Cell-based Gene Therapy Represents ‘Hope’ for X... - AMN EASIER


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Cell-based Gene Therapy Represents ‘Hope’ for X-ALD Patients, Review Study Suggests

KennyInPA profile image

Definitely hopeful for the future of ALD/AMN!


13 Replies

"Encoded adrenoleukodystrophy protein (ALDP)". This is the first I've heard of it. Is this the enzyme responsible for breaking down the fatty acids?

I hope that this new therapy will eliminate the chance of AMN in adulthood. Regardless, this is exciting news!

That’s for males up to 17 correct

KennyInPA profile image
KennyInPA in reply to Roker1176

The Lenti D trial was for under 17, but I’ve heard chatter about stem cells for adults as well hopefully sometime in the near future!

How would this be different than stem cell therapy, like they do in Panama? monkeybus any idea?

This is great stuff, isn't it?

Jason, as I understand it, they pull out your own cells, fix the defective gene, then pump it back into you. No issues with tissue-type matching. A perfect match. Halts the disease. I'll settle for a halt

It is the fixing of the ABCD1 gene that's the key, isn't it. Doesn't say how they do it. Might be Crispr. Doesn't matter to me.

When can I sign up? Put me to the front of the queue, I am sure you'll all stand aside.

This kind of treatment won't be cheap. Cost an arm and a leg to develop it, and they'll want to recoup their investment. Who'll pay for this? Then again, it may be a simple laboratory procedure.

ahhh, so that would mean, ALL of your cells are scrubbed of ABCD1 gene?

They correct the mutation in your ABCD1 gene. Then they pipe it back into you.

Makes perfect sense. I'm going to look into this and write it up on my Website.

pretty invasive, like a BMT...lots of problems from that, man.

Just sayin

I'm with you, brother.

But we gots a choice. Let this disease keep marching on, getting progressively worse and worse. Spasticity, pain, neuropathy, spasms, blindness, epilepsy, sexual dysfunction, on and on. Have I missed out any symptoms? Probably.

The thing about a BMT is you'll never get an exact tissue type match (unless you have a twin brother).

So you'll take a chance on a donor that's got a similar match, they'll give you chemo to kill your immune system, pump in someone else's stem cells and you hope for the best.


But, if it is your own DNA they are sticking into you, your body will just accept it as is. Take your DNA out, fix it up, correct the ABCD1 gene, inject it back in.

Won't cure us, but if it freezes the disease as is, I'll buy that.

Jason, I'm 48. My son just watched Star Wars. I saw it in the cinema 40 years ago. FORTY YEARS AGO. Oh no.

I can still walk, Got my fatigue and neuropathy under control.

Lyrica, Modafinil, 4-AP, Gabapentin, Keppra, laxatives twice a week, Viagra as and when.

But I can function. If I can freeze my disease as it is now, I'll settle for that. Because you never know what tomorrow will bring..

Some of my symptoms came on slowly, others hit me quick, literally overnight. I had mild spasticity, then I was on holiday, at the beach, and bang, my legs were like jelly.

Was at work, What was it? Six weeks ago, normal day, then, bang, I had a two hour epileptic seizure. Woke up strapped to a bed. Spent a week in hospital. Every day is different with this disease. I read this board, some of us in chronic pain, wheelchairs, blindness, et al. Look back through the old posts, some of us have passed on. This f*cking disease.

I am currently looking for a bone marrow donor. If I can find a decent match, I'll go for it. Take a chance. I cannot allow this disease to continue attacking me.

We'll see what happens. If I have to pay, so be it.

It is good that we do have options, and other treatments are emerging. I can remember when I had zero hope.

Could you find a hospital/doctor to perform a BMT with your symptoms the way they are now? Also, it's fucking expensive!


why not try for stem cell treatment in a place like Panama first? That's a lot less coin,

monkeybus you never answered my question, I'm offended :)

My insurance wouldn't cover anything experimental. Panama/Mexico would qualify as 100% unproven.

But, you know me, I'd try anything.

Was milking my insurance today. Went to see a decent neurologist. I printed this out and showed him.

He said he'd just been to a major conference in the USA about this very thing.

I asked him about getting it done. He did a no comment.

He did say how it was moving into major clinical trials.

And that, was that..

He wrote me a prescription for the usual Lyrica, Gabapentin, Keppra and Modafinil.

Total cost of today's treatment, $900. Beat that. Thank God I've got insurance. This disease could bankrupt any of us.

Still, let's all stay alive and wait this one out. I have a good feeling about this.

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