Kanwal Mallhi, MD

Gene therapy is a novel, cutting-edge approach that seeks to prevent or treat disease by delivering a healthy copy of a gene to your body’s cells

The cells of your body contain thousands of genes which dictate the color of your hair and eyes, how tall you grow, how your body creates the proteins and cells it needs and how well they function. 

Faulty, damaged or missing genes can lead to diseases like cancer, sickle cell disease, neurofibromatosis, and congenital deafness. Sometimes, you can be born with these abnormal genes; sometimes a gene change called a mutation happens later.

Gene therapy is a new way to provide a healthy copy of a gene to your body’s cells in order to:

  • Replace a missing or damaged gene that is causing a problem
  • Add genes to the body to help treat disease
  • Turn off genes that are causing problems

Some cellular therapies that treat cancer, such as chimeric antigen receptor (CAR) T-cell therapies are also considered gene therapies. Roswell Park has many CAR T-cell therapies to treat several types of cancer including, leukemia, lymphoma and multiple myeloma.

Conditions treated with gene therapy

Gene therapy is an emerging type of treatment and most options available to patients are still under study in clinical trials. To date, FDA approved gene therapies are available to treat the following conditions:

  • Cancer
  • Sickle cell disease
  • Beta thalassemia

Gene therapies at Roswell Park

Currently, Roswell Park offers an FDA-approved gene therapy for treating sickle cell disease and beta thalassemia. Both these conditions are caused by defective genes that prevent the body from producing normal hemoglobin, a protein in red blood cells that carries oxygen to the body’s tissues. Fixing or replacing the gene to produce normal hemoglobin treats the disease.

  • Casgevy (exagamglogene autotemcel) is approved for both sickle cell disease and beta thalassemia. It uses a unique gene editing technology (called CRISPR/Cas9) to edit the patient’s own stem cells DNA in patients’ blood stem cells in a laboratory. This directs the cells to increase production of fetal hemoglobin (HbF) that helps to prevent red blood cells from becoming sickle shaped.

With this type of therapy, a patient’s own blood stem cells are collected (in a process similar to donating blood) prior to treatment and sent to a specialized lab where the genes in the cells are modified. Patients undergo high-dose chemotherapy to remove cells from their bone marrow, so they can be replaced with their new, genetically altered cells through an infusion.

Why Roswell Park?

Cell and gene therapies are revolutionizing care for patients with cancer and certain blood disorders, and Roswell Park, at the forefront of this work, is the only authorized treatment center in the region that can provide them.

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