Abstract 1009P
Background
Among novel strategies for treatment of solid tumors (ST), cell and/or gene therapy (CT/GT) products are increasingly under investigation, also based on encouraging results observed in lymphoid cancers with industry-manufactured CAR-T-cells. Available information on ongoing studies in ST is limited, due to the variety of programs and infrastructures involved in advanced therapy medicinal products (ATMPs) manufacturing and delivery.
Methods
This study aimed to describe the current landscape of CT/GT developments for treatment of ST from Jan 2018 to Dec 2020 by means of a web-based questionnaire circulated within the ESMO and EBMT centers.
Results
147 questionnaires were returned from 53 countries, 22% of the respondents were involved in CT/GT trials during the study period and 16% indicated their intention to start a CT/GT program. The majority of the active centers treated only adults (88%), while a minority exclusively or partially treated children (12%); 50% of the centers treated 1-5 pts, while a quarter enrolled more than 20 pts. Almost half of the studies were dedicated to melanoma or lung cancer; GI tract tumors, bone sarcomas, head & neck and gynecological cancers were also targeted. Evaluated ATMPs were mainly ex-vivo manipulated T lymphs, cultured and, in more than 50% of the cases, gene-modified either with CAR sequence or TCR transgene. TILs were the most frequently used non-gene modified products. In as many as 67% of the cases, ATMPs were combined with other treatment modalities, largely represented by ICIs. Small scale cell and gene engineering was mostly performed onsite by point-of-care manufacturing facilities. A minority of the studies were supported by EU funding.
Conclusions
Our survey shows that, although increasingly used, gene-modified T-cells represent little more than 50% of ATMPs employed in ST. Many clinical trials are based on point-of-care ATMPs production at academic centers, although industry-sponsored trials are running in at least half of the centers. In perspective, while waiting for breakthrough cellular products to treat ST, the field may benefit from network models for ATMPs production in academic centers.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
S. Peters: Consultation/Advisory role: AbbVie, Amgen, AstraZeneca, Bayer, Beigene, Biocartis, Bio Invent, Blueprint Medicines, Boehringer Ingelheim, Bristol-Myers Squibb, Clovis, Daiichi Sankyo, Debiopharm, Eli Lilly, Elsevier, F. Hoffmann-La Roche/Genentech, Foundation Medicine, Illumina, Incyte, IQVIA, Janssen, Medscape, Merck Sharp and Dohme, Merck Serono, Merrimack, Mirati, Novartis, PharmaMar, Phosplatin Therapeutics, Pfizer, Regeneron, Sanofi, Seattle Genetics, Takeda, Vaccibody; Talk in a company’s organized public event: AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb, e-cancer, Eli Lilly, F. Hoffmann-La Roche/Genentech, Illumina, Medscape, Merck Sharp and Dohme, Novartis, PER, Pfizer, Prime, RTP, Sanofi, Takeda; Receipt of grants/research supports: (Sub)investigator in trials (institutional financial support for clinical trials) sponsored by Amgen, AstraZeneca, Biodesix, Boehringer Ingelheim, Bristol-Myers Squibb, Clovis, F. Hoffmann-La Roche/Genentech, GSK, Illumina, Lilly, Merck Sharp and Dohme, Merck Serono, Mirati, Novartis, and Pfizer, Phosplatin Therapeutics.