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Leukemia survivor Emma Whitehead  (Source: allvoices.com)
Drug company Novartis has provided $20 million for a research center

Pennsylvania researchers have found a way to use immune cells in the battle against cancer, and ended up saving a young leukemia patient's life.

A team from the University of Pennsylvania, which was led by Dr. Carl June, created a new cancer treatment that involves the altering of a patient's T-cells. Once modified, the T-cells are placed back into the patient to fight cancer cells.

The treatment works like this: millions of a patient's T-cells, which is a type of white blood cell, are removed and equipped with new genes and a disabled form of the HIV virus. The HIV virus was used because it does a great job of carrying genetic material into T-cells.

The modified T-cells, also called chimeric antigen receptor cells, are reprogrammed to attack a protein called CD-19, which can be found on the surface of most healthy or malignant B-cells. B-cells are a part of the immune system that become malignant in leukemia.

Once a patient receives this treatment, they become very ill. That's how you know the T-cells are working, because the chemicals that are released from the T-cells trigger cytokine-release syndrome (aka, severe fevers and chills).

These symtpoms nearly killed 7-year-old Emma Whitehead, who was diagnosed with leukemia when she was only five. After several attempts at chemotherapy, which resulted in relapses, Whitehead was admitted to the Children's Hospital of Philadelphia, where she received June's experimental treatment. It had never been performed on a human before.

The cytokine-release syndrome sent Whitehead's temperature up to 105, and she had to be placed on a ventilator. Just as it seemed there was no hope, June discovered that Whitehead's levels of the cytokine interleukin-6 were ridiculously high, leading to the worsening side effects of the treatment. June used a drug called tocilizumab, which is typically for rheumatoid arthritis, to lower the interleukin-6 levels.

On May 2, after being unconscious for a week, Whitehead woke up on her seventh birthday. She has been cancer-free ever since.

While the method was particularly successful for Whitehead, it has had mixed results with other patients since then. So far, about a dozen patients have had the treatment. Three adults with chronic leukemia are cancer-free, another four adults have improved but do not have full remissions, a child improved and relapsed, and another two adults had no results at all.

June and the research team believe the mixed results may be due to either a flawed batch of T-cells or that not all leukemic cells have the CD-19 protein that the T-cells are designed to attack.

Nevertheless, drug company Novartis has shown interest in the method and offered to pay $20 million for a research center on the University of Pennsylvania's campus to bring the treatment to market. What's more shocking about the investment is that the new T-cell treatment isn't a "one-size fits all" kind of deal; rather, it costs about $20,000 per patient to produce engineered T-cells specific to each individual.

But the treament has changed the life of Whitehead, who is now back in school with her peers and capable of doing the things a healthy child should be able to do.

Source: The New York Times



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Cool
By Paj on 12/14/2012 8:15:15 AM , Rating: 2
Good on this girl for agreeing to try this experimental treatment - it cant have been easy.

Sounds like a promising development, despite the mixed results in other patients.




RE: Cool
By FITCamaro on 12/14/2012 8:33:35 AM , Rating: 2
My mom is having a bone marrow transplant in March. Not sure which would be rougher. That or this treatment. But I'm glad my mom is able to have the transplant vs just getting chemo every few years for the rest of her life.


RE: Cool
By Breathless on 12/14/2012 9:59:00 AM , Rating: 5
Take it from me...

My wife died last year from complications after a bone marrow transplant. Here is an extremely simple version of what happened. The first transplant failed (they think the CMV virus caused it to fail), they tried again (this time using an experimental treatment similar to the one in this article to combat the CMV virus), and the second attempt seemed to work. The problem is that she had already been in the hospital so long, and with the lack of movement, she developed Pneumonia. The Pneumonia caused her to need to be intubated. She got several infections, her kidneys shut down, and the rest is history. I say this not to scare you, but to give you the following advice....

1) KEEP HER MOVING. It was a fight and constant negotiation to keep her moving, but she didn't move enough, and didn't eat enough

2) WATCH THE DOCTORS AND NURSES LIKE A HAWK. Frequently, they do not follow protocol, and aren't careful enough about germs. I could tell you a few instances that would make you sick. DON'T BE AFRAID TO BE A DOUCHE BAG ABOUT IT AND GIVE THEM AN ATTITUDE IF NECESSARY so that they know to be extra careful with you and your mom. Be extremely nice to them until they break the rules, then give them an attitude if/when necessary. KEEP HER FROM GETTING INFECTIONS!!! You should probably not leave the room very often.

3) KEEP HER DRINKING AND EATING. MAKE HER EAT. FULLY prep her beforehand that she will eat even if and when she doesn't feel like it. Have her eat HIGH FIBER FOODS so that she keeps pooping. My wife had several abdominal obstructions as a result of the medication slowing down her digestive system, combined with the lack of eating and lack of moving. She blew up like a balloon and experienced extreme uncontrollable pain for a large part of her hospital stay because of this.

4) STAY WITH HER. Get her an iPad (or some kind of tablet) to keep her sane and stay with her in the room. Work it out with your job and make it happen. If you get fired from your job, FUDGE IT. Would you rather have a job or a mom? Constant moral support is crucial. Don't use the public bathroom. Use the bathroom in her room and clean it well after you are done. No sense in bringing in the germs from God knows how many other people use those bathrooms. Better that she use the bathroom after you have made sure it is clean then you bring in any outside germs unnecessarily. I argued this point with them and they said "you know... you're right!". This was common sense to me so I don't know why it wasn't the norm.

What you and her are about to go through is not easy, but with some mental preparation and both of you "sticking to your guns" even when its really tough, you can beat it. If the first transplant was successful, my wife would have made it. If she didn't get all the infections after the second transplant, she would have made it.


RE: Cool
By Wolfpup on 12/14/2012 10:49:22 AM , Rating: 2
Thanks for posting this. I'm not even up to reading all of it right now, it's so scary. I'm so sorry that you and she had to go through that, but it's awesome you're sharing it, could help others! I'm truly sorry you lost her :(


RE: Cool
By Breathless on 12/14/2012 11:21:22 AM , Rating: 5
No problem, and if you need more advice / moral support, feel free to contact me - breathlessrap@gmail.com

There is plenty more valuable advice I could give.


RE: Cool
By FITCamaro on 12/14/2012 11:46:48 AM , Rating: 2
She is a pharmacist and is aware of the potential risks and problems that can result and what certain medications do.

But it will be in Houston whereas I live in Charleston. So I won't be able to be there. I'm not sure yet if my dad will be staying with her during it or not. One of my aunt's (who is the donor) might be as well.


RE: Cool
By FITCamaro on 12/14/2012 11:47:15 AM , Rating: 2
But thanks for the info.

I'm sorry about your wife. I'm sure that wasn't fun to watch and experience.


RE: Cool
By Lord 666 on 12/14/2012 12:56:17 PM , Rating: 2
Good luck to your mom.

Huge thanks for the other guy for sharing the advice unfortunately learned from his wife. Life would not be kind to that medical team if there were egregious errors with my immediate family.


RE: Cool
By neihrick1 on 12/14/2012 3:11:59 PM , Rating: 2
I might be subjected to this if i relapse again. Where at in Houston, MDA? Thats where I went. On 2nd transplant now, 1 year out. Good luck to her.


RE: Cool
By Breathless on 12/14/2012 1:59:37 PM , Rating: 3
I would implore your dad to be there at all times with her.

There was more than one time that if I wasn't physically present in the room overnight she probably would have died a lot sooner from having difficulty breathing and not being able to push the call button. I hope everything works out for you and your family


RE: Cool
By sonoran on 12/15/2012 10:33:57 AM , Rating: 2
They're exploring similar therapies for lymphoma.

quote:
My mom is having a bone marrow transplant in March.
Hopefully she's having an autologous transplant? Not a picnic (I just went through one 6 months ago), but not nearly as tough as an allo transplant. Best wishes to you both.

As for that "$20,000 for creating the T cells", that's not that big an expense compared to existing therapies. One injection of Mozibil (just to mobilize stem cells for collection for transplant) was about $25,000.


RE: Cool
By fic2 on 12/14/2012 6:46:55 PM , Rating: 2
I hope this treatment works out. The 3 year old son of a close friend lost his battle with leukemia in February after battling for 9 months. He was ok'd for an experimental treatment at St. Jude in Tennessee and actually flew out there (great risk itself since just being among the germy population could have killed him at that point since he had virtually no white blood cells) but was sent back because he had a fungal infection.

Becoming aware of the world of childhood leukemia is an eye opener. Not enough research is being done since 95% of it is focused on adult cancers - specifically breast cancer.


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