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Dr. Alice Bertaina completed her MD degree at the University of Pavia in Italy, her fellowship in hematopoietic stem cell transplantation (HSCT) at the Bambino Gesù Children’s Hospital in Rome, and her PhD degree in Immunology and Biotechnology at Tor Vergata University in Rome. Until joining Stanford University in 2017, she was Head of the Stem Cell Transplant Unit in the Department of Hematology and Oncology at the Bambino Gesù Children’s Hospital in Rome (this institution currently has the largest number of children transplanted with hematopoietic progenitors/stem cells in Europe).Dr. Bertaina is an expert in the field of allogeneic HSCT in pediatric patients affected by hematological malignancies or nonmalignant disorders. In particular, she has pioneered the novel approach of graft manipulation based on the physical elimination of alfa/beta T cells and B cells. Dr. Bertaina has excellent clinical and biological expertise, as demonstrated by her publications in the field of pediatric hematology and oncology. Moreover, she is expert in different aspects of immunological reconstitution of children given an allograft of hematopoietic stem cell, paying particular attention to innate immunity.
Dr. Bertaina is a highly experienced clinician and will play a key role in supporting Section Chief Dr. Rajni Agarwal and Clinical Staff in the Stem Cell Transplant Unit at Lucile Packard Children’s Hospital. She will also continue her research on immune recovery and miRNA, understanding the mechanisms underlying immune reconstitution, Graft-versus-Host Disease (GvHD), and leukemia relapse after allogeneic HSCT in pediatric patients affected by hematological malignant and non-malignant disorders.
Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Adults With T-allo10 Cells Addback
The purpose of this study is to determine the safety of a cell therapy, T-allo10, after
αβdepleted-HSCT in the hopes that it will boost the adaptive immune reconstitution of the
patient while sparing the risk of developing severe Graft-versus-Host Disease (GvHD).
The primary objective of Phase 1 is to determine the recommended Phase 2 dose (RP2D)
administered after infusion of αβdepleted-HSCT in children and young adults with hematologic
A Phase 1b extension will occur after dose escalation, enrolling at the RP2D for the T-allo10
cells determined in the Phase 1 portion to evaluate the safety and efficacy of infusion of
T-allo10 after receipt of αβdepleted-HSCT. Additionally, Phase 1b aims to explore
improvements in immune reconstitution.
All participants on this study must be enrolled on another study: NCT04249830
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Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults
The purpose of the CliniMACS® TCRαβ-Biotin System and CliniMACS® CD19 is to improve the
safety and efficacy of allogeneic HLA-partially matched related or unrelated donors HSCT when
no matched donors are available, to treat malignant and nonmalignant disorders for which HSCT
is the recommended best available therapy. Initially this device will be used in a
single-center, open-label, single-arm, phase II clinical trial to evaluate the efficacy of
haploidentical PBSC grafts depleted of TCRα/β+ and CD19+ cells using the CliniMACS®
TCRαβ/CD19 System in children and adults with hematological and non-hematological
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
The application of experimental hematopoietic cell transplantation (HCT) therapy in
sickle-cell disease (SCD) must strike a balance between the underlying disease severity and
the possibility of a direct benefit of the treatment, particularly in pediatric populations.
Clinical studies in adults with SCD have focused on interventions that prolong survival and
improve the quality of life. Unlike children, adults with SCD are much more likely to have a
debilitating complication. As a result, the risk/benefit ratio of HCT is very favorable in
adults, particularly if an approach to HCT that defines an acceptable level of toxicity can
Whereas hematopoietic stem cell transplantation (HSCT) remains the only curative treatment
currently available for patients with SCD, the morbidity, the frequent irreversible damage in
target organs and the mortality reported in the natural course of patients with severe SCD
are strong incentives to perform HSCTs in younger age groups. For those who lack a matched
related donor, CB transplant is an appealing option, but despite been less problematic, CB
accessibility related to cell dose of appropriately matched cord blood unit (CBU) remains a
significant issue. Through a 7-day culture process of a CBU's hematopoietic stem cell HSCs
with the UM171 compound, the total cell dose is increased mitigating this limitation.
UM171-CB expansion (ECT-001-CB) allows a greater CB accessibility, the selection of better
matched cords that might translate into favourable clinical outcomes as reported in previous
trials, including a lower risk of graft-versus-host disease. After CB selection and ex-vivo
expansion, ECT-001-CB transplant will follow a myeloablative reduced-toxicity conditioning
regimen consisting of rATG, busulfan and fludarabine with doses of all agents optimized to
the individual using model-based dosing and will be followed by standard supportive care and
GVHD prophylaxis consisting of tacrolimus and MMF.
Stanford is currently not accepting patients for this trial.
For more information, please contact David Shyr, MD, .
Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell
disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa
and beta cells that comes from a partially matched family donor. The study will assess
whether immune cells, called T cells, from the family donor, that are specially grown in the
laboratory and given back to the patient along with the stem cell transplant can help the
immune system recover faster after transplant. As a safety measure these T cells have been
programmed with a self-destruct switch so that they can be destroyed if they start to react
against tissues (Graft versus host disease).
Stanford is currently not accepting patients for this trial.
For more information, please contact Cancer Clinical Trials Office (CCTO), 650-498-7061.
KIR Favorable Mismatched Haplo Transplant and KIR Polymorphism in ALL/AML/MDS Allo-HCT Children
This is a phase II, open-label, non-randomized, prospective study of haploidentical
transplantation using KIR-favorable donors for children with acute lymphoblastic leukemia
(ALL), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic
hematopoietic cell transplantation (HCT). The relationship of KIR2DL1 polymorphisms to
survival in children with these diseases undergoing any approach to allogeneic HCT during the
study time frame will also be determined.