Treatment for HD

The Huntington's Scene In New Zealand

Site Maintained by
Graham Taylor

Site Resources available from the Homepage
NZ HD Diagnosis, Care & Support Key Site Resources Research Newsletters Books	About Huntingtons Disease HD Resources About the Associations Youth

Treatments for Huntingtons Disease:

Near or Distant Future?

The following excerpts are taken from a clinical research panel at the 1998 HDSA Convention, chaired by Ira Shoulson, M.D., Louis C. Lasagna Professor of Experi-mental Therapeutics at the University of Rochester and Chair, Huntington Study Group Executive Committee.

DR. SHOULSON:

"There are several levels in which therapy is developed and applied for human conditions, and Huntington’s Disease is no exception. What we want to do is move from what we call symptom-atic treatments to treating the disease at a more fundamental level, both at the level of the brain and perhaps even at the level of gene expression.

"Obviously we would like to develop therapies that are active... either just when the illness is being detected or, even better, just prior to the illness being detected, especially in individuals who are known to carry the Huntington’s Disease gene but who have not yet shown features of HD.

"[This] is the concept of neuroprotective therapies ...aimed at either slowing the progression of Huntington’s Disease once the disease has ap-peared, or better yet, postponing or preventing onset in those who carry the gene."

CARE-HD STUDY

"CARE-HD is a study that was developed by the Huntington Study Group under the leadership of Karl Kieburtz and Walter Koroshetz. CARE-HD stands for Co-enzyme Q10 And Remacemide Evalua-tion in Huntington’s Disease.

"Co-enzyme Q10 is a naturally occurring sub-stance, very important in the mitochondria — the little energy house within all cells that is impor-tant in terms of normal energy transmission. A variety of evidence suggests that this particular compound may be helpful in terms of rectifying the energy derangement and perhaps improving Huntington’s Disease in the longer term, over a period of a few years.

"The other compound, Remacemide, is a drug that has actually been under development for many years, most recently by Astra Pharmaceuti-cals. This is a drug that blocks glutamate recep-tors and may potentially have some value in the long term in patients with Huntington’s Disease, again in terms of slowing the progression of illness.

"The CARE-HD study has what we call a two-by-two factorial design, whereby research subjects are randomly assigned to one of four treatment groups: either active Remacemide alone, active Co-enzyme Q 1 0 alone, both active compounds, or double placebo. In all instances there are adequate controls so that at the end of the study we’ll be able to determine with a high degree of confidence whether or not there has been any benefit. For each person who consents to partici-pate, the study is going to last at least two and a half years. This is really the first large-scale study aimed at slowing the progression of earlyHuntington’s Disease in a controlled fashion.

"When we began enrolling patients last July, it was expected that enrolment would take a little over a year. [In fact], half the patients were en-rolled in the first three months and it took the remaining seven or eight months to enrol the rest (not because there is a lack of patients, but we just hadn’t developed adequate parity between all the participating sites in the Huntington Study Group). This enrolment period has been a phe-nomenal success and we are really pleased with the response (enrolment of 347 subjects was completed in June 1998).

RILUZOLE

"Another compound that we hope to be studying soon is called Riluzole. It’s a compound that is actually already available for the treatment of amyotrophic lateral sclerosis - ALS or Lou Gehrig’s Disease. We have an application pend-ing to look at the safety and tolerability of this compound in Huntington’s Disease that is going to be reviewed by the Food and Drug Administra-tion this September. Dr. Fred Marshall at the University of Rochester is the principal investiga-tor.

"I should tell you that Riluzole is a drug that has very modest effects in ALS, [but] we are interested in the drug in two ways. One, it may have some utility in Huntington’s Disease. Also, there is a mouse model for ALS ... and the drug has shown some benefit in that model. So we might use this opportunity to find out how you move from drugs in an animal model to human research.

" [Riluzole] is not used by all patients with ALS... and we would certainly not recommend it in patients with Huntington’s Disease until we are convinced that it’s sufficiently safe and tolerable and [there is] some indication that it might be effective.

HUNTINGTON STUDY GROUP

"I just want to conclude this part by mentioning the Huntington Study Group. This is an organiza-tion that is about six years old that was formed by clinical investigators and research scientists in an effort to improve treatments and strategies for patients with Huntington’s Disease in a more collective, cooperative fashion. It’s an interna-tional group but the bulk of the sites are in the U.S. and Canada and we are very pleased with the support that we have from our sites. We’re very grateful to HDSA in particular, which has really been our major supporter. "I should tell you that the Huntington Study Group met in tandem with the HDSA Coalition for the Cure [last December]. This year at our annual meeting in November, not only will we be meeting in tandem but we are going to have a joint meeting so that the basic scientists and the clinical researchers will have an even greater opportunity to share with each other strategies and new information that’s not been published, and perhaps to even develop some joint ven-tures." Acknowledgement: "Toward A Cure", September 1998 Huntington Disease Society Of America (via the Internet).