HIV vs. Cancer: Altered Immune Cells Beat Leukemia

June 20, 2013 10:00 AM

"Emma Whitehead was near death from acute lymphoblastic leukemia but is now in remission after an experimental treatment at the Children’s Hospital of Philadelphia."

The New York Times has a feature from December 2012 and this incredible story was the subject of a short film as part of a GE/cinelan-sponored Vimeo series of 3 minute documentaries on "big ideas"
posted by 3rdparty (10 comments total)

Oh, those interleukins. I've got several friends on immunosuppressants for Rheumatoid Arthritis Disease; I wonder how this breakthrough reflects in looking at some of their treatment options.

Interesting about the involvement of Novartis; I suppose they're taking the long view, looking at the broader impacts of knowledge/technology transfer fostered by this push.
posted by tilde at 10:21 AM on June 20, 2013

This is fascinating stuff. Every day, your body destroys millions of cancer cells with an evolutionary fine-tuned system to distinguish self from not-self. Escape from this "surveillance" is what allows a cancer to develop.

Immunotherapy for cancer seems so theoretically sound, but the practical implication of it has struggled. This area, and melanoma, are two areas that seem to be showing some benefit.

This is the original abstract.

The headline is a bit mis-leading in that the new T-Cells aren't made to target cancer cells specifically (which would be the holy grail), but rather target ALL of a patients B-cells, the progenitor cell of the leukemia. Current, antibody based therapies (Rituximab) have the same target, but I imagine with less efficacy

The B-cells are a key part of your immune system and I imagine there will be pretty serious infectious complications down the road for these patients.
posted by cacofonie at 10:49 AM on June 20, 2013 [3 favorites]

Dr. Michel Sadelain... said: “These T-cells are living drugs. With a pill, you take it, it’s eliminated from your body and you have to take it again.” But T-cells, he said, “could potentially be given only once, maybe only once or twice or three times.”

This is truly phenomenal, and it troubles me greatly that it's being framed (even in part) by the metric of market viability. Here's why:

"The economic model is totally acceptable," [the president of Novartis Oncology] said.

Yeah, I'm not sure what would be "economically acceptable" about a drug that only applies to certain cancer patients, is incredibly expensive to produce, and that the users only have to pay for once.

The drug companies would be forced to make the prices incredibly high just to maintain the production infrastructure, then they'd have to increase that in order to make a profit, and they'd have to make it profitable enough that it wouldn't get culled from the company budget during the seemingly-biweekly restructurings.

Even if they can reduce the costs somewhat as the research advances, I just don't see this as succeeding on its capitalist merits. The only ways I see that happening are:

1. As a "flagship" product that earns so much goodwill for Novartis as the cure for fucking cancer that it drives sales of their other drugs,
2. If the research (retroviral immune reinforcement) can become the basis to cheaply develop lots of cures, with Novartis somehow maintaining a monopoly or being willing to take a fair slice of a gigantic pie,
3. By Novartis funding this research under the good-faith assumption that they'll get to empty patients' bank accounts, until the populace pulls the rug out from them by demanding legislation that undermines Novartis's market protections, and/or
4. Because influential people at Novartis are willing to risk their careers to make this happen regardless of profitability, and the other influential people at Novartis are too oblivious to stop them.

But I have to say, I really don't want to get into a situation where the only the rich are able to cure their cancer, and then standards for carcinogens in consumer products suddenly collapse for some unknown reason.
posted by Riki tiki at 11:04 AM on June 20, 2013 [1 favorite]

Riki: The article mentions that it's main comparison is against the bone marrow transplant, which is the current "hail-mary pass" of hematolgic malignancies and is a really expensive, impractical (finding donors and the like), and painful (graft versus host disease, etc) modality that, economically, wouldn't take much to surpass..
posted by cacofonie at 11:16 AM on June 20, 2013 [1 favorite]

"The treatment very nearly killed her. But she emerged from it cancer-free, and about seven months later is still in complete remission. She is the first child and one of the first humans ever in whom new techniques have achieved a long-sought goal — giving a patient’s own immune system the lasting ability to fight cancer.

Three adults with chronic leukemia treated at the University of Pennsylvania have also had complete remissions, with no signs of disease; two of them have been well for more than two years, said Dr. David Porter. Four adults improved but did not have full remissions, and one was treated too recently to evaluate. A child improved and then relapsed. In two adults, the treatment did not work at all. "

Totally fucking breathtaking. Wow. One day, cancer, one day soon.
posted by marienbad at 11:21 AM on June 20, 2013 [1 favorite]

From the article: Dr. June said that producing engineered T-cells costs about $20,000 per patient — far less than the cost of a bone-marrow transplant.

Though this sounds like a lot of money, we spend about the same amount (or more) per year on ongoing treatments for many, many chronic diseases. Infliximab, the infusion drug which is given to treat rheumatoid arthritis, ulcerative colitis, Crohn's disease, ankylosing spondylitis, etc costs about that much (depending on dosage) and is given for the rest of your life, not just once or twice to induce remission. I don't have personal experience with it but I imagine many chemotherapies are in the same price range already. This is not the million dollar only for rich people cure for cancer, by a longshot.
posted by telegraph at 11:24 AM on June 20, 2013 [1 favorite]

This is truly phenomenal, and it troubles me greatly that it's being framed (even in part) by the metric of market viability. Here's why:

Using the National Institute for Clinical Excellence (NICE) guidelines which determine which treatments the NHS pays, for we can guess the maximum that the NHS would be willing to spend on it.

NICE generally fund drugs that cost less than between £20k - £30k per Quality Adjusted Life Year (QALY), given the emotional resonance and political sensitivity of childhood cancer we will pick the top end of that range.

Assume that because of the immune complications we 'Quality Adjust' the remaining years of life to 0.9.

Assume that it effects a total cure for 50% of patients and has no effect on the rest. I think that this understates the actual data but I don't have time to look at the actual distributions and this is just a quick and dirty estimate.

An effective cure for Leukemia, administered to a five year-old like Emma Whitehead would add about 80 years to her life.

That means that the NHS would fund this if it cost less than £30k * 0.9 * 80 * 50% which equals about a million pounds. Of course there are costs associated with administering and monitoring the drug, follow-up costs, etc. Still, that's not bad, is it?

My guess is that Novartis is hoping that this opens up a whole family of treatments.
posted by atrazine at 11:24 AM on June 20, 2013 [3 favorites]

I guess I didn't read that as clearly as I should have, cacofonie, and I'm laughably far from being an expert on these matters. When it succeeds, does marrow transplantation tend to be a "one-off" solution where the patient ideally needs minimal continued treatment? Who tends to bear the "extra" costs like identifying potential donors?
posted by Riki tiki at 11:26 AM on June 20, 2013

telegraph: "Though this sounds like a lot of money, we spend about the same amount (or more) per year on ongoing treatments for many, many chronic diseases. Infliximab, the infusion drug which is given to treat rheumatoid arthritis, ulcerative colitis, Crohn's disease, ankylosing spondylitis, etc costs about that much (depending on dosage) and is given for the rest of your life, not just once or twice to induce remission."

That's kinda what I'm getting at, telegraph. An expensive treatment is far more profitable than an expensive cure, which is itself far, far more profitable than a cheap prevention. I'm slightly encouraged by the responses to my comment, but remain extremely concerned about the incentive models of market-based health care.
posted by Riki tiki at 11:34 AM on June 20, 2013

An expensive treatment is far more profitable than an expensive cure, which is itself far, far more profitable than a cheap prevention. I'm slightly encouraged by the responses to my comment, but remain extremely concerned about the incentive models of market-based health care.

The reality of drug development is that it's driven far more by just finding something, anything that works, is safe, and is at least as effective as other products on the market than it is driven by trying to find something that wrings the most profit possible out of patients. It's not like drug companies have a surfeit of new treatment options coming out of the pipeline that they are picking and choosing among. This, for example, is not a conversation that has ever happened:

Scientist: "The Phase 3 trial results are in. Our new treatment showed excellent results, as did our cure for the same disease."
Manager: "I was afraid that might happen. Put the treatment on the market and bury the research on the cure. We want to maximize profits, after all! If the treatment hadn't worked out we might have gone with the cure, but since we can make more money this way, we'll only sell the treatment."

Instead it's more like this:

Scientist: "The results for the dozens of in vitro, animal model, Phase 1, Phase 2, and Phase 3 trials that we have running simultaneously for multiple approaches to preventing, treating, and curing various diseases have come in. Most of them failed, but a few have shown good results or gotten approval to move forward."
Manager: "Great. Move them all forward, even the slightly questionable ones. That's how we make money, after all."
posted by jedicus at 11:52 AM on June 20, 2013 [4 favorites]