I receive several comments a week on my articles as well as many email questions. This morning I responded to an inquiry ... “I wasn't going to ask this as I can instantly give a bucket load of reasons why it can't be answered. But, I'm interested to know if you've ever wondered yourself. I think the question stems back to a piece you wrote on there being no money in curing rare diseases but it's something I've pondered since. How much would it cost to find a cure?”
Before responding, I had to consider everything that I know and heard about Kennedy’s Disease research. I do not believe anyone could ever put a realistic cost on financing research to find a treatment or cure. The issue, in my opinion, is that not enough funding is made available for Kennedy’s Disease research because it is a rare disorder that does not garner much public attention. Major pharmaceutical companies do not consider this type research a potential "cash cow" so they sit back and wait for some university to discover the treatment or cure and then perhaps buy the patent. When the potential market (patient base) for a treatment or cure is roughly 1-in-40,000 across the world, it must be difficult for any corporation to justify the research expense unless there is some major return on investment afterwards.
If you read my blog last week, you saw the Dr. Taylor (and several others) published a paper on some research that looks very promising. Paul will be our guest on the October 16th KDA chat room and will amplify and answer questions about this research. In my opinion, smaller companies, for example AndroScience, have been looking for funding to continue their initial research on a potential treatment for Kennedy’s Disease. ASC-J9 is something that looks promising, but I am sure AndroScience has to focus their attention on the acne cure and only dabble in other potential benefits of this compound.
In earlier articles I mentioned the time it takes to move a potential treatment or cure from the test tube, through fly and mouse models, to other testing, and eventually to human trials ... IF the drug continues to show promise and is safe. I also understand that for those of us living with this mutation that remaining patient is very difficult as you see yourself, or a loved one, slowly decline in capabilities and health.
Since the KDA is an all-volunteer organization and our donation base is rather small compared to the major non-profits (e.g., MDA, Heart Association, Diabetes Foundation, United Way, etc.), we try to encourage younger researchers to focus on Kennedy’s Disease research by providing “seed money” in hopes these scientists can continue their research while having time to pursue larger grant opportunities. If we ever grew enough to be able to offer larger grants, we would probably focus our funding on projects like ASC-J9, IGF-1 for muscles, and now this latest research by Paul Taylor. Until that end, we are a small fish in a very large pond and “hope” is mostly what we have to offer.