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Gene therapy to treat cancer

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The New York Times is featuring an article this week about an experimental treatment for leukemia that could show a world of promise for current or future sufferers of leukemia and hopefully other types of cancer. In the method, which was used recently on William Ludwig, 65, of Bridgeton, N.J., doctors extracted a billion of Ludwig’s T-cells, altered them to identify and attack his cancer cells and then replanted the “tweaked” cells back into his body.

After the procedure, Ludwig experienced some fever and other symptoms but after a few weeks, the fever was gone and his cancer was in remission. The treatment was said to have killed two pounds of cancer cells, and a year hence, Ludwig is still cancer free.

This part of the article briefly explains the nuts and bolts of the procedure:

To make T-cells search out and destroy cancer, researchers must equip them to do several tasks: recognize the cancer, attack it, multiply, and live on inside the patient. A number of research groups have been trying to do this, but the T-cells they engineered could not accomplish all the tasks. As a result, the cells’ ability to fight tumors has generally been temporary.

The University of Pennsylvania team seems to have hit all the targets at once. Inside the patients, the T-cells modified by the researchers multiplied to 1,000 to 10,000 times the number infused, wiped out the cancer and then gradually diminished, leaving a population of “memory” cells that can quickly proliferate again if needed.

The researchers said they were not sure which parts of their strategy made it work — special cell-culturing techniques, the use of H.I.V.-1 to carry new genes into the T-cells, or the particular pieces of DNA that they selected to reprogram the T-cells.

This is of particular interest to me because I myself have been the recipient of a successful bone marrow transplant using a then-experimental method for treating immune deficiency. In my case, from birth I lacked the necessary B-cells of healthy people. Thus, the slightest contact with germs could make me very sick. I essentially lacked a functioning immune system. As a 4-year-old at Memorial Sloan Kettering Cancer Center in New York (the one mentioned in The Times article), I received a type of unmatched bone marrow transplant in which the attacker T-cells were “spun off” from the donor’s blood cells and bound to a compound called lectin. The necessary B-cells were then transplanted into my system, and I eventually began staving off illness on my own. I was the first patient to have undergone a successful unmatched bone marrow transplant without being confined to a sterile room or suit (A la, the droll and ill-conceived movie, “Bubble Boy.” I say “droll” because there isn’t much amusing at all about a person that is confined to a sterile environment for any period of time.)

I only mention my case briefly because it’s hard to overstate how important research into cell modification, gene therapy and stem cell research could be in treating and curing some of the most destruction diseases of our time, from Lou Gehrig’s, to cancer, immune deficiencies, Parkinson’s and others, yet, most of the evangelical people in this nation are worried about protecting the interests of clusters of undifferentiated cells.

I leave this post with a lengthy but cogent and piercing passage from Sam Harris (from “The End of Faith“) on the efficacy of embryonic stem cell research and why this research, and other similar avenues of study, should be immediate and rigorous:

Consider the present debate over research on human embryonic stem cells.  The problem with this research, from the religious point of view is simple:  it entails the destruction of human embryos. The embryos in question will have been cultures in vitro (not removed from a woman’s body) and permitted to grow for three to five days. At this stage of development, an embryo is called a blastocyst and consists of about 150 cells arranged in a microscopic sphere. Interior to the blastocyst is a small group of about 30 embryonic stem cells. These cells have two properties that make them of such abiding interest to scientists: as stem cells, they can remain in an unspecialized state, reproducing themselves through cell division for long periods of time (a population of such cells living in culture is known as a cell line); stem cells are also pluripotent, which means they have the potential to become any specialized cell in the human body – neurons of the brain and spinal chord, insulin-producing cells of the pancreas, muscle cells of the heart, and so forth.

Here is what we know. We know that much can be learned from research on embryonic stem cells. In particular, such research may give us further insight into the processes of cell division and cell differentiation. This would almost certainly shed new light on those medical conditions, like cancer and birth defects, that seem to be merely a matter of processes gone awry. We also know that research on embryonic cells requires the destruction of human embryos at the 150-cell stage. There is not the slightest reason to believe, however, that such embryos have the capacity to sense pain, to suffer, or to experience the loss of life in any way at all. What is indisputable is that there are millions of human beings who do have these capacities, and who currently suffer from traumatic injuries to the brain and spinal chord. Millions more suffer from Parkinson’s and Alzheimer’s diseases. Millions more suffer from stroke and heart disease, from burns, from diabetes, from rheumatoid arthritis, from Purkinje cell degeneration, from Duchenne muscular dystrophy, and from vision and hearing loss. We know that embryonic stem cells promise to be a renewable source of tissues and organs that might alleviate such suffering in the not too distant future.

Enter faith:  we now find ourselves living in a world in which college-educated politicians hurl impediments in the way of such research because they are concerned about the fate of  single cells. Their concern is not merely that a collection of 150 cells may suffer its destruction. Rather, they believe that even a human zygote (a fertilized egg) should be afforded all the protections of a fully developed human being. Such a cell, after all, has the potential to become a full developed human being. But given our recent advances in the biology of cloning, as much can be said of almost every cell in the human body. By the measure of a cell’s potential whenever the president scratches his nose he is now engaged in a diabolical culling of souls.

Out of deference to some rather poorly specified tenets of Christine doctrine (after all, nothing in the Bible suggests that killing human embryos, or even human fetuses, is the equivalent of killing a human being), the U.S. House of Representatives voted effectively to ban embryonic stem-cell research on February 27, 2003.

No rational approach to ethics would have led us to such an impasse. Our present policy on human stem cells has been shaped by beliefs that are divorced from every reasonable intuition we might form about the possible experience of living systems. In neurological terms, we surely visit more suffering upon this earth by killing a fly than by killing a human blastocyst, to say nothing of a human zygote (flies, after all, have 100,000 cells in their brains alone).  Of course, the point at which we fully acquire our humanity, and or capacity to suffer, remains an open question. But anyone who would dogmatically insist that these traits must arise coincident with the moment of conception has nothing to contribute, apart from his ignorance, to this debate. Those opposed to therapeutic stem-cell research on religious grounds constitute the biological and ethical equivalent of a flat-earth society. Our discourse on the subject should reflect this. In this area of public policy alone, the accommodations that we have made to faith will do nothing but enshrine a perfect immensity of human suffering for decades to come.

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