Gene editing saves girl dying from leukaemia in world first | Hacker News Reader

Gene editing saves girl dying from leukaemia in world first

newscientist.com

192 points_freu27 comments

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_e...

[2] https://en.wikipedia.org/wiki/Zinc_finger_nuclease

[3] https://en.wikipedia.org/wiki/CRISPR

[4] http://www.ninds.nih.gov/disorders/mitochondrial_myopathy/mi...

[5] http://www.nature.com/nm/journal/v19/n9/full/nm.3261.html

[6] http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3992073/

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.

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.

Why global warming?

Really? You're going to be that person?

Oh, so it's political. Never mind then.

(If it is not political, I still would like to see the answer.)

Because it's 2015

same reason gun control is a good idea?

Because many people will die in horrible ways from its effects.

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.

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.slate.com/blogs/quora/2013/02/07/where_do_the_mil...

An ounce of prevention is worth a pound of cure. And yet we still sell cigarettes. And though WHO proved processed meats cause cancer the deli counter line is still very long. There are things that you can teach people to prevent doing, don't stick a knife in the electric socket, and then there are the things that take a monumental effort to change.

Prevention education is important, but I wonder if the meat counter will be restricted to 18 and over to purchase and come with a warning from the surgeon general.

I'm being unusually sarcastic, but I've had this conversation several times in my own head and I can't figure out a solution.

Leaded gasoline was poising the nation, but no one stopped driving. Only when the government forced the corporations to change did the lead poising stop.

You can't make someone change, you can't force an individual to change but if you never allow it to enter their world then it has been prevented right?

The obvious solution is to change incentives. The solution that will actually work, but will bring endless accusations of cruelty and heartlessness and Godwin's law, is simple:

Bring back shaming. Encourage fat shaming. Encourage alcohol shaming. We still encourage smoke-shaming, and it has resulted in a reduction in smoking. We need to stop being so afraid and sensitive to shame. It is a natural human tendency to police behaviors with negative externalities via shame.

Pass all the laws you want, but social shame is far more effective.

This reminds me of a Black Mirror episode where at some point in the future, fat shaming was brought to such an extreme, that everybody's job was just to pedal stationary bikes. And if you slacked, you got fat, and if you got fat, you got fired, and then you had to wear a bright yellow uniform and appear on fat shaming reality tv shows and clean up everybody's trash.

Shaming would only make things worse. From my experience alcoholics and over-eaters do not respond well to negative social stimuli. i.e. they will eat and drink more.

And yet we still sell cigarettes. And though WHO proved processed meats cause cancer the deli counter line is still very long.

And we still go skiing, and we still take nonessential car trips. Things have benefits as well as costs.

I'd love to know what the benefits of cigarettes are.

It's so common I start to think society is too dumb-smart and optimize for cure to idiotic problems rather refactoring itself.

My condolences. My 2 year old daughter was recently diagnosed with Leukemia as well, it's a horrible message to get and I wish your family well.

One thing we try to remember is that those five year cohorts lack all the advances in the previous half decade. The five year survival percentage could have improved dramatically!

Look, I'm not comfortable discussing my own circumstances, but making it to mid-30s has been a huge blessing. Getting diagnosed after 40 years on this earth is a tough pill to swallow. I can't disagree that it feels like one of the most devastating things that could happen.

Yet, you know, there are actually cancers that strike kids before they even have a chance to reach puberty. One in particular is Rhabdomyosarcoma which is a cancer of muscle tissue that essentially is a death sentence. The treatments that exist are limited, and funding does exist to work on it, but it's not a well known issue until it hits a family, which means funding is pretty much dependent on foundations willing to spend money on a hard challenge that isn't glamorous.

What do I mean by glamorous? I mean breast cancer. Pancreatic cancer. Colon cancer. The things that are likely to hit a large group of people.

I sincerely hope the best for your sister, I really do. She's about to enter a world which is a new perspective on life. Over time I've learned that a lot of people don't value what they have until it's in jeopardy, and when the bubble gets burst, when mortality is staring strong, that's when it's time to get real, to bite down on the belt and take the pain if it means survival. Stay strong, and tomorrow holds a future that none of us can predict.

Pancreatic cancer research is far from "glamorous" and is sorely underfunded. Despite having one of the highest mortality rates of any cancer, it has some of the lowest funding.

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.

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.

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!

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.

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

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/

Reply on news.ycombinator.com