I've read with great interest the recent passionate pleas from members ofour HD community for everyone to get involved with clinical trials. Seemslike lists of potential participants would have been prepared years ago inanticipation of what was going to be needed in the future by theorganization who had the most knowledge about what was coming down thepipeline and what new needs and resources increasing clinical trials wouldhave in the future- more leadership and preparation than putting up thepassive http://www.hdtrials.org/ website (which still hasn't been widely advertised)and expecting everyone to stumble on it and register! Had we had moreinspiration and leadership then, we would already be better prepared TODAYto actively enroll everyone in appropriate clinical research studies!Encouraging everyone to participate today is good- it's only about two tothree years too late in my opinion! I developed a forward-looking plan several years ago that recommended thatwe immediately begin focusing on identifying, "befriending," helping andrecruiting the largest pool of potential clinical trial participantsavailable- those who are at-risk! I was scoffed at and ridiculed.Visualize in your mind a picture of a huge iceberg: those already diagnosedwith HD represent the small tip of the iceberg (that is visible to the nakedeye)! However, the group represented by the largest part of the iceberg-the part underwater, is the very large number of individuals who areat-risk! They represent, by far, the biggest or "bulk" of the iceberg! The current Center's of Excellence (COE's) are okay but they are too littletoo late and are geared primarily for those who are already symptomatic. Myplan was to establish COE's who would also be caring for those who wereat-risk, before they get fired, divorced, arrested for embezzling, etc. Ithink you see my point. It doesn't take much insight to realize that thevast majority of our "potential" research participants for current as wellas future clinical trials are going to come from this untapped naturalresource called the "at-risk group" whom we still know relatively nothingabout! I know that here in Arizona, I know most of those who are in thisgroup. I also know who is at-risk and showing symptoms, gene positive butnot symptomatic, gene positive and a little symptomatic, etc., etc. I hopeyou can see my point. Who is putting on the mantel to become the inspiredleaders we need today to organize such an effort? A few groups and organization around the country know a lot about the sheep in their folds,but the majority still don't!
Thursday, March 19, 2009
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I couldn't agree with you more Heather; thank you for highlighting my biggest pet peeve with the HD establishment. Can you please post a link to your forward-looking plan that you referenced -- I would like to take a look. But I feel just as strongly in the HD community’s lack of assertiveness in taking control of their future. [I feel a rant coming on] Motivating the "at-risk group" lies at the heart of any hope for a treatment, but we can't expect that treatment to kick down our door and land in our lap, nor can we expect that treatment to come in a handy little pill that we can take with a smoothie. More than likely, any truly effective treatment for our generation is going to have to come from aggressive therapies that will require direct insertion into the effected parts of the brain. Pills are systemic and can't penetrate deep into the HD-effected parts of the brain, which means it will have to be delivered manually. The good news is that HD is very consistent and strikes the same part of the brain (unlike Alz and PD), which makes these aggressive treatments much safer and more effective. The bad news is that, while researches are actively (and excitedly) addressing these more direct scenarios, they have no access to people willing to undergo these studies. This is a place where the "tip-of-the-iceberg group" can also be helpful. In the meantime, every person that is at-risk, gene-positive, and/or gene-negative MUST MUST MUST MUST register at HDTRIALS.org.
ReplyDeleteBottom line: no volunteers = no treatments or therapies.
And while we’re on the topic: please people, get tested. It’s not required for hdtrials, but it would help tremendously. It is critical that researchers know where the populations are so that they can plan their trials and provide services. Hiding in the shadows is so 20th century. Cancer is familial, and yet, I've never heard of a person not wanting to find out if they have a tumor (so much so that the “at risk” get tested annually), and allow themselves to be blasted with radiation even if there’s only a chance of a tumor.
Scientists cannot solve this for us; they can only solve this with us.