Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases

["Dave Farber" <farber () gmail com>]

> Begin forwarded message:
>
> From: Dewayne Hendricks <dewayne () warpspeed com>
> Subject: [Dewayne-Net] Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases
> Date: July 12, 2018 at 6:52:26 PM GMT+9
> To: Multiple recipients of Dewayne-Net <dewayne-net () warpspeed com>
> Reply-To: dewayne-net () warpspeed com
>
> Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases
> Scientists report that they have discovered a way to tweak genes in the body’s immune cells by using electrical 
> fields.
> By Gina Kolata
> Jul 11 2018
> <https://www.nytimes.com/2018/07/11/health/gene-editing-cancer.html>
>
> For the first time, scientists have found a way to efficiently and precisely remove genes from white blood cells of 
> the immune system and to insert beneficial replacements, all in far less time than it normally takes to edit genes.
>
> If the technique can be replicated in other labs, experts said, it may open up profound new possibilities for 
> treating an array of diseases, including cancer, infections like H.I.V. and autoimmune conditions like lupus and 
> rheumatoid arthritis. 
>
> The new work, published on Wednesday in the journal Nature, “is a major advance,” said Dr. John Wherry, director of 
> the Institute of Immunology at the University of Pennsylvania, who was not involved in the study. 
>
> But because the technique is so new, no patients have yet been treated with white blood cells engineered with it. 
>
> “The proof will be when this technology is used to develop a new therapeutic product,” cautioned Dr. Marcela Maus, 
> director of cellular immunotherapy at Massachusetts General Hospital. 
>
> That test may not be far away. The researchers have already used the method in the laboratory to alter the abnormal 
> immune cells of children with a rare genetic condition. They plan to return the altered cells to the children in an 
> effort to cure them. 
>
> Currently, scientists attempting to edit the genome often must rely on modified viruses to slice open DNA in a cell 
> and to deliver new genes into the cell. The method is time-consuming and difficult, limiting its use.
>
> Despite the drawbacks, the virus method has had some success. Patients with a few rare blood cancers can be treated 
> with engineered white blood cells — the immune system’s T-cells — that go directly to the tumors and kill them. 
>
> This type of treatment with engineered white cells, called immunotherapy, has been limited because of the difficulty 
> of making viruses to carry the genetic material and the time needed to create them.
>
> But researchers now say they have a found a way to use electrical fields, not viruses, to deliver both gene-editing 
> tools and new genetic material into the cell. By speeding the process, in theory a treatment could be available to 
> patients with almost any type of cancer.
>
> “What takes months or even a year may now take a couple weeks using this new technology,” said Fred Ramsdell, vice 
> president of research at the Parker Institute for Cancer Immunotherapy in San Francisco. “If you are a cancer 
> patient, weeks versus months could make a huge difference.” 
>
> “I think it’s going to be a huge breakthrough,” he added.
>
> The Parker Institute already is working with the authors of the new paper, led by Dr. Alexander Marson, scientific 
> director of biomedicine at the Innovative Genomics Institute — a partnership between University of California, San 
> Francisco and the University of California, Berkeley — to make engineered cells to treat a variety of cancers. 
>
> In the new study, Dr. Marson and his colleagues engineered T-cells to recognize human melanoma cells. In mice 
> carrying the human cancer cells, the modified T-cells went right to the cancer, attacking it.
>
> The researchers also corrected — in the lab — the T-cells of three children with a rare mutation that caused 
> autoimmune diseases. The plan now is to return these corrected cells to the children, where they should function 
> normally and suppress the defective immune cells, curing the children.
>
> The technique may also hold great promise for treating H.I.V., Dr. Wherry said. 
>
> The H.I.V. virus infects T-cells. If  they can be engineered so that the virus cannot enter the T-cells, a person 
> infected with H.I.V. should not progress to AIDS. Those T-cells already infected would die, and the engineered cells 
> would replace them.
>
> Previous research has shown it might be possible to treat H.I.V. in this way. “But now there is a really efficient 
> strategy to do this,” Dr. Wherry said.
>
> [snip]
>
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