A cancer treatment that one experienced called the ‘most titillating thing I’ve seen in my lifetime’ just got approved
The US Food and Drug Administration just approved a cutting-edge cancer therapy.
On Wednesday, the FDA approved Novartis’ Kymriah, also known as tisagenlecleucel, a treatment for pediatric acute lymphoblastic leukemia.
"I think this is the most arousing thing I’ve seen in my lifetime," said Dr. Tim Cripe, an oncologist who was part of the FDA advisory committee panel that voted in July to recommend the drug’s approval.
The very personalized treatment is called CAR T-cell therapy (CAR is brief for chimeric antigen receptor). It’s a type of cancer immunotherapy, which harnesses the bod’s immune system to take on cancer cells. It eliminates a person’s cells, reengineers them, then puts them back in their bod to attack cancer cells.
"We’re injecting a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer," the FDA commissioner, Scott Gottlieb, said in a statement. "Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving."
The one-time treatments won’t come cheap — Novartis said it would cost $475,000, however it also said in a news release Wednesday that it was working with the Centers for Medicare and Medicaid Services to come up with a payment system that reflects how well the drug works in a person. As part of the collaboration, CMS will have to pay "only when pediatric and youthfull adult [acute lymphoblastic leukemia] patients react to Kymriah by the end of the very first month," Novartis said.
Novartis’ therapy is one of two cutting-edge treatments for blood cancers that are poised to get approved by the end of the year.
The FDA is also expected to make a decision about another CAR-T therapy, from Kite Pharma, which just got acquired by Gilead Sciences, for aggressive B-cell non-Hodgkin lymphoma. Kite said in February that of the one hundred one patients in its trial, 36% had a finish response to the treatment (meaning the cancer had disappeared) after six months.
It’s a type of cancer that Novartis also wants to get approval to treat. In June, Novartis released data from its Phase two trial of tisagenlecleucel in adult patients with diffuse large B-cell lymphoma, an aggressive form of lymphoma. Of the fifty one patients in the trial, twenty three had either a accomplish response or a partial response (meaning their tumor displayed signs of shrinking).
Fresh treatments like Kymriah face some other challenges:
- Manufacturing the drugs is no puny feat, considering the personalized treatment requires taking out a person’s cells, reprogramming them, then reinserting them. The company that can do that quickly and securely could have the advantage in the competitive CAR-T therapy space.
- Trials of these kinds of treatments have been deadly in the past. In May, Kite disclosed that one person had died in a clinical trial for its late-stage CAR-T therapy from cerebral edema, a condition in which excessive fluid causes the brain to erect. And last year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.
FDA approves Novartis CAR-T cancer treatment very first gene therapy – Business Insider
A cancer treatment that one experienced called the ‘most arousing thing I’ve seen in my lifetime’ just got approved
The US Food and Drug Administration just approved a cutting-edge cancer therapy.
On Wednesday, the FDA approved Novartis’ Kymriah, also known as tisagenlecleucel, a treatment for pediatric acute lymphoblastic leukemia.
"I think this is the most titillating thing I’ve seen in my lifetime," said Dr. Tim Cripe, an oncologist who was part of the FDA advisory committee panel that voted in July to recommend the drug’s approval.
The very personalized treatment is called CAR T-cell therapy (CAR is brief for chimeric antigen receptor). It’s a type of cancer immunotherapy, which harnesses the assets’s immune system to take on cancer cells. It eliminates a person’s cells, reengineers them, then puts them back in their assets to attack cancer cells.
"We’re injecting a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer," the FDA commissioner, Scott Gottlieb, said in a statement. "Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving."
The one-time treatments won’t come cheap — Novartis said it would cost $475,000, tho’ it also said in a news release Wednesday that it was working with the Centers for Medicare and Medicaid Services to come up with a payment system that reflects how well the drug works in a person. As part of the collaboration, CMS will have to pay "only when pediatric and youthfull adult [acute lymphoblastic leukemia] patients react to Kymriah by the end of the very first month," Novartis said.
Novartis’ therapy is one of two cutting-edge treatments for blood cancers that are poised to get approved by the end of the year.
The FDA is also expected to make a decision about another CAR-T therapy, from Kite Pharma, which just got acquired by Gilead Sciences, for aggressive B-cell non-Hodgkin lymphoma. Kite said in February that of the one hundred one patients in its trial, 36% had a accomplish response to the treatment (meaning the cancer had disappeared) after six months.
It’s a type of cancer that Novartis also wants to get approval to treat. In June, Novartis released data from its Phase two trial of tisagenlecleucel in adult patients with diffuse large B-cell lymphoma, an aggressive form of lymphoma. Of the fifty one patients in the trial, twenty three had either a accomplish response or a partial response (meaning their tumor displayed signs of shrinking).
Fresh treatments like Kymriah face some other challenges:
- Manufacturing the drugs is no petite feat, considering the personalized treatment requires taking out a person’s cells, reprogramming them, then reinserting them. The company that can do that quickly and securely could have the advantage in the competitive CAR-T therapy space.
- Trials of these kinds of treatments have been deadly in the past. In May, Kite disclosed that one person had died in a clinical trial for its late-stage CAR-T therapy from cerebral edema, a condition in which excessive fluid causes the brain to erect. And last year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.
