The Food and Drug Administration, USA, declared its affirmation over gene based therapy this year.
The FDA issued an order which permitted the use of Kymriah or as it is famously known among scientists, “the living drug”.
What is Kymriah?
The process entails genetic modification of immune cells of the patients. These genetically modified cells then help in curing cancer.
It is used in a patient whose cancer has relapsed twice or more.
However, it is allowed only on the patients who are young adults up to the age of 25 or children affected with lymphoblastic leukemia, who don’t respond to the normal treatment.
What is lymphoblastic leukaemia?
This cancer is most common in the USA among children. The cancer affects the bone marrow and the blood of the patients.
As per the statistics, around 1300 patients are diagnosed with the disease every year. Most of them are of the age of 20 or below.
FDA’s statement over the approval
FDA commissioner Scott Gottlieb said that the approval has opened a new arena in medical field. He also stated that the new technologies have the abilities to change the face of medicine.
The new innovations can help in providing cure for the incurable ones. Director of FDA called this treatment a medicinal coup.
He said that the treatment offered the patients a new option.
How does this treatment work?
This treatment involves gene and cells technology. T-cells (which are also known as immune cells) of the patients are first removed.
Then the next step is undertaken in a lab where the genetic modification of the T-cells is done. These genetically modified cells cure the leukemia cells.
Next stage involves the infusion of these genetically modified cells into the patients.
The therapy is named CAR-T cell therapy.
Why this treatment?
The treatment has shown positive results in 83% of the 63 patients. These patients have had been through several relapses or were unresponsive to the standard treatment.
Hence, the FDA finally approved it in the month of July. However, the treatment has also shown some adverse effects on the patients.
Some of them suffered from problems of immune system like cytokine-release syndrome.
The FDA has approved only 32 places where the treatment will be carried out. These clinics and hospitals have been specially trained to administer the drug.
How big will be the hole made in your wallet because of this treatment?
Novartis, the company who developed the drug, estimated the cost to be $475,000. The treatment is the ultimate thing for the patients who respond to it.
It has also been stated officially by the company that the ones who remain unaffected by the treatment will not have to pay.
The company is making special efforts to make the treatment more feasible for the needy ones.