Lentiviral therapy

[Intensor]

Who wants to go to China for testing?ichgcp.net/ru/clinical-tria...

[monkeybus]

Lentiviral Gene Therapy X-ALD

September 18, 2019 Updated: Shenzhen Geno-Immune Medical Institute

Lentiviral gene therapy for X-linked adrenoleukodystrophy (X-ALD)

This is a phase I/II clinical trial of gene therapy for the treatment of X-linked adrenoleukodystrophy using the highly safe, highly effective, self-inactivating lentiviral vector TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of a clinical gene transfer protocol.

Study Overview

Status

Unknown

Conditions

X-linked adrenoleukodystrophy

Intervention/treatment

Genetic: Intracerebral LV Gene Therapy

Detailed description

X-linked adrenoleukodystrophy (X-ALD) is a severe neurological disease caused by mutations in the ABCD1 gene encoding the peroxisomal ATP-binding cassette transporter (ABCD1). ABCD1 is responsible for the transport of CoA-activated very long chain fatty acids (VLCFAs) to peroxisomes for degradation. X-ALD is clinically characterized by two main phenotypes: adrenomyeloneuropathy (AMN) and inflammatory cerebral ALD. This disease is most common in men. Approximately 50% of heterozygous women show some symptoms later in life. Approximately two-thirds of patients with ALD will have the childhood cerebral form of the disease, which is the most severe form. The disease is characterized by normal development in early childhood, followed by a rapid degeneration into a vegetative state. Patients with ALD are usually treated with hematopoietic stem cell transplantation (HSCT) from a matched healthy donor. However, HSCT must be performed at a very early stage of the disease, which limits therapeutic options in juvenile or adult forms of ALD. This trial aims to treat ALD using a self-inactivating lentiviral vector with improved safety and efficacy carrying a functional ABCD1 gene to correct a genetic defect. By intracerebral injection, a lentiviral vector with a normal ALD gene is delivered to correct the pathologies associated with this genetic defect.

The main objectives are to assess the safety of the advanced self-inactivating lentiviral vector TYF-ABCD1, the clinical protocol for in vivo gene transfer and the effectiveness of the degrading metabolite in patients during treatment, the assessment of vector integration sites, and, finally, the long-term correction of patient behavior during the disease.

[nikolahn in reply to monkeybus]

Sounds better than Minoryx stupid story - “to freez” the young pacients brain damage state.

Please define me the meaning of young and to freeze.

[monkeybus]

Just to add a bit more meat to this.

Here is a non-Google translated text of the trial. It's the usual stuff.

ichgcp.net/clinical-trials-...

szgimi.org/product.php?id=7

That is their website. All of 10 patients enrolled in this trial. Trial is long finished. No results posted. I am used to that.

If you fancy putting your name down, [e-mail] is the place to head to. They got English and Russian speaking staff. From what I can make out, this is a private clinic.

The doctors have all been published. Seems on the up, though the English language website is full of dead links and doesn't have an SSL certificate.

All the time I spent traveling in Asia, a decent China hospital doesn't scare me one bit. The Chinese know their science, and they are pouring billions into it.

I'll mail them tomorrow and find out what they are selling. It is in Shenzhen. Plenty of money/tech/research there.

[3WheelWonder in reply to monkeybus]

yeah, I was wondering why you put up incomplete information😁

American hospitals scared the hell out of me

[monkeybus in reply to 3WheelWonder]

Try a British hospital. I spent a very long 8 hours waiting in one when I took my mother in there as an emergency last year. Eight hours, then we gave up.

I mailed that China clinic. I'll post up the hard-sell that they try on me.

The website put me off, though. If they can't manage the most basic Web design skills, it's unlikely I'd let them stick a needle in my arm. Still, we have to look up, not down. Be interesting to see what they say. They'd have to try hard to beat the $50,000 price tag that the Panama clinic quoted me.

[3WheelWonder in reply to monkeybus]

is this for stem cells?

[monkeybus in reply to 3WheelWonder]

Here is the reply...

The ALD lentiviral gene therapy treatment has ended the phase I safety trial. The result is encouraging. The intrathecal lentiviral injection is safe, and observed brain white matter regression after treatment.

This positive result encouraged the 301 team to expand the treatment to global patients in a Global Reach Program. Due to limited trial fund, international patients fund own treatment, yet the lentiviral product itself is supplemented and free to the trial candidates. The total estimated fees is around 50000 USD which includes all domestic expenses, lodging, hospital procedures (anesthesia and treatment, hospitalization up to a week), excluding meals and unpredictable medical expenses.

The 301 IRB although approved and treated one adult AMN, the IT LV injection of the GLOBAL REACH PROGRAM is mainly open to pediatric patients, although adult AMN patients may benefit more. We are currently sorting out an adjunct program to accommodate AMN patients. If you are interested, we will assist with the evaluation process.

Shall I file this one under my semi-regular WE ARE CURED series of posts?

[3WheelWonder in reply to monkeybus]

confirmed: logged under WE ARE CURED series of posts?

[Kranal in reply to monkeybus]

Thanks monkeybus for all your down to earth replies, and up-to-date information. My son (33 years) has lived with adult cALD and AMN for 8 years, and we have a realistic outlook now. Kranal

[nikolahn]

👍

Any word about neuropathy treatment?

[monkeybus in reply to nikolahn]

That's the entire reply I received. No promises about anything. Don't build your hopes up.