The Dilemma of Proactive

One of Jerry Lampson’s final efforts at Lighthouse was to create a listing of agents of potential benefit for HD people. Because he knew that many HD people were trying these agents without guidance (or acknowledgement) from national HD organizations, he did what he could to compile a list with the information available to him. What follows is a similar effort that I hope he would find satisfactory.

Though Huntington’s scientists have made remarkable progress in molecular biology and have identified dozens of agents that help in models of Huntington’s; and a few have now made it through pilot human clinical trials: None have made it through definitive human clinical trials. As such, our HD national organizations have not and can not make any “definitive” recommendation other than the centuries old “nothing” for treatment in our disease.

So that leaves this generation of HD between “the rock and the hard place”. The choices are: (1) to do nothing and wait, with results that are all too predictable, or (2) try those agents (that are available either over the counter or by prescription) that have been reported to show benefit either in models of Huntington’s disease or in small (pilot) trials of Huntington’s people. Of course the best alternative would be to take these agents as part of a clinical trial effort, but if no such trials are offered, HD people aren’t sure where to go with their questions.

Presented below in table form are a few of the agents that fit into “potential benefit” category. Be cautioned that potential benefit doesn’t mean sure benefit, but also know that HD people have the right to decide that “potential” benefit is better than “nothing” the only “sure” alternative. But if you decide to try one or more of these (or other) agent(s); be responsible; educate yourself and inform and educate your healthcare provider.

First a few disclaimers: (1) This list doesn’t contain all reported agents.(2) I’m biased and will likely include my favorites. (3) I’ll give rationale and supporting (but not all) references for each agent chosen (click on the agent name). (4) If you take an agent make sure you read the side effect portion. Know what the risks are too. (5) “Cocktails” of agents work best in mouse models, but DON’T USE THEM ALL. Mixing large numbers of these agents may (and likely will) be harmful. (6) I am not including agents that have “risky” side effect profiles, even if FDA approved. This includes drugs that would usually be used by cancer or transplant specialty doctors.

Over time I will update the list of agents of potential benefit and I’ll add a list of agents of potential harm.

Lastly, this list is not meant as HD Lighthouse or my personal recommendation for definitive treatment; because we unfortunately know there is none for this disease. This list is meant to catalogue some of the information that is available from scientific publications and put it in a user-friendly form so that HD people (and their healthcare providers) can access it. While acknowledging that the scientific information for human therapy of HD is still in its infancy; informed decisions are better decisions.

Source: LaVonne Veatch Goodman, M.D.

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