Randy and Bob,
I believe you both saw this on ALSTDI forum, where I posted it March 2. Nothing has changed. The FDA will do what they’re going to do—with or without Italian data. This is no longer an issue of science (data about whether Iplex is or is not pre-proven and good for those with ALS). It’s simply an issue of whether or not they can deny a terminal population a medication that is already FDA approved—nothing else. That’s an issue of state statutes, federal law and FDA practice—all of which they (FDA) are now violating. And those physicians who will write off-label Rx’s or INDs will do so with or without Italian data—which they find suspect under the best of situations. And those who won’t write off-label or INDs will not start doing so because they see data from an Italian “observation.”
Anyone reading this can use their time more constructively by writing to your legislators and to FDA, using as a template that which you have previously seen from several people—me, Eddie Esparza and others. Or, write a new and different message demanding the right to use Iplex.
From ALSTDI FORUM March 2, 2009
Dear Randy,When reading the following, please bear in mind that I am neither an apologist nor spokesperson for Insmed. Because of my interest in Iplex as a potential treatment for ALS, I have been able to make some communication inroads with Insmed and others related to this issue. I think, from your post quoted just below, and others from you more recently, it is advisable to share some thoughts on the subject.“I feel like a broken record and apologies if this has been covered in another post, but it has now been almost 3 months since the Iplex rally, at which time we were told (those of us who attended) that the Italian data would be released soon. I understood that it was this data that was to be the predicate of further activities and pursuits of obtaining it in the U.S. and maybe elsewhere. Have I missed something? Has this data been released or, if not, any indication as to when this will happen? I suppose "soon" could be anytime, but with each passing day an no news on this front, it looks more and more like either there was misinformation, miscommunication or attempts by one or more parties to mislead and spread false hope. I hope I'm wrong about this, of course.” Questioner ALS-TDI forum 02/09/09In response to the above and other of your posts and emails:1. When I asked for the data on December 22, Insmed stated they have turned over the results to two separate researchers for analysis and comparison to historical placebos. They had, just then, received the data from Italy after “numerous requests.”2. When I asked again on January 19, 2009, they stated both companies were having difficulty analyzing the information received from Italy because of the difficult nature of translating data from many different areas of Italy (20), many different age and progression groups, many different analyses by physicians treating the patients (I think it was 20 physicians) and few patients in the study (only 60 because they excluded all others who weren’t on Iplex for at least a certain time period). In other words, it was never purported to be a clinical test study—but rather “an observation.”3. While the information is unsatisfactory to me, and perhaps to you, it’s also understandable even by those of us who would prefer a clear set of study data and results from a well-conducted double-blind test study.4. Those with ALS don’t always have the luxury of time that a clinical test study requires. In the case of Myotrophin, an inferior drug for the purpose of treating ALS, it took from 1997 to 2009 for the results to be finalized. Even then, the results were unclear and marred by ineffective dosages and contradictory results from three different trials (in US, EU and again US).5. Therefore, those with diseases such as ALS (little-understood, no apparent cure, almost always treated unsuccessfully) are sometimes required to try a medication even without the benefit of prior test data if there is some, or any, reason to feel the treatment has promise. In the case of Iplex, there are several foundations, or at least suggestions, to feel the drug has promise. 6. You already know what those are, but just in case: prior usage by eight US patients in early 2007; continuing usage by now of up to 180 Italian patients paid for by the Italian government (a cost I doubt they take lightly); the results thus far of use by those with Myotonic muscular dystrophy (University of Rochester—a continuing study); scientific foundations previously provided you and others regarding the underlying benefits of IGF-1/IGFbp-3 vs free IGF-1; hope for Iplex when other hope doesn’t exist.7. Insmed has nonetheless stated that, from the little information on hand, there appears to have been a positive effect from at least 50% of those who were in the 60-patient group. That is my recollection of the specific words, and while it may be ever-so-slightly off, the meaning was clear—50% of those with ALS treated with anything is a success. I didn’t think to ask, at the time, if the remaining 50% were “stable, declining, or otherwise.”8. I was asked, at the time of the conversations above, not to publicize the information because it was unconfirmed and to some extent conjecture. Further, Insmed, as a public company, is unable to release such information without qualification because it can be construed as “forward-looking statements”, a legal issue for public companies. I do feel that enough time has passed from the conversations that it can be disseminated.9. If this is, in anyone’s view, “misinformation, miscommunication or attempts by one or more parties to mislead and spread false hope”, then so be it. Steve Byer