A cure? OMG OMG OMG
As a lot of you know, I was diagnosed over four years ago (on the day after I turned 50) with Chronic Leukocytic Leukemia ( CLL); you can live for decades with the stuff, but it’s bad news. I have had recurrent hospitalizations since then - horrible trouble with opportunistic infections (greatly reduced immune system) and it just drains me of energy in general. And there’s always that sword of Damocles hanging over my head that it could suddenly go into overdrive…and switch to a more fast acting version. Or whatever.
Most of the time, I don’t actively think about it. I can’t. I don’t dare do my usual research on the disease, because the variable nature of the thing from person to person, each with their different stories, is such that I’d go into deep depression worrying about any and all of it. What I do just do the maintenance stuff to ward off problems as a standard (like brushing teeth or washing hands) that I don’t think about.
It also means that I can’t read or watch stuff about leukemia in general without it depressing me; I just avoid it. And I’d give plenty for a cure.
And now, one may be right around the corner: there’s a story on tonight’s NBC Nightly News on it, and a similar one on Today.. see also the articles from the researchers at Penn in the New England Journal of Medicine and Science: Translational Medicine.
The Penn scientists targeted chronic lymphocytic leukemia (CLL), the most common type of the blood disease. It strikes some 15,000 people in the United States, mostly adults, and kills 4,300 every year. Chemotherapy and radiation can hold this form of leukemia at bay for years, but until now the only cure has been a bone marrow transplant. A bone marrow transplant requires a suitable match, works only about half the time, and often brings on severe, life-threatening side effects such as pain and infection.
In the Penn experiment, the researchers removed certain types of white blood cells that the body uses to fight disease from the patients. Using a modified, harmless version of HIV, the virus that causes AIDS, they inserted a series of genes into the white blood cells. These were designed to make to cells target and kill the cancer cells. After growing a large batch of the genetically engineered white blood cells, the doctors injected them back into the patients.
In similar past experimental treatments for several types of cancer the re-injected white cells killed a few cancer cells and then died out. But the Penn researchers inserted a gene that made the white blood cells multiply by a thousand fold inside the body. The result, as researcher June put it, is that the white blood cells became “serial killers” relentlessly tracking down and killing the cancer cells in the blood, bone marrow and lymph tissue.
As the white cells killed the cancer cells, the patients experienced the fevers and aches and pains that one would expect when the body is fighting off an infection, but beyond that the side effects have been minimal.
Apparently the holdup on wider testing is - funding, with grant money from the US Government so heavily whacked on. For all those who want to cut the heck out of medical research funds, this sort of thing is the result…people die. People suffer. But a wealthy couple set up a charity to do this sort of groundbreaking research, and it paid off…God bless them.
From the UPenn press release:
The work was supported by the Alliance for Cancer Gene Therapy, a foundation started by Penn graduates Barbara and Edward Netter, to promote gene therapy research to treat cancer, and the Leukemia & Lymphoma Society.
Would I go through that sort of ‘fevers, aches and pains’ for weeks to get a CURE for this monster? God, yes. In a heartbeat. No question. Better being sick as a dog for a few weeks while the T-cells do their work than to have a bomb in your body that drains you down all the time. Over 4000 lives a year could be saved by this research directly with CLL, and many more with any allied treatments for other cancers that come from this.
A quote from a Webmd article on side effects and results:
The treatment was not a walk in the park for patients. One of the three patients became so ill from the treatment that steroids were needed to relieve his symptoms. The steroid rescue may be why this patient had only a partial remission.
“Those engineered T cells don’t hug the cells to death. They release an array of substances, nasty things that have evolved to clear virus- infected cells from your body,” Galipeau says. “But now they are using this to melt down a couple of pounds worth of tumor burden, you will get some side effects.”
One of the patients, whose case is reported in the New England Journal of Medicine, described his experience in a University of Pennsylvania news release… He was diagnosed with CLL at age 50; 13 years later his treatment was failing. Facing a bone-marrow transplant, he jumped at the chance to enter Porter’s clinical trial of CAR T cells.
“It took less than two minutes to infuse the cells and I felt fine afterward. However, that fine feeling changed dramatically less than two weeks later when I woke up one morning with chills and a fever,” he says. “I was sure the war was on. I was sure the CLL cells were dying.”
A week later the patient was still in the hospital when Porter brought him the news that the CLL cells had disappeared from his blood.
Is he cured? Doctors hate to declare a cure until patients have been cancer-free for at least five years. But there are signs the CAR-T cells persist in patients’ immune memory, ready to mop up any CLL cells that reappear.
And there’s a big downside. The CAR T cells that fight CLL also kill off normal B lymphocytes. These are the cells that the body needs to make infection-fighting antibodies.
As long as the CAR T cells persist - which may be for the rest of patients’ lives - patients will require regular infusions of immune globulin.
You know what? I already have monthly immunoglobulin infusions to fight infections, so what else is new? And there’s always a good shot that the treatment will get refined so as to eliminate or greatly reduce this problem.
More stories on this:
- at usnews.com
- at webmd.com