Clinical program director: Prof. dr. Josef Vormoor
Clinical program
Within our hemato-oncology program, we treat children and young people with leukemia, lymphoma and certain other related blood disorders, such as bone marrow failure and histiocytosis. Our stem cell transplantation team (a collaboration with the Wilhelmina Children’s Hospital) is embedded within our program.
Our ultimate goal is to cure every child with leukemia/lymphoma with optimal quality of life. Close links between research and clinical care are essential to enable innovation and we have established several disease specific and cross-cutting teams via our Máxima Comprehensive Childhood Cancer Center (M4C). Through these M4C groups, we run a broad portfolio of innovative clinical trials.
A key focus of our program has been the implementation and development of immunotherapy (including cellular immunotherapy, such as CAR T cell therapy) for children with hematological malignancies.
Dissecting and targeting the bone marrow envrionment
Josef Vormoor's research is focused on understanding the interactions between acute lymphoblastic leukemia (ALL) blasts and their microenvironment. Using single cell sequencing, we have identified adipocyte progenitors as a novel key niche component in ALL. We also study how RAS mutations help ALL cells to reprogram their (immune) micro-environment. In our Research Institute, we work closely together with a group of top scientists, all with a common focus on the bone marrow micro-environment: Prof. Monique den Boer, dr Mirjam Belderbos and Prof. Olaf Heidenreich. Our mutual aim is to identify vulnerabilities in the interaction of the leukemic blasts with the bone marrow environment that can be exploited for novel therapies.
2. George SL, Lynn C, Stankunaite R, Hughes D, Sauer CM, Chalker J, Waqar Ahmed S, Oostveen M, Proszek PZ, Yuan L, Shaikh R, Jamal S, Brew A, Tall J, Rogers T, Clifford SC, Vormoor J, Shipley JM, Tweddle DA, Jones C, Willis C, Burke GAA, Vedi A, Howell L, Johnston R, Rees H, Adams M, Jesudason A, Ronghe M, Elliott M, Ross E, Makin G, Campbell-Hewson Q, Grundy RG, Turnbull J, Wilson S, Lee V, Gray JC, Stoneham S, Gatz SA, Marshall LV, Angelini P, Anderson J, Cresswell GD, Graham TA, Al-Lazikani B, Cortes-Ciriano I, Kearns P, Hutchinson JC, Hargrave D, Jacques TS, Hubank M, Sottoriva A, Chesler L. Stratified Medicine Paediatrics: Cell free DNA and serial tumour sequencing identifies subtype specific cancer evolution and epigenetic states. Cancer Discov. 2024 Dec 19. doi: 10.1158/2159-8290.CD-24-0916.
3. Rossig C, Pearson AD, Vassal G, Scobie N, Bird N, Blanc P, Vormoor HJ, Calkoen FG, Locatelli F, Bufalo FD, Rives S, Jacoby E, Balduzzi A, Bourquin JP, Baruchel A; International BFM Study Group (I-BFM), Innovative Therapies for Children with Cancer (ITCC), and the European Society for Paediatric Oncology (SIOPE). Chimeric Antigen Receptor (CAR) T-Cell Products for Pediatric Cancers: Why Alternative Development Paths Are Needed. J Clin Oncol. 2024 Jan 20;42(3):253-257. doi: 10.1200/JCO.23.01314. Epub 2023 Nov 27.
4. Slade D, Menne T, Irving J, Johnson S, Kearns PR, Plummer R, Savage J, Shenton G, , Veal GJ, Vormoor B, Vormoor J*, Billingham L*. Selumetinib in combination with dexamethasone for the treatment of relapsed/refractory RAS-pathway mutated paediatric and adult Acute Lymphoblastic Leukaemia (SeluDex): Study protocol for an international, two-phase, two-group, dose finding with expansion phase I/II trial. BMJ Open. 2022 Mar 4;12(3):e059872. doi: 10.1136/bmjopen-2021-059872. *Common senior authors
5. Pal D, Blair H, Boyd S, Angel HS, Nizami S, Isa A, Beckett M, Nelson R, Wilson A, Singh M, Sankar S, Tirtakusuma R, Sirintra N, Knill C, Fuller A, McNeill H, Russell L, Schwab C, Zhous P, Sinclair P, Coxhead J, Filby A, Halsey C, Allan JM, Harrison JC, Moorman A, Heidenreich O, Vormoor J. An ex vivo human bone marrow milieu identifies CDH2 as a driver of niche-mediated treatment resistance in leukaemia. Cell Rep Med. 2022 Aug 16;3(8):100717. doi: 10.1016/j.xcrm.2022.100717.
