Many articles on Spinal Muscular Atrophy…..What do they Mean?

It seems that every day, there is an unending supply of new articles posted on the web regarding Spinal Muscular Atrophy. Some articles are great………..some are horrendous…some posts seem to be unintelligible while many seem to contradict one another. While some still are very well written, but they may only be a partial view of a program which could lead to misinterpretation of the data. We try to stay as up to date on the research as possible………Our little secret……yup we are giving it up………..is to set up a Google Alert for Spinal Muscular Atrophy. All right…..OK……the truth is many SMA families already have a google alert set up for SMA and this tip is for newer families.  It is extremely easy to do just by using this link. In seconds  you can be assured that you will always be one of the first to read the latest articles on SMA research. The important thing to realize is that not all articles are worth reading! You must be careful of what is posted…..for example just the other day this link popped up on Google Alert on IPS Stem Cells.The post claims that IPS stem cells are now curing SMA. By now many of us already understand that Stem Cells Research has a way to go before it can be considered a viable treatment for SMA. Yet this article made some extraordinary claims. I recently saw a post on the proven efficacy of Neurotrophic support using stem cells. The article sounded very promising and the use of 36% extension of life made this therapy seem so enticing. However, when we use our SMA Mathematics we see that a 36% extension of life in a mouse model that lives 15 days, translates to a 5-6 day extension of life when administered early.

There was an excellent update on SMA Research and the historical timeline of this disease posted to the Archives of Neurology. The article spoke about where we have been and what future therapies are on the horizon for our community. The article included results on prior therapies as well as future ones. The article posted some great findings using AAV9 delivery of SMN1, however the articles interpretation could lead to a biased view by the reader because some  information was not included:

“This resulted in SMN1 expression in 60% of spinal cord motor neurons and complete rescue of motor function and strength, muscle physiology, and life span, so that the treated mice had an average life span of more than 400 days compared with approximately 16 days in the untreated animals. This approach has been reproduced by other groups using both adeno-associated virus, serotypes 8 and 9 constructs to deliver SMN1 to motor neurons.2526,32 An important caveat to this approach is that injections done in the mice have their maximal effect on postnatal day 1; the effect falls off rapidly with advancing age so that injections on day 5 had only a partial effect, while injections on postnatal date 10 had no effect.”

As an SMA parent who is reading this article it becomes very clear that a particular part of this paper stands out. If my opinions were based solely on this sentence I might have a different view of this program.

An important caveat to this approach is that injections done in the mice have their maximal effect on postnatal day 1; the effect falls off rapidly with advancing age so that injections on day 5 had only a partial effect, while injections on postnatal date 10 had no effect.”

With limited knowledge an SMA parent might make a presumption on how this will translate to our kids. However if we look at the larger picture, including what we know which has already been presented, the landscape changes. When we move into a larger animal…….much closer to a human…..a non-human primate, we see in some cases up to 90% transduction of motor neurons. The primate however did not have SMA. So the big question is how does this impact SMA? There is still a big part of the equation that has yet to be filled in. When a non-sma mouse was injected with AAV9 what were the results? Did we see a greater transduction of motor neurons? Was the window to target motor neurons larger in a healthy mouse? The answer to that question is the results and timeline in an SMA Mouse and a healthy mouse were ……………..identical!

The important thing to understand is that there are many many questions that are yet to be answered. We must push forward the most promising programs with supportive data to the clinic to really determine how they will or will not impact our community. If a program is showing lack luster data from the start ……move on and find something with better data and advance that program. We look forward to effectively and efficiently raising the necessary funding to push the most promising programs towards the clinic.

Comments

  1. Teri Taylor says:

    Once again, well said. Thank you.

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