Hey terry, you're just going to need to retire this angie pseudonym once and for all. No one bought it. I dare you to bring it out again.
Even the horse doc knows this...thanks rehd, who is now recommending a buy on INSM.
Quote from rehd concerning the case of Angie Cacchillo:
1. signed and informed consent paper to enroll in the 6 month long Phase IIb MMD trial of 2007-2009. This study was coordinated by Dr. Richard Moxley of Rochester U based upon a DM1 mutation diagnosis.
2. Informed consent delineates a precise dose, duration and parameters for treatment. Mrs. Cacchillo went through that term of treatment and improved according to the primary endpoint (walking further and of longer duration). Unfortunately most of the other patients in the trial did not improve according to that primary endpoint.
3. If most patients that enrolled had improved I am sure that INSM would have applied and been granted an "extension" of the Phase IIb trial to be part of a longer than 6 month Phase III clinical trial. If you notice on ClinicalTrialsDOTgov, there is an extension protocol for the CF/Pa patients that complete 6 rounds of treatment with Arikace. But this protocol is not currently activated and thus constitutes a "signal" as to the future of Arikace in being used for longer term for CF therapy. Many are all awating that protocol being activated because it will be a sign that the primary and secondary endpoints of Arikace have (probably) been met.
4. The contents and boundaries of an informed consent statement are internationally agreed upon and can be found on any website you wish (FDA, EMEA and even WHO). Mrs. C's lawsuit was dismissed originally because a clinical trial approved by the FDA (i.e., the MMD trial) cannot be altered from the parameters originally agreed upon with the FDA. It cannot automatically be transformed into and EAP, compassionate treatment protocol for ALS because one patient got more benefit from treatment than another.
Sorry for Mrs. C, but the protocol was specific and she signed. That included being part of discontinuing the protocol if most of the patients did not benefit and the protocol ends because of that outcome. People have to remember that 10% of the patients treated with Laetrile had their tumors shrink. 90% had no benefit.
This will not be a factor in INSM's near future. But it still should have been a more focused study on Duchenne's or adult hypotonia without neurodegeneration. Then Mrs. C would still be on treatment.