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Childhood Cancer and Blood Disorders

32
Publications
71.88
%Q1
66
Number of citations
59.26
% Open Access Publications
24
Ongoing Projects
1
Number of Presented Theses
69
Number of Active Clinical Trials
CC&BD pioneers research into novel drugs and biomarkers aimed at treating pediatric cancers and hematological disorders. Our multidisciplinary approach spans from laboratory breakthroughs to bedside applications. With three dedicated research laboratories focusing on sarcomas, neural tumors, and rare anemias, we cover diverse areas of study. Our clinical research initiatives encompass a Personalized medicine program, a clinical trials unit, and hematopoietic stem cell transplantation (HSCT) and cellular/gene therapies. Our team has earned recognition through prestigious human resources programs, including Rio Hortega, Sara Borrell, and PhD-VHIR fellowships. In a major milestone, we are initiating the first-in-child clinical trial of ABTL0812 for neuroblastoma treatment. Furthermore, we have participated in several national and international research projects, demonstrating our commitment to advancing scientific innovation and improving therapeutic options for pediatric patients.

eCORE

  • Advanced Therapies and Advanced Interventions, Nanomedicine, Transplant & Donation
  • Cancer
  • Personalized Medicine, Innovative Diagnostics, Molecular Imaging & Digital Health
  • Woman & Child Health & Rare Diseases

Team

Group Leader
Lucas Moreno Martín-Retortillo

Principal Investigator (PI)
M Mar Mañu, Josep Roma, Miguel F Segura, Aroa Soriano, Cristina Diaz de Heredia

Researchers
María J Pérez García, Ariadna Boloix, Khloud Elsharawy, Patricia Zarzosa, Anna Llort, Anna Collado, Raquel Hladun, Lorena Valero, Pablo Velasco, Constantino Sábado, Luis Gros, Thais Murciano, Paula Pérez, Laura Murillo, Blanca Espinosa, Angela Menarguez, María Pérez-Torres Lobato, Marina Ortiz, Laura Alonso, Maria Isabel Benítez, Luz Uria, Gabriela Guillen, Melissa Panesso, Marina Bonfill, Anna Salo, Maria Oliveras

PhD Students
Amira Idrizovic, Valeria Rizzuto, Guillem Pons, Julia Sansa, Lia García, Adrià Molero Valenzuela, Marta Miera

Lab Technicians
Paula Cabré

Nursing and Technical Staff
Laura Romero, Sergio Manresa, Andrea Vilaplana, Carolina A Torres Barbosa, María de los Ángeles Rodriguez, Claire Diout, Elena Andetta, Carlota Aguilera, Raquel Andreu, Raquel Anta

Selected Publications

Moreno L , Weston R , Owens C , Dominique Valteau-Couanet , Marion Gambart , Victoria Castel , C Michel Zwaan , Karsten Nysom , Nicolas Gerber , Aurora Castellano , Genevieve Laureys , Ruth Ladenstein , Jochen Rössler , Guy Makin , Dermot Murphy , Bruce Morland , Sucheta Vaidya , Estelle Thebaud , Natasha van Eijkelenburg , Deborah A Tweddle , Giuseppe Barone , Julie Tandonnet , Nadege Corradini , Pascal Chastagner , Catherine Paillard , Francisco J Bautista , Soledad Gallego Melcon , Bram De Wilde , Lynley Marshall , Juliet Gray , Susan A Burchill , Gudrun Schleiermacher , Louis Chesler , Andrew Peet , Martin O Leach , Kieran McHugh , Roisin Hayes , Neil Jerome , Hubert Caron , Jennifer Laidler , Nicola Fenwick , Grace Holt , Veronica Moroz , Pamela Kearns , Simon Gates , Andrew D J Pearson , Keith Wheatley ; ITCC and SIOPEN
Bevacizumab, Irinotecan, or Topotecan Added to Temozolomide for Children with Relapsed and Refractory Neuroblastoma: Results of the ITCC-SIOPEN BEACON-Neuroblastoma Trial
J Clin Oncol. 2024 Apr 1;42(10):1135-1145
DOI: 10.1200/JCO.23.00458.
IF: 44.54

Locatelli F, Antmen B, Kang HJ, Koh K, Takahashi Y, Kupesiz A, Dias Matos MGA, Chopra Y, Bhat S, Im HJ, Güngör T, Lu MY, Stefanelli T, Rosko C, St Pierre A, Burock K, Smith Y, Sinclair K, Diaz-de-Heredia C.
Ruxolitinib in treatment-naive or corticosteroid-refractory paediatric patients with chronic graft-versus-host disease (REACH5): interim analysis of a single-arm, multicentre, phase 2 study.
Lancet Haematol. 2024 Aug;11(8):e580-e592
DOI: 10.1016/S2352-3026(24)00342-9
IF: 24.7

Gallo-Oller G, Pons G, Sansa-Girona J, Navarro N, Zarzosa P, García-Gilabert L, Cabré-Fernandez P, Guillén Burrieza G, Valero-Arrese L, Segura MF, Lizcano JM, Sánchez de Toledo J, Moreno L, Gallego S, Roma J.
TRIB3 silencing promotes the downregulation of Akt pathway and PAX3-FOXO1 in high-risk rhabdomyosarcoma
Exp Hematol Oncol. 2024 Apr 5;13(1):38.
DOI: 10.1186/s40164-024-00503-9.
IF: 11.4

Murphy C, Devis-Jauregui L, Struck R, Boloix A, Gallagher C, Gavin C, Cottone F, Fernandez AS, Madden S, Roma J, Segura MF, Piskareva O.
In vivo cisplatin-resistant neuroblastoma metastatic model reveals tumour necrosis factor receptor superfamily member 4 (TNFRSF4) as an independent prognostic factor of survival in neuroblastoma.
LoS One. 2024 May 29;19(5)
DOI: 10.1371/journal.pone.0303643
IF: 3.3

Al-Samkari H, Shehata N, Lang-Robertson K, Bianchi P, Glenthøj A, Sheth S, Neufeld EJ, Rees DC, Chonat S, Kuo KHM, Rothman JA, Barcellini W, van Beers EJ, Pospíšilová D, Shah AJ, van Wijk R, Glader B, Mañú Pereira MDM, Andres O, Kalfa TA, Eber SW, Gallagher PG, Kwiatkowski JL, Galacteros F, Lander C, Watson A, Elbard R, Peereboom D, Grace RF.
Diagnosis and management of pyruvate kinase deficiency: international expert guidelines.
Lancet Haematol. 2024 Mar;11(3):e228-e239.
DOI: 10.1016/S2352-3026(23)00377-0
IF: 24.7

Selected Projects

MONALISA: A SIOPEN pragmatic clinical trial to MOnitor NeuroblastomA relapse with LIquid biopsy Sensitive Analysis
Principal Investigator: Lucas Moreno
Agency: European Commission
Funding: 84,800 € (VHIR)
Period: 2023-2027

ERDERA: European Rare Disease Research Alliance
Principal Investigator: M Mar Mañú
Agency: European Commission
Funding: 1,080,135.58 €
Period: 2024-2031

NeuVac – a multiepitope mRNA vaccine for high-risk neuroblastoma
Principal Investigator: Miguel F Segura
Agency: Neuroblastoma UK
Funding: £208,887
Period: 2024-2026

Development of prognostic and therapeutic tools targeting epigenetic regulators in metastatic neuroblastoma
Principal Investigator: Miguel F Segura
Agency: ISCIII
Funding: 300,000 €
Period: 2024-2026

Development of a novel and potent strategy for blocking the Hedgehog pathway to treat pediatric sarcomas
Principal Investigator: Josep Roma
Agency: AGAUR
Funding: 50,000 €
Period: 2024-2025

Patents

Compounds for use in preventing or treating cancer
Priority Number: PCT/EP2019/067557
Priority Date: 29/06/2018
Applicants: VHIR/LEITAT/SELABTEC

Peptides for the treatment of cancer and/or metastasis.
Priority Number: EP19383084.1
Priority Date: 05/12/2019
Applicants: Vall Hebron Research Institute (VHIR)/BCN peptides.

Nanovesicles and its use for nucleic acid delivery.
Priority Number: PCT/EP2020/063195
Priority Date: 12/05/2020
Applicants: VHIR / CSIC / CIBER

A pharmaceutical combination for the treatment of cancer
Priority Number: WO2018EP62554
Priority Date: 09/08/2017
Applicants: Ability Pharmaceuticals S.L

Selected Dissemination Activities

Fira dels somnis
Place: Balaguer (Lleida)
Date: 05/04/2024
Brief: Experimental workshop

Science & High School
Place: High-school from IE Greda. Olot
Date: 01/01/1970
Brief: Master class & Experimental workshop

EURODIS
Place: VHIR. Neural tumors Lab
Date: 06/06/2024
Brief: Lab Visit

Women in Science
Place: Barcelona
Date: 01/01/1970
Brief: Experimental workshop at Primary school students from ADEDI school.

Primary school students from ADEDI school
Place: High-school students from IE Frederic Mistral Sarrià
Date: 01/01/1970
Brief: Primary school students from ADEDI school

VHIR Annual Report 2024