HD Lighthouse Contributing Editor's Comment:

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Posted to the HDL: 25 Mar 2006

A Perspective for Treating HD Today

Jim Tretheway

Since my wife Laura was diagnosed with Huntington’s Disease in March of 2002, I’ve had the opportunity to meet many wonderful HD families dealing with this disease. I’ve also had discussions with a number of the very dedicated neurologists who specialize in HD…they are all saints in my book.

We HD families are also fortunate to have many outstanding researchers working in the field. The Huntington’s Disease Society of America and its local chapters provide important family support services plus major funding for research to find an effective treatment or cure. We are kept continually updated on these research efforts by marvelous, family-led organizations like HD Lighthouse, Huntington’s Disease Advocacy Center, and the Hunt-Dis online discussion group. Other organizations like the Hereditary Disease Foundation, High Q, the Cure HD Initiative and the Huntington Study Group are all working to find effective treatments. Huntington’s Disease Drug Works, led by a physician with HD in her family, is running a two year clinical trial using a combination of nutritional supplements.

Like most HD caregivers, I’m committed to help my wife live a full life with this disease by treating the symptoms and attempting to delay the progression of HD for as long as possible. So what should we do when some of us are told by our medical doctors that they can’t offer a cure or even a treatment that has been proven to slow the course of the disease?

Right after diagnosis, Laura and I learned that the 2002 HDSA annual meeting was being held in Columbus, Ohio in a couple of months time. We made reservations and attended the meeting. I can highly recommend the HDSA annual meetings for all HD families. They are well organized and we learned as much from talking with other family members as we did from the official program. Dr. Karl Kieburtz made a presentation at the Columbus meeting which provided the direction we needed. He is a leading HD researcher and clinical neurologist. He described a simple but useful method for evaluating treatment options that your doctor may not suggest. I’d like to share it here for other HD families to consider.

The Kieburtz Clinical Concept

Data

The first comment Dr. Kieburtz made was that HD researchers are generating a lot of new data. The results often are encouraging, but at times they appear contradictory. And the research typically is done in animal models like fruit flies and mice that may react quite differently than humans. The take-home message: Data alone can be very confusing, and should not lead us to try treating ourselves in a manner similar to what was done in a research experiment, at least without additional considerations.

Dr. Kieburtz went on to show the following equation:

Data + Context = Information

And then he explained what he meant.

Context

Context means understanding the limitations of the environment where the data are obtained. Fruit flies, zebra fish and mice are not the same as humans. The research treatments in these animal models may not be measured directly against HD, but against "HD-like" symptoms. In the short span of the experiments, no useful evaluation of side effects may have been possible to do. And what is safe for a mouse that lives for four months may not be safe for a human to take every day for several decades.

Information

Dr. Kieburtz pointed out that data plus the context in which they are obtained may yield information. The information may or may not be useful, depending on the interests of the individual.

Biologists. They want to know the mechanisms by which a drug or other treatment works. Early stage experiments often provide useful information for them.

Medical Doctors. They may not care about the method of action of a treatment, but whether it is safe and effective in patients at a statistically significant level. Usually this information is not obtained until after years long, FDA-approved, clinical trials.

Patients. They care less about statistical significance, but simply whether the treatment has a chance to work for them. It can take years to get the statistically significant data that medical doctors or the FDA demand. HD patients don't have years to wait. The information they need to act may come long before our doctors obtain useful information. This is often the major difference between the perspective of HD patients and their doctors.

Then Dr. Kieburtz provided a second equation:

Information + Cautious Guidance = Informed Action

Cautious Guidance

Taking unproven drugs or high doses of nutritional supplements would not be a wise course of action based only on the information an HD family member obtains from a research program. Our decisions need to be tempered with Cautious Guidance.

Cautious Guidance involves the following:

• Avoid harm. But accept that HD is serious and that this warrants taking some risk with unproven treatments.
• Avoid needless expense. The patient doesn't exist in a vacuum. Unreasonably high expenses for unproven treatments that destroy an individual's or family's finances are not appropriate. The definition of unreasonable will vary depending on each person's financial situation.
• Take advantage of possible benefits. Some possible non-prescription drug treatments for HD, like using the fish oil ingredients EPA and DHA, or using Creatine to try to protect the energy producing part of neurons, are not very costly. They also normally are well-tolerated by the body within a range of doses that have been established over time for other purposes. Certain risks can be monitored by regular blood tests or other means. Your doctor may not recommend these treatments because there may not be definitive clinical evidence of effectiveness. Keep an open mind to trying them under the philosophy of "probably won’t hurt, but might help". At the same time we need to be careful to understand the safe dosage levels and be observant of any side effects.
• Consider alternative choices. Right now there appear to be many different pathways that might slow down or stop the progression of HD. If costs or side effects prevent you using certain treatment options, consider trying others. And since nothing right now looks like a magic bullet against the disease, consider combining more than one treatment option in the hope that one works or that the combination is better than one treatment alone.

  Informed Action

  Informed Action refers to the individual decisions that you take to treat HD. Informed action may involve bringing HD information to your doctor along with details on drug treatment options so that you can discuss these together. It may also involve taking nutritional supplements. In most cases it should involve a well defined, regular program of physical exercise. Informed Action is dependent on your own interest and ability to follow the HD research and understand its context. It's dependent on your own body and how it is affected by treatment options. And it can be dependent on other factors, such as your personal financial situation. We can expect that the Informed Action that one individual takes may vary significantly from that of someone else. Those affected by HD and their advisors must be careful not to become judgmental of the treatment choices of others, nor should they become missionaries for any particular unproven treatment.

  Revisit the decision. New information on HD treatments is coming in all the time. Some treatments that may have been promising at the early stage, may now look less likely after additional research or clinical trials have been done. You may observe side effects in yourself that outweigh the unproven nature of possible benefits.

  Revisit your treatment decisions on a regular basis, but give them a chance to work. And remember that with HD, stabilization of the symptoms or slowing the rate of decline may be considered a success. New research results may suggest changes in your treatment program. In the absence of any side effects demanding immediate attention, a good schedule is to review your treatment program every six months.

  Taking It from Here

  So what does this mean for Laura and me? We continue to look at the research studies and clinical trials as they are being reported, not just in the U.S., but also overseas. The HD Lighthouse (http://www.hdlighthouse.org) is a great place to go if you don’t have access to the research reports directly. The editors who contribute to the HD Lighthouse try to summarize and interpret the results of research that otherwise might be too technical for many of us.

  The HD Lighthouse also summarizes treatment recommendations that individuals with HD can consider taking today, for this generation. Look at them carefully. If your doctor is open to considering unproven treatments, discuss them with her or him. Then take the action that you are comfortable with, even if it is to do nothing at the present. You can revisit the decision at any time.

  Tracked on the Lighthouse:
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