FDA Approves Very first Gene Therapy For Leukemia
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson. Steve Gschmeissner/Science Source hide caption
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson.
The Food and Drug Administration on Wednesday announced what the agency calls a “historic act” — the very first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer.
The drug was approved to treat children and youthfull adults up to age twenty five suffering from a form of acute lymphoblastic leukemia who do not react to standard treatment or have suffered relapses.
The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About Trio,100 patients who are twenty and junior are diagnosed with ALL each year.
“We’re coming in a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a written statement.
“Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability to treat and even cure many intractable illnesses,” Gottlieb said.
The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It’s also known as CAR-T cell therapy.
“Kymriah is a first-of-its-kind treatment treatment that fills an significant unmet need for children and youthfull adults with this serious disease,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
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“Not only does Kymriah provide these patients with a fresh treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials,” Marks said in the FDA statement.
The treatment, which is also called CTL019, produced remission within three months in eighty three percent of sixty three pediatric and youthfull adult patients. The patients had failed to react to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.
The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
In addition, the treatment will be primarily available only at thirty two hospitals and clinics that have been specially trained in administering the therapy.
Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who react. People who do not react within a month would not be charged, and the company said it is taking extra steps to make sure everyone who needs the drug can afford it
But some patient advocates criticized the cost nevertheless.
“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive,” says David Mitchell, founder and president of Patients For Affordable Drugs. “Let’s reminisce, American taxpayers invested over $200 million in CAR-T’s discovery.”
Correction Aug. 30, 2017
A previous version of this story referred to the treatment as CTL109. The correct name is CTL019.
CAR-T Therapy For Leukemia Wins FDA Approval: Shots – Health News: NPR
FDA Approves Very first Gene Therapy For Leukemia
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson. Steve Gschmeissner/Science Source hide caption
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson.
The Food and Drug Administration on Wednesday announced what the agency calls a “historic activity” — the very first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer.
The drug was approved to treat children and youthful adults up to age twenty five suffering from a form of acute lymphoblastic leukemia who do not react to standard treatment or have suffered relapses.
The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About Three,100 patients who are twenty and junior are diagnosed with ALL each year.
“We’re injecting a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a written statement.
“Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability to treat and even cure many intractable illnesses,” Gottlieb said.
The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It’s also known as CAR-T cell therapy.
“Kymriah is a first-of-its-kind treatment treatment that fills an significant unmet need for children and youthfull adults with this serious disease,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
Shots – Health News
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
“Not only does Kymriah provide these patients with a fresh treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials,” Marks said in the FDA statement.
The treatment, which is also called CTL019, produced remission within three months in eighty three percent of sixty three pediatric and youthfull adult patients. The patients had failed to react to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.
The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
In addition, the treatment will be primarily available only at thirty two hospitals and clinics that have been specially trained in administering the therapy.
Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who react. People who do not react within a month would not be charged, and the company said it is taking extra steps to make sure everyone who needs the drug can afford it
But some patient advocates criticized the cost nevertheless.
“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive,” says David Mitchell, founder and president of Patients For Affordable Drugs. “Let’s reminisce, American taxpayers invested over $200 million in CAR-T’s discovery.”
Correction Aug. 30, 2017
A previous version of this story referred to the treatment as CTL109. The correct name is CTL019.
CAR-T Therapy For Leukemia Wins FDA Approval: Shots – Health News: NPR
FDA Approves Very first Gene Therapy For Leukemia
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson. Steve Gschmeissner/Science Source hide caption
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson.
The Food and Drug Administration on Wednesday announced what the agency calls a “historic activity” — the very first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer.
The drug was approved to treat children and youthfull adults up to age twenty five suffering from a form of acute lymphoblastic leukemia who do not react to standard treatment or have suffered relapses.
The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About Three,100 patients who are twenty and junior are diagnosed with ALL each year.
“We’re coming in a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a written statement.
“Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability to treat and even cure many intractable illnesses,” Gottlieb said.
The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It’s also known as CAR-T cell therapy.
“Kymriah is a first-of-its-kind treatment treatment that fills an significant unmet need for children and youthfull adults with this serious disease,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
Shots – Health News
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
“Not only does Kymriah provide these patients with a fresh treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials,” Marks said in the FDA statement.
The treatment, which is also called CTL019, produced remission within three months in eighty three percent of sixty three pediatric and youthful adult patients. The patients had failed to react to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.
The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
In addition, the treatment will be originally available only at thirty two hospitals and clinics that have been specially trained in administering the therapy.
Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who react. People who do not react within a month would not be charged, and the company said it is taking extra steps to make sure everyone who needs the drug can afford it
But some patient advocates criticized the cost nevertheless.
“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive,” says David Mitchell, founder and president of Patients For Affordable Drugs. “Let’s reminisce, American taxpayers invested over $200 million in CAR-T’s discovery.”
Correction Aug. 30, 2017
A previous version of this story referred to the treatment as CTL109. The correct name is CTL019.
CAR-T Therapy For Leukemia Wins FDA Approval: Shots – Health News: NPR
FDA Approves Very first Gene Therapy For Leukemia
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson. Steve Gschmeissner/Science Source hide caption
Scientists have created a treatment in which genetically modified T cells, shown in blue, can attack cancer cells, shown in crimson.
The Food and Drug Administration on Wednesday announced what the agency calls a “historic activity” — the very first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer.
The drug was approved to treat children and youthfull adults up to age twenty five suffering from a form of acute lymphoblastic leukemia who do not react to standard treatment or have suffered relapses.
The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About Three,100 patients who are twenty and junior are diagnosed with ALL each year.
“We’re injecting a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a written statement.
“Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability to treat and even cure many intractable illnesses,” Gottlieb said.
The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It’s also known as CAR-T cell therapy.
“Kymriah is a first-of-its-kind treatment treatment that fills an significant unmet need for children and youthfull adults with this serious disease,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
Shots – Health News
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
“Not only does Kymriah provide these patients with a fresh treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials,” Marks said in the FDA statement.
The treatment, which is also called CTL019, produced remission within three months in eighty three percent of sixty three pediatric and youthful adult patients. The patients had failed to react to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.
The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
In addition, the treatment will be primarily available only at thirty two hospitals and clinics that have been specially trained in administering the therapy.
Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who react. People who do not react within a month would not be charged, and the company said it is taking extra steps to make sure everyone who needs the drug can afford it
But some patient advocates criticized the cost nevertheless.
“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive,” says David Mitchell, founder and president of Patients For Affordable Drugs. “Let’s recall, American taxpayers invested over $200 million in CAR-T’s discovery.”
Correction Aug. 30, 2017
A previous version of this story referred to the treatment as CTL109. The correct name is CTL019.