IPLEX, And the IND Process

Dear friends,

A very dear and close friend, Kathy Thompson a.k.a. fighting mom (Team IPLEX Capt.), posted this excellent retort on ALSTDI last night. This says it all...,


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On behalf of PALS that are genuinely interest in Iplex or the IND process - I am going to try to clarify this issue and present THE FACTS - but I do not have the time or energy to 'debate' this issue. I am doing this because I remember what it was like to first join this forum - searching for hope and searching for answers to questions. There are too many posts regarding Iplex - where assumptions are stated as if they were facts - and this is not fair. So I will drop in this post and answer questions of sincerely interested PALS.

Briefly - The expanded access program or compassionate use is a mechanism set up by Congress to help allow patients with serious or life threatening diseases access medications that may not be available through any other mechanism and when there are not alternative treatment options. There are different types of IND's but the IND's we filed for Iplex are Emergency Single Patient IND and are prepared by each patient's doctor - with that doctor agreeing that he/she believes in the possiblity of the drug and will oversee the patient's 'safety' and that the patient does not have an alternative treatment option. Then an IRB is required and this 'medical institution' is required to work with or over the doctor to ensure the patient's safety while taking the drug. You can 'google' the information about this process if you would like to do so.

Usually the Single Patient Ind's are for drugs that are not yet FDA approved - but still in a phase of clinical trial. If a drug is FDA approved - in almost all cases - your doctor can write the prescription and you can get the drug 'off-label' for a disease or condition that the drug is not FDA approved for (example - Myotrophin/Increlex is FDA approved for SGS in children - but many ALS patients take it off-label at higher doses than in the 2008 ALS trial - in hopes of slowing ALS).

Iplex is FDA approved !- and under 'normal' circumstances ALS patients should be able to get Iplex 'off-label' with a simple prescription. However - due to the patent settlement Iplex's marketing status is still discontinued (it can not be sold to children for the indication it was FDA approved for). The Iplex marketing status is based on a CORPORATE issue. Usually, the FDA marketing status is discontinued because of safety issues (Vioxx) or the drug has gone generic so there is an alternative drug. It is because of the 'discontinued marketing status' that PALS must file a Single Patient IND and have an IRB (institutional review board) to oversee the safety and administration of the medication. These guidelines were established because usually 'discontinued marketing status = safety question or alternative options. Iplex is safe and there is nothing else like it (per FDA description) but 'discontinued is discontinued' thus the IND/IRB requirement.

The FDA had no dispute with the method or content of the IND's that were filed for Iplex. We believe the person who put our IND's on 'clinical hold' made a mistake and held ALS patients to the conditions of IND approval for 'serious disease/conditions' vs. their 'life threatening disease' conditions and guidelines. By Congressional and FDA's own guidelines - ALS is an immediately life threatening disease (see below).

So -- what are patients that filed IND's for Iplex doing to resolve this matter. In early February, myself and another patient each drove 4 hours to meet with an FDA deputy director and an FDA patient service person. We were treated politely and we were very polite back (hard to do when someone is holding your child's life in their hands)- but left the meeting with 'they would look into it' and we have not heard back from the persons in the meeting (and yes - we did send follow-up emails).

I learned from an FDA person not involved in our case - that the proper response to a clinical hold is to file an 'appeal'. They recommended hiring an attorney familiar with the FDA. So - I spoke with 4 excellent Washington DC FDA attornies and they agreed that our clinical hold was incorrect and that we needed an attorney to file our appeal. So...I selected an attorney to handle my appeal. He will also handle the appeal of any other PALS who would like to use his services (or he will work with anyone else's appeal attorney). Our appeal was filed with the FDa in mid-February and I think our appeal is excellent. The FDA has 30 days to respond. If the FDa does not help us - then we are prepared to go to court.

When I chose the name 'fighting mom' I just meant that in terms of trying to fight for the health, happiness and safety of my son. I certainly did not want to fight the FDA or anyone else. I simply want my son to have the right to try Iplex and pray that it would help him. I also want this for any other PALS that would like to try it. There is NO other alternative for my son. He does not qualify for any clinical trials as his length of disease and disease progression excludes him from current trials (or we would be there!). I am very very sad to say - that since filing the 'emergency' IND for Iplex on Dec. 16 - my son's disease progression has drastically accelerated - but we have to try - that is just who we are.

I hope this helps bring you up to speed and clarifies the situation. We know many of you do not believe in Iplex and that is okay - but at least wish us well in this journey. We will never know until we try....

FDA IND Guidelines
https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfCFR/CFRSearch.cfm?fr=3 12.34
excerpt:
(2) Serious disease. For a drug intended to treat a serious disease, the Commissioner may deny a request for treatment use under a treatment protocol or treatment IND if there is insufficient evidence of safety and effectiveness to support such use.

(3) Immediately life-threatening disease. (i) For a drug intended to treat an immediately life-threatening disease, the Commissioner may deny a request for treatment use of an investigational drug under a treatment protocol or treatment IND if the available scientific evidence, taken as a whole, fails to provide a reasonable basis for concluding that the drug:

(A) May be effective for its intended use in its intended patient population; or

(B) Would not expose the patients to whom the drug is to be administered to an unreasonable and significant additional risk of illness or injury.

(ii) For the purpose of this section, an "immediately life-threatening" disease means a stage of a disease in which there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment.
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