At Your Local Pharmacy The molecule ursodeoxycholic acid (UDCA) effectively treats HD in transgenic mice with the human HD gene. The generic drug name for UDCA is ursodiol. Brand names for UDCA include; in the US, Actigall, Urso; in the UK; Destolit, Urdox, Ursofalk, Ursogal; in South Africa, Ursotan.

The bear has a rich place in astronomy, mythology and in our hearts but not in our medicine.

Recently the constellation Ursa Major, the Big Bear, was in the news. One of the stars in Ursa Major may have a hospitable planet. It is easy to see this as a good omen for those concerned about HD. It takes delusion to suggest that bears anywhere are being slaughtered so HD can be treated. In the US we love our bears. Bears are protected by law that provides large fines and prison time for any one who would harm a bear for a treatment of any kind.

UDCA For Huntington's Disease

Clifford J. Steer, M.D. Professor of Medicine and Genetics, Cell Biology and Development M.D. Degree: University of Minnesota Fellowship Training: NIH Residency Training: University of Minnesota Specialty: Hepatology Research Interests: Liver regeneration, apoptosis, gene therapy, gene repair. A PubMed search returns 100 publication for author Steer CJ.

In a telephone conversation with this editor, Professor Steer provided vital information to those considering the proactive treatment of HD with ursodiol.

About five years ago Professor Steer was aware of the use of UDCA to treat certain types of liver diseases. He considered that UDCA might prevent liver cell death. His lab work showed that UDCA did indeed protect liver cells and much more. In culture, UDCA protected every type of cell tested from a varity of different agents that produce cell death by apoptosis. The agent, 3-nitropropionic acid (3NPA), was used to challenge neuronal cells and causes a condition in mammals similar to HD. In the world of good science, testing 3NPA impaired rats was a logical extension to the finding of Steer's lab work.

Again the Steer and his valued collaborators struck pay dirt. The UDCA treated 3NPA rat models of HD did spectacularly better than untreated rats. The 3NPA HD model was a useful test system. Treatments that work on this rat model, could conceivably work in humans. The path to human treatment must leave no rat and/or mouse unturned. Now, building on a long series of lab successes, the next step was to test two dozen of Bates' transgenic mice that have the human mutant HD gene.

The lab experiments found that the treatment worked for the mice carrying the human gene. Now there is solid hope for the effective UDCA treatment of many neurological conditions. UDCA is almost without side effects, our bodies even produce it in small amounts. It can be prescribed off label by any medical doctor. Can science get any better?

Ethics

We have not had the necessary series of clinical trial that would prove the efficacy and safety of UDCA for the treatment of HD. No one will argue that these trials are not necessary. These trials are another extension to the successes of Steer's lab. Drug trials are expensive of both time and money.

What about the thousands of folks now carrying the mutated gene? Untreated the progression of HD is relentless. HD, like a thief in the night, takes a little of a person each day. When nothing else is left HD takes the last heart beat.

UDCA is a very safe, available drug. The promise that it will treat HD in humans is based on solid science. It is hopeful that UDCA will stop the HD thief in the night. It is simply not ethical to ask folks to suffer the progression on HD while waiting for drug trials.

The Bet

Professional Help

I appreciate that there are people who would like to support my laboratory and the projects. They are certainly welcomed to contact me directly. My colleagues and I also have ongoing projects involving acute stroke as well as ALS."

Clifford J. Steer, M.D.
Professor of Medicine and
Genetics, Cell Biology, and Development
Director, Molecular Gastroenterology Program
University of Minnesota Medical School
Mayo Mail Code 36
Mayo Building, Room A536
420 Delaware Street S.E.
Minneapolis, MN 55455
Tel: 612-624-6648; Fax: 612-625-5620
E-mail: steer001@tc.umn.edu

Work To Be Done

The current best guess dose for UDCA is 50 mg/kg/day. Dr. Steer wants to do a more definitive study that will precisely measure UDCA spinal fluid levels. Despite a rich UDCA literature, this experiment has not been done. This will aid humans in the proactive UDCA treatment and ease mice experiments planned for Steer's Lab.

At some future date a few volunteers may be needed for phase I HD UDCA trials.

Funding

Source:HDL, Ursodiol Treats Huntington's Disease -- 13-Sep-2002 00:00 GMT

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