Gene Therapy for Leukemia Recommended for Approval by FDA Advisory Committee

The Food and Drug Administration (FDA) Advisory Committee recommended the approval of the experimental therapy Tisagenlecleucel, also known as CTL019, to treat pediatric and adolescent patients with the most common form of leukemia in this age group: acute lymphoblastic leukemia (ALL).

Thirty years ago this disease was considered deadly in all cases; however, medical advances have achieved survival rates of 75 percent. This percentage could increase even more if the FDA gives final approval to the use of the first gene therapy to be available in the market.

The treatment consists of extracting immune cells from the patient and freezing them, that are then sent to a laboratory for genetic modification to increase their disease-fighting capabilities. Once modified, the cells are re-introduced into the patient’s body where they can identify and attack the cancer.

This recommendation was based on clinical results of 63 patients with ALL who received the treatment between April 2015 and August 2016. According to the findings the cancer disappeared in 82 per cent of the treated patients.

If the FDA approves the use of CTL019, it would be the first authorization of a series of personalized gene-modification therapies that are being developed to fight different types of cancer. The CTL019 treatment was created by scientists at the University of Pennsylvania and Novartis Pharmaceuticals.

Leukemia is a type of cancer of the blood that begins in the bone marrow, the soft tissue in the interior of the bones where blood cells are produced. Its main types are: Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Chronic Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML) and Hairy Cell Leukemia.

Further genomic research

In 2010, Carlos Slim Foundation and the Broad Institute of MIT and Harvard launched SIGMA, the Iniciative for Genomic Medicine in the Americas. This unprecedented alliance also included the Mexican National Institute of Genomic Medicine and 15 other health and research institutions across the country. Its aim is to promote the benefits of genomic research for the populations of Mexico and the rest of Latin America through:

• Researching the genetic causes of the main diseases that afflict these populations: diabetes mellitus type 2 and cancer.
• Promote genomic research in Mexico by training scientists and promoting the development of tools for genomic diagnosis and treatment with genomic technology.

To date, SIGMA is one of the most efficient partnerships in genomic research in the World. It has helped to highlight the relevance of including the Mexican and Latin American genomes in large scale genetic studies. Through SIGMA it has been possible to discover variants in genes that are specific to these populations that are highly relevant for the development of diabetes and certain types of cancer.