The global Fanconi anemia gene therapy program will continue to be available for patients in the USA and Europe during 2018 and 2019. In Europe, a new study, known as FANCOLEN-2, will be available at CIEMAT/Hospital del Niño Jesús in Madrid, Spain, starting in the Fall of 2018. In the USA, a highly similar study will become available at Stanford University in late 2018 or early 2019. Both of these clinical studies will incorporate key learnings from the recent gene therapy studies conducted by the Spanish Network on Fanconi Anemia and Fred Hutchinson Cancer Research Center (Seattle).
As of July 2018, three patients with Fanconi anemia complementation group A (FANCA) have been treated at Fred Hutch (Seattle) and six FANCA patients have received treatment at CIEMAT/Hospital del Niño Jesús (Madrid). Both studies involved collection of patients’ hematopoietic stem cells and transduction with a very similar lentiviral vector followed by infusion of the stem cells without any chemotherapy conditioning. There were some differences in the way that stem cells were removed from patients and purified, and also in the precise manner in which the transduction was performed. Key takeaways from these important clinical studies include:
The global FA gene therapy programs going forward will utilize the lentiviral vector developed at CIEMAT and will be available for FANCA patients ages 1 through 12 who have not developed severe bone marrow failure. The gene therapy studies are intended to provide prevention of bone marrow failure and potentially the need for an allogeneic bone marrow transplant.
The gene therapy will be administered without conditioning chemotherapy, and will require a (relatively) short hospital stay to collect stem cells over an approximate 6-day period, followed by a single intravenous administration of gene-corrected hematopoietic cells over the course of one hour or less. Patients treated to-date have been able to leave the hospital within 1-2 days after receiving the investigational therapy. Following treatment, study visits, including blood tests, are scheduled over the course of three years, with more frequent visits planned in the weeks and months immediately following the investigational therapy.
These studies are anticipated to begin patient enrollment in Spain during the Fall of 2018 (at CIEMAT/Hospital del Niño Jesús, Madrid), with enrollment at University College London/ Great Ormond Street Hospital in England to follow. US enrollment at Stanford University will begin in either late 2018 or early in 2019. Both the US and European programs are sponsored by Rocket Pharma (New York, USA). Assistance will be available for families to help with travel, housing accommodations, and other support., both for initial treatment and subsequent follow-up visits.
The principal investigators for the studies are as follows:
Ciemat (Madrid, Spain):
Juan Bueren, PhD: program director
Paula Río, PhD: associated investigator
Susana Navarro, PhD: associated investigator
Hospital del Niño Jesús (Madrid, Spain)
Julian Sevilla, MD PhD: principal investigator
Hospital Val d’Hebrón (Barcelona, Spain)
Cristina Díaz de Heredia, MD, PhD, co-principal investigator
UCL/Great Ormond Street (London, England):
Adrian Thrasher, PhD: program director
Claire Booth, MBBS, MSc, PhD: principal investigator
Stanford University (Palo Alto, CA, USA):
Maria Grazia Roncarolo, MD: program director
Sandeep Soni, MD: principal investigator
Agnieszka Czechowicz, MD PhD: co-investigator
Additional centers in the EU/US may participate in the program in the future, and Hans-Peter Kiem, Pamela Becker, Jennifer Adair, and other colleagues from Fred Hutch will continue to serve as key advisors for the gene therapy studies.
If you or your family member may be interested in enrolling in the gene therapy trials during the Fall of 2018 and would like to learn more, please contact:
Dr. Juan Bueren: firstname.lastname@example.org
Dr. Julian Sevilla: email@example.com
Dr. Agnieszka Czechowicz: firstname.lastname@example.org
Dr. Gayatri Rao: email@example.com
This article was written by participating institutions: CIEMAT, Fred Hutch, Stanford, UCL and Rocket Pharma.