Some special sessions that we have introduced in the past years, were again met with great enthusiasm, and were highly rated by attendees. These sessions were perfectly within the EBMT spirit and goals. EBMT delivers a comprehensive portfolio of educational activities covering the needs of both the youngest investigators (“How to get published”) and the most experienced ones (“Best clinical trials of the year”). EBMT is also a global transplant and cellular therapy society. The session entitled “EBMT global collaborations: East and West raising the bar together” allowed top scientists from all over the globe to exchange experiences for the benefit of patients. Please see below to read the outcomes of the following special sessions.
The EBMT Legal and Regulatory Affairs Committee special session
The Legal and Regulatory Affairs Committee (LRAC) was established in mid-2017 as part of the EBMT’s increasing interaction with the European Union and the European Medicines Agency. The Annual Meeting in Lisbon was perhaps its first full presentation in public when it was included in the programme for the Cell Therapy Day. Compared to the earlier cell therapy sessions, the room was quieter, perhaps an sadly accurate reflection of congress attendees’ priorities, but still proved to be a very interesting session with Esteve Trias’ explanation of the EuroGTP II project
, an update on the evaluation of the Cell and Tissue Directives
by Deirdre Fehily of the European Commission and an interesting talk on how can we facilitate the delivery of advanced therapies to patients (including the REGenableMED project
by Aurelie Mahalatchimy.
One view is that currently three issues dominate:
- data to support regulatory and health technology assessment needs
- regulations of existing and novel therapies
- costs and access to care.
The EBMT considers that it has a multi-faceted role in relation to these issues and regulators:
- The EBMT network is made up of the experts in these diseases (many qualify as rare diseases) and their treatments
- The EBMT registry is a unique resource in relation to these treatments for research and follow-up purposes
- The EBMT has demonstrated its commitment to safe and efficient care through the successful JACIE standards and accreditation programme
- International collaboration is in EBMT’s ‘DNA’ since its foundation in the early 1970’s.
EBMT is a volunteer-led professional society with limited staff resources and so the greatest challenge faced by organisations like EBMT is their real capacity to engage and interact in a meaningful and consistent way. To date we have probably ‘punched above our weight’ and have achieved recognition by European regulatory bodies of our expertise and resources such as the Registry. In recent years, the society has taken a more active line on access to new therapies through issuing a number of firm position statements. Alliances have been formed with similar organisations in the area of Substances of Human Origin (SoHO) to raise our profile as professional societies. However, further mobilisation of resources will be necessary in the future to build and maintain momentum and to demonstrate ourselves as a steady and reliable stakeholder or even to assume a role as a leader among our sister societies. A certain amount of creative thinking will be necessary, not least of all, to better share the workload among a wider group of motivated experts.
If professionals do not involve themselves in regulatory issues, they run the risk of being ‘ambushed’ by regulations that will not facilitate their work as physicians and investigators. The EBMT has managed to ‘open a door’ to the European institutions in recent years on the basis of goodwill and responsiveness but more work needs to be done to keep these channels open in the future so that the EBMT will continue to be considered a valued stakeholder. Therefore the LRAC warmly welcomes interest and participation from experts from within the EBMT network.
For further information, please write to Prof. Christian Chabannon email@example.com
or Eoin McGrath firstname.lastname@example.org
CAR-T cells and beyond: future perspectives
CAR-T cells were certainly one of if not the hottest topic covered at this year Annual Meeting. Many sessions included or were entirely devoted to the presentation of preclinical or clinical results obtained with this new class of medicinal products. This special session - chaired by the current and past Presidents of the Local Organizing Committees for the two latest editions of the EBMT Annual meetings - attempted to illustrate three of the most pressing issues in the field:
- Beyond already demonstrated clinical efficacy when targeting CD19, and to a lesser extent BCMA, are there evidences that other tumour antigens can be efficiently targeted, and thus that other types of blood malignancies can be controlled through CAR-T cell therapies?
- Are other categories of immune cells than T-cells amenable to genetic manipulations leading to express CAR and enhance anti-tumour recognition / cytotoxicity?
- Are there alternatives to costly manufacturing by industry in order to facilitate patients’ access to CAR-T Cells, and improve sustainability for healthcare payers and our societies at large?
Dr Carlos Ramos from Baylor College of Medicine and Texas Children’ Hospital in Houston, TX, USA, beautifully illustrated the first issue, through his presentation of CAR-T Cells that target CD30. He presented the result of a pilot clinical trial, demonstrating acceptable tolerance and documenting response in a fraction of patients with Hodgkin’s disease or CD30+
non-Hodgkin’s lymphoma. He also discussed factors that could improve CAR-T Cells efficacy by overcoming the tumour / tumour microenvironment immune suppression mechanisms or by improving CAR- T cells trafficking to tumour sites.
Dr Kathy Rezvani from MD Anderson Cancer Center, Houston, TX, USA, then showed that Natural Killer cells are also amenable to genetic engineering, and can express a chimeric antigen receptor. Of particular interest is the possibility to manufacture “off-the-shelf” – and thus readily available – allogeneic immune cellular therapy medicinal products with potentially reduced alloreactivity as compared to allogeneic T cells. Dr Rezvani presented results of a phase I/II clinical study where third party CAR-NK cells targeting CD19 were manufactured from umbilical cord blood.
Finally, Dr Alvaro Urbano-Ispizua from Hospital Clinic, University of Barcelona, Spain presented his team’s experience with the manufacturing of autologous CAR-T cells in an academic facility complying with good manufacturing practices and other sets of rules that apply to substantial cell processing / gene engineering of hematopoietic cells. This allowed treating 15 patients with CD19+
lymphoid malignancies, age 3 – 54, with an apparently comparable range of efficacy and side-effects as has been reported in the literature during the conduct of industry-sponsored clinical trials.
The CAR-T cell field is fast growing, in particular due to large investments made by pharma companies in the field of cellular therapies, but also because of relentless efforts of academic research groups that conduct both preclinical and clinical work. It has brought the demonstration that CAR-T cell therapies can induce responses and potentially cure a significant fraction of patients affected with advanced or poor prognosis lymphoid malignancies. The packed room for this session was a testimony that the field is eagerly awaiting further information supporting the possibility of a commercial success and a broader applicability for other types of malignancies. There is no doubt that the next editions of the EBMT Annual Meeting will provide a forum where progress will be discussed and raise high interest from delegates.
Social Media Session
The social media session reflected the high level of engagement that occurred on the twitterverse at #EBMT18. Highlights of the session included a perspective by 2017 EBMT Young Ambassador Dr. Khaled Ghanem - @khaledghanem, who reflected on the opportunities that have come his way through the cloud and the positive role of virtual networking on advancing his career. Attendees then participated in a robust panel discussion with Ms. Cynthia Chmielewski - @MyelomaTeacher, a multiple myeloma patient who has successfully leveraged social media as a tool for advocating for and sharing medical information with patients, and with BMT Twitter experts Prof. Mohamed Mohty - @Mohty_EBMT, Prof. Miguel Perales - @DrMiguelPerales, and Prof. Jaap Boelens - @JJ_boelens. Questions ranged from the basic “how to use social media” to very thoughtful discussions on managing physician-patient relationships and maintaining professionalism on various social media platforms. To top off things, Ms. Melanie Chaboissier - @TheEBMT, gave a behind the scenes tour of how the EBMT uses social media to promote and share information and its activities. There was a lot of “tweeting” at the session, which was hopefully noticed by conference attendees, and was definitely heard worldwide (on Twitter)!
By Prof. Navneet Majhail @BldCancerDoc
Watch the short video about #EBMT18, a hashtag in review