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Multiple Myeloma CAR-T Cell Immunotherapy Update

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“Side effects (from CAR-T cell therapy) can be life-threatening, however. They include high fevers, crashing blood pressure, lung congestion and neurological problems. 

No one wants CAR-T cell therapy to live up to its promise more than I do. I am a long-term survivor of an incurable blood cancer called multiple myeloma. MM is a blood cancer like the cancers discussed below. Like all chemotherapy development however, the challenge is to figure out the risk/reward proposition for you or your loved one.

Image result for image of car-t cells

In the study for car-t cell approval discussed in the NYTimes article linked and excerpted below the in addition to serious life threatening collateral damage car-t cell therapy caused two deaths.

My conclusions of the current state of car-t cell chemotherapy development for MM:

  1. As of the writing of this post, Car-t cell therapy is not available for multiple myeloma survivors yet. Great to start with blood cancers but nothing for MM yet.
  2. Once Car-T cells are available for MM patients it is clear that the short, long-term and late stage side effects can be severe.
  3. Car-t cell therapy appears to be incredibly expensive. It is difficult to know just how expensive it will be for MMers at this point.
  4. For the first time ever a drug company is discussing the possibility of drug pricing based on performance…

My experience as a long-term MM survivor is that car-t cell development is relatively young. Most if not all chemotherapy development involves challenges like the ones outlined below. I plan to blog about the ongoing development of this potential chemo breakthrough in the future.

Have you been diagnosed with multiple myeloma? What stage? What symptoms are you experiencing?

Thank you,

David Emerson

  • Multiple Myeloma Survivor
  • MM Cancer Coach
  • Director PeopleBeatingCancer

Recommended Reading:

F.D.A. Approves Second Gene-Altering Treatment for Cancer

“The new therapy, Yescarta, made by Kite Pharma, was approved for adults with aggressive forms of a blood cancer, non-Hodgkin’s lymphoma, who have undergone two regimens of chemotherapy that failed…

About 3,500 people a year in the United States may be candidates for Yescarta. It is meant to be given once, infused into a vein, and must be manufactured individually for each patient. The cost will be $373,000

Side effects can be life-threatening, however. They include high fevers, crashing blood pressure, lung congestion and neurological problems. In some cases, patients have required treatment in an intensive care unit. In the study that led to the approval, two patients died from side effects. Doctors have learned to manage them better, but it takes training and experience…

Novartis is expected to ask the F.D.A. to approve Kymriah for lymphoma and other blood cancers as well, and may vary its price depending on how well it works for those diseases…Kite also plans to seek approval for other blood cancers, but does not plan to vary Yescarta’s price, said Ms. Cassiano…

The company (Kite Pharma) also hopes that Yescarta will eventually be approved for earlier stages of lymphoma, rather than being limited to patients with advanced disease who have been debilitated by multiple types of chemotherapy that did not work

The study that led to approval enrolled 111 patients at 22 hospitals; 101 of them received Yescarta. They had one of three diseases: diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma or transformed follicular lymphoma.

Initially, 54 percent had complete remissions, meaning that their tumors disappeared. Another 28 percent had partial remissions, in which tumors shrank or appeared less active on scans. After six months, 80 percent of the 101 were still alive.

With a median follow-up of 8.7 months, 39 percent of the 101 were still in complete remission — a much higher rate than achieved with earlier treatments — and 5 percent still had partial remissions…

Tina Bureau, a fifth-grade teacher from Queensbury, N.Y., was one of the lymphoma patients in the study. Previously, she’d had several types of chemotherapy…

Last spring, she had the T-cell treatment at the Dana-Farber Cancer Institute and Brigham and Women’s Hospital in Boston. The side effects were ferocious.

“You don’t even recognize your family members,” Ms. Bureau said. “I had some bleeding on my brain, and had to be put in intensive care. The week it was happening, I don’t remember a lot. It was much more difficult for my family than me.”

Within a month, she had a complete remission, which has continued. She is back at work, full time.

“Yes, it can pose life-threatening problems,” Ms. Bureau said. “But when you’re in a situation where your life’s threatened anyway, I don’t feel you have anything to lose.”

What’s the Total Cost of One CAR T-Cell Treatment?

“Tisagenlecleucel (Kymriah, Novartis) was priced at $475,000, and axicabtagene ciloleucel (Yescarta, Kite Pharma) was priced at $373,000.

However, these are prices for the drug products alone. On top of this comes the cost of administering and managing a patient undergoing CAR T-cell treatment, which include leukapherisis, lymphodepletion therapy, and management of the adverse effects of CAR T.

Now, for the first time, there is an estimate of the total mean cost of the treatments in the United States: $510,963 for tisagenlecleucel, and $402,647 for axicabtagene ciloleucel…

The nondrug costs are $400,000 to $450,000, which is just about equal to the drug costs, he said. Thus, these additional costs double the total cost of the therapy.

“They are underestimating the non-T-cell costs by at least fivefold,” Silver told Medscape Medical News

Failed Alzheimer’s Drug Might Boost CAR T-Cell Therapy for Multiple Myeloma

“The relatively new type of cancer treatment know as CAR T-cell therapy is already being used to treat some people with leukemia and lymphoma. This form of immunotherapy has also shown promise as a treatment for multiple myeloma, although it’s not yet approved by the Food and Drug Administration (FDA) for that use…”



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