The Very first CAR-T Therapy Drug Is Approved and Recommended by FDA Experts Unanimously on a 10-0 Vote!
Novartis’ chimeric antigen receptor T cell therapy (CAR-T) was supported and approved unanimously (10-0) on July 12th by the U.S. FDA Advisory Committee experts. This vote marks the key milepost of this kind of experimental treatment. FDA is expected to give final approval decision on October 3rd. Carl June, a scientist from the University of Pennsylvania who led the drug development said that this may open a fresh chapter in CAR T immunotherapy—”a truly viable drug.”
CAR-T manufactured by Novartis is only for a puny number of children and adolescents with leukemia who get no responses from standard treatment. These patients often have severe prognosis, and the key tests from almost ten countries, 83% of the patients get into remission. After one year, Two/Three of patients are still in remission stage. Childhood leukemia is only embark studied by industrial and academic field. Kite pharmaceutical company, located in Santa Monica City of California, had applied for approval in the treatment of non-Hodgkin’s lymphoma, and Novartis was following. Researchers are also exploring CAR-T for leukemia therapy and numerous myeloma. They are solving a more difficult challenge that this therapy can be used for tumor in lung or brain.
CAR-T cell therapy extracting a white blood cells (immune system guards) called T cells from patients’ blood, freezing and sending to large manufacturing plant in Morris Plains city, Fresh Jersey. There, modified HIV fragment was used for genetic modification of T cells, so that T cell can find and attack cancer cells. The modified cell was frozen again and re-injected into the patient. Once in the assets, the number of T cells enlargened.
Doctors and researchers were excited obviously. Oncologists at University of Pennsylvania and Stephen Schuster who led Novartis Lymphoma Research said, “we are saving those patients who let all the scientists were at a loss to maintain their lives three or four years ago.” Novartis’ lymphoma research and Kite’s pharmaceutical test displayed that drug treatment can make about 1/Three of patients with advanced disease (all treatment options have been fatigued) eased.
However, with enthusiasm, the safety, cost and complexity of therapy are urgent.
The company has not disclosed the price of treatment, but analysts predict that the disposable infusion is about $300 thousand to $600 thousand. Brad Loncar (a company whose investment fund is focused on the development of immunotherapy) hoped that the spend will not lead to a rebound. He said, “CAR-T is not EpiPen. It is truly beyond the limit and the frontiers of science”.
However, the fattest concern is security issue. Accelerated immune system becomes an effective anti-cancer agent, but it was also a danger on patients. Many serious side effects need us to worry about. Stephan Grupp, from Children’s Hospital of Philadelphia, said, “securely treatment is the core; validity problems will be resolved, but safety requires a lot of attention.” Grupp was the directory leading pediatric research and early test of Novartis hospital cancer immunotherapy program.
One of the most common side effects are cytokine release syndrome, which can cause high fever and symptoms of anxiety. In some cases, it may be too dangerous to patients in intensive care. Another major concern is neurotoxicity, which may cause improvised confusion or potentially fatal brain edema. Juno Therapeutics shut down its CAR-T program after the death of five patients with brain edema. Novartis official said there was no brain edema in the test of Novartis.
In order to ensure the safety of patients, Novartis had no typical plans to promote products as widely as possible. Instead, the company will appoint thirty to thirty five medical centers to implement treatment. Many medical staff participated in clinical trials, and got a lot of training of Grupp.
With the terrible practice from Emily Whitehead five years ago, Grupp said he and his staff understood the side effects of CAR-T therapy and how to treat them. Emily had recurred two times of acute lymphoblastic leukemia with routine treatment. Grupp suggested to her parents to let Emily be the very first one receiving the experimental treatment. After treatment, the Emily’s temperature rose, blood pressure was dropped, and stayed in ICU of hospital ultimately for two weeks. When Grupp assured that Emily was not likely to live another day, he got the laboratory test results which demonstrated that the surge in interleukin six protein made her immune system attack the bod. The doctor determined to give a tocilizumab immune suppressing drugs. She improved in a few hours. On the 2nd day, she woke up on her seven bday. The tester demonstrated that her cancer had disappeared.
CAR-T cell therapy’ authorization will represent the 2nd major advance in the treatment of immune less than ten years. In 2011, FDA approved the very first prep of fresh drugs called immune check point. From then on, five other drugs were also approved. There is a big difference inbetween the two treatments. Checkpoint inhibitors are for solid tumors, such as malignant melanoma, lung cancer and bladder cancer, while CAR-T cell therapeutics is for blood disease. Albeit checkpoint inhibitors are readily available, each patient receives the same drug, while CAR-T cell therapies is based on a custom-built bod. Many immune treatment experts believe that when researchers find the combination method, cancer will achieve the largest progress.
For University of Pennsylvania team, CAR-T cell story can be traced back to a few decades ago. There was known as a National Naval Medical Center, where June and Bruce Levine researcher engaged in the explore of fresh HIV treatment. In this process, they came up with an enhanced T cell to make it more powerful and more adequate. Two playmates moved to Philadelphia in one thousand nine hundred ninety nine for cancer research. Two years later, June’s wifey died of ovarian cancer, which motivated him to work stiffer in this field. In the next few years, researchers around the country obtained a T cell and a series of attractive discoveries.
About 2010, Bill Ludwig became the very first patient receiving CAR-T cell therapy in the University of Pennsylvania. Another two people received treatment in the near future. One is still in remission, and the other one died of recurrence. After the three patients, researchers at University of Pennsylvania spent all money but cannot have more treatment. In order to enhance people’s interest for this therapy and access to funds, they determined to publish research results. In August, 2011, an article published on fresh England Journal of Medicine (NEJM) caused a storm to bring fresh resources to them. The pediatric trial and success of Emily therapy opened a fresh spring. After six months, the University of Pennsylvania transferred the technology to Novartis in exchange for financial support, including a fresh cell manufacturing facility.
FDA’s approval seems imminent, and researchers have played an significant role in the development and testing of treatments.
Very first gene therapy – ‘a true living drug’ – on the cusp of FDA approval – The Washington Post
The Very first CAR-T Therapy Drug Is Approved and Recommended by FDA Experts Unanimously on a 10-0 Vote, Biolabs Blog
The Very first CAR-T Therapy Drug Is Approved and Recommended by FDA Experts Unanimously on a 10-0 Vote!
Novartis’ chimeric antigen receptor T cell therapy (CAR-T) was supported and approved unanimously (10-0) on July 12th by the U.S. FDA Advisory Committee experts. This vote marks the key milepost of this kind of experimental treatment. FDA is expected to give final approval decision on October 3rd. Carl June, a scientist from the University of Pennsylvania who led the drug development said that this may open a fresh chapter in CAR T immunotherapy—”a truly viable drug.”
CAR-T manufactured by Novartis is only for a puny number of children and adolescents with leukemia who get no responses from standard treatment. These patients often have severe prognosis, and the key tests from almost ten countries, 83% of the patients get into remission. After one year, Two/Trio of patients are still in remission stage. Childhood leukemia is only begin studied by industrial and academic field. Kite pharmaceutical company, located in Santa Monica City of California, had applied for approval in the treatment of non-Hodgkin’s lymphoma, and Novartis was following. Researchers are also exploring CAR-T for leukemia therapy and numerous myeloma. They are solving a more difficult challenge that this therapy can be used for tumor in lung or brain.
CAR-T cell therapy extracting a white blood cells (immune system guards) called T cells from patients’ blood, freezing and sending to large manufacturing plant in Morris Plains city, Fresh Jersey. There, modified HIV fragment was used for genetic modification of T cells, so that T cell can find and attack cancer cells. The modified cell was frozen again and re-injected into the patient. Once in the figure, the number of T cells enhanced.
Doctors and researchers were excited obviously. Oncologists at University of Pennsylvania and Stephen Schuster who led Novartis Lymphoma Research said, “we are saving those patients who let all the scientists were at a loss to maintain their lives three or four years ago.” Novartis’ lymphoma research and Kite’s pharmaceutical test demonstrated that drug treatment can make about 1/Three of patients with advanced disease (all treatment options have been weakened) eased.
However, with enthusiasm, the safety, cost and complexity of therapy are urgent.
The company has not disclosed the price of treatment, but analysts predict that the disposable infusion is about $300 thousand to $600 thousand. Brad Loncar (a company whose investment fund is focused on the development of immunotherapy) hoped that the spend will not lead to a rebound. He said, “CAR-T is not EpiPen. It is indeed beyond the limit and the frontiers of science”.
However, the largest concern is security issue. Accelerated immune system becomes an effective anti-cancer agent, but it was also a danger on patients. Many serious side effects need us to worry about. Stephan Grupp, from Children’s Hospital of Philadelphia, said, “securely treatment is the core; validity problems will be resolved, but safety requires a lot of attention.” Grupp was the directory leading pediatric research and early test of Novartis hospital cancer immunotherapy program.
One of the most common side effects are cytokine release syndrome, which can cause high fever and symptoms of anxiety. In some cases, it may be too dangerous to patients in intensive care. Another major concern is neurotoxicity, which may cause improvised confusion or potentially fatal brain edema. Juno Therapeutics shut down its CAR-T program after the death of five patients with brain edema. Novartis official said there was no brain edema in the test of Novartis.
In order to ensure the safety of patients, Novartis had no typical plans to promote products as widely as possible. Instead, the company will appoint thirty to thirty five medical centers to implement treatment. Many medical staff participated in clinical trials, and got a lot of training of Grupp.
With the terrible practice from Emily Whitehead five years ago, Grupp said he and his staff understood the side effects of CAR-T therapy and how to treat them. Emily had recurred two times of acute lymphoblastic leukemia with routine treatment. Grupp suggested to her parents to let Emily be the very first one receiving the experimental treatment. After treatment, the Emily’s temperature rose, blood pressure was dropped, and stayed in ICU of hospital ultimately for two weeks. When Grupp assured that Emily was not likely to live another day, he got the laboratory test results which showcased that the surge in interleukin six protein made her immune system attack the assets. The doctor determined to give a tocilizumab immune suppressing drugs. She improved in a few hours. On the 2nd day, she woke up on her seven bday. The tester displayed that her cancer had disappeared.
CAR-T cell therapy’ authorization will represent the 2nd major advance in the treatment of immune less than ten years. In 2011, FDA approved the very first prep of fresh drugs called immune check point. From then on, five other drugs were also approved. There is a big difference inbetween the two treatments. Checkpoint inhibitors are for solid tumors, such as malignant melanoma, lung cancer and bladder cancer, while CAR-T cell therapeutics is for blood disease. Albeit checkpoint inhibitors are readily available, each patient receives the same drug, while CAR-T cell therapies is based on a custom-made bod. Many immune treatment experts believe that when researchers find the combination method, cancer will achieve the largest progress.
For University of Pennsylvania team, CAR-T cell story can be traced back to a few decades ago. There was known as a National Naval Medical Center, where June and Bruce Levine researcher engaged in the probe of fresh HIV treatment. In this process, they came up with an enhanced T cell to make it more powerful and more adequate. Two fucking partners moved to Philadelphia in one thousand nine hundred ninety nine for cancer research. Two years later, June’s wifey died of ovarian cancer, which motivated him to work stiffer in this field. In the next few years, researchers around the country obtained a T cell and a series of attractive discoveries.
About 2010, Bill Ludwig became the very first patient receiving CAR-T cell therapy in the University of Pennsylvania. Another two people received treatment in the near future. One is still in remission, and the other one died of recurrence. After the three patients, researchers at University of Pennsylvania spent all money but cannot have more treatment. In order to enhance people’s interest for this therapy and access to funds, they determined to publish research results. In August, 2011, an article published on fresh England Journal of Medicine (NEJM) caused a storm to bring fresh resources to them. The pediatric trial and success of Emily therapy opened a fresh spring. After six months, the University of Pennsylvania transferred the technology to Novartis in exchange for financial support, including a fresh cell manufacturing facility.
FDA’s approval seems imminent, and researchers have played an significant role in the development and testing of treatments.
Very first gene therapy – ‘a true living drug’ – on the cusp of FDA approval – The Washington Post