Children’s Hospital of New Orleans Stem Cell Transplant, Cellular,
and Gene Therapies Program
What is a stem cell transplant?
Hematopoietic stem cell transplantation (HSCT) is often the best and sometimes
only chance for cure for patients with certain cancers or blood disorders.
The process involves replacing someone’s defective blood stem cells
with new healthy stem cells. HSCT is often called a bone marrow transplant
because the blood stem cells live within the bone marrow. The stem cells
can be collected from the blood or bone marrow from a patient’s
own body (autologous) or from someone else (allogeneic). Prior to receiving
the new cells, a patient must go through chemotherapy and/or radiation
to eliminate their defective stem cells. Receiving the stem cells does
not require surgery as the new cells are administered through an IV similar
to a blood transfusion. The process takes weeks to months and involves
close care from a highly specialized, experienced multidisciplinary team.
What is cellular therapy?
Cell therapy is a process of training the immune system to attack specific
cells, including cancer cells. Chimeric Antigen Receptor T-cell therapy
(CAR T) is the most common cellular therapy and revolutionized the management
of difficult to treat cancers like leukemia and lymphoma. Many cellular
therapies use the patient’s own cells however some require a donor.
After collecting and training the cells, they are given back to the patient
through an IV where they begin seeking out and killing the unwanted cells.
What is gene therapy?
Gene therapy is an approach to prevent or treat diseases by correcting
the genetic mutations responsible for the diseases. The number of diseases
that are currently able to be treated with gene therapy is growing rapidly
and includes immunodeficiencies, sickle cell disease, thalassemia, and
others. The process involves collecting a patient’s own cells and
then changing the genes to turn them off, replace them with a normal healthy
gene, or introducing new genes depending on the disease. These cells are
then given back to patient through an IV to treat their disease.
Diseases we treat:
We treat a variety of cancerous and non-cancerous diseases including but
not limited to those listed below.
Leukemia and Lymphoma
ALL
AML
Hodgkin’s lymphoma
Non-Hodgkin’s lymphomas
Myelodysplastic syndrome (MDS)
Hemophagocytic lymphohistiocytosis (HLH)
Aggressive cancers requiring high doses of chemotherapy
Neuroblastoma
Brain tumors
Aggressive or metastatic sarcomas
Bone Marrow Failure syndromes
Severe aplastic anemia
Paroxysmal nocturnal hemoglobinuria
Fanconi anemia
Dyskarytosis congenita
Diamond Blackfan anemia
Sickle cell anemia
Thalassemia
Severe combined immunodeficiency disorder (SCID)
Immune Dysregulation Syndromes
IPEX
Wiskott Aldrich syndrome
Chronic granulomatous disease
X-linked lymphoproliferative disorder (XLP)
Who we are?
The multidisciplinary team at CHNOLA have extensive experience at taking
care of stem cell transplant and cellular and gene therapy patients. We
provide an individualized approach to treat our patients so that each
patient receives the best possible care for their disease. The mission
is to care for our patients with a family centered approach which utilizes
all resources available. Our multidisciplinary team includes physicians,
nurses, social workers, nutritionists, pharmacists, physical therapists,
psychologists, and blood bank personnel.
Why choose us?
Dr. Lolie Yu started the hematopoietic stem cell transplant program at
CHNOLA in 1989. With over 30 years’ experience in curing patients
with cancer and blood diseases, CHNOLA is the premier center for pediatric
stem cell transplant and cellular therapy in Louisiana and treats more
patients than any other pediatric center in the state. The survival rate
for our patients have historically met or exceeded the expectations set
by the National Marrow Donor Program (NMDP). We are the only pediatric
program accredited by the Foundation for the Accreditation of Cellular
Therapies and the only pediatric CAR-T certified program in Louisiana.
We are passionately dedicated to ensuring that the patients in the Gulf
South region have access to the most advanced and comprehensive therapies
possible. To achieve this goal, our program is affiliated with transplant
centers across the country through collaboration with the Children’s
Oncology Group (COG), Pediatric Blood and Marrow Transplant Consortium
(PBMTC), Pediatric Immunodeficiency Treatment Consortium (PIDTC), the
Blood and Marrow Transplant Clinical Trials Network (BMTCTN), and the
Sickle Cell Transplant Advocacy and Research Alliance (STAR).
We are also focused on supporting our families through the long journey
of transplant and thereafter. This includes services with psychology,
psychiatry, and child life therapy while in the hospital. Following discharge,
patients who live away from New Orleans have available housing on our
campus through the Ronald McDonald House and Hog’s House. To help
manage potential long-term complications, we also offer the only pediatric
survivorship clinic in the Gulf South.
Newly Approved Gene Therapy Treatment Center For Sickle Cell Disease &
Chronic Pain
Children's Hospital New Orleans is a recently activated treatment center
for both Bluebird Bio and Vertex for gene therapy, which received FDA
approval for patients over 12yrs with sickle cell disease and chronic
pain in December 2023. For Vertex, Children's Hospital New Orleans is
currently one of only ten centers in the United States to be activated.
For both Bluebird Bio and Vertex, Children's is the only pediatric center
in Louisiana, Arkansas, Mississippi, Alabama, Georgia, and Florida. Louisiana
has one of the largest sickle cell populations with approximately 1,500
kids under the age of 18 affected. In addition to Bone Marrow Transplant,
this now gives multiple curative options to this debilitating disease
and makes Children's Hospital New Orleans a destination for advanced treatment
of Sickle Cell Disease. Both companies also have products for patients
with Beta-Thalassemia who are transfusion dependent.
For more information regarding the hematopoietic stem cell transplant center
at Children's Hospital, please contact the Hematology/Oncology office at
504.896.9740.
Hematology Oncology, Pediatric Hematology and OncologyView Profile
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