Healthy Living

FDA Approves Breakthrough Gene Therapy Treatment for Lymphoma

FDA Approves Breakthrough Gene Therapy Treatment for Lymphoma

Researchers have been working around the clock to find new and improved treatments for different cancers. Drugs, experiments, natural remedies, and more treatment options have been heavily explored in the search for a cure. Recently a new form of treatment has been developed by researchers that alters the genetic makeup that may cause certain cancers and pathogens. Even more importantly, a Food and Drug Administration panel recently unanimously endorsed this treatment, which could lead to FDA approval as soon as September.

The Treatment

The treatment, called tisagenlecleucel (CTL019) which has been successfully tested on B-cell acute lymphoblastic leukemia. The treatment is based on Chimeric Antigen Receptor T-Cell (CAR-T) therapy. This type of procedure extracts T cells from the blood of a patient before genetically modifying them to target cancer cells. The cells are then infused back into the patient. The procedure takes an estimated 22 days, and the T cells begin to multiply after they are infused back into the body. These T-cells target the CD-19 protein, which is believed to be an indicator of white blood cells for leukemia and lymphoma. 


The creator of the treatment, Novartis, claim that the drug is aimed at treating leukemia in patients age 3-25. They then performed a series of trials testing the drug's efficacy. The study began with 88 patients, although 16 patients discontinued before the infusion due to a rapid escalation of their disease. Most of these patients were children or young adults. After 3 months 83% of patients achieved complete remission (no signs of the disease). Data evaluations also showed relapse-free survival and a high probability of survival after getting the treatment.

The findings were not all positive, however. 47% of patients experienced cytokine release syndrome. Essentially, the infused T-cells release what are called cytokines that can lead to the creation of inflammatory agents. This inflammation can lead to pain and/or reduced mobility in patients, and around 15% also experienced minor neurologic events. Thankfully no deaths due to the cytokines or neurologic events were reported during the study.


CTL019 would be the first form on gene therapy approved by the FDA. This revolutionary therapy could be the first step in finding a cure for leukemia, the most common form of childhood cancer in the United States. Novartis is asking the FDA for permission to treat leukemia patients ages 3 to 25 years old.

Although CTL019 has some potentially dangerous side effects, it has not been shown to cause serious injury or death. The chance of getting complete remission may very well be worth the risk. This is a very important treatment, and could pave the way for the FDA approving similar treatments for other cancers like lymphoma or cancer tumors.

Signs of Childhood Cancer

Finding out your child has cancer is every parent's worst nightmare. Many parents would refuse to entertain the concept, and their fear is understandable. However, it is important to get your child the help they need as quickly as possible if they do in fact have a form of childhood cancer. Below are a few telltale signs that your child might be in the beginning stage of a form of cancer. If your child experiences any of these symptoms you should take them to a doctor right away, as they are all caused by some form of serious illness even if it isn't cancer.

  1. Fatigue - Perpetual fatigue and feelings of being unwell (also known as malaise) could indicate that a child is developing some form of cancer. If your child is constantly lethargic, always lacking energy, and frequently getting fever or infections then you should take them to a pediatrician as soon as possible. All of this points to cancer cells infiltrating the bone marrow, which could be serious.
  2. Prolonged Fever - Fever that lasts more than two weeks with no identifiable cause is a common sign of some form of cancer. This fever could lead to night sweats, malaise, bone pain, or weight loss. Fever that lasts this long is never a good sign, and requires a pediatrician to see your child quickly.
  3. Recurring Infections - Frequent infections that are resistant to treatment could be a sign of a form of cancer. If your child has taken over the counter medication and antibiotics with little to no effect, the infections could be due to cancer cells taking over the body. If these infections won't go away, seeing a doctor becomes very necessary.
  4. Anorexia and Weight Loss - Children with developing cancers often find themselves with a significant loss of appetite. This will usually result in concerning weight loss in the children. In some cases, children may not change their eating habits at all and still experience weight loss. This is because the immune system spends tons of energy trying to fight off cancer cells, and the body ends up burning more calories than are being consumed.
  5. Swelling of Lymph Nodes - The swelling of lymph nodes, also called lymphadenopathy, is the body telling you that something is wrong. The swelling of the lymph nodes is not uncommon, as any common virus or illness will cause them to swell. Lymph nodes swelling can be a good sign as it means your body is fighting infections. It only becomes particularly troublesome when the lymph nodes are swollen for longer than a month and are showing no response to antibiotics. Most of the time lymph nodes that are swollen are also tender. However, lymph nodes in cancer patients tend to not be tender, which gives another sign that something is wrong.
  6. Headaches - A sudden onset of constant headaches and migraines could be problematic. These headaches are often severe and can possibly wake the child during the night. If the headaches get worse when lying down that is a telltale sign that something is putting pressure on the brain (usually a tumor of some sort).
  7. Struggling in School - Obviously a worsening school performance can be due to a million factors. When combined with one of the symptoms above it could spell trouble. Cancer will lead to a lack of energy which makes it harder to concentrate, and it could also lead to memory loss.
  8. Personality and Mood Changes - Cancer takes both a physical and a mental toll on children. Children with cancer will often become much more irritable and short-tempered out of nowhere, and may have a higher chance of acting out. Children may also be sad more frequently, and not engage in activities that they once enjoyed. While this is a sign of cancer, it could also be a sign of mental health issues. Either way your child should see a pediatrician.
  9. Abdominal Mass - A mass discovered on the abdomen is often signs of a malignant tumor. This can often cause constipation, vomiting, and abdominal pain. It should be clear that any unexplainable masses found on your child should be checked out immediately.

Final Thoughts

CTL019 could be the first approved gene therapy for cancer patients, and could particularly help children with leukemia. Although it is still in the clinical stages, it's results so far have been very promising. There remains hope that childhood cancer will be a thing of the past in the near future. For more information on recent developments and information, be sure to check out the rest of our website.