Last week, the Federal Drug Administration took historic action in giving their approval for a revolutionary living drug treatment used for treating children with Acute Lymphocytic Leukemia, also known as Acute Lymphoblastic Leukemia.

Yes, this is a form of cancer and by being called ‘acute’ means that it is aggressive, developing quickly and if not properly treated can be fatal within a few months.

Cancer is generally defined as when cells begin to grow out of control, generally in a form different than the original cells. It can happen anywhere in the body and may develop from any kind of cell.

Leukemia is a term used when cancer cells begin forming in the white blood cells, generally as they are manufactured in the bone marrow. From there, they travel through the blood stream to virtually every part of the body, where they can stay and begin to grow. White blood cells are one of the body’s main immune system tools for fighting many diseases, but when the white blood cells become the source of the disease, it’s extremely difficult to fight.

Lymphocytic or lymphoblastic is used when these cancerous white blood cells gather in the lymph nodes and begin to grow.

In acute lymphocytic leukemia, the cancerous white blood cells are not able to mature and quickly accumulate. It generally occurs in children.

Most treatments for this type of aggressive and deadly cancer involves strong drugs, administered through an IV into the blood vessels. In the majority of cases, the side effects from these drugs can be quite severe, often causing a host of other health concerns.

And sadly, a growing number of children either can’t tolerate the harsh chemotherapy or they don’t respond to the standard chemotherapy used. And sometimes if they initially respond but later on the cancer returns, they frequently don’t respond to a second or third round of chemo.

The new treatment approved by the FDA is designed to provide a treatment for those who can’t tolerate or fail to respond to standard chemotherapy. It’s called CAR-T cell therapy, or Kymriah.

A simple explanation is that doctors extract T cells from the patient. T cells are another vital tool in the body’s immune system. They genetically modify the patient’s T cells so that they only attack and kill off the leukemia cells. Then they put these genetically modified T cells back into the patient and allow them what they have been modified to do.

How well does this new technology work?

During trials, it was tried on 63 pediatric and young adult patients. In 83% of the cases, the CAR-T cell therapy resulted in remission of the cancer within 3 months. There is one possible side effect known as cytokine-release syndrome, where the body’s immune system overreacts to the modified T cells, so the FDA is requiring a strong warning accompany the treatment.

The newly approved CAR-T cell therapy is not cheap, coming with a price tag of $475,000. However, the price of a bone marrow transplant can easily run over $800,000. The company spent over $200 million developing the new treatment. Additionally, the company providing the CAR-T therapy, says that if any patient does not respond to the one-time treatment after a month, they will not be charged.

The treatment will now be available at 32 hospitals and clinics nationwide with more to receive the special training in the near future. The company also hopes to receive FDA approval on a similar treatment for adult leukemia and they hope to use the same technology to develop treatments for other forms of cancer.

Considering that about 3,100 new cases of acute lymphocytic leukemia are diagnosed every year, the new genetic ‘living drug’ is sure save thousands of young lives.