Any experiences of gene therapy trials for neurodiverse children?

[OttoGraph]

Im not sure where to put this

But has anyone's neurodiverse child had gene therapy? obviously for a gene mutation, this gene therapy is in the trial stages still

Child is under neurologist at a children's hospital and the chid has been put forward for gene therapy ( obviously with permission) ,

Looking for any advice, experiences etc

I haven’t researched gene therapy for my ND child, could you share your genuine research sources please?

what do you mean?

the child has been put forward by a neurologist at a children’s hospital for gene therapy.

im not a neurologist, they do the research etc

Gene therapy for what exactly? Neurodiversity?? Or a different condition?

A gene mutation, I wasn’t aware gene therapy could be used on other conditions

Your child wouldn’t be entered into a trial without you being given an information pack and consent forms. So what is the trial?
What is the research being conducted and the goal?
What is required from participants?
Do you get an update?
Which specialist is conducting this trial?
At which facility?

I have entered multiple genetic trials due to a genetic disorder, as have both my children, one of whom has a neurodivergent disorder.
The fact that you don’t understand what I mean by what is your research into gene therapy for neurodivergence is deeply concerning

I'm guessing your child has a genetic disorder that has neurodivergence as one of the features of the disorder? And the gene therapy is to try to address the genetic fault known to cause this specific disorder? I only have experience from the science side, not the patient/parent side so that's probably not what you're looking for, but I wish you well as your navigate this for your child ❤️

I’m not doing the gene therapy, the neurological team will do that., that’s not concerning to me.

I just came here to see if anyone had children who had gene therapy, don’t really want to share with you all the details you’re asking for

Thank you, yes child has gene mutation and the therapy is to try to correct this. Yes the gene mutation comes with comorbilities that include neurodivergence.

was just looking for any other parents that had experience of this

thanks

Some of these posts are unnecessary.
op neurodivergence means someone’s brain and sensory processing etc are different to those who perform in a neuronormative (socially accepted/expected) way. It can include diagnosed conditions but covers a whole range of presentations including eg mental illness.
im guessing that the gene therapy is not aimed at the neurodivergence specifically however that presents but the gene mutation? Maybe asking if people have had experience of gene therapy might get more helpful replies?

You're definitely the arsehole here.

Well I am (well, was...) a geneticist @Dingledanglebinglebongle and I don't understand why you're being so aggressive to the OP. Why don't you spell out your concerns instead of bombarding her with intrusive questions and rudeness?

@TofuTuesday the neurodivergence aspect may well be relevant though as it may affect how the child understands what is happening medically, or how they process the experience, compared with a NT child with a genetic condition.

Gene therapy is only suitable for monogenic conditions, which are only responsible for a very small proportion of autism diagnoses. Gene therapy for these named conditions is in its early trial stages so only an extremely small number of children in the U.K. might be involved.

Sorry I don't think I'm actually helping. @OttoGraph are you involved with any genetics-focused patient support groups like Unique? Might be the best way of connecting with people in your position, given that gene therapy trials by their nature are quite rare and usually involve small numbers of patients.

Edited for typo

JohnnyAndTheTaxDemand

thanks, not heard of Unique, off to look that up - I was just after others experiences and as you says its only a small number of patients I guess

I know they do gene therapy for SMA1 and sickle cell, but don't think they have the neurodiverse aspect as a comorbidity to the gene mutation

Yes gene therapy is used for many conditions

Do you mean for genes to be mutated to 'remove' the genes which are thought to be associated with ND?

I think its fairly obvious OP has a child who has been entered into for this and she just wants people to share stories of any of their experiences

She doesnt need the Spanish Inquisition.

Yes of course but I’m not guessing that the gene therapy is aiming to resolve neurodivergence (suspect is being used as shorthand for autism or ADHD).

Firstly, I'm assuming the OP doesn't want to share very private medical information online.

Secondly, you have no idea as to the education or background of the OP. People who speak English as a second language, or aren't massively literate or educated also have kids who have conditions and get put forward for trials. Those people might not go home and pull out the latest academic peer reviewed literature. They might go on mumsnet for some advice. Give her a break.

I'm guessing the gene therapy isn't 'for' the neurodiversity. But interesting some kids who are diagnosed as (for example) autistic actually have an underlying genetic condition. I guess if you could cure the genetic condition the symptoms might fall away.

Gene therapy for neurodiversity per se should theoretically be possible but I gather isn't being pursued due to accusations of eugenics.

I have ND kids and I absolutely wouldn't do gene therapy for that. That would change a fundamental part of who they are. If they wanted to do it when older, that would be their choice.

On the other hand, if there was gene therapy for the autoimmune medical condition, I would be all over finding out more about that, though I'd be wary of something so new.

MiniatureHouse

they have been in picu 3 x now and twice the emergency bell going off for them. It’s is so scary & I find this hard and if the gene therapy can have a high percentage of preventing more visits to picu, which is likely, I’d rather go down that route. I appreciate everyone has different views.

I've been thinking about this and I'm not sure it would be possible to use gene therapy for the ND aspect of a condition. Gene therapy normally relates to fixing a faulty 'recipe' for cells, I think. For example, with SMA, the gene therapy can stop the degeneration of neurones, but it can't reverse the destruction of the ones that were already destroyed.

Autism is accepted to be a physical condition - the inherent wiring of the brain is altered. You can't undo that by changing genes.

Similarly, DD1 has a brain malformation and it has now been suggested that she has a genetic variation that may be responsible. No amount of gene therapy is going to redistribute her grey and white matter concentrations throughout the areas of her brain that are affected. She's lived with autism and ADHD symptoms for over 20 years. I can't imagine that these could now be reversed.

I don't even know who DD1 would be if she didn't have her ND conditions. She just wouldn't be her. Can you imagine, after 20 years, if her learning disability was reversed? She'd have to learn to read properly, write properly, construct paragraphs, discuss matters of the world with adult themes, learn proper social etiquette - it would be like plonking an alien in the world and expecting them to know how to function.