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By Babita Jyoti

M.D. on 09/12/2017

Newly Approved Gene Therapy for Acute Lymphocytic Leukemia

Recently, Dr. Ackerman was brought onto Jacksonville’s Channel 4 News to discuss a newly approved cancer-fighting gene therapy that offers hope to children and young adults suffering from Acute Lymphocytic Leukemia (ALL). I wanted to expand on this topic for all of you and review the potential life-saving benefits of this promising new therapy.

ALL is an aggressive blood and bone marrow cancer, and one of the most common childhood cancers. Approximately 5900 cases of ALL are diagnosed each year in the USA. Although remission is achieved in 80-90% of these cases, unfortunately, around 20% of these relapse.

Kymriah is the first gene therapy to be approved by the U.S. Food and Drug Administration in patients age 25 and younger with ALL that is resistant to conventional treatment therapies or has returned following previous treatment.

Kymriah is a one-time treatment that uses the patient’s own cells to fight the cancer. T-cells, which are the work horses of our immune system, are separated from the patient’s own blood and genetically engineered to produce receptors on the cell surface called chimeric antigen receptor (CAR). These are then expanded in special laboratories into millions of cells before being infused back into the patient. These genetically modified CAR-T cells then seek out and target the cancer cells that harbor the antigen on their surface. Patients using Kymriah will also receive a lymho-depleting chemotherapy regimen prior to this this infusion. An international study has shown that 83 percent of patients who underwent this treatment achieved remission within three months.

Statements issued by Novartis, the manufacturer of Kymriah, indicate the treatment will eventually be available at 32 locations. Twenty hospitals are expected to undergo training and be ready to administer the treatment within one month.

Like most treatments, the new T-cell therapy is associated with specific side effects that include cytokine release syndrome (an overreaction of the immune system) along with other potential long-term complications, some of which are still unknown.

The approval of the new gene therapy marks the beginning of a different approach to cancer treatment and will pave the way for other gene and cell therapies.

I’m excited and encouraged to see the emergence of new therapies for caner. It is my hope that this therapy could be modified to treat lymphomas, which share similar traits with ALL, and also developed to fight other non-blood cancers. 

For questions about gene therapy, please contact me at jyotimd@ackermancancer.com.

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