FDA Approves First-of-Its-Kind Cancer Treatment

August 30, two thousand seventeen — The FDA has for the very first time approved a treatment that uses a patient’s own genetically modified cells to attack a type of leukemia, opening the door to what the agency calls “a fresh frontier” in medicine.

The approval Wednesday permits a process known as CAR T-cell therapy to be used in children or youthfull adults fighting an often fatal recurrence of the most common childhood cancer — B-cell acute lymphoblastic leukemia.

And it clears the way for a fresh treatment to fighting cancer by harnessing the body’s immune system — a long-sought purpose of medical researchers.

“This is a fantasy come true,” says Henry Fung, MD, director of the Fox Pursue Cancer Center-Temple University Hospital Bone Marrow Transplant Program. “It’s now limited to one disease in children only, but that platform potentially can benefit a lot of different types of cancer patients, particularly blood cancer patients.”

‘A Fresh Frontier’

FDA Commissioner Scott Gottlieb, MD, called the approval of the therapy — brand named Kymriah — a “fresh frontier in medical innovation.”

In a news conference on Wednesday, Gottlieb said the FDA had seventy six active investigational fresh drug applications related to CAR T-cell products, and more than five hundred for gene therapy products are being studied for a diversity of ailments, ranging from genetic disorders to autoimmune diseases, diabetes, cancer, and HIV.

“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,” Gottlieb says.

Fung, who’s also vice chairman of hematology/oncology at Fox Pursue, says the treatment could help patients hammer back an illness that has resisted conventional treatments like chemotherapy and radiation, leaving them facing death. “This is the breakthrough of the century,” he says.

And Hetty Carraway, MD, an acute leukemia doctor at the Cleveland Clinic, says the freshly approved therapy represents a very first step for a fresh way of treating cancer. “If it can bring this kind of paradigm to other types of cancers, that’s indeed where I think the larger implications are,” she says.

Before This Procedure, I Was Planning My Own Funeral

Taking the Fight to Cancer

B-cell acute lymphoblastic leukemia attacks the blood cells that make antibodies, which help your bod fight off disease. Most of the time, it’s treated successfully with chemotherapy, radiation, or by transplants of bone marrow, which produces blood cells. But in some cases, treatment fails to strike back the cancer, or it comes back. When that happens, the odds of survival fall to as little as one in Ten.

The fresh treatment is a one-time infusion developed by researchers at the University of Pennsylvania and the pharmaceutical company Novartis. Officially known as chimeric antigen receptor T-cell therapy, it starts with doctors extracting disease-fighting white blood cells, known as T cells, from a patient’s blood. The cells are frozen and shipped to a laboratory, where they’re genetically engineered to attack a specific protein on the cancerous B cells.

They’re then put back into the assets, where they seek out and ruin cancer cells. And because they’re cells taken from the patient’s own figure, there’s no need for anti-rejection drugs, which are needed after transplants.

“This is truly combining everything together,” Fung says. “This is truly using patients’ own immune cells to fight cancer.”

Dangerous Side Effects Remain a Concern

The therapy can have dangerous side effects — mainly a condition known as cytokine release syndrome (CRS). That happens when T cells release a lot of a chemical messenger into the bloodstream. This affects the vascular system, causing high fevers and acute drops in blood pressure. More than 60% of patients in clinical trials had side effects due to cytokine release, Novartis reported, but none of those reactions were fatal.

We believe this treatment can switch the world. But we know some children relapse and we know children who didn’t make it.

Tom Whitehead, father of cancer patient Emily Whitehead

Emily Whitehead, the very first pediatric patient to attempt the therapy in 2011, had such a bad reaction primarily that she was in a coma for fourteen days. Her doctors told the family to say their good-byes.

“They believed she had less than a 1-in-1,000 chance of surviving to the next morning,” says her father, Tom Whitehead.

As a last hope, doctors gave Emily the arthritis drug Actemra (tocilizumab), which blocks one of the main inflammatory signals driving the CRS. On Wednesday, the FDA also approved Actemra as a treatment for CRS. In fact, under the conditions of approval, doctor’s can’t use CAR T therapy unless they also have Actemra on mitt to manage side effects.

Within twelve hours, Emily embarked to recover. She has been cancer free for five years.

Because of the side effects, Kymriah won’t be available everywhere. Hopsitals and clinics will have to be specially certified to administer the treatment. Doctors and other staff will also have get extra training before they can prescribe it.

“We know and expect that type of side effect will happen, and we know that we can successfully manage it,” she says. “But it needs to be managed by people who are familiar with this type of side effect and how best to support patients,” Carraway says.

Other side effects included anemia, nausea, diarrhea, and headaches.

In three trials involving about one hundred fifty people, the remission rates were 69%, 83%, and 95%. A total of seventeen patients died after receiving the treatment; fourteen of them from the disease and three from infections, according to documents the company filed with the FDA.

“We believe this treatment can switch the world,” says Tom Whitehead, who frequently speaks about his daughter’s practice and testified before the FDA about the treatment. He also helps raise money for children’s cancer research through The Emily Whitehead Foundation. “But we know some children relapse and we know children who didn’t make it.”

Big Possibilities and a Big Price Tag

Another concern is the price tag associated with the therapy: The process is reported to cost as much as $475,000.

In a press release, the Center for Medicare and Medicaid Services (CMS) announced that it was exploring “innovative payment modes and arrangements” for Kymriah and other potentially life-saving treatments.

In a news release, Novartis, the company that makes Kymriah, said it was collaborating with CMS on an outcomes-based treatment to pricing, which would mean that the company would only be reimbursed if a patient responds to the therapy by the end of the very first month of treatment.

“Certainly, it’s far and above the expense that we typically see for drugs,” Carraway says. But current treatments can also run into the low six figures, sometimes with little success. The number of patients with relapsed acute lymphoblastic leukemia is puny, “and the options for them in their youthful lives are pretty limited.”

We hope CAR T is the end of it all.

Patrick Thislethwaite, father of cancer patient Liam

“Our hope is we’ll get better at making these medications, and hopefully, with time, the cost of this will decrease,” she adds.

Novartis spokeswoman Julie Masow says the company will do “everything we can” to help get the treatment to patients who need it.

“We are cautiously considering the adequate price for CTL019, taking into consideration the value that this treatment represents for patients, society, and the health care system, both near-term and long-term, as well as input from outward health economic experts,” Masow says.

The therapy was produced “via pioneering technology and a sophisticated manufacturing process,” she says — however, “We recognize our responsibility in bringing this innovative treatment to patients.”

‘He’s Commenced School’

One of the more latest patients to have CAR T-cell therapy is 5-year-old Liam Thistlethwaite. He has been cancer free for four months since commencing the therapy to treat his acute lymphoblastic leukemia.

Very first diagnosed shortly before his 2nd bday, Liam had gotten thirty two months of different kinds of chemotherapy drugs to poison the cancer out of his petite bod. The treatment is harsh but almost always successful. Doctors told Liam’s parents he had a 96% chance of a cure if he could finish it.

Liam Thistlethwaite in a hospital bed.

But eight months later, Liam’s cancer came back, with a vengeance. Leukemia cells spread to his spinal fluid. Tumors grew on two glands in his brain.

Liam’s doctor, Ching-Hon Pui, MD, chairman of the Oncology Department at St. Jude, had recently been to a medical conference that discussed the results of the CAR T-cell therapy. He persuaded Children’s Hospital of Philadelphia to put him on its waiting list, which was about six months long at the time.

Because Liam was relatively healthy and had a low cancer cargo when he was treated, his father thinks he avoided some of the most severe side effects of the therapy. He spiked very high fevers and spent a few days in the hospital but pulled through.

“He’s embarked school. He’s doing wonderfully,” says Patrick Thistlethwaite.

Major Questions Remain Despite Optimism

One of the unanswered questions is how long CAR T cells can last in the figure. In some patients, they’ve persisted for as long as five years. Others have their cells die in weeks or months. Another big question is whether the cancer will come back if the CAR T cells are gone.

Liam, this year, ready for the very first day of school.

The Thistlethwaites say it was very hard to know whether to attempt CAR T on a toddler.

“Our physician truly felt that we’d have the same odds, so to speak, as going into a stem cell transplant with powerful radiation. He believed CAR T to have high side effects up front, but no high long-term side effects,” Patrick Thistlethwaite says.

They knew radiation to Liam’s brain and spinal cord could cause long-term harm.

“We still have those options,” Patrick says. “We hope we never have to use them.”

“We hope CAR T is the end of it all.”

Sources

National Cancer Institute: “CAR-T Cells.”

Leukemia and Lymphoma Society: “Relapsed and Refractory ALL.”

American Cancer Society: “Cancers that Develop in Children.”

News release: “Novartis CAR-T cell therapy CTL019 unanimously (10-0) recommended for approval by FDA advisory committee to treat pediatric, youthful adult r/r B-cell ALL.”

Henry Fung, MD, director, Fox Pursue Cancer Center-Temple University Hospital Bone Marrow Transplant Program.

Hetty Carraway, MD, acute leukemia doctor, Cleveland Clinic.

Ching-Hon Pui, MD, chairman, Department of Oncology, St. Jude Children’s Research Hospital.

FDA.gov: “Slides for the July 12, two thousand seventeen Meeting of the Oncologic Drugs Advisory Committee (ODAC).”

Tom Whitehead, The Emily Whitehead Foundation. Drug maker Novartis is a sponsor of the foundation’s upcoming Believe Ball, which raises money for children’s cancer research.

FDA Approves First-of-Its-Kind Cancer Treatment

The approval Wednesday permits a process known as CAR T-cell therapy to be used in children or youthful adults fighting an often fatal recurrence of the most common childhood cancer — B-cell acute lymphoblastic leukemia.

And it clears the way for a fresh treatment to fighting cancer by harnessing the body’s immune system — a long-sought objective of medical researchers.

‘A Fresh Frontier’

FDA Commissioner Scott Gottlieb, MD, called the approval of the therapy — brand named Kymriah — a “fresh frontier in medical innovation.”

In a news conference on Wednesday, Gottlieb said the FDA had seventy six active investigational fresh drug applications related to CAR T-cell products, and more than five hundred for gene therapy products are being studied for a multitude of ailments, ranging from genetic disorders to autoimmune diseases, diabetes, cancer, and HIV.

Before This Procedure, I Was Planning My Own Funeral

Taking the Fight to Cancer

B-cell acute lymphoblastic leukemia attacks the blood cells that make antibodies, which help your bod fight off disease. Most of the time, it’s treated successfully with chemotherapy, radiation, or by transplants of bone marrow, which produces blood cells. But in some cases, treatment fails to hammer back the cancer, or it comes back. When that happens, the odds of survival fall to as little as one in Ten.

They’re then put back into the figure, where they seek out and ruin cancer cells. And because they’re cells taken from the patient’s own figure, there’s no need for anti-rejection drugs, which are needed after transplants.

“This is indeed combining everything together,” Fung says. “This is truly using patients’ own immune cells to fight cancer.”

Dangerous Side Effects Remain a Concern

We believe this treatment can switch the world. But we know some children relapse and we know children who didn’t make it.

Tom Whitehead, father of cancer patient Emily Whitehead

Emily Whitehead, the very first pediatric patient to attempt the therapy in 2011, had such a bad reaction originally that she was in a coma for fourteen days. Her doctors told the family to say their good-byes.

“They believed she had less than a 1-in-1,000 chance of surviving to the next morning,” says her father, Tom Whitehead.

Within twelve hours, Emily began to recover. She has been cancer free for five years.

Other side effects included anemia, nausea, diarrhea, and headaches.

Big Possibilities and a Big Price Tag

Another concern is the price tag associated with the therapy: The process is reported to cost as much as $475,000.

“Certainly, it’s far and above the expense that we typically see for drugs,” Carraway says. But current treatments can also run into the low six figures, sometimes with little success. The number of patients with relapsed acute lymphoblastic leukemia is petite, “and the options for them in their youthful lives are pretty limited.”

We hope CAR T is the end of it all.

Patrick Thislethwaite, father of cancer patient Liam

“Our hope is we’ll get better at making these medications, and hopefully, with time, the cost of this will decrease,” she adds.

Novartis spokeswoman Julie Masow says the company will do “everything we can” to help get the treatment to patients who need it.

‘He’s Began School’

One of the more latest patients to have CAR T-cell therapy is 5-year-old Liam Thistlethwaite. He has been cancer free for four months since kicking off the therapy to treat his acute lymphoblastic leukemia.

Very first diagnosed shortly before his 2nd bday, Liam had gotten thirty two months of different kinds of chemotherapy drugs to poison the cancer out of his petite figure. The treatment is harsh but almost always successful. Doctors told Liam’s parents he had a 96% chance of a cure if he could finish it.

Liam Thistlethwaite in a hospital bed.

But eight months later, Liam’s cancer came back, with a vengeance. Leukemia cells spread to his spinal fluid. Tumors grew on two glands in his brain.

“He’s commenced school. He’s doing wonderfully,” says Patrick Thistlethwaite.

Major Questions Remain Despite Optimism

One of the unanswered questions is how long CAR T cells can last in the bod. In some patients, they’ve persisted for as long as five years. Others have their cells die in weeks or months. Another big question is whether the cancer will come back if the CAR T cells are gone.

Liam, this year, ready for the very first day of school.

The Thistlethwaites say it was very hard to know whether to attempt CAR T on a toddler.

“Our physician truly felt that we’d have the same odds, so to speak, as going into a stem cell transplant with intense radiation. He believed CAR T to have high side effects up front, but no high long-term side effects,” Patrick Thistlethwaite says.

They knew radiation to Liam’s brain and spinal cord could cause long-term harm.

“We still have those options,” Patrick says. “We hope we never have to use them.”

“We hope CAR T is the end of it all.”

Sources

National Cancer Institute: “CAR-T Cells.”

Leukemia and Lymphoma Society: “Relapsed and Refractory ALL.”

American Cancer Society: “Cancers that Develop in Children.”

News release: “Novartis CAR-T cell therapy CTL019 unanimously (10-0) recommended for approval by FDA advisory committee to treat pediatric, youthfull adult r/r B-cell ALL.”

Henry Fung, MD, director, Fox Pursue Cancer Center-Temple University Hospital Bone Marrow Transplant Program.

Hetty Carraway, MD, acute leukemia doctor, Cleveland Clinic.

Ching-Hon Pui, MD, chairman, Department of Oncology, St. Jude Children’s Research Hospital.

FDA.gov: “Slides for the July 12, two thousand seventeen Meeting of the Oncologic Drugs Advisory Committee (ODAC).”

Tom Whitehead, The Emily Whitehead Foundation. Drug maker Novartis is a sponsor of the foundation’s upcoming Believe Ball, which raises money for children’s cancer research.

FDA Approves First-of-Its-Kind Cancer Treatment

And it clears the way for a fresh treatment to fighting cancer by harnessing the body’s immune system — a long-sought objective of medical researchers.

‘A Fresh Frontier’

FDA Commissioner Scott Gottlieb, MD, called the approval of the therapy — brand named Kymriah — a “fresh frontier in medical innovation.”

In a news conference on Wednesday, Gottlieb said the FDA had seventy six active investigational fresh drug applications related to CAR T-cell products, and more than five hundred for gene therapy products are being studied for a multitude of ailments, ranging from genetic disorders to autoimmune diseases, diabetes, cancer, and HIV.

And Hetty Carraway, MD, an acute leukemia doctor at the Cleveland Clinic, says the freshly approved therapy represents a very first step for a fresh way of treating cancer. “If it can bring this kind of paradigm to other types of cancers, that’s truly where I think the larger implications are,” she says.

Before This Procedure, I Was Planning My Own Funeral

Taking the Fight to Cancer

B-cell acute lymphoblastic leukemia attacks the blood cells that make antibodies, which help your figure fight off disease. Most of the time, it’s treated successfully with chemotherapy, radiation, or by transplants of bone marrow, which produces blood cells. But in some cases, treatment fails to hammer back the cancer, or it comes back. When that happens, the odds of survival fall to as little as one in Ten.

They’re then put back into the assets, where they seek out and ruin cancer cells. And because they’re cells taken from the patient’s own bod, there’s no need for anti-rejection drugs, which are needed after transplants.

“This is indeed combining everything together,” Fung says. “This is truly using patients’ own immune cells to fight cancer.”

Dangerous Side Effects Remain a Concern

We believe this treatment can switch the world. But we know some children relapse and we know children who didn’t make it.

Tom Whitehead, father of cancer patient Emily Whitehead

“They believed she had less than a 1-in-1,000 chance of surviving to the next morning,” says her father, Tom Whitehead.

Within twelve hours, Emily embarked to recover. She has been cancer free for five years.

Other side effects included anemia, nausea, diarrhea, and headaches.

Big Possibilities and a Big Price Tag

Another concern is the price tag associated with the therapy: The process is reported to cost as much as $475,000.

We hope CAR T is the end of it all.

Patrick Thislethwaite, father of cancer patient Liam

“Our hope is we’ll get better at making these medications, and hopefully, with time, the cost of this will decrease,” she adds.

Novartis spokeswoman Julie Masow says the company will do “everything we can” to help get the treatment to patients who need it.

“We are cautiously considering the suitable price for CTL019, taking into consideration the value that this treatment represents for patients, society, and the health care system, both near-term and long-term, as well as input from outer health economic experts,” Masow says.

‘He’s Began School’

One of the more latest patients to have CAR T-cell therapy is 5-year-old Liam Thistlethwaite. He has been cancer free for four months since embarking the therapy to treat his acute lymphoblastic leukemia.

Very first diagnosed shortly before his 2nd bday, Liam had gotten thirty two months of different kinds of chemotherapy drugs to poison the cancer out of his puny figure. The treatment is harsh but almost always successful. Doctors told Liam’s parents he had a 96% chance of a cure if he could finish it.

Liam Thistlethwaite in a hospital bed.

But eight months later, Liam’s cancer came back, with a vengeance. Leukemia cells spread to his spinal fluid. Tumors grew on two glands in his brain.

Liam’s doctor, Ching-Hon Pui, MD, chairman of the Oncology Department at St. Jude, had recently been to a medical conference that discussed the results of the CAR T-cell therapy. He coaxed Children’s Hospital of Philadelphia to put him on its waiting list, which was about six months long at the time.

“He’s began school. He’s doing wonderfully,” says Patrick Thistlethwaite.

Major Questions Remain Despite Optimism

Liam, this year, ready for the very first day of school.

The Thistlethwaites say it was very hard to know whether to attempt CAR T on a toddler.

“Our physician truly felt that we’d have the same odds, so to speak, as going into a stem cell transplant with mighty radiation. He believed CAR T to have high side effects up front, but no high long-term side effects,” Patrick Thistlethwaite says.