Why the FDA approved Kymriah  a CAR
Kymriah
The drug  called  Kymriah   is a highly personalized cancer treatment called CAR T-cell therapy (CAR is short for chimeric antigen receptor).
The FDA approved  Kymriah  to treat pediatric acute lymphoblastic leukemia in people up to age 25.
The one-time treatment isn't cheap: Novartis  which makes  Kymriah   on Wednesday said the price would be $475 000  which is lower than many expected. Only about 600 people a year could get this treatment  based on Wednesday's approval. Even so  the approval has industry experts and cancer doctors excited.
How  Kymriah  works
 Kymriah  isn't your run-of-the-mill pill â or even a biologic drug  like insulin â that can be mass produced. Since the therapy is made from a person's own immune system  the process can take about three weeks.
While that's a one-time process  it's not the end of the road. About half of the patients in a Kymriah study got cytokine-release syndrome  a response to the reprogrammed cells running loose in the body. It can cause high fevers and flu-like symptoms  and it can be life-threatening. If they get it  patients need to be admitted to intensive-care units  which can bring additional costs. (The FDA on Wednesday expanded the approval of Actemra  a rheumatoid-arthritis drug that can also treat cytokine-release syndrome.)
In a trial of 63 patients treated with Kymriah  83% were in remission after three months  and 64% were still in remission after a year.
Kymriah is the first CAR-T cell therapy to get approved  and several more are in the works. Kite Pharma  Juno Therapeutics  and Bluebird Bio are among a growing group of biotech companies working with CAR-T. Kite  which was recently acquired by Gilead Sciences  is expected to hear back from the FDA about its treatment for aggressive B-cell non-Hodgkin lymphoma by November.
"We're at the very beginning of what's going to be a big new field of medicine " said  David Epstein  who helped license Kymriah from the University of Pennsylvania while at Novartis .
 Epstein  left Novartis in 2016 as CEO of its pharmaceuticals divisions.  He 's now the executive chairman of Rubius Therapeutics  a biotech firm that's also working with cell therapy to develop treatments like  Kymriah  that don't have to be as personalized. The hope is that one day doctors will be able to prescribe a cell therapy and use it that same day instead of waiting weeks to get it back.
 Epstein  said  he  envisioned cell therapies having much shorter life cycles than traditional drugs. Instead of getting a better  updated therapy for a disease every decade or so  we might begin to see second-generation cell therapies in a few years.
Those updates could increase the number of patients that cell therapies can treat. The 600 or so people a year eligible for  Kymriah  now could become tens of thousands  but  it  would require some major changes   Epstein  said.
For one  the treatments would have to treat more types of cancer than the one  Kymriah  was approved for on Wednesday.
And to affect more people  the cell therapies would need to go beyond blood cancers. Right now  that's where most of the big successes have come from  but that could one day include solid tumors and maybe even autoimmune diseases like Type 1 diabetes   Epstein  said.
The drug  called   [TGT] is a highly personalized cancer treatment called CAR T-cell therapy (CAR is short for chimeric antigen receptor).
The FDA approved  Kymriah  to treat pediatric acute lymphoblastic leukemia in people up to age 25.
The one-time treatment isn't cheap: Novartis  which makes   [TGT] on Wednesday said the price would be $475 000  which is lower than many expected. Only about 600 people a year could get this treatment  based on Wednesday's approval. Even so  the approval has industry experts and cancer doctors excited.
How   [TGT] works
  [TGT] isn't your run-of-the-mill pill â or even a biologic drug  like insulin â that can be mass produced. Since the therapy is made from a person's own immune system  the process can take about three weeks.
While that's a one-time process  it's not the end of the road. About half of the patients in a  [TGT]  study got cytokine-release syndrome  a response to the reprogrammed cells running loose in the body. It can cause high fevers and flu-like symptoms  and it can be life-threatening. If they get it  patients need to be admitted to intensive-care units  which can bring additional costs. (The FDA on Wednesday expanded the approval of Actemra  a rheumatoid-arthritis drug that can also treat cytokine-release syndrome.)
In a trial of 63 patients treated with Kymriah  83% were in remission after three months  and 64% were still in remission after a year.
 [TGT]  is the first CAR-T cell therapy to get approved  and several more are in the works. Kite Pharma  Juno Therapeutics  and Bluebird Bio are among a growing group of biotech companies working with CAR-T. Kite  which was recently acquired by Gilead Sciences  is expected to hear back from the FDA about its treatment for aggressive B-cell non-Hodgkin lymphoma by November.
"We're at the very beginning of what's going to be a big new field of medicine " said   [TGT] 
 [TGT] left Novartis in 2016 as CEO of its pharmaceuticals divisions.   [TGT]  's now the executive chairman of Rubius Therapeutics  a biotech firm that's also working with cell therapy to develop treatments like  Kymriah  that don't have to be as personalized. The hope is that one day doctors will be able to prescribe a cell therapy and use it that same day instead of waiting weeks to get it back.
  [TGT] said   [TGT]   envisioned cell therapies having much shorter life cycles than traditional drugs. Instead of getting a better  updated therapy for a disease every decade or so  we might begin to see second-generation cell therapies in a few years.
Those updates could increase the number of patients that cell therapies can treat. The 600 or so people a year eligible for   [TGT] now could become tens of thousands  but   [TGT]   would require some major changes   Epstein  said.
For one  the treatments would have to treat more types of cancer than the one  Kymriah  was approved for on Wednesday.
And to affect more people  the cell therapies would need to go beyond blood cancers. Right now  that's where most of the big successes have come from  but that could one day include solid tumors and maybe even autoimmune diseases like Type 1 diabetes    [TGT] said.