Category: Treatment
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Viral Delivery
I’m changing my tune and jumping on the CRISPR bandwagon, with the stipulation that for polyQ SCA, it must be futuristically deployed with viral delivery—i.e., nanoparticles cannot be effective enough. The time horizon for treating SCA(3) is beyond my lifetime, but at least it’s possible to imagine it working eventually on presymptomatic children. Neuronal death…
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FDA-Approved ASO Therapies as of 2017
As of 2017, there have been six FDA-approved antisense oligonucleotide (ASO)-based therapies on the market. These are all for non-brain diseases: diseases of the retina (2), liver (1), and three others. Number six (spinal muscular atrophy) is the closest we get to the brain. As I mentioned earlier, Ionis Pharmaceuticals is in the early stages of…
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SCA Therapeutics Development
This page lists some therapeutics (mostly drugs; sorted by company name) being developed for spinocerebellar ataxia (SCA)—leaning towards SCA3, which I have—and Huntington’s disease (HD). There are reasonably no claims to stop, prevent, or cure the diseases. The National Ataxia Foundation’s take on this. Why include Huntington’s disease? The SCA that I have (SCA3) and…
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How to Make a Treatment Seem Better Than It Is
The key is to shroud the treatment among others that are low-risk with clear short-term benefits and thus make it impossible to attribute specific improvements to specific treatments. For example, end a treatment session with a massage and ask for treatment feedback either during or immediately after the massage. Two more examples follow. The first…
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Why I’m Skeptical of Troriluzole for SCA3
I’m skeptical of a 20-plus-year-old drug (riluzole)—shown mostly ineffective for SCA3 (and SCA2)—in a new prodrug package (named BHV-4157 in 2016; also called trigriluzole in 2017; renamed to troriluzole in 2018) being then on-label repurposed for SCA/SCA3. This seems to me to be primarily a money-generating move, and yes, I do accept that, because I accept…
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CRISPR and Crunchier
My understanding of CRISPR/Cas9 is simple: it’s a gene-editing tool that can change the DNA in the genome of an individual cell, like genomic search-and-replace, thereby modifying the cell before it performs its function(s), such as replication (cell division in non-neurons) and protein creation (gene expression). 2017-10-25 note: CRISPR/Cas13 operates on RNA instead of DNA,…
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Shut Up, Genes (ASOs and RNAi)
Antisense oligonucleotides (ASOs; sometimes abbreviated as AONs) are a form of gene silencing discovered in 1978. The first ASO drug was approved by the FDA 20 years later in 1998. Another form of gene silencing is RNA interference (RNAi) and was discovered in 1998. The first RNAi drug was approved by the FDA 20 years…
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Clearer Thoughts on a Cure
Some of the spinocerebellar ataxias, including SCA3, are genetically similar to Huntington’s disease (HD) in that they involve CAG repeats on a chromosome. I’ve found the HD community to be more informed and practical than the SCA community, where: There’s not a misinformation campaign against accurately conveying disease prevalence, as there is for SCA. Sadly,…
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What is BHV-4157?
2018-08-14. Trigriluzole was renamed to troriluzole. On 2018-11-02, most references to trigriluzole and BHV-4157 were removed from the Biohaven website. 2017-October: After SCA trials failed, Biohaven was quick to remove some SCA-related information from their website (hence, some broken URLs). See here. Later, they resumed testing after doubling the dose. 2017-April (or so): Biohaven removed…
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Stranger in a Strange Land
The world would be a strange place if everyone in it grokked the ataxia family of diseases. What does the average person have to gain by educating themselves on one rare disease, if it doesn’t affect them, and there are thousands of rare diseases? Nothing. I don’t know if this is a real quote, but…