the opportunity for innovative industry/healthcare provider margin-friendly partnerships for sustainable patient access to life saving living cell therapies.

The mission critical contribution of an engaged technical workforce is central to success of all of these plausible commercial deployment schemes and the mission of ISCT in meeting that goal is exemplified by forthcoming regional knowledge transfer activities to be held in near future including the ISCT ANZ regional meeting August 2024 to be held in Queenstown, NZ and the ISCT Europe regional meeting September 2024 in Gothenburg, Sweden. 

Importantly, the abstract submission window is now open for the forthcoming ISCT 2024 international meeting in Vancouver, May 2024. All members, including technologists, are encouraged to contribute!

Jacques Galipeau, MD
President, ISCT
Associate Dean for Therapeutics Development 
University of Wisconsin School of Medicine and Public Health

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