No doubt Cancer is a deadly disease. Nowadays we quite often see people suffering from different types of Cancer. Breast Cancer and Uterine Cancer is quite prevalent among women while Prostate Cancer is more common among men. The above three types are more or less manageable. You can actually remove the specific body parts to avoid further risks. Leukemia as well can be managed in the same way, i.e., by replacing blood and chemotherapy.

Now let us come to the basics, you can think of overcoming Cancer like how Chickenpox and Polio were eliminated over a period. But if Cancer is in your genes, you’re certain to be a victim of Cancer as well. A lot of experiments are done worldwide day after day.

But not all are making it to the market and made available to the people who have Cancer. Thanks to FDA’s approval for New Gene Therapy Treatment which is to treat pediatric patients who have acute lymphoblastic leukemia (ALL). So what is this all about? What is the success rate and are there any potential risks involved?

What is Gene Altering Therapy?

The recent new Gene Altering Therapy Treatment in the United States received Food and Drug Administration (FDA) approval. This new treatment is a dawn of a new era of cancer therapy. The therapy can be used to treat some pediatric patients suffering from ALL.

ALL is the most common childhood cancer in the USA that affects more than 5,000 children every year. It is a blood and bone marrow cancer in children. ALL causes the immature WBCs (white blood corpuscles/cells) to grow out of control. The condition can progress quickly and may prove out to be fatal within few months if not treated.

The gene altering treatment is called tisagenlecleucel. It can treat patients up to 25 years of age who did not respond to initial chemotherapy or who have had a relapse post-treatment. This occurs in over 20 percent of children suffering from ALL. Till now, there were very limited treatment options available for these patients.

Though the FDA approval applies to a very small group of patients, oncologists believe it is a significant beginning. Dr. Mikkael Sekeres, MD, Director of the Leukemia Program at the Cleveland Clinic Cancer Center reportedly said, “The therapy is remarkable and a vanguard of cancer therapy that we are going to see more and more in the future.”

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How does the therapy work?

Tisagenlecleucel which is an immunotherapy treatment can be used for patients suffering from ALL that affects B-cells, a type of lymphocytes. The therapy works by modifying the immune cells collected from the patient’s blood genetically. A gene is added to a protein known as chimeric antigen receptor (CAR).

Then the treatment team modifies the blood and returns it to the patient to do its job in identifying and destroying the leukemia cells.

According to Dr. Sekeres, this treatment is a paradigm shift, a real step forward in personalized treatment for cancer. Addressing the physicians, he remarked that he is hopeful that this is just the beginning.

Impressive Success Rates in Clinical Trial

As any medical product development or new medical therapy cannot be implemented without a successful clinical trial, so the clinical trial was also performed for tisagenlecleucel. Though the clinical trial of the revolutionary treatment was a small one that included 63 pediatric patients, still the results were awe-inspiring. Dr. Sekeres confirmed that within three months of treatment, patients had an overall remission rate of around 83 percent.
The patients were followed up only about five months after receiving the treatment. The patients for ALL are considered to be cured if the patients stay free of the condition for four years after the end of treatment.

How much does tisagenlecleucel cost?

The treatment will be given to each person individually. It has been predicted to cost about $475,000.

Potential Side Effects

Though Tisagenlecleucel sounds promising, it comes with few potential serious side effects. It may cause a condition known as cytokine release syndrome (CRS) which causes high fever and flu-like symptoms. Sometimes CRS may turn out to be life-threatening. In response, FDA has expanded the use of tocilizumab, to treat CRS in children above two years of age. Various studies show that it can eliminate the CRS symptoms in many patients within two weeks.

Tisagenlecleucel may also cause some neurological problems. Few minor side effects that may occur within a month of treatment include low blood pressure, infections, decreased oxygen levels, kidney damage, etc.

Gene therapy such as tisagenlecleucel that uses a patient’s own cells to fight cancer is the most promising approach available to treat ALL at present. It also holds enormous potential to treat many other challenging health conditions in the near future. Before opting for the gene therapy, it is recommended you consult your child’s pediatrician as they can best help you determine if he or she is suitable to get treated by the therapy.

Author Bio:

Aravind Sundaram is a Mumbai based Content Specialist who has spent 6+ years romping around the healthcare, medical device, manufacturing, content and software industries. He is currently a Content Specialist at Technosoft Innovations, Inc. where he documents and presents the latest researches and innovations of the company in the medical devices industry. In his spare time, he is an avid reader, hobbyist, and enjoys sharing medical product development knowledge and experience via his posts and articles.

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