On Wednesday the Food and Drug Administration announced according to the agency’s words a “historical action” – the very first approval in the USA of a cell-based gene therapy.
The Food and Drug Administration approved Kymriah or as the scientists call it the “living drug.” Why this name? Because Kymriah involves using immune cells that are genetically modified from patients in order to attack their cancer.
This living drug was approved in order to treat young adults up to the age of 25 and children who have acute lymphoblastic leukemia and who have suffered relapses or don’t respond to the standard treatment.
This disease is actually a cancer of bone marrow and blood and in the USA is the most common childhood cancer. In fact, each year around 1,300 patients who are 20 years old and younger are diagnosed with ALL.
In a written statement the FDA Commissioner Scott Gottlieb said that this is a beginning of new frontier regarding medical innovation. This medical innovation can reprogram a patient’s cells in order to attack deadly cancer.
According to Gottlieb, the new technologies like cell and gene therapies have the potential to change medicine. Also to make an inflection point regarding our ability to treat and maybe even cure some intractable illnesses.
The Treatment
How does this treatment work? The treatments involve removing the T cells also known as immune system cells from each patient. Then the cells are genetically modified in the lab in order to kill and attack the leukemia cells.
The cells that are already genetically modified are infused back into the patients. The name of this treatment is CAR-T cell therapy.
The director of the Food and Drug Administration’s Center for Biologics Evaluation and Research, Peter Marks, says that the Kymriah treatment approach is first of its kind. And that is fills the crucial unmet need for young adults and children with this severe disease.
In the FDA statement, Marks notes that Kymriah provides the patients with a new option for treatment in the area otherwise poor with options. Also, this treatment option has shown survival rates and remission in clinical trials.
Why This Treatment?
The treatment, i.e. CTL019 within 3 months produced remission in 83 % of 63 young adult and pediatric patients.
These patients had suffered relapses or failed to respond to the standard treatment. Because of these results, the FDA advisory panel suggested the approval in July.
However, the treatment comes with risks like a dangerous overreaction of the immune system, i.e. cytokine-release syndrome.
Therefore, FDA requires strong warnings.
The Treatment and Its Cost
Also, this treatment initially will be available, but only at 32 clinics and hospitals. These clinics and hospitals have gone under special training in administering this therapy.
According to Novartis, that developed the drug, this one-time treatment for patients who respond will cost $475,000.
Those people that within a month do not respond will not have to pay. In addition, the company said that they are taking extra steps to make sure that everyone who needs this drug can afford it.
Nevertheless, the statement of the company some patient advocates criticized the cost of the treatment.
The president and founder of Patients for Affordable Drugs, David Mithcell, notes that the decision of Novartis to set the price for one treatment of $475,000 for some is restraint. But he believes that it is actually excessive.
And he notes that the people should not forget that the American taxpayers invested more than $200 million for the CAR-T discovery.
Source NPR | WebMD
The Food and Drug Administration approved Kymriah or as the scientists call it the “living drug.” Why this name? Because Kymriah involves using immune cells that are genetically modified from patients in order to attack their cancer.
This living drug was approved in order to treat young adults up to the age of 25 and children who have acute lymphoblastic leukemia and who have suffered relapses or don’t respond to the standard treatment.
This disease is actually a cancer of bone marrow and blood and in the USA is the most common childhood cancer. In fact, each year around 1,300 patients who are 20 years old and younger are diagnosed with ALL.
In a written statement the FDA Commissioner Scott Gottlieb said that this is a beginning of new frontier regarding medical innovation. This medical innovation can reprogram a patient’s cells in order to attack deadly cancer.
According to Gottlieb, the new technologies like cell and gene therapies have the potential to change medicine. Also to make an inflection point regarding our ability to treat and maybe even cure some intractable illnesses.
The Treatment
How does this treatment work? The treatments involve removing the T cells also known as immune system cells from each patient. Then the cells are genetically modified in the lab in order to kill and attack the leukemia cells.
The cells that are already genetically modified are infused back into the patients. The name of this treatment is CAR-T cell therapy.
The director of the Food and Drug Administration’s Center for Biologics Evaluation and Research, Peter Marks, says that the Kymriah treatment approach is first of its kind. And that is fills the crucial unmet need for young adults and children with this severe disease.
In the FDA statement, Marks notes that Kymriah provides the patients with a new option for treatment in the area otherwise poor with options. Also, this treatment option has shown survival rates and remission in clinical trials.
Why This Treatment?
The treatment, i.e. CTL019 within 3 months produced remission in 83 % of 63 young adult and pediatric patients.
These patients had suffered relapses or failed to respond to the standard treatment. Because of these results, the FDA advisory panel suggested the approval in July.
However, the treatment comes with risks like a dangerous overreaction of the immune system, i.e. cytokine-release syndrome.
Therefore, FDA requires strong warnings.
The Treatment and Its Cost
Also, this treatment initially will be available, but only at 32 clinics and hospitals. These clinics and hospitals have gone under special training in administering this therapy.
According to Novartis, that developed the drug, this one-time treatment for patients who respond will cost $475,000.
Those people that within a month do not respond will not have to pay. In addition, the company said that they are taking extra steps to make sure that everyone who needs this drug can afford it.
Nevertheless, the statement of the company some patient advocates criticized the cost of the treatment.
The president and founder of Patients for Affordable Drugs, David Mithcell, notes that the decision of Novartis to set the price for one treatment of $475,000 for some is restraint. But he believes that it is actually excessive.
And he notes that the people should not forget that the American taxpayers invested more than $200 million for the CAR-T discovery.
Source NPR | WebMD
“A DREAM COME TRUE” – The Long Expected Cancer Treatment Finally Approved by FDA!
Reviewed by admin
on
September 28, 2017
Rating:
Reviewed by admin
on
September 28, 2017
Rating:
No comments: