Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthful adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the assets, the cells multiply exponentially and go hunting for the CD19 protein, which emerges on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the embark of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis investigate experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to utterly severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that examine also had neurological problems, including seizures and delirium. But there were no cases of fatal brain erection, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. However the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment hammer back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthful adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the bod, the cells multiply exponentially and go hunting for the CD19 protein, which shows up on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the embark of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis examine experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to enormously severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that investigate also had neurological problems, including seizures and delirium. But there were no cases of fatal brain full salute, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. However the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment hammer back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthful adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the assets, the cells multiply exponentially and go hunting for the CD19 protein, which emerges on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the begin of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis examine experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to utterly severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that investigate also had neurological problems, including seizures and delirium. But there were no cases of fatal brain erection, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. Tho’ the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment hammer back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthfull adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the figure, the cells multiply exponentially and go hunting for the CD19 protein, which shows up on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the commence of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis investigate experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to utterly severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that probe also had neurological problems, including seizures and delirium. But there were no cases of fatal brain erection, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. Tho’ the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment hit back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most titillating thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthful adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the figure, the cells multiply exponentially and go hunting for the CD19 protein, which emerges on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the embark of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis probe experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to enormously severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that explore also had neurological problems, including seizures and delirium. But there were no cases of fatal brain full salute, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. Tho’ the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment strike back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthfull adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the figure, the cells multiply exponentially and go hunting for the CD19 protein, which emerges on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the commence of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis probe experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to enormously severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that examine also had neurological problems, including seizures and delirium. But there were no cases of fatal brain erection, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. Tho’ the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment strike back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
The inwards track on Washington politics.
*Invalid email address
Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthful adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the figure, the cells multiply exponentially and go hunting for the CD19 protein, which shows up on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the commence of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis explore experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to enormously severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that explore also had neurological problems, including seizures and delirium. But there were no cases of fatal brain full salute, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. However the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment hammer back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.
FDA panel recommends approval of CAR T-cell therapy – The Washington Post
Novel cancer treatment wins endorsement of FDA advisers
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Emily Whitehead was the very first child treated with a fresh type of cancer therapy that uses the patient’s own genetically modified immune cells to fight cancer. She has been in remission from acute lymphoblastic leukemia for five years. Her parents, Thomas and Kari Whitehead, are strong advocates for the treatment. (Sean Simmers/For The Washington Post)
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for despairingly ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a fresh path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most arousing thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and youthfull adults whose leukemia doesn’t react to traditional treatments — a group that numbers six hundred or so patients a year in this country. But the treatment also is being tested for a range of diseases from non-Hodgkin lymphoma and numerous myeloma to solid tumors.
If cleared by the FDA, it would be the very first gene therapy approved in the United States. But unlike traditional gene therapy, the fresh treatment doesn’t substitute disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inwards the assets, the cells multiply exponentially and go hunting for the CD19 protein, which shows up on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated twenty two days, Novartis officials told the committee Wednesday.
From the commence of Wednesday’s meeting, committee members made clear that they were not worried about the treatment’s efficacy, which has been well established — eighty three percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to treat possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis explore experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to enormously severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that explore also had neurological problems, including seizures and delirium. But there were no cases of fatal brain erection, as occurred in another company’s trial, Grupp said.
To attempt to ensure safety, Novartis is limiting the therapy’s availability to thirty to thirty five medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to go after patients for up to fifteen years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. However the FDA isn’t required to go after the guidance of its advisory committees, it usually does.
David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. “It represents a paradigm shift in treating cancers,” said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment hammer back her daughter’s cancer and brought back “the sparkle” in her eyes. And while she died three months later, “our children deserve this chance,” Kappen said.
For other parents, there were more satisfied outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he suffered on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.
Thomas Whitehead, whose daughter was the very first pediatric patient to receive the treatment, gasped up while telling panel members about Emily’s practice. She got CAR T-cell therapy when she was six and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.
“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she’s standing right beside me,” said Whitehead, his voice cracking.