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New and Emerging Therapies

St. Jude Children’s Research Hospital is committed to developing more effective, less-toxic treatments for cancer, sickle cell disease, neurological diseases, and other life-threatening childhood illnesses. 

Sculpture of child climbing DNA double-helix

These emerging therapies are sometimes called targeted therapies or precision medicine. 

From the start, St. Jude brought scientists and medical experts together under the same roof. They worked to make discoveries and develop new treatments to save the lives of children around the world. This work continues today. 

In our labs and in collaboration with researchers around the world, St. Jude scientists work on new ways to kill cancer cells without harming healthy cells. We are working on gene therapies, including gene editing, that could be used to correct the underlying cause of some diseases including cancers, and blood disorders.  

We are also developing new therapies for immune disorders and for neurodegenerative diseases. 

Treatment names and approaches change with new and better therapies, but the goal remains the same: the longest and healthiest life for young people facing life-threatening diseases. 

Gene therapy

Gene therapy has promise for the treatment of sickle cell diseases, blood disorders, and neurodegenerative diseases caused by an error in the bodies genetic instructions. The approach uses tools to either edit the problem gene or to deliver a working copy of that gene to patients.  

DNA sequences called CRISPR are the basis of gene editing tools. The technology makes it possible to treat diseases such as sickle cell disease by editing the genes involved. We are working to expand this emerging therapy to children with bone marrow failure syndromes and other genetic blood disorders. 

St. Jude researchers helped to pioneer another approach to gene therapy. Rather than editing genes, this method uses a vector, usually an altered virus, to introduce a healthy copy of a defective gene. The working copy of the gene is packaged into the vector and then inserted into the patient’s own blood stem cells. The gene-corrected blood stem cells are given back to the patient into a vein. The technique has been used to treat the bleeding disorder hemophilia B and the immune disorder severe combined immunodeficiency (SCID). 

Another form of gene-directed therapy is called antisense oligonucleotide or ASO. An ASO is a small piece of DNA-like material that can correct the way a faulty gene functions. Some ASO’s have been approved for use in neurological conditions like Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD).  

Neuroscience researchers at St. Jude are taking this well understood therapy and applying it to rare genetic diseases. They are working toward addressing an individual child’s unique genetic variant or „typo“ by creating an ASO therapy specific for that child’s disease.


Immunotherapy is a treatment that uses the immune system to fight cancer. Immunotherapies are standard treatment for certain childhood cancers. Chimeric antigen receptor (CAR) T cells are one example of how immunotherapy is changing treatment of leukemia and lymphoma.  

Other treatments involve antibodies. They are made by immune cells to find and help to destroy tumor cells. A patient’s antibodies are collected and then engineered in the laboratory to better target the patient’s cancer cells. The specialized antibodies are being combined with cytokines, another immunotherapy, to treat the childhood solid tumor neuroblastoma. Cytokines are molecules that help to activate patients’ immune systems against their cancer.  

Researchers are working on more immunotherapies to treat childhood cancer. The emerging therapies include molecules called checkpoint inhibitors to stop tumor cells from turning off a patient’s immune system. 

Cancer treatment vaccines are also in the works. Unlike vaccines to prevent influenza, COVID-19 and other infectious diseases, treatment vaccines aim to help the immune system find and destroy cancer cells. 

Efforts are also underway to expand direct infusion of immune cells therapy for treatment of solid tumors and brain tumors. This approach commonly involves collecting either a patient’s T cells or immune cells called natural killer or NK cells. The T cells are engineered to express a protein or CAR on their cell surface to recognize and destroy cancer cells. The CAR T cells are then returned to the patient through an infusion. 

St. Jude is leading the way in scientific advances to understand and treat life-threatening childhood diseases. Our research drives new therapies, moving them from the laboratory to the clinic. Our goal is saving children at St. Jude and worldwide and give them back their life.