Wednesday, February 27, 2013

How do we prove to the FDA that MSF Works?

            This was a pretty crucial question to answer. Without having a good idea of how to show quantitative memory improvement, an Alzheimer's treatment would be dead in the water, and while Dr. Moss had designed numerous experiments as well as run a clinical trial for efficacy, all of these were with small samples. The testing for memory improvement needed to be streamlined so that we could easily collect data on as many as two hundred subjects through quick and easy tasks that wouldn't put too much strain on either the Alzheimer's patient or their caretaker. One of the big concerns when running large scale trials is that if your tests are too onerous, you'll have a large dropout rate and may end up with an insufficiently representative sample to satisfy a regulatory body like the FDA.
            With this in mind we contacted Dr.Edward Awh and Dr. Edward Vogel at the University of Oregon. Both Eds have existing labs that study visual working memory and attention, two major faculties damaged by the progression of Alzheimer's disease. They accepted the task of putting together FDA compliant tasks that could demonstrate a change in a person's basic physiological ability to remember things, even after repeated exposure to the same procedure (ie, they can account for the learning effect).

Excerpted from an email to me by Dr.  Vogel:
The plan for assessing the efficacy of MSF by testing memory functioning in Alzheimer's patients.

Our general plan for doing this involves two basic components:
1. Clinician-administered tests (once every 3-4 weeks during the study) which will include the ADAS-Cog (a battery of cognitive tests for alzheimer's that has been the standard measure in nearly all clinical trials of cholinesterase inhibitors); and one or two questionnaires regarding Daily living activities and function to be filled out by the patient and caregiver. These tests should take approximately 45mins to 1 hr to complete.

2. Remotely-administered tests via computer (once per week during the study) of memory and cognition which will include assessment of picture memory, paired-association with words, delayed free recall of words, and the COWAT (Controlled Oral Word Association Test) which measures the individual's ability to fluently generate words of a particular type (e.g., say all the words you can think of that start with the letter "F"). Together, these tests will take approximately 20mins to complete.     

            He ended the email suggesting that they should be finished with the test development process by the end of March, putting us well on schedule to proceed as soon as the long term, FDA compliant toxicity studies are completed.

I'll admit I'm kind of curious to take one of these memory tests myself. I'll probably keep the results confidential though.

Friday, February 8, 2013

The Story So Far

There are about thirty years of backstory to this project which you should read about either in Dr. Moss' book, or for a quick overview, in the excerpts section on the brain-tools website.  The part where we (braintools) came into the picture, however, was in October of 2012. Dr. Moss and Dr. Summerton had already agreed to collaborate on finally turning the drug Dr. Moss had developed and tested into a workable treatment for memory loss in Alzheimer's patients. The trouble that's faced Dr. Moss over so many years was the lack of patent protection afforded to the MSF drug, making it cost-prohibitive for any big pharmaceutical company to carry through a 100 million dollar phase three clinical trial, despite its unprecedented efficacy in restoring the memories of Alzheimer's patients. Dr. Summerton had discovered a long forgotten congressional bill called the Orphan Drug act, passed in 1983, which was developed for the specific purpose of getting highly useful, but non profitable drugs on the market. The bill was originally developed to cover drugs that treated life threatening illnesses that affected a very small patient base like Muscular Dystrophy, but since has been expanded to cover any drug that can provide great social benefit but not enough profit to cover the costs of clinical trials. The Orphan Drug act mandates that the FDA collaborate with drug development groups to help minimize the cost of clinical trials and streamline the process, while simultaneously offering a seven year guarantee of protected market status to help recoup losses. For us this ends up being four years of market protection as phase two and three clinical trials are expected to take about three years.

Because major pharmaceutical companies have passed on the drug, we at brain-tools may be the last hope to get MSF (Methane SulfonylFluoride) into the market. Our plan is discussed in detail here, but the key element is the reliance on crowd funding instead of a traditional equity investment. With funds raised through contributions we will not be accruing large debt to venture capitalists. Between this and the reduced costs of clinical trials through the Orphan Drug Act we can offer a contractual guarantee that we will keep prices of MSF affordable to everyone, even those without health insurance or on social security. This is a guarantee that other avenues of drug development cannot offer. Perhaps an even bigger advantage of this method is that it will open the path for other companies to follow a crowd funding/Orphan Drug act model, allowing other potentially lifesaving drugs that have fallen through the cracks to make it into the market.

It's an exciting prospect, to say the least.