ASO – Potential ataxia treatment?

Nobody will say it out loud yet, because there are still many unknowns, so I will say it.

Hey, there MIGHT be a cure!

I guess. It seems that yes, there is.

I am not going to convince you of the correctness of my suspicions, but I will share the information I have gained.

I am talking about gene therapy based on the action of antisense oligonucleotides, which are probably effective in reducing the harmful activity of excess proteins that are produced by ataxia. Ok, that may sound complicated so I hope this graphic will help you understand:

Unfortunately, I do not remember the author – it is from the NAF conference. If you happen to know, please let me know!

I will often cite various scientific works here because they best reflect the essence of things. So try to stay focus and understand these complicated scientific materials.

 „ASOs are short, synthetic, single-stranded strings of nucleic acids of approximately 8 to 50 nucleotides in length (…) Recent years have shown great promise for ASO therapies in an application for neurodegenerative disorders.”

There is a strong suspicion that this therapy is simply working. How do we know this?

* This approach is used to treat a disease called SMA with a drug called nusinersen and, well, it works (article)

* This approach has been tested in ataxia mice and, well, it works (article)

* ASO treatment to lower ataxin-1 levels doesn’t cause unwanted side effects in a SCA1 mouse model (article)

* at the NAF conference this approach was often referred to as highly promising

* there is a facility called VICO therapeutics, which received research funding from European institutions and the US FDA

„VICO’s lead antisense oligonucleotide product is designed to suppress these mutant proteins and slow or halt disease progression. A major strength of VICO’s approach is the broad applicability to different polyQ diseases and the preferential reduction of mutant relative to normal polyQ proteins.”

References supporting VICO’s RNA modulating therapeutics:

Mrs. Susan Perlman at the NAF conference in Las Vegas said there will be a cure for ataxia in 5 years.

We will within the next five years have at least one approved disease-modifying drug for ataxia

Susan Perlman

Everyone started clapping and I had tears in my eyes. Dr. Perlman does not cancel her statement but emphasizes that events such as the global Covid epidemic have extended all research by a year. However, I believe that yes, the drug will come. The drug is here, it exists, but it needs time to be tested, approved, and commercialized. Therefore, all we can do is wait and dream. But it seems we are very close to a solution.

Of course, there are still a lot of other questions – will it work for other ataxias? Will it work for people with severe ataxia? How many doses do we need? It is still unknown and we can only guess.

Ataxia Cure? So what does it mean to us? – read here

I have different sources of knowledge, but I mainly use these:

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