BBC News, James
Gallagher, Health and science reporter, 11 July 2013
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| Errors in the genetic code can have deadly consequences |
The study,
in the journal Science, showed the three patients were now going to school.
A second
study published at the same time has shown a similar therapy reversing a severe
genetic disease affecting the immune system.
Gene
therapy researchers said it was a "really exciting" development.
Both
diseases are caused by errors in the patient's genetic code - the manual for
building and running their bodies.
Decline
Babies born
with metachromatic leukodystrophy appear healthy, but their development starts
to reverse between the ages of one and two as part of their brain is destroyed.
Wiskott-Aldrich
syndrome leads to a defective immune system. It makes patients more susceptible
to infections, cancers and the immune system can also attack other parts of the
body.
The
technique, developed by a team of researchers at the San Raffaele Scientific
Institute in Milan, Italy, used a genetically modified virus to correct the
damaging mutations in a patient's genes.
Bone marrow
stem cells are taken from the patient then the virus is used to 'infect' the
cells with tiny snippets of DNA which contain the correct instructions. These
are then put back into the patient.
Three
children were picked for treatment from families with a history of
metachromatic leukodystrophy, but before their brain function started to
decline.
Dr
Alessandra Biffi told the BBC: "The outcome has been very positive,
they're all in very good condition, with a normal life and going to
kindergarten at an age when their siblings were unable to talk.
"It is
something which is very pleasing to us."
'New era'
She said
that all treatments had side effects and these patients needed to be followed
for longer, but the evidence so far suggested the treatment was safe.
Gene
therapy is a field that has promised far more than it has delivered and has
been hampered by serious concerns about safety.
Dr Biffi
said lessons had been learnt from previous failings: "Experience showed
that gene therapy could be improved and we could be at the starting point for a
new era to achieve more than we did in the past."
In the
other study, published simultaneously in the journal Science, symptoms such as
repeat infections and eczema had lessened in the three patients treated.
Prof Bobby
Gaspar, from Great Ormond Street Hospital in London is working on a Medical
Research Council trial using gene therapy as a treatment for adenosine
deaminase deficiency - which also leads to immune problems.
He told the
BBC News website: "This is really exciting. Metachromatic leukodystrophy
is a very significant neural degeneration which cannot be cured in any other
way and now the study shows they can live relatively normal lives.
"It
raises the prospect that other diseases can be treated in the same way."
Prof Luigi
Naldini, who leads the San Raffaele Telethon Institute for Gene Therapy, said:
"Three years after the start of the clinical trial the results obtained
from the first six patients are very encouraging.
"The
therapy is not only safe, but also effective and able to change the clinical
history of these severe diseases.
"After
15 years of effort and our successes in the laboratory, but frustration as
well, it's really exciting to be able to give a concrete solution to the first
patients."
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Kryon Q&A
Question: Dearest Kryon: I've read the question and answer on people with diseases of the mind - for example, Alzheimers and dementia. But I have more questions. You say that people have chosen this path and that the lessons are for us. As I work with these people, I'm wondering if there is any stage where there could be a reversal of the condition, and if so, with what methods? The people in the hostel are so drugged up, and there's a mind-set with the authorities that no "alternate" therapies work - although they're using colored lights. (Sadly, the diversional therapist told me she doesn't know what color therapy is.)
From a spiritual point of view, what is the best way to work with these people - talk to them as though they were normal, or go along with their imaginings? I've been told that they need to be kept quiet, especially toward evening. However, I've found that with one woman who mostly paces saying very little, the more childlike I am (dancing and singing makes her happy), the more she talks. I could go on and on - could you please enlighten me further?
Answer: I will answer the second part first. Love those who are in this condition. Find out what makes them smile... and then make them smile. The best you can do in a facilitation of this condition is to somehow create joy. Even in their confusion they can laugh at situations and be creative. They'll also remember you better as the one who creates this emotion. Each is very different, but in general, try to find their "happy" button and push it as often as you can. They will remember that.
Right now you're perched upon some important discoveries that will be able to reverse these conditions to a large degree. But just as the paraplegic who regains their nerve connections must than relearn how to walk, suffering much pain, there will be this attribute with a regeneration of the mind. Even if new cells are created, they won't necessarily have the old memories, but they can be trained to be healthy and be ready for new memories.
So someday these will have the ability to halt the progress of the degeneration of cells that are being taken, and instead grow new pathways around them. Some will be able to "reconnect" to certain kinds of memories (like recognition) but will have to relearn what the association of recognition actually means. So history and events might have to be studied and relearned... sometimes even things like reading, also. The pain will be that the individual will regain mental health and will realize exactly what has happened.
Your stem-cell research is very important, and you're reaching a point where you'll be able to use birth cells that aren't embryonic, but every bit as potent for research... thereby sidestepping all moral issues. Look for this in the next few years.
Question: Dearest Kryon: I've read the question and answer on people with diseases of the mind - for example, Alzheimers and dementia. But I have more questions. You say that people have chosen this path and that the lessons are for us. As I work with these people, I'm wondering if there is any stage where there could be a reversal of the condition, and if so, with what methods? The people in the hostel are so drugged up, and there's a mind-set with the authorities that no "alternate" therapies work - although they're using colored lights. (Sadly, the diversional therapist told me she doesn't know what color therapy is.)
From a spiritual point of view, what is the best way to work with these people - talk to them as though they were normal, or go along with their imaginings? I've been told that they need to be kept quiet, especially toward evening. However, I've found that with one woman who mostly paces saying very little, the more childlike I am (dancing and singing makes her happy), the more she talks. I could go on and on - could you please enlighten me further?
Answer: I will answer the second part first. Love those who are in this condition. Find out what makes them smile... and then make them smile. The best you can do in a facilitation of this condition is to somehow create joy. Even in their confusion they can laugh at situations and be creative. They'll also remember you better as the one who creates this emotion. Each is very different, but in general, try to find their "happy" button and push it as often as you can. They will remember that.
Right now you're perched upon some important discoveries that will be able to reverse these conditions to a large degree. But just as the paraplegic who regains their nerve connections must than relearn how to walk, suffering much pain, there will be this attribute with a regeneration of the mind. Even if new cells are created, they won't necessarily have the old memories, but they can be trained to be healthy and be ready for new memories.
So someday these will have the ability to halt the progress of the degeneration of cells that are being taken, and instead grow new pathways around them. Some will be able to "reconnect" to certain kinds of memories (like recognition) but will have to relearn what the association of recognition actually means. So history and events might have to be studied and relearned... sometimes even things like reading, also. The pain will be that the individual will regain mental health and will realize exactly what has happened.
Your stem-cell research is very important, and you're reaching a point where you'll be able to use birth cells that aren't embryonic, but every bit as potent for research... thereby sidestepping all moral issues. Look for this in the next few years.
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