Johns Hopkins All Children's Hospital

Center for Cell and Gene Therapy for Non-Malignant Conditions

Our center provides gene therapy and transplant solutions for children with noncancerous blood and bone marrow disorders. See which conditions we can treat.

Stem cell and bone marrow transplants are often the best treatment for a variety of serious, nonmalignant (not cancerous) conditions. For children with sickle cell disease, primary immunodeficiencies and metabolic disorders, a transplant offers hope of a cure.

The Center for Cell & Gene Therapy for Nonmalignant Conditions (CCGNC) is home to a skilled, multispecialty team. Immunologists, oncologists, hematologists and bone marrow transplant specialists collaborate on these complex cases. Our experts treat rare, nonmalignant disorders using gene therapy and transplantation.

Center for Cell & Gene Therapy for Nonmalignant Conditions: Why Choose Us?

This unique center is part of the Johns Hopkins All Children’s Cancer & Blood Disorders Institute in St. Petersburg, Florida. Thanks to groundbreaking research, our teams are consistently improving outcomes for all our young patients.

When your child comes to the CCGNC, you can expect:

  • Collaborative approach: Our research and treatment teams include skilled specialists who are all experts in their fields. They work together to determine the most effective treatments for each child. Meet the cell and gene therapy team.
  • Pioneering research: Our work with gene therapy and cell processing has made it possible to cure many children with nonmalignant conditions. We are continually working to learn more about these conditions and find innovative ways to treat them.
  • Advances in bone marrow transplant: Thanks to our evolving techniques, stem cell and bone marrow transplants are now an option for more children. We use advanced cell processing methods to remove cells that can cause a transplant to fail, while preserving cells that fight infection. This allows us to perform successful transplants even when a perfectly matched bone marrow donor isn’t available. Find out more about our Stem Cell & Bone Marrow Transplant Program.
  • Innovative gene therapy: Gene therapy can be lifesaving for several genetic conditions, such as sickle cell disease. We can take a child’s bone marrow cells, genetically modify them to correct the inherited mutation, and then re-infuse them into the child’s blood.
  • Access to clinical trials: As a member of Johns Hopkins Medicine, based in Baltimore, we offer access to leading-edge research studies and trials — right here on Florida’s west coast. We also work with the Children’s Oncology Group, National Bone Marrow Donor Program and other groups working on cutting-edge research to improve patient outcomes. Learn more about our current clinical trials.
  • Support through the process: If your child is a candidate for cell and gene therapy, we’ll invite your family to visit our Stem Cell and Bone Marrow Transplant Clinic. Our experts will answer your questions, explain the process and help you prepare for your child’s treatment.  

Nonmalignant Conditions We Treat

At the Center for Cell & Gene Therapy, our goal is to provide the most advanced treatment options for various rare conditions, including:

Hemoglobinopathies

Hemoglobinopathies such as sickle cell disease and thalassemia are inherited conditions. These disorders affect the number and shape of red blood cells in the body. Bone marrow transplants can cure these conditions. We also offer gene therapy approaches developed in collaboration with other institutions.

Learn more about our Sickle Cell Program.

Bone Marrow Failure Syndromes

Bone marrow failure syndromes may be inherited or acquired. This means a child may be born with a disorder or develop it later on. In these rare blood disorders, the bone marrow fails to produce certain types of blood cells. Children with these syndromes have a very high risk of developing many life-threatening complications, including leukemia or other cancers.

A bone marrow transplant can cure some of the blood disorders related to these syndromes.

Bone marrow failure syndromes we treat include:

  • Diamond-Blackfan anemia
  • Dyskeratosis congenita
  • Fanconi anemia
  • Severe aplastic anemia
  • Severe congenital neutropenia
  • Shwachman-Diamond syndrome 

Severe Combined Immunodeficiency (SCID) and Leaky SCID

SCID is a rare genetic condition that causes life-threatening problems with the immune system. This type of primary immune deficiency is usually diagnosed shortly after birth.

Children with SCID have too few lymphocytes (a type of white blood cell) or lymphocytes that do not work properly. Without enough properly functioning lymphocytes, the immune system can’t fight off infections. Leaky SCID is a less severe, but still serious, form of the disorder.

Bone marrow transplants (BMT) can cure SCID. We also offer gene therapy as a treatment for certain types of SCID.

Non-SCID Primary Immunodeficiencies

Children with primary immunodeficiencies have weakened immune systems and are unable to fight off infection. They get more frequent infections that last longer and are harder to treat. Children with primary immunodeficiencies are also prone to:

  • Inflammation
  • Anemia
  • Digestive problems
  • Growth and development issues

Bone marrow transplants may cure some types of primary immunodeficiency.

Non-SCID primary immunodeficiencies we treat include:

  • Chronic granulomatous disease (CGD)
  • Leukocyte adhesion deficiency
  • MonoMac syndrome
  • DOCK8 deficiency
  • Zap70 deficiency
  • Common variable immunodeficiency (CVID)
  • Wiskott-Aldrich syndrome (WAS)
  • NEMO deficiency

Immune Dysregulation Disorders

This group of rare, inherited disorders causes an overactive immune system. Instead of fighting off infection, an overactive immune system attacks your own body. Immune dysregulation disorders cause dangerous inflammation and organ damage.

A bone marrow transplant may cure some dysregulation disorders. We also offer several targeted treatments that help dampen out-of-control immune responses.

Immune dysregulation disorders we treat include:

  • Immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome
  • CTLA-4 deficiency
  • LRBA deficiency
  • STAT gain-of-function (STAT1 and STAT3)
  • X-linked lymphoproliferative disease (XLP)
  • Hemophagocytic lymphohistiocytosis (HLH)
  • Systemic Langerhans cell histiocytosis (LCH)

Collaborative Care

The experts at the Center for Cell & Gene Therapy coordinate treatments with bone marrow transplant, immunology and other specialists. Together, we provide comprehensive care for patients with conditions that require a bone marrow transplant.

Learn more about our Immunology/BMT Clinic and Immune Dysregulation Clinic.

Resources

These organizations offer support and resources for families:

Contact Us

To find out more about our Center for Cell & Gene Therapy for Nonmalignant Conditions, call us at 727-767-3984. We serve families throughout the greater Tampa Bay area and beyond.

Read more:

Brothers with Life-Threatening Genetic Disease now Thriving

Brothers Jerrick, 5, and Evian, 2, were diagnosed with the same rare, life-threatening genetic immunodeficiency. They received bone marrow transplants at Johns Hopkins All Children’s, and are now back to being healthy, energetic kids. The physicians who have treated them are also researching the disease to continue to advance treatments for future patients.

Read More

 


The Johns Hopkins All Children’s Center for Cell and Gene Therapy for Non-Malignant Conditions is supported by the Love McKinley Foundation.