2016-11-15

An anonymous reader quotes a report from Popular Mechanics:
A team of Chinese scientists from Sichuan University in Chengdu have become the first to inject a person with cells modified with the gene-editing tool CRISPR-Cas9. The trial involved modifying a patient's own immune system cells to make them more effective at combating cancer cells and then injecting them back into the patient. The Chinese trial was approved back in July, and United States medical scientists also plan to use CRISPR as an experimental treatment for cancer patients in early 2017. The CRISPR-Cas9 "tool" is a DNA construct that can be injected into any organism -- in this case, human immune system T cells -- to modify the genome of that organism. It works in three steps: an RNA sequence guides the CRISPR construct to the correct part of the organism's DNA, the Cas9 enzyme "cuts out" that segment of DNA, and then, as an optional third step, a new DNA sequence can be inserted to replace the deleted segment of the genome. In the case of the Chinese trial, conducted October 28 at the West China Hospital in Chengdu, only the first two steps of the CRISPR-Cas9 process were carried out. Immune system cells were extracted from a patient with metastatic lung cancer, and then the gene code that produces a protein called PD-1 was deleted by the Cas9 enzyme. PD-1 instructs T cells to stop or slow an immune system response, and cancer cells can take advantage of this protein to trick the body into responding to the ailment with less than full force. Once the PD-1 protein was removed with CRISPR, the edited cells were cultivated to increase their numbers and then injected back into the patient. This is the first of two injections for the patient, and an additional nine patients in the trial will receive between two and four injections of edited cells, depending on their individual conditions. Carl June, scientific advisor for the planned U.S. trial,
told Nature: "I think this is going to trigger 'Sputnik 2.0,' a biomedical duel in progress between China and the United States, which is important since competition usually improves the end product."

Re:Do it, do it now!

By SemperUbi



2016-Nov-15 23:50

• Score: 5, Informative
• Thread

This particular use of CRISPR-Cas9 should be pretty low risk. Tumors are often surrounded by lymphocytes, cells which ordinarily have the power to kill cancer cells -- but many cancers give off signals that cause the lymphocytes to ignore tumors cells and let them grow. It's like the mafia buying off the local cops. Modifying tumor-reactive lymphocytes ex vivo might wake them up and help them do their job and go after the tumor again. In a patient with terminal cancer, this treatment should be pretty low risk and could give him a bit longer to live.

This is excellent, excellent, excellent news

By ShooterNeo



2016-Nov-16 01:04

• Score: 4, Insightful
• Thread

I saw the ugly truth of biomedical research when I was in grad school. TLDR, while every last stinking one of us has every possible motivation to spend EVERY spare penny not keeping us alive in the near term on the research (since it's the only thing that has the slightest chance of making sure we continue living past a mere 7-10 decades), there are 2 nasty problems :

1. Due to extreme amounts of government and institutional red tape, nothing gets done. Nothing. All those stories you read of brain implants? Basically never going to happen. That's because the way the legal system works is, institution administrators always have to ask "can WE be blamed if this goes wrong?" Basically, if the research kills someone but ultimately saves 1000 lives, our courts won't give any credit to the 1000 lives saved, it's all about slamming the institution for making an error. Also, the government has a very poor model for assessing results. If a drug works on cancer that has failed every other treatment, you don't need a trial with 1000+ participants. Cancers that reach that stage don't just disappear for no reason. A trial with 20 people is enough if 10 of them get up and leave with their tumors destroyed. This is a very strong effect and one that shouldn't require the one size fits all approach the FDA demands.

2. Most medical spending is on overpriced procedures and drugs and equipment that all suck.

Re:This is excellent, excellent, excellent news

By ShanghaiBill



2016-Nov-16 01:13

• Score: 4, Insightful
• Thread

1. Due to extreme amounts of government and institutional red tape, nothing gets done. Nothing. All those stories you read of brain implants? Basically never going to happen.

It will happen, just not in America. In China, stuff gets done.

America will take at least 30 years to build a high speed train from SF to LA, at a cost of $100-300B.
China built the high speed train from Shanghai to Beijing (twice the distance from SF to LA) in 3 years, at a cost of $32B.

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