Kennedys Disease Community
IGF1 SBMA study results

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11-15-2012, 11:05 PM
IGF1 SBMA study results
I found this article on MDA's website. The results of the study were first published August of this year. Looks very encouraging.

Article Highlights:
Mice with a disease resembling spinal-bulbar muscular atrophy (SBMA) that were treated with an insulin-like growth factor 1-based compound had better motor function, slower weight loss, improved muscle health and increased survival time.
Recent studies have shown that IGF1 works in SBMA by reducing toxicity caused by a mutation in the androgen receptor (AR) gene; it also helps promote muscle and nerve growth.
The new findings suggest that IGF1 potentially may be effective in ameliorating the disease.

Iplex (IGF1 and IGF1 binding protein)is approved for use in children with growth failure due to IGF deficiency. Iplex has also been used in other disease including ALS and Type 1 myotonic muscular dystrophy. While no improvement in muscle function was observed they write that the data suggests Iplex is reasonably safe for human use. In Type 1 MMD the patients did show improvements in lean body mass and metabolism.

The article finishes up by saying that SBMA may be a better candidate for treatment with Iplex not because of the non specific benefits to muscles and nerve survival but because Iplex changes the behavior of the abnormal AR protein.

Here is the link to the article.

Here is the link to the study.
11-16-2012, 03:43 PM
Jeff, the IGF-1 studies began several years ago at NIH and reports have been published several times. My "Living with Kennedy's Disease" blog has several articles on IGF-1 including an interview with one of the lead researchers in this study. NIH is hoping to have a clinical trial within the next two years if current trials continue to go well.

It continues to look promising and appears to be especially effective if started pre-or-early in the onset. Watch for additional information in the months to come.
01-31-2013, 02:23 PM
Deer Antler Velvet

I added this article to my blog ' Living with Kennedy's Disease' Living with Kennedy's Disease' and felt it needed to be shared in the forum.

A major issue with those of us living with a disease that does not have a treatment or cure is some of us become desperate. We read about something that works for another similar disease, or is advertised that it works in building muscle, and we think, “Why not give it a try?”

Well, there are plenty of reasons to ‘not give it a try’. The drug or concoction is not always safe or proven to work. It could also be dangerous, especially if we are not aware of the dosage. Finally, what other problems (side effects) might it cause?

deer antlerThe last few years IGF-1 for muscles and ASC-J9 are two drugs that have been discussed by researchers as having a potential benefit for Kennedy’s Disease (SBMA or Spinal Bulbar Muscular Atrophy). They are both currently being tested with small mammals. One of the big issues with both drugs is how to decide upon a dosage that will work, but will do no harm. And, if it does work, another issue is how to make the drug available (injection, pill, IV, etc.) and convenient.
Another issue with those of us living with Kennedy’s Disease concerns the mutation in our DNA. Some drugs will not help, and could possibly do more harm, because of this defect.

IGF-1Recently, several articles have been published about athletes taking a banned substance called IGF-1 or a supplement made from deer antler velvet. The athletes are using the drug to improve their strength (in other words, cheating). Several people with Kennedy’s Disease have asked about using IGF-1 that you can by over the internet or in a foreign country.

We consulted with a member of the KDA’s Scientific Review Board concerning the IGF-1 you can buy over the internet. We received the following reply: “I do not recommend the sublingual IGF-1 advertised on the internet site you sent. The IGF-1 (being test) in mice and the IGF-1 currently approved for human use are only available by injection, and neither has yet been shown to be safe and effective in KD patients.”

Being someone who is not getting any younger, I understand the lure of a potential treatment for Kennedy’s Disease. I also realize that patience is a virtue. NIH is testing both the drugs mentioned above. Hopefully there will be a clinical trial for one or both in the next couple of years. If and when it does become available for human use, it will be safe and effective.

02-15-2013, 01:38 PM

ASC-J9 are currently having human trials at the NIH. I have no idea how long something likes this takes, though, nor do I know the progress they are making.
02-15-2013, 02:21 PM
Ed Meyertholen
HI raxel19 - I do not believe that there are human trials of ASC-J9 at this time. The only ongoing trial is the exercise trial.
02-15-2013, 06:38 PM
Ed is correct, there is small animal testing taking place in hopes of moving to a clinical trial in the next one to two years.
02-19-2013, 10:07 AM
Hi there,

The same day I posted about ASC-J9, Dr.Chang answered my email re: the status of this potential treatment. He said it was currently in human trials at the NIH and to await results. Perhaps I was given wrong information or perhaps the public is not privy to all that goes on at the NIH. Let's hope it's the latter.
02-19-2013, 10:36 AM
Ed Meyertholen
There is a trial dealing with ASC-J9 as a topical cream for acne which used humans. You can search for clinical trials at NIH here- The only ongoing trial for SBMA is the exercise trial. In addition, if there were a clinical trial for ASC-J9, they would need patients with KD and imo, it would hard to hide this from the KDA - I expect the KDA would be a primary source of volunteers as it was for the dutasteride and exercise trials.

This message has been edited. Last edited by: Bruce,
02-19-2013, 10:40 AM
Understood. Sorry for an confusion. I specifically asked Dr.Chang about ASC-J9 for Kennedy's so assumed his answer referred to that. Sorry.
02-19-2013, 10:53 AM
Ed Meyertholen
No reason to be sorry, Raxel19, we all want this to proceed!!

I am not sure what Dr. Chang was referring to but it may be this grant - here is a link to the announcement of the preclnical trials sponsored by NIH - This is a 3 year grant (awarded in early 2011).

The following is from the announcement:

"This $3.8 Million cooperative translational research grant will leverage expertise from the NINDS and draw upon ASC’s innovative approach to targeting the mutant androgen receptor (AR). The goals of the grant will be to first validate an orally administered ARD enhancer drug is efficacious in the SBMA transgenic animal model, and further, to complete preclinical toxicology, safety pharmacology, and ADME studies necessary in supporting of an IND filing to commence human clinical studies. To date, ASC has provided robust proof of concept data using an ARD enhancer compound; demonstrating treatment ameliorates cardinal features of SBMA neuromuscular pathology, restores functional activity, and improves survival in a SBMA transgenic mouse model."
02-19-2013, 12:38 PM
Thank you, Ed! I have seen this article and am hopeful we will have more updates sooner than later!