7 ms·

Gene editing saves girl dying from leukaemia in world first

192 points by cedricr 11y ago

dang 11y ago
Also https://news.ycombinator.com/item?id=10518432 https://news.ycombinator.com/item?id=10518432. (Edit: not a duplicate, but an article with additional info.)
medymed 11y ago
Would be fascinating if they could harvest healthy host progenitor cells, destroy the leukemic cells with another persons's engineered off-the-shelf graft which has a couple of chemically (e.g. tetracycline) inducible kill-switches, and wait until the graft destroys the cancer to turn on the kill switch and replant the patient's stored cells. This could prevent lifelong graft versus host disease, but the risk of death/complications from an additional retransplant or the reemergence of dormant cancer cells may be too great to make it worth the risk.
- 2skep 11y ago
  There are other platforms that do that. Take a look at Kiadis which can identify activated T cells and kill them and then reinfuse rest of white cells back into the patient.
deleted 11y ago
[deleted]
drjackal 11y ago
On Monday I got the worst phone call of my life. My sister, who is in her 40s has been diagnosed with Leukemia. The variant she has is Acute Myeloid Leukemia (AML) which has a 5 year survival rate of only about 30%. She seemed healthy just a few days before diagnosis, she was just a bit tired and had low appetite. Then she noticed some abnormality in her vision and that's when she did a blood test and found extremely low hemoglobin count. Shows how quickly your world can come crashing. Cancer is awful. Cancer and global warming are where most of the worlds research dollars should be going. This story brings hope for the future.
- cema 11y ago
  Why global warming?
  - coob 11y ago
    Really? You're going to be that person?
    - cema 11y ago
      Oh, so it's political. Never mind then. (If it is not political, I still would like to see the answer.)
  - skylan_q 11y ago
    Because it's 2015
  - mccracken 11y ago
    same reason gun control is a good idea?
  - anonymfus 11y ago
    Because many people will die in horrible ways from its effects.
- mngrey 11y ago
  I wish you both all the very best in this awful situation. I have been in the same position as you and in the same position as her. Your love and support will be so important to her.
- rrdharan 11y ago
  With respect, and empathy given that I have lost several close family relatives to cancer, I disagree with you. Many cancers are in fact overfunded relative to their societal impact. Moreover too much money is spent on chasing cures rather than prevention: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3411479/ http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3411479/ http://www.slate.com/blogs/quora/2013/02/07/where_do_the_millions_of_cancer_research_dollars_go_every_year.html http://www.slate.com/blogs/quora/2013/02/07/where_do_the_mil...
NotSammyHagar 11y ago
This is an incredible medical advance, the old idea from at least the 70s actually working. It will take a long time until we can apply this to most diseases or conditions. There are so many different causes and interactions behind medical problems, even those that can be attacked by genetics + specially designed org like this. I'm sad my mom's cancer couldn't be treated by something like this. Live long enough, and we'll keep seeing amazing things!
- nonbel 11y ago
  They call this (also CRISPR) gene editing, but interestingly when I looked at the literature whats going on is always this: 1) Cells are put in a dish 2) Gene "editing" tech is added to the dish 3) Most, if not the vast majority of the cells die. Or this information is simply not reported, even if the authors did report it in other papers so they know it is important. 4) The surviving cells consist largely of those with "edited" genes. Control cells, who were not exposed to the "editing" conditions, also have a small percentage of "edited" genes. It sounds to me like they are selectively killing the cells that are not already mutants. I read around 8 papers on CRISPR, none of them addressed this possibility (or mention it and cite someone who did address it). Of course, in terms of getting the desired mutants for the clinic it works just as well. Misunderstanding why it works is not good though.
fasteo 11y ago
In this particular case, they have used TALEN, but we have two additional gene editing techniques that show promise. Namely, - ZFN [2] - CRISPR [3] I have great hopes in all of them and in fact, my genetic condition [4] has already been fixed in-vitro using both mitoTALENs [5] and ZFN [6]. Exciting times for medicine. Too bad that we have a long, desperate bureaucratic burden ahead. [1] https://en.wikipedia.org/wiki/Transcription_activator-like_effector_nuclease https://en.wikipedia.org/wiki/Transcription_activator-like_e... [2] https://en.wikipedia.org/wiki/Zinc_finger_nuclease https://en.wikipedia.org/wiki/Zinc_finger_nuclease [3] https://en.wikipedia.org/wiki/CRISPR https://en.wikipedia.org/wiki/CRISPR [4] http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mitochondrial_myopathy.htm http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mi... [5] http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html [6] http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/ http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/
- yarou 11y ago
  It truly is an exciting time to live in. CRISPR in particular looks promising for editing human embryos, and we may be on the cusp of a post-cancer world with CAR T-Cell Therapy. One of my coworkers, who has the same cancer as my deceased father, is going for an experimental treatment involving CAR T-Cell therapy. Unfortunately, there will always be a bureaucratic burden, but I doubt it will restrict the progress of these types of technologies and innovation.
dnautics 11y ago
I'm not sure how this is a 'world first'. Gene therapy has from time to time been used to treat childhood leukemias for a long time, e.g.: http://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/ http://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/

192 points by cedricr 11y ago

dang 11y ago

Also https://news.ycombinator.com/item?id=10518432 https://news.ycombinator.com/item?id=10518432. (Edit: not a duplicate, but an article with additional info.)

medymed 11y ago

Would be fascinating if they could harvest healthy host progenitor cells, destroy the leukemic cells with another persons's engineered off-the-shelf graft which has a couple of chemically (e.g. tetracycline) inducible kill-switches, and wait until the graft destroys the cancer to turn on the kill switch and replant the patient's stored cells. This could prevent lifelong graft versus host disease, but the risk of death/complications from an additional retransplant or the reemergence of dormant cancer cells may be too great to make it worth the risk.

2skep 11y ago
There are other platforms that do that. Take a look at Kiadis which can identify activated T cells and kill them and then reinfuse rest of white cells back into the patient.

deleted 11y ago

[deleted]

drjackal 11y ago

On Monday I got the worst phone call of my life. My sister, who is in her 40s has been diagnosed with Leukemia. The variant she has is Acute Myeloid Leukemia (AML) which has a 5 year survival rate of only about 30%. She seemed healthy just a few days before diagnosis, she was just a bit tired and had low appetite. Then she noticed some abnormality in her vision and that's when she did a blood test and found extremely low hemoglobin count. Shows how quickly your world can come crashing. Cancer is awful. Cancer and global warming are where most of the worlds research dollars should be going. This story brings hope for the future.

cema 11y ago
Why global warming?
- coob 11y ago
  Really? You're going to be that person?
  - cema 11y ago
    Oh, so it's political. Never mind then. (If it is not political, I still would like to see the answer.)
- skylan_q 11y ago
  Because it's 2015
- mccracken 11y ago
  same reason gun control is a good idea?
- anonymfus 11y ago
  Because many people will die in horrible ways from its effects.
mngrey 11y ago
I wish you both all the very best in this awful situation. I have been in the same position as you and in the same position as her. Your love and support will be so important to her.
rrdharan 11y ago
With respect, and empathy given that I have lost several close family relatives to cancer, I disagree with you. Many cancers are in fact overfunded relative to their societal impact. Moreover too much money is spent on chasing cures rather than prevention: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3411479/ http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3411479/ http://www.slate.com/blogs/quora/2013/02/07/where_do_the_millions_of_cancer_research_dollars_go_every_year.html http://www.slate.com/blogs/quora/2013/02/07/where_do_the_mil...

NotSammyHagar 11y ago

This is an incredible medical advance, the old idea from at least the 70s actually working. It will take a long time until we can apply this to most diseases or conditions. There are so many different causes and interactions behind medical problems, even those that can be attacked by genetics + specially designed org like this. I'm sad my mom's cancer couldn't be treated by something like this. Live long enough, and we'll keep seeing amazing things!

nonbel 11y ago
They call this (also CRISPR) gene editing, but interestingly when I looked at the literature whats going on is always this: 1) Cells are put in a dish 2) Gene "editing" tech is added to the dish 3) Most, if not the vast majority of the cells die. Or this information is simply not reported, even if the authors did report it in other papers so they know it is important. 4) The surviving cells consist largely of those with "edited" genes. Control cells, who were not exposed to the "editing" conditions, also have a small percentage of "edited" genes. It sounds to me like they are selectively killing the cells that are not already mutants. I read around 8 papers on CRISPR, none of them addressed this possibility (or mention it and cite someone who did address it). Of course, in terms of getting the desired mutants for the clinic it works just as well. Misunderstanding why it works is not good though.

fasteo 11y ago

In this particular case, they have used TALEN, but we have two additional gene editing techniques that show promise. Namely, - ZFN [2] - CRISPR [3] I have great hopes in all of them and in fact, my genetic condition [4] has already been fixed in-vitro using both mitoTALENs [5] and ZFN [6]. Exciting times for medicine. Too bad that we have a long, desperate bureaucratic burden ahead. [1] https://en.wikipedia.org/wiki/Transcription_activator-like_effector_nuclease https://en.wikipedia.org/wiki/Transcription_activator-like_e... [2] https://en.wikipedia.org/wiki/Zinc_finger_nuclease https://en.wikipedia.org/wiki/Zinc_finger_nuclease [3] https://en.wikipedia.org/wiki/CRISPR https://en.wikipedia.org/wiki/CRISPR [4] http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mitochondrial_myopathy.htm http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mi... [5] http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html [6] http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/ http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/

yarou 11y ago
It truly is an exciting time to live in. CRISPR in particular looks promising for editing human embryos, and we may be on the cusp of a post-cancer world with CAR T-Cell Therapy. One of my coworkers, who has the same cancer as my deceased father, is going for an experimental treatment involving CAR T-Cell therapy. Unfortunately, there will always be a bureaucratic burden, but I doubt it will restrict the progress of these types of technologies and innovation.

dnautics 11y ago

I'm not sure how this is a 'world first'. Gene therapy has from time to time been used to treat childhood leukemias for a long time, e.g.: http://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/ http://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/