International Research with Amatruda Lab


OBJECTIVE

Finding a cure for childhood germ cell tumors in case of relapse, and treatments with less toxicity for affected patients.

RELEVANCE

In the absence of specific treatments for Childhood Germ Cell Tumor, these are treated with adult treatments, which involve great toxicity for children and teenagers, as was the case with Anita. It is imperative to be able to continue research to offer tailor-made treatments for children that will enhance their quality of life and improve current survival rates.

STATUS AND SCOPE

COMPLETED

The program was developed between Sant Joan de Déu Research Foundation and Amatruda Lab.

PROJECT START: 2014

BUDGET

15.462 €

(in 2014)

Dr. James F. Amatruda, MD PhD, Amatruda Lab (Los Angeles, California).

"Our research is dedicated to developing more effective treatments for childhood germ cell tumors . We are using new genome sequencing technologies in order to study tumors at the DNA level in order to discover mutations in the tumor that cause it to grow out of control."

ASOCIACIÓN ANITA CREATES A NEW RESEARCH PROJECT FOR CHILDHOOD GERM CELL TUMORS

International relations were established as a result of the search for new options for patients who currently have no alternatives.

The relationship between Dr. Jaume Mora, with his team at the Sant Joan de Déu Research Foundation, and Dr. James F. Amatruda, along with his team at Amatruda Lab, and the entire group of American germ cell tumor researchers, was born due to the case of ANITA in 2014 who was in need to seek new advances and options at a very difficult time in her disease... after 4 relapses and having tried countless treatments that had not been effective.

Anita's parents prompted the creation of a high-level international research and training program, led by Dr. Jaume Mora and his team, to search for a Senior Investigator who could join Dr. Amatruda's research to try to accelerate the discovery of a solution for refractory patients.

This collaboration not only focused on research, but also involved the contribution of germ cell tumor samples from the Tumor Bank of the Children's Oncology Laboratory of Sant Joan de Déu hospital for incorporation into the international study led by Dr. Jim Amatruda.

James Amatruda, MD, PhD - Interim Chief and Interim Director, Cancer and Blood Disease Institute, Head, Basic and Translational Research, Cancer and Blood Disease Institute, Dr. Kenneth O. Williams Chair in Cancer Research, Children's Hospital Los Angeles, Professor of Pediatrics and Medicine, Keck School of Medicine of USC.

Clinical Interests: Solid tumors in childhood including sarcomas, germ cell and Wilms' tumors.

The association with Jim Amatruda, led years later to the discovery of Andrew J. Murphy and his study on microRNAs, which has allowed today to open a new research on the M371 TEST-GCT Pediatric Biomarker at the Sant Joan de Déu Research Foundation, which will bring significant benefits to a large group of patients with childhood germ cell tumors.

In addition, the relationship established with the European group through the microRNAs and the English group associated with Jim, has allowed greater participation in international research, thanks to the initiative of Asociación Anita.

Despite tireless efforts, significant progress in the treatment of these refractory tumors has not yet been achieved. However, progress has been made in the field of microRNAs in terms of diagnosis and prognosis, an important achievement in itself.

International collaboration and perseverance remain critical to further progress in the research and treatment of refractory childhood germ cell tumors.

Photo: Anita at 5 years old, 2014

After facing 10 surgeries, 27 cycles of chemotherapy (21 different chemo treatments), 32 radiotherapy sessions, being hospitalized more than 39 times, and undergoing an auto bone marrow transplant; her body could not take any more toxicity and adult treatments.

On December 19, 2014 Anita passed away, without any of the existing protocols and treatments - even to this day - being effective. Anita is and always will be our LIGHT and SOURCE OF INSPIRATION.

Photo: Anita at 5 years old, 2014.

"INTEGRATED GENOMIC ANALYSIS REVEALS ABERRATIONS IN WNT SIGNALING IN GERM CELL TUMORS IN CHILDREN AND TEENAGERS" - JAMES AMATRUDA

ACKNOWLEDGMENTS

We thank the patients and families who contributed biological samples to this study. Supported by grants from the National Cancer Institute (CA135731 to J.F.A., CA151284 to J.N.P., T32 CA099936 to J.B. and U10180886, U24CA114766 and U10CA180899 in support of the Children's Oncology Group), an award from the St. Baldrick's Foundation Consortium (to A.L.F., M.D. .K. and J.F.A.) and the Cancer Prevention and Research Institute of Texas (RP110394 to J.F.A.). James Amatruda acknowledges the contribution of the Asociación Anita for the work on GCT at the Sant Joan de Déu hospital in Barcelona.

You can download the full report of the work of this successful research or consult it online.