Josef Vormoor

Key publications

• Key publications

  Key publications

  1. Vormoor B, Savage J,Kristunas C, Johnson S, Ferrao Blanco MN, Shenton G, Lancaster D, Castleton A, Fielding AK, Latif A_L, Morley N, Strullu M, Tuckuviene R, Kearns P, Jennings C, Barnett S, Veal GJ, Irving J, Billingham L*, Menne T*, Vormoor J*. Responses can be achieved with a combination of the MEK inhibitor selumetinib and dexamethasone in patients with relapsed/refractory RAS-pathway mutated acute lymphoblastic leukemia: Results of a parallel cohort, dose-finding and expansion phase I/II trial. EJC Pediatric Oncology. 2026 in press. *common senior authors

  Study results of our SeluDex trial: despite recruitment challenges, the trial achieved a 44% response rate (CR/CRi) in a small highly refractory cohort, confirming our preclinical hypothesis and providing a rationale for further evaluation of MEK inhibitors in ALL.

  2. Derevyanko PK, Swart LE, Mata Casimiro LD, van Oort A, du Plessis M, van den Brink L, Ashtiani M, Zwaan CM, Krippner-Heidenreich A, Bonifer C, Schiffelers R, Vormoor J, Kellaway SG, Heidenreich O. Fusion Gene Depletion Eliminates Stemness and Induces Bidirectional Differentiation of Acute Myeloid Leukemia. Blood. 2025 Dec 11;146(24):2963-2978. doi: 10.1182/blood.2025028988.

  From my research collaboration with Professor Heidenreich: here, we demonstrate the essential role of RUNX1::RUNX1T1 in driving and sustaining the leukemia, confirming this oncogenic fusion gene as a suitable target for therapy.

  3. Ferrao Blanco MN, Kazybay B, Belderbos M, Heidenreich O, Vormoor HJ. Distinct Stromal Cell Populations Define the B-cell Acute Lymphoblastic Leukemia Microenvironment. Leukemia. 2025 Nov;39(11):2622-2639. doi: 10.1038/s41375-025-02734-z.

  Using single cell RNA sequencing and ex vivo modelling we identified adipogenic progenitors (often called: Adipo-CAR) as a key and hitherto neglected component of the ALL niche.

  4. 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. 2025 Apr 2;15(4):717-732. doi: 10.1158/2159-8290.CD-24-0916.

  When I was still Chair of Paediatrics at Newcastle University, I was involved in setting up he Stratified Medicine Program for children with cancer in the UK. These are the first results!

  5. 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.

  An important expert statement from our European Phase 1 network (ITCC) and our European professional organization (SIOPe) on the challenges and opportunities for establishing CAR T cell trials for rare pediatric cancers.

  6. 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.

  [Accompanied by a Preview article to highlight our work]

  Here, we identified N-cadherin (CDH2) as a key & targetable adhesion molecule involved in the interaction of mesenchymal stroma cells (MSC) and ALL; in addition, we established iPSC-derived MSC as a renewable source for MSC (iMSC) for ex vivo modelling of the bone marrow niche.

  7. 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

  This manuscript describes the design of our academically developed SeluDex trial. The innovative design allows recruitment of ALL patients across the age barrier (children & adults) and is based on our own pre-clinical research.