‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
Picture of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen. Eye of Science/Science Source hide caption
Photo of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen.
A fresh kind of cancer treatment that uses genetically engineered cells from a patient’s immune system to attack their cancer lightly cleared a crucial hurdle Wednesday.
A Food and Drug Administration advisory committee unanimously recommended that the agency approve this “living drug” treatment for children and youthfull adults who are fighting a common form of leukemia. The agency doesn’t have to go after the committee’s recommendation but usually does.
The treatment takes cells from a patient’s assets, modifies the genes, and then reinfuses those modified cells back into the person who has cancer. If the agency approves, it would mark the very first time the FDA has approved anything considered to be a “gene therapy product.”
The treatment is part of one of the most significant developments in cancer research in decades — finding ways to corset the bod’s own immune system to fight cancer. And while it has generated much hope, there are some concerns about its safety over the long term — and its cost.
Even so, several of the committee members were unusually enthusiastic in explaining their 10-0 vote recommending approval.
“This is the most arousing thing I’ve seen in my lifetime,” said Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus, Ohio.
Shots – Health News
Cancer Immunotherapy At A Crossroads
“This is a major advance and is ushering in a fresh era in treating children,” agreed Dr. Malcolm Smith, associate branch chief for pediatric oncology at the National Cancer Institute.
For years, scientists have attempted to use drugs that stimulate the immune system to fight cancer, and have had only modest success.
In latest years, however, scientists developed a fresh generation of “immunotherapy” drugs that have produced outstanding results for a broad range of cancers by letting out the bod’s natural defense system.
The fresh treatment is known as CAR-T cell immunotherapy. It works by removing key immune system cells known as T cells from the patient so scientists can genetically modify them to seek out and attack only cancer cells. That’s why some scientists refer to this as a “living drug.”
Doctors then infuse millions of the genetically modified T cells back into the patient’s assets so they can attempt to obliterate the cancer cells and hopefully leave healthy tissue unscathed.
“It’s truly a paradigm shift,” said Dr. David Lebwohl, who goes the CAR-T Franchise Global Program at the drug company Novartis, which is seeking the FDA’s approval for the treatment. “It represents a fresh hope for patients.”
The drug endorsed by the advisory panel is known as CTL019 or tisagenlecleucel. It was developed to treat children and youthfull adults ages three to twenty five who have relapsed after undergoing standard treatment for B cell acute lymphoblastic leukemia, which is the most common childhood cancer in the United States.
While this blood cell cancer can be very curable, some patients fail to react to standard treatments; and a significant proportion of patients practice relapses that don’t react to follow-up therapies.
“There is a major unmet medical need for treatment options” for these patients, Dr. Stephen Thirst, who helped examine at the Children’s Hospital of Philadelphia, told the committee.
Shots – Health News
Training The Immune System To Fight Cancer Has 19th-Century Roots
In the main investigate that the company submitted as evidence in seeking FDA approval, doctors at twenty five sites in eleven countries administered the treatment to eighty eight patients. The patients, ages three to 23, had failed standard treatment or experienced relapses and failed to react to follow-up standard treatment. CTL019 produced remissions in eighty three percent of patients, the company told the committee.
“This product has been shown to result in a high rate of response,” Lebwohl said.
Still, while those results are encouraging, the treatment has raised concerns.
The treatment can produce a life-threatening adverse reaction known as a “cytokine release syndrome,” in which the immune system attacks vital organs. In the past, a handful of patients who were getting similar treatments developed by other companies died from serious brain full salute.
Albeit those sorts of complications did occur in some patients receiving CTL019, the patients recovered and there were no fatalities, the company says.
There are also concerns about possible long-term complications. Scientists use a virus to make the genetic switches in the T cells, raising fears about possible long-term side effects.
Because of the safety concerns, the company plans to proceed to go after the medical progress of patients receiving the treatment. The company is also planning to originally make the treatment available only at about thirty to thirty five hospitals that have had the necessary training and expertise to produce and administer the elaborate treatment.
Another big concern is the cost. While Novartis will not estimate the price it will ultimately put on the treatment, some industry analysts project it will cost $500,000 per infusion.
Nevertheless, during a public comment period several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval.
“I’m blessed most of all for the patients who will benefit from this therapy,” said Dr. Carl June of the University of Pennsylvania Perelman School of Medicine, who developed the therapy.
FDA Advisers Endorse Genetically Engineered Immunotherapy For Cancer: Shots – Health News: NPR
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
Pic of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen. Eye of Science/Science Source hide caption
Picture of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen.
A fresh kind of cancer treatment that uses genetically engineered cells from a patient’s immune system to attack their cancer lightly cleared a crucial hurdle Wednesday.
A Food and Drug Administration advisory committee unanimously recommended that the agency approve this “living drug” treatment for children and youthfull adults who are fighting a common form of leukemia. The agency doesn’t have to go after the committee’s recommendation but usually does.
The treatment takes cells from a patient’s figure, modifies the genes, and then reinfuses those modified cells back into the person who has cancer. If the agency approves, it would mark the very first time the FDA has approved anything considered to be a “gene therapy product.”
The treatment is part of one of the most significant developments in cancer research in decades — finding ways to corset the figure’s own immune system to fight cancer. And while it has generated much hope, there are some concerns about its safety over the long term — and its cost.
Even so, several of the committee members were unusually enthusiastic in explaining their 10-0 vote recommending approval.
“This is the most titillating thing I’ve seen in my lifetime,” said Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus, Ohio.
Shots – Health News
Cancer Immunotherapy At A Crossroads
“This is a major advance and is ushering in a fresh era in treating children,” agreed Dr. Malcolm Smith, associate branch chief for pediatric oncology at the National Cancer Institute.
For years, scientists have attempted to use drugs that stimulate the immune system to fight cancer, and have had only modest success.
In latest years, however, scientists developed a fresh generation of “immunotherapy” drugs that have produced awesome results for a broad range of cancers by releasing the assets’s natural defense system.
The fresh treatment is known as CAR-T cell immunotherapy. It works by removing key immune system cells known as T cells from the patient so scientists can genetically modify them to seek out and attack only cancer cells. That’s why some scientists refer to this as a “living drug.”
Doctors then infuse millions of the genetically modified T cells back into the patient’s bod so they can attempt to obliterate the cancer cells and hopefully leave healthy tissue unscathed.
“It’s truly a paradigm shift,” said Dr. David Lebwohl, who goes the CAR-T Franchise Global Program at the drug company Novartis, which is seeking the FDA’s approval for the treatment. “It represents a fresh hope for patients.”
The drug endorsed by the advisory panel is known as CTL019 or tisagenlecleucel. It was developed to treat children and youthful adults ages three to twenty five who have relapsed after undergoing standard treatment for B cell acute lymphoblastic leukemia, which is the most common childhood cancer in the United States.
While this blood cell cancer can be very curable, some patients fail to react to standard treatments; and a significant proportion of patients practice relapses that don’t react to follow-up therapies.
“There is a major unmet medical need for treatment options” for these patients, Dr. Stephen Thirst, who helped examine at the Children’s Hospital of Philadelphia, told the committee.
Shots – Health News
Training The Immune System To Fight Cancer Has 19th-Century Roots
In the main explore that the company submitted as evidence in seeking FDA approval, doctors at twenty five sites in eleven countries administered the treatment to eighty eight patients. The patients, ages three to 23, had failed standard treatment or experienced relapses and failed to react to follow-up standard treatment. CTL019 produced remissions in eighty three percent of patients, the company told the committee.
“This product has been shown to result in a high rate of response,” Lebwohl said.
Still, while those results are encouraging, the treatment has raised concerns.
The treatment can produce a life-threatening adverse reaction known as a “cytokine release syndrome,” in which the immune system attacks vital organs. In the past, a handful of patients who were getting similar treatments developed by other companies died from serious brain full salute.
Albeit those sorts of complications did occur in some patients receiving CTL019, the patients recovered and there were no fatalities, the company says.
There are also concerns about possible long-term complications. Scientists use a virus to make the genetic switches in the T cells, raising fears about possible long-term side effects.
Because of the safety concerns, the company plans to proceed to go after the medical progress of patients receiving the treatment. The company is also planning to primarily make the treatment available only at about thirty to thirty five hospitals that have had the necessary training and expertise to produce and administer the sophisticated treatment.
Another big concern is the cost. While Novartis will not estimate the price it will ultimately put on the treatment, some industry analysts project it will cost $500,000 per infusion.
Nevertheless, during a public comment period several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval.
“I’m glad most of all for the patients who will benefit from this therapy,” said Dr. Carl June of the University of Pennsylvania Perelman School of Medicine, who developed the therapy.
FDA Advisers Endorse Genetically Engineered Immunotherapy For Cancer: Shots – Health News: NPR
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
Photo of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen. Eye of Science/Science Source hide caption
Photo of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen.
A fresh kind of cancer treatment that uses genetically engineered cells from a patient’s immune system to attack their cancer lightly cleared a crucial hurdle Wednesday.
A Food and Drug Administration advisory committee unanimously recommended that the agency approve this “living drug” treatment for children and youthful adults who are fighting a common form of leukemia. The agency doesn’t have to go after the committee’s recommendation but usually does.
The treatment takes cells from a patient’s figure, modifies the genes, and then reinfuses those modified cells back into the person who has cancer. If the agency approves, it would mark the very first time the FDA has approved anything considered to be a “gene therapy product.”
The treatment is part of one of the most significant developments in cancer research in decades — finding ways to corset the figure’s own immune system to fight cancer. And while it has generated much hope, there are some concerns about its safety over the long term — and its cost.
Even so, several of the committee members were unusually enthusiastic in explaining their 10-0 vote recommending approval.
“This is the most titillating thing I’ve seen in my lifetime,” said Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus, Ohio.
Shots – Health News
Cancer Immunotherapy At A Crossroads
“This is a major advance and is ushering in a fresh era in treating children,” agreed Dr. Malcolm Smith, associate branch chief for pediatric oncology at the National Cancer Institute.
For years, scientists have attempted to use drugs that stimulate the immune system to fight cancer, and have had only modest success.
In latest years, however, scientists developed a fresh generation of “immunotherapy” drugs that have produced astounding results for a broad range of cancers by letting out the bod’s natural defense system.
The fresh treatment is known as CAR-T cell immunotherapy. It works by removing key immune system cells known as T cells from the patient so scientists can genetically modify them to seek out and attack only cancer cells. That’s why some scientists refer to this as a “living drug.”
Doctors then infuse millions of the genetically modified T cells back into the patient’s figure so they can attempt to obliterate the cancer cells and hopefully leave healthy tissue unscathed.
“It’s truly a paradigm shift,” said Dr. David Lebwohl, who goes the CAR-T Franchise Global Program at the drug company Novartis, which is seeking the FDA’s approval for the treatment. “It represents a fresh hope for patients.”
The drug endorsed by the advisory panel is known as CTL019 or tisagenlecleucel. It was developed to treat children and youthful adults ages three to twenty five who have relapsed after undergoing standard treatment for B cell acute lymphoblastic leukemia, which is the most common childhood cancer in the United States.
While this blood cell cancer can be very curable, some patients fail to react to standard treatments; and a significant proportion of patients practice relapses that don’t react to follow-up therapies.
“There is a major unmet medical need for treatment options” for these patients, Dr. Stephen Thirst, who helped examine at the Children’s Hospital of Philadelphia, told the committee.
Shots – Health News
Training The Immune System To Fight Cancer Has 19th-Century Roots
In the main investigate that the company submitted as evidence in seeking FDA approval, doctors at twenty five sites in eleven countries administered the treatment to eighty eight patients. The patients, ages three to 23, had failed standard treatment or experienced relapses and failed to react to follow-up standard treatment. CTL019 produced remissions in eighty three percent of patients, the company told the committee.
“This product has been shown to result in a high rate of response,” Lebwohl said.
Still, while those results are encouraging, the treatment has raised concerns.
The treatment can produce a life-threatening adverse reaction known as a “cytokine release syndrome,” in which the immune system attacks vital organs. In the past, a handful of patients who were getting similar treatments developed by other companies died from serious brain erection.
Albeit those sorts of complications did occur in some patients receiving CTL019, the patients recovered and there were no fatalities, the company says.
There are also concerns about possible long-term complications. Scientists use a virus to make the genetic switches in the T cells, raising fears about possible long-term side effects.
Because of the safety concerns, the company plans to proceed to go after the medical progress of patients receiving the treatment. The company is also planning to originally make the treatment available only at about thirty to thirty five hospitals that have had the necessary training and expertise to produce and administer the sophisticated treatment.
Another big concern is the cost. While Novartis will not estimate the price it will ultimately put on the treatment, some industry analysts project it will cost $500,000 per infusion.
Nevertheless, during a public comment period several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval.
“I’m blessed most of all for the patients who will benefit from this therapy,” said Dr. Carl June of the University of Pennsylvania Perelman School of Medicine, who developed the therapy.
FDA Advisers Endorse Genetically Engineered Immunotherapy For Cancer: Shots – Health News: NPR
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
Photo of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen. Eye of Science/Science Source hide caption
Pic of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen.
A fresh kind of cancer treatment that uses genetically engineered cells from a patient’s immune system to attack their cancer lightly cleared a crucial hurdle Wednesday.
A Food and Drug Administration advisory committee unanimously recommended that the agency approve this “living drug” treatment for children and youthful adults who are fighting a common form of leukemia. The agency doesn’t have to go after the committee’s recommendation but usually does.
The treatment takes cells from a patient’s assets, modifies the genes, and then reinfuses those modified cells back into the person who has cancer. If the agency approves, it would mark the very first time the FDA has approved anything considered to be a “gene therapy product.”
The treatment is part of one of the most significant developments in cancer research in decades — finding ways to corset the assets’s own immune system to fight cancer. And while it has generated much hope, there are some concerns about its safety over the long term — and its cost.
Even so, several of the committee members were unusually enthusiastic in explaining their 10-0 vote recommending approval.
“This is the most arousing thing I’ve seen in my lifetime,” said Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus, Ohio.
Shots – Health News
Cancer Immunotherapy At A Crossroads
“This is a major advance and is ushering in a fresh era in treating children,” agreed Dr. Malcolm Smith, associate branch chief for pediatric oncology at the National Cancer Institute.
For years, scientists have attempted to use drugs that stimulate the immune system to fight cancer, and have had only modest success.
In latest years, however, scientists developed a fresh generation of “immunotherapy” drugs that have produced outstanding results for a broad range of cancers by releasing the figure’s natural defense system.
The fresh treatment is known as CAR-T cell immunotherapy. It works by removing key immune system cells known as T cells from the patient so scientists can genetically modify them to seek out and attack only cancer cells. That’s why some scientists refer to this as a “living drug.”
Doctors then infuse millions of the genetically modified T cells back into the patient’s bod so they can attempt to obliterate the cancer cells and hopefully leave healthy tissue unscathed.
“It’s truly a paradigm shift,” said Dr. David Lebwohl, who goes the CAR-T Franchise Global Program at the drug company Novartis, which is seeking the FDA’s approval for the treatment. “It represents a fresh hope for patients.”
The drug endorsed by the advisory panel is known as CTL019 or tisagenlecleucel. It was developed to treat children and youthfull adults ages three to twenty five who have relapsed after undergoing standard treatment for B cell acute lymphoblastic leukemia, which is the most common childhood cancer in the United States.
While this blood cell cancer can be very curable, some patients fail to react to standard treatments; and a significant proportion of patients practice relapses that don’t react to follow-up therapies.
“There is a major unmet medical need for treatment options” for these patients, Dr. Stephen Thirst, who helped examine at the Children’s Hospital of Philadelphia, told the committee.
Shots – Health News
Training The Immune System To Fight Cancer Has 19th-Century Roots
In the main investigate that the company submitted as evidence in seeking FDA approval, doctors at twenty five sites in eleven countries administered the treatment to eighty eight patients. The patients, ages three to 23, had failed standard treatment or experienced relapses and failed to react to follow-up standard treatment. CTL019 produced remissions in eighty three percent of patients, the company told the committee.
“This product has been shown to result in a high rate of response,” Lebwohl said.
Still, while those results are encouraging, the treatment has raised concerns.
The treatment can produce a life-threatening adverse reaction known as a “cytokine release syndrome,” in which the immune system attacks vital organs. In the past, a handful of patients who were getting similar treatments developed by other companies died from serious brain full salute.
Albeit those sorts of complications did occur in some patients receiving CTL019, the patients recovered and there were no fatalities, the company says.
There are also concerns about possible long-term complications. Scientists use a virus to make the genetic switches in the T cells, raising fears about possible long-term side effects.
Because of the safety concerns, the company plans to proceed to go after the medical progress of patients receiving the treatment. The company is also planning to primarily make the treatment available only at about thirty to thirty five hospitals that have had the necessary training and expertise to produce and administer the elaborate treatment.
Another big concern is the cost. While Novartis will not estimate the price it will ultimately put on the treatment, some industry analysts project it will cost $500,000 per infusion.
Nevertheless, during a public comment period several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval.
“I’m blessed most of all for the patients who will benefit from this therapy,” said Dr. Carl June of the University of Pennsylvania Perelman School of Medicine, who developed the therapy.
FDA Advisers Endorse Genetically Engineered Immunotherapy For Cancer: Shots – Health News: NPR
‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle
Pic of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen. Eye of Science/Science Source hide caption
Pic of a CAR-T cell (reddish) attacking a leukemia cell (green). These CAR-T lymphocytes are used for immunotherapy against cancer (CAR stands for chimeric antigen receptor). After the proliferation of the CAR-expressing T cells, they are transfused back into the patient and can directly detect the cancer cells carrying the antigen.
A fresh kind of cancer treatment that uses genetically engineered cells from a patient’s immune system to attack their cancer lightly cleared a crucial hurdle Wednesday.
A Food and Drug Administration advisory committee unanimously recommended that the agency approve this “living drug” treatment for children and youthful adults who are fighting a common form of leukemia. The agency doesn’t have to go after the committee’s recommendation but usually does.
The treatment takes cells from a patient’s bod, modifies the genes, and then reinfuses those modified cells back into the person who has cancer. If the agency approves, it would mark the very first time the FDA has approved anything considered to be a “gene therapy product.”
The treatment is part of one of the most significant developments in cancer research in decades — finding ways to corset the bod’s own immune system to fight cancer. And while it has generated much hope, there are some concerns about its safety over the long term — and its cost.
Even so, several of the committee members were unusually enthusiastic in explaining their 10-0 vote recommending approval.
“This is the most arousing thing I’ve seen in my lifetime,” said Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus, Ohio.
Shots – Health News
Cancer Immunotherapy At A Crossroads
“This is a major advance and is ushering in a fresh era in treating children,” agreed Dr. Malcolm Smith, associate branch chief for pediatric oncology at the National Cancer Institute.
For years, scientists have attempted to use drugs that stimulate the immune system to fight cancer, and have had only modest success.
In latest years, however, scientists developed a fresh generation of “immunotherapy” drugs that have produced astounding results for a broad range of cancers by letting out the bod’s natural defense system.
The fresh treatment is known as CAR-T cell immunotherapy. It works by removing key immune system cells known as T cells from the patient so scientists can genetically modify them to seek out and attack only cancer cells. That’s why some scientists refer to this as a “living drug.”
Doctors then infuse millions of the genetically modified T cells back into the patient’s bod so they can attempt to obliterate the cancer cells and hopefully leave healthy tissue unscathed.
“It’s truly a paradigm shift,” said Dr. David Lebwohl, who goes the CAR-T Franchise Global Program at the drug company Novartis, which is seeking the FDA’s approval for the treatment. “It represents a fresh hope for patients.”
The drug endorsed by the advisory panel is known as CTL019 or tisagenlecleucel. It was developed to treat children and youthful adults ages three to twenty five who have relapsed after undergoing standard treatment for B cell acute lymphoblastic leukemia, which is the most common childhood cancer in the United States.
While this blood cell cancer can be very curable, some patients fail to react to standard treatments; and a significant proportion of patients practice relapses that don’t react to follow-up therapies.
“There is a major unmet medical need for treatment options” for these patients, Dr. Stephen Thirst, who helped examine at the Children’s Hospital of Philadelphia, told the committee.
Shots – Health News
Training The Immune System To Fight Cancer Has 19th-Century Roots
In the main explore that the company submitted as evidence in seeking FDA approval, doctors at twenty five sites in eleven countries administered the treatment to eighty eight patients. The patients, ages three to 23, had failed standard treatment or experienced relapses and failed to react to follow-up standard treatment. CTL019 produced remissions in eighty three percent of patients, the company told the committee.
“This product has been shown to result in a high rate of response,” Lebwohl said.
Still, while those results are encouraging, the treatment has raised concerns.
The treatment can produce a life-threatening adverse reaction known as a “cytokine release syndrome,” in which the immune system attacks vital organs. In the past, a handful of patients who were getting similar treatments developed by other companies died from serious brain full salute.
Albeit those sorts of complications did occur in some patients receiving CTL019, the patients recovered and there were no fatalities, the company says.
There are also concerns about possible long-term complications. Scientists use a virus to make the genetic switches in the T cells, raising fears about possible long-term side effects.
Because of the safety concerns, the company plans to proceed to go after the medical progress of patients receiving the treatment. The company is also planning to primarily make the treatment available only at about thirty to thirty five hospitals that have had the necessary training and expertise to produce and administer the sophisticated treatment.
Another big concern is the cost. While Novartis will not estimate the price it will ultimately put on the treatment, some industry analysts project it will cost $500,000 per infusion.
Nevertheless, during a public comment period several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval.
“I’m glad most of all for the patients who will benefit from this therapy,” said Dr. Carl June of the University of Pennsylvania Perelman School of Medicine, who developed the therapy.