Is it about optimizing the delivery system, or finding the right targets to test on in bigger animals first?
There are so many people closecto dying in cancer, that finding volunteers for a Phase 1 test should be easy.
Within that first year he was put on a trial that added another 2 or so years to his life. Then during that time another experimental treatment was created and tested on him and that gave him another couple of years. A decade and a half later here we are, he still has cancer but his family (wife and two kids) has got to keep him around a while longer.
He’s been “patient one” on a number of studies, some have helped him extend his life expectancy, others have done nothing. Sometimes he has the mental and physical energy to start a trial and other times he does not.
The cancer has never gone away, and there have been so many moments of false hope that I’m never really sure where he sits mentally anymore (he really doesn’t want to talk about cancer when we’re together and I don’t think anyone can blame him) but I know he’s grateful for the chance to add more time onto his life when the experimental treatments are available.
Still though, it's a better time to have cancer than ever I suppose.
There was one treatment that they were going to trial my friend on that would give him another expected extension but basically make his digestive tract stop working so he’d be pooping out of a tube into a bag the rest of his life but he declined, said it just wasn’t worth it.
There have been a couple times that’s been the call he’s made and I appreciate why.
Cancer sucks; why it is so hard for us to beat is so frustrating. need more research focused on this.
Good luck to him
It's very rare that everyone in the control group dies and everyone in the experimental group survives. Usually the result is mixed.
The problem is that medical doctors sometime make bad guess about how many years the person will survive. That's one of the reason to have a control group, so the error in the guess compensate.
A good friend of mine signed up for a trial as an alternative treatment for her aggressive breast cancer, everyone's hopes went up (trials tend to have that effect on people) but "nothing happened". She passed away 6 months after the trial started without knowing if a placebo was used. It's painful anecdata, but the few people I know that got into cancer studies as an alternative to their regular treatment, none ever had the study knowingly influence their outcome. Yet, in every case, the study was the one-hundred-percent animic lifeboat to grab on to.
Participating in a trial is about helping science just as, or more than, it is about helping yourself.
As an aside, I’ll have to be careful here because I think I might be confusing some of the trials he was a part of with actual treatments he received. It’s been a big mix of throwing a lot of different things at the wall.
One reason RNA drugs are so exciting is that their manufacture is simple and can be standardized, cutting the GMP manufacturing step to a few weeks instead of a year or more. Small-molecule drugs are ”bespoke” in that each needs its own, different, manufacturing process. In contrast, all RNA molecules are essentially chemically identical, differing only in the ordering of the nucleotides.
Then there is safety testing in animals. Then regulatory approval. Then starting the phase I trial. The trial might take a year or more. Then you need to show the drug works compared to some standard treatment, which in a phase 3 trial in breast cancer takes 3 years and many hundreds of patients at least. Longer if you want to show it prolongs overall survival. Then the drug needs to be approved.
There is also the matter of funding but there is a lot of money for early stage biotechs at the moment.
The 2014 result only showed that gene editing before birth might prevent cancers.
"yet it doesn’t seem to be important for normal development"
Gene space is huge. My cancer is cured, but now I'm dying of mange. So that's why that mouse kept licking itself. Big ask for someone else. They have trouble getting blood thinners, statins and beta blockers right. And there's no genetics there. It's stuff people ate out in the woods for hundreds of thousands of years.
> There are so many people close to dying in cancer finding volunteers for a Phase 1 test should be easy
I take the opposite approach. These people have few days left. It's a time to make things nice. It can get much worse quick. Cancer, which can be managed, is nowhere near as bad as it can get.
Disease tends to hit all at once. It's really just dying. Cancer, strokes, heart attacks and infections all tend to gang up at once. The trial will kill these people no doubt. If you have a ticket out of the hospital, take it! Don't squander it trying to wash mud.
Also the patient pays a lot for clinical trials. Hundreds of thousands of dollars.
Here take this medicine, we are fairly confident it won't kill you. Here is your $100k bill. Oh? Can't afford that? No trial for you!
Ancillary routine FDA approved testing that accompanies the trials may be billed to insurance in some cases? I don’t know about that. But a trial for a novel cancer treatment will be free, if not even include an honorarium of some kind to defray costs to the patient for participating.
I've had someone in the family who got cancer. It was basically how you said it would be. They lived their life to the fullest, not thinking about the untreatable cancer. Do whatever is fun and makes you happy.
At some point it got bad. No more living your life. There were bad moment. There were better moments. There were worse moments. I only saw it from afar until the very end. The last bit must have been hell for the closest family. It really is just dying. Slowly. Painfully. And all society and doctors try to do is to prolong the suffering.
I'm being unfair to some people. I do know the palliative care team did their best. All volunteer doctors. Power to them.
https://www.mskcc.org/cancer-care/clinical-trials/frequently...
My guess for the delay is production process and delivery mechanisms. There are delivery mechanisms where the combinatory effects lead to some serious toxicity.
Plus, how long do mice live for? I'm sure they are still taking down observations and collecting data. The article submission may be to help with funding.