FDA Advisory Committee Recommends Approval of Gene Therapy against Leukemia

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

Thirty years ago this disease was considered a deadly; however, medical advances have achieved survival rates of 75 percent. This percentage could increase if the FDA finally approves the use of the first gene therapy that would be available in the market.

The treatment consists of extracting immune cells from the patient and freezing them. Subsequently, they are sent to the laboratory where a genetic modification is done, so they can fight the disease. Once modified, the cells are re-introduced into the patient’s body and there they can identify and attack the cancer.

This recommendation was based on clinical results of 63 patients who received the treatment between April 2015 and August 2016. The findings were convincing, leukemia disappear 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 the blood cells form. 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. This unprecedented alliance also included the National Institute of Genomic Medicine and 15 other health and research institutions across the country. Its aim is promoting the benefit 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 researchers and promoting the development of tools for genomic diagnosis and treatment with genomic technology.

The results of the first phase of research have confirmed the presence in the Mexican genome of 57 genes associated with type 2 diabetes mellitus in populations of other countries; In addition, two new genes –specific to the Mexican population– linked to type 2 diabetes were discovered.