I was amazed - and, needless to say, very happy - to read of a young girl in England who's now cancer-free, thanks to a revolutionary new treatment.
A teenage girl's incurable cancer has been cleared from her body in the first use of a revolutionary new type of medicine.
. . .
The team at Great Ormond Street used a technology called base editing, which was invented only six years ago.
. . .
Base editing allows scientists to zoom to a precise part of the genetic code and then alter the molecular structure of just one base, converting it into another and changing the genetic instructions.
The large team of doctors and scientists used this tool to engineer a new type of T-cell that was capable of hunting down and killing Alyssa's cancerous T-cells.
They started with healthy T-cells that came from a donor and set about modifying them.
- The first base edit disabled the T-cells targeting mechanism so they would not assault Alyssa's body
- The second removed a chemical marking, called CD7, which is on all T-cells
- The third edit was an invisibility cloak that prevented the cells being killed by a chemotherapy drug
The final stage of genetic modification instructed the T-cells to go hunting for anything with the CD7 marking on it so that it would destroy every T-cell in her body - including the cancerous ones. That's why this marking has to be removed from the therapy - otherwise it would just destroy itself.
. . .
"She's the first patient to be treated with this technology," said Prof Waseem Qasim, from UCL and Great Ormond Street.
He said this genetic manipulation was a "very fast-moving area of science" with "enormous potential" across a range of diseases ... Sickle-cell anaemia, for example, is caused by just one base change that could be corrected.
So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.
There's more at the link.
This news is simply mind-boggling. There are dozens, possibly hundreds, of medical conditions for which there is currently no treatment possible except palliative care. If base editing can eliminate them at the source, in our own bodies, that will open up new lives to untold numbers of people who were staring death in the face, with no alternative and no hope.
One of the most difficult things a pastor has to do is to try to help and comfort families and patients faced with that kind of problem. I've had to do it on occasion, and it's heartbreaking. To have to tell a young child that she's going to die, in a way that she can understand - if not accept - and then to have to tell her parents that no, their daughter won't be coming home again . . . I never, ever want to have to do that again. Perhaps, with this new technique, it'll become less common. I hope and pray so, anyway.
Meanwhile, for those battling this sort of disease have new hope. For example, in the USA, millions of black people are fighting sickle cell anemia, for which there is no medication to provide a cure. I hope this technology spreads here, and all over the world, as fast as possible. A lot of lives are at stake.
Peter
8 comments:
'Before receiving the treatment, Alyssa said: ‘Once I do it, people will know what they need to do, one way or another, so doing this will help people – of course I’m going to do it'
These days, too many are described as heroes. It's an honour to see a real one.
Millions of Americans are not fighting sickle cell anemia:
https://www.ncbi.nlm.nih.gov/books/NBK482384/
Sigh! Yes, this is a great news, but remember: this is also gene-based therapy! These type of therapies will always be at least somehow controversial: where is the border between correcting a problem and playing God?
Don't get me wrong: I'm all for finding means for correcting genetic diseases but these therapies need to be systematically and very thoroughly tested before being largely deployed. Otherwise we'll be in the same situation as the COVID "vaccines": shoddy patchwork at best...
The other problem is the temptation to "improve" the existing human genome, thus quickly coming to "custom-tailored children" or similar "initiatives". I'm pretty sure the military is positively salivating over "optimized combat enhancement therapies" or some-such nonsense...
Einstein was right when he said that “Technological progress is like an axe in the hands of a pathological criminal.” ...
While I agree "millions" are too high, the article listed says 1) 300,00 are born in the USA every year in the USA. 2) There are about 100,000 victims with sickle cell in the USA. If both are true, the death rate among children is fantastically high. Looks like most people live with it, and don't talk about it, and/or are not devestated by it.
A crisis is indeed serious. One can imagine eliminating the affected blood cells, my understanding is that not all blood cells are affected. But that would be a continuous need - every three months or more.
I believe the problem lies in the bone marrow which is producing the sickle cells. It might not be feasible to eliminate a high percentage of those.
Nice, I'll have to check with my Oncologist, thanks. Acute Lymphoblastic Leukemia is also known as Acute Lymphocytic Leukemia, before now it was a (rather short) death sentence. I actually have a closely related cancer, Chronic Lymphocytic Leukemia, the important difference is it is slow, one of the slowest cancers actually and in general people my age who are diagnosed with it most always die of something else, it was the primary cause of death of my paternal grandmother, however. For me I just get my blood work checked every year but this is worth keeping an eye on.
Very good to read..Thanks for posting this.
Agreed... sometimes, the medical achievements we are capable of make me very proud of my species.
Then, there are other times...
The College of Physicians and Surgeons in Ontario Canada, is apparently mandating that its members consider (and administratively designate) people who refuse the covid vaxx to be mentally ill.
If you also own firearms, this might be a problem as far as meeting the base requirements for the associated license is concerned.
This will also permit such a person to apply for assisted suicide.
Don't get sick.
Mike in Canada
This is a miracle cure for cancer. I've been expecting it for decades, and I'm glad to see it finally worked - even once. The problem is it requires designing a unique genetic therapy for each patient, so mass deployment is going to be very difficult.
Sickle cell anemia is NOT cancer, and it won't be treated by this exact technique. It's an mutation of the gene for hemoglobin, producing hemoglobin that is inadequate for the normal job of carrying oxygen in your red blood cells, but is poisonous to the malaria germ, which dines on red blood cells. If your two sets of chromosomes include one sickle-cell gene and one normal gene, you have 50% normal hemoglobin and 50% changed. You are pretty much immune to malaria and can thrive in malarial tropical swamps, and you have enough oxygen-carrying capacity to live and work hard in the lowlands. Extreme exercise at high altitudes such as mountain-climbing is out, but that's not a problem for 99% of people. Don't live somewhere like Denver or Flagstaff, and it's likely you'll never even know you have one sickle-cell gene.
But if two carriers (each with one sickle-cell gene) marry, on the average 1/4 of their children will have two normal genes (and are susceptible to malaria), 1/2 will have one of each and be normally OK, and 1/4 will have two sickle-cell genes and (absent extreme medical intervention) die from inadequate blood oxygen capacity. In the old days in some African swamp, that meant that if you are a carrier, half your children were healthy and survived, while the other half would mostly die young - but that puts you ahead of non-carriers, who either died of malaria or were sickly with sickly or dying children. But it really sucks when you are Minnesota and don't have to worry about malaria but one of your kids is dying of sickle-cell because you and your wife both happen to be carriers.
So currently, you can prevent sickle cell in your children by genetic testing yourself and everyone you might meet before you might meet them and fall in love. Like that's going to happen... You can hope the known treatments happen to work well on your kids, or try a bone marrow transplant, which might grow normal red blood cells but always has adverse effects on the immune system. Or you can hope for some kind of gene therapy to be developed. This would NOT be the one used in this case, but would have to be something like removing bone marrow and genetically changing it to eliminate the sickle-cell genes, until there was enough to kill the patient's bone marrow and replace it - rather than transplanting someone else's bone marrow (which is never a 100% match for immunity), you fix the patient's own bone marrow and put it back. That would do it, but it's a huge job.
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