CAR-T gene therapy for leukemia clears FDA hurdle
Last Updated Jul 13, two thousand seventeen 11:16 AM EDT
A panel of cancer experts has voted unanimously in favor of a leukemia treatment which could be the very first gene therapy available in the U.S.
The Food and Drug Administration advisory panel voted 10-0 on Wednesday to recommend approval of the treatment developed by the University of Pennsylvania and Novartis Corp. The one-time treatment would be for children and youthfull adults with advanced leukemia.
The therapy could be the very first of a wave of treatments custom-made to target a patient’s cancer . Called CAR-T, it involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells to recognize and ruin cancer and injecting them back into the patient.
The FDA is not required to go after the panel’s recommendation but often does.
FDA panel to vote on revolutionary gene therapy for cancer
“It’s a pretty amazing fresh treatment,” Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, said on “CBS This Morning.” “They take the white [blood] cells out of a child with cancer, they send them to [a lab in] Fresh Jersey, and they put in a gene to reprogram these cells to attack the cancer.”
The vote came after lengthy discussion and impassioned prayers from the fathers of two youthfull patients whose lives were saved by the therapy. The one-time leukemia treatment would be for children and youthful adults with the most common form of childhood cancer, known as ALL.
“Our daughter was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pennsylvania. His daughter Emily, now 12, was the very first child to receive the experimental therapy, five years ago. “We believe when this treatment is approved, it will save thousands of children’s lives around the world.”
In a key test, results were far better than chemotherapy and even newer types of cancer drugs. Of the fifty two patients whose results were analyzed, eighty three percent had finish remission, meaning their cancer vanished. Most patients suffered serious side effects. Eleven patients died, four from side effects and seven from their leukemia.
The FDA is expected to determine whether to approve the Novartis treatment in the next few months. The drugmaker is seeking approval to use the treatment for patients aged three to twenty five with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to react to standard treatment. That happens to more than six hundred patients in the U.S. each year. At that point, they have limited options – all more toxic than the CAR-T therapy – and survival chances are slender. ALL accounts for a quarter of all cancers in children under age 15.
After decades of setbacks and disappointments in efforts to fix, substitute, or switch genes to cure diseases, several companies are near the finish line in a race to bring CAR-T and other types of gene therapy to patients. Kite Pharma also has a CAR-T therapy under FDA review and Juno Therapeutics and others are in late stages of testing.
In March, CBS News chief medical correspondent Dr. Jon LaPook reported on the results of a clinical trial in which one hundred one patients with advanced lymphoma who had failed previous therapy received the Kite Pharma CAR-T treatment. About eight months after a single treatment, thirty nine percent of patients had no evidence of cancer.
“That’s actually fairly remarkable knowing that at best only one out of ten of these patients could have finish disappearance of their lymphoma with standard chemotherapy,” said Dr. Frederick Locke, who helped lead the trial.
CAR-T therapy starts with filtering key immune cells called T cells from a patient’s blood. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancer cells. Millions of copies of the fresh T cells are grown in the lab and then injected into the patient’s bloodstream where they can seek out and demolish cancer cells. Doctors call it a “living drug” – permanently altered cells that proceed to multiply in the figure to fight the disease.
During the patient testing, the entire process took about sixteen weeks on average, which can be too long a wait for some despairingly ill patients, the FDA advisers noted during Wednesday’s meeting in Silver Spring, Maryland. Drug company officials said they can now produce a treatment and get it to a patient in about three weeks.
Novartis said in a statement after the vote that it has long believed CAR-T therapy could “switch the cancer treatment paradigm.”
“It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy,” said the Penn team’s leader, Dr. Carl June.
The cost of CAR-T therapy is likely to be hundreds of thousands of dollars, but it’s only given once. Typically, cancer patients take one or more drugs until they stop working, then switch to other drugs, so treatment – and side effects – can go on for years.
The treatment’s short-term side effects, including fever and hallucinations, are often intense as the assets’s revved-up immune system goes on the attack. The long-term side effects of the treatment are unknown. It’s also unclear if patients whose cancer goes into remission will be cured or will have their cancer come back eventually. The FDA panel recommended that patients who get the treatment be monitored for fifteen years.
Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and infrequent gene-linked diseases. A few products have been approved elsewhere – one for head and neck cancer in China in two thousand four and two in Europe, most recently GlaxoSmithKline’s Strimvelis. That was approved last year for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.
UniQure’s Glybera was approved for a infrequent enzyme disorder. It was used only once in five years, likely due to its $1 million-plus price tag, so uniQure is pulling it from the market.
© two thousand seventeen CBS Interactive Inc. All Rights Reserved. This material may not be published, broadcast, rewritten, or redistributed. The Associated Press contributed to this report.
CAR-T gene therapy for leukemia clears FDA hurdle – CBS News
CAR-T gene therapy for leukemia clears FDA hurdle
Last Updated Jul 13, two thousand seventeen 11:16 AM EDT
A panel of cancer experts has voted unanimously in favor of a leukemia treatment which could be the very first gene therapy available in the U.S.
The Food and Drug Administration advisory panel voted 10-0 on Wednesday to recommend approval of the treatment developed by the University of Pennsylvania and Novartis Corp. The one-time treatment would be for children and youthful adults with advanced leukemia.
The therapy could be the very first of a wave of treatments custom-made to target a patient’s cancer . Called CAR-T, it involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells to recognize and demolish cancer and injecting them back into the patient.
The FDA is not required to go after the panel’s recommendation but often does.
FDA panel to vote on revolutionary gene therapy for cancer
“It’s a pretty amazing fresh treatment,” Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, said on “CBS This Morning.” “They take the white [blood] cells out of a child with cancer, they send them to [a lab in] Fresh Jersey, and they put in a gene to reprogram these cells to attack the cancer.”
The vote came after lengthy discussion and impassioned prayers from the fathers of two youthfull patients whose lives were saved by the therapy. The one-time leukemia treatment would be for children and youthfull adults with the most common form of childhood cancer, known as ALL.
“Our daughter was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pennsylvania. His daughter Emily, now 12, was the very first child to receive the experimental therapy, five years ago. “We believe when this treatment is approved, it will save thousands of children’s lives around the world.”
In a key test, results were far better than chemotherapy and even newer types of cancer drugs. Of the fifty two patients whose results were analyzed, eighty three percent had finish remission, meaning their cancer vanished. Most patients suffered serious side effects. Eleven patients died, four from side effects and seven from their leukemia.
The FDA is expected to determine whether to approve the Novartis treatment in the next few months. The drugmaker is seeking approval to use the treatment for patients aged three to twenty five with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to react to standard treatment. That happens to more than six hundred patients in the U.S. each year. At that point, they have limited options – all more toxic than the CAR-T therapy – and survival chances are slender. ALL accounts for a quarter of all cancers in children under age 15.
After decades of setbacks and disappointments in efforts to fix, substitute, or switch genes to cure diseases, several companies are near the finish line in a race to bring CAR-T and other types of gene therapy to patients. Kite Pharma also has a CAR-T therapy under FDA review and Juno Therapeutics and others are in late stages of testing.
In March, CBS News chief medical correspondent Dr. Jon LaPook reported on the results of a clinical trial in which one hundred one patients with advanced lymphoma who had failed previous therapy received the Kite Pharma CAR-T treatment. About eight months after a single treatment, thirty nine percent of patients had no evidence of cancer.
“That’s actually fairly remarkable knowing that at best only one out of ten of these patients could have finish disappearance of their lymphoma with standard chemotherapy,” said Dr. Frederick Locke, who helped lead the trial.
CAR-T therapy starts with filtering key immune cells called T cells from a patient’s blood. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancer cells. Millions of copies of the fresh T cells are grown in the lab and then injected into the patient’s bloodstream where they can seek out and ruin cancer cells. Doctors call it a “living drug” – permanently altered cells that proceed to multiply in the figure to fight the disease.
During the patient testing, the entire process took about sixteen weeks on average, which can be too long a wait for some despairingly ill patients, the FDA advisers noted during Wednesday’s meeting in Silver Spring, Maryland. Drug company officials said they can now produce a treatment and get it to a patient in about three weeks.
Novartis said in a statement after the vote that it has long believed CAR-T therapy could “switch the cancer treatment paradigm.”
“It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy,” said the Penn team’s leader, Dr. Carl June.
The cost of CAR-T therapy is likely to be hundreds of thousands of dollars, but it’s only given once. Typically, cancer patients take one or more drugs until they stop working, then switch to other drugs, so treatment – and side effects – can go on for years.
The treatment’s short-term side effects, including fever and hallucinations, are often intense as the bod’s revved-up immune system goes on the attack. The long-term side effects of the treatment are unknown. It’s also unclear if patients whose cancer goes into remission will be cured or will have their cancer comeback eventually. The FDA panel recommended that patients who get the treatment be monitored for fifteen years.
Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and infrequent gene-linked diseases. A few products have been approved elsewhere – one for head and neck cancer in China in two thousand four and two in Europe, most recently GlaxoSmithKline’s Strimvelis. That was approved last year for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.
UniQure’s Glybera was approved for a infrequent enzyme disorder. It was used only once in five years, likely due to its $1 million-plus price tag, so uniQure is pulling it from the market.
© two thousand seventeen CBS Interactive Inc. All Rights Reserved. This material may not be published, broadcast, rewritten, or redistributed. The Associated Press contributed to this report.
CAR-T gene therapy for leukemia clears FDA hurdle – CBS News
CBS News Logo
CAR-T gene therapy for leukemia clears FDA hurdle
In this photo taken March 21, 2017, a nurse reaches for blood samples taken from a patient receiving a kind of immunotherapy known as CAR-T cell therapy at the Fred Hutchinson Cancer Research Center in Seattle.
Last Updated Jul 13, two thousand seventeen 11:16 AM EDT
A panel of cancer experts has voted unanimously in favor of a leukemia treatment which could be the very first gene therapy available in the U.S.
The Food and Drug Administration advisory panel voted 10-0 on Wednesday to recommend approval of the treatment developed by the University of Pennsylvania and Novartis Corp. The one-time treatment would be for children and youthfull adults with advanced leukemia.
The therapy could be the very first of a wave of treatments custom-made to target a patient’s cancer . Called CAR-T, it involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells to recognize and ruin cancer and injecting them back into the patient.
The FDA is not required to go after the panel’s recommendation but often does.
FDA panel to vote on revolutionary gene therapy for cancer
An FDA panel meets Wednesday to determine whether to recommend the very first government-approved gene therapy. CAR T-cell treatment uses the bod’s own .
“It’s a pretty amazing fresh treatment,” Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, said on “CBS This Morning.” “They take the white [blood] cells out of a child with cancer, they send them to [a lab in] Fresh Jersey, and they put in a gene to reprogram these cells to attack the cancer.”
The vote came after lengthy discussion and impassioned prayers from the fathers of two youthful patients whose lives were saved by the therapy. The one-time leukemia treatment would be for children and youthfull adults with the most common form of childhood cancer, known as ALL.
“Our daughter was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pennsylvania. His daughter Emily, now 12, was the very first child to receive the experimental therapy, five years ago. “We believe when this treatment is approved, it will save thousands of children’s lives around the world.”
In a key test, results were far better than chemotherapy and even newer types of cancer drugs. Of the fifty two patients whose results were analyzed, eighty three percent had accomplish remission, meaning their cancer vanished. Most patients suffered serious side effects. Eleven patients died, four from side effects and seven from their leukemia.
The FDA is expected to determine whether to approve the Novartis treatment in the next few months. The drugmaker is seeking approval to use the treatment for patients aged three to twenty five with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to react to standard treatment. That happens to more than six hundred patients in the U.S. each year. At that point, they have limited options – all more toxic than the CAR-T therapy – and survival chances are slender. ALL accounts for a quarter of all cancers in children under age 15.
After decades of setbacks and disappointments in efforts to fix, substitute, or switch genes to cure diseases, several companies are near the finish line in a race to bring CAR-T and other types of gene therapy to patients. Kite Pharma also has a CAR-T therapy under FDA review and Juno Therapeutics and others are in late stages of testing.
In March, CBS News chief medical correspondent Dr. Jon LaPook reported on the results of a clinical trial in which one hundred one patients with advanced lymphoma who had failed previous therapy received the Kite Pharma CAR-T treatment. About eight months after a single treatment, thirty nine percent of patients had no evidence of cancer.
“That’s actually fairly remarkable knowing that at best only one out of ten of these patients could have accomplish disappearance of their lymphoma with standard chemotherapy,” said Dr. Frederick Locke, who helped lead the trial.
CAR-T therapy starts with filtering key immune cells called T cells from a patient’s blood. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancer cells. Millions of copies of the fresh T cells are grown in the lab and then injected into the patient’s bloodstream where they can seek out and ruin cancer cells. Doctors call it a “living drug” – permanently altered cells that proceed to multiply in the figure to fight the disease.
During the patient testing, the entire process took about sixteen weeks on average, which can be too long a wait for some despairingly ill patients, the FDA advisers noted during Wednesday’s meeting in Silver Spring, Maryland. Drug company officials said they can now produce a treatment and get it to a patient in about three weeks.
Novartis said in a statement after the vote that it has long believed CAR-T therapy could “switch the cancer treatment paradigm.”
“It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy,” said the Penn team’s leader, Dr. Carl June.
The cost of CAR-T therapy is likely to be hundreds of thousands of dollars, but it’s only given once. Typically, cancer patients take one or more drugs until they stop working, then switch to other drugs, so treatment – and side effects – can go on for years.
The treatment’s short-term side effects, including fever and hallucinations, are often intense as the bod’s revved-up immune system goes on the attack. The long-term side effects of the treatment are unknown. It’s also unclear if patients whose cancer goes into remission will be cured or will have their cancer come back eventually. The FDA panel recommended that patients who get the treatment be monitored for fifteen years.
Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and uncommon gene-linked diseases. A few products have been approved elsewhere – one for head and neck cancer in China in two thousand four and two in Europe, most recently GlaxoSmithKline’s Strimvelis. That was approved last year for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.
UniQure’s Glybera was approved for a uncommon enzyme disorder. It was used only once in five years, likely due to its $1 million-plus price tag, so uniQure is pulling it from the market.
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