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FDA approves 1st drug for Huntington's disease
"Federal drug regulators Friday approved a medication to treat a major symptom of Huntington's disease, marking the first time since the disorder was first described in a Long Island family 136 years ago that any kind of treatment has been available in the United States.
In Huntington's, a rare, devastating condition, brain cells degenerate because of a genetic miscue easily passed from one generation to the next. The disorder results in jerky, involuntary movements known as chorea.
The drug tetrabenazine controls the chorea, which affects about 90 percent of people with the disease. It was approved under the Food and Drug Administration's orphan products program, which is aimed at developing treatments for conditions affecting fewer than 200,000 people. Huntington's disease affects 30,000 people nationwide.
"I think this is a big deal both in terms of having something to offer patients with Huntington's disease and ... because people have been trying to find something for Huntington's patients for decades," said Dr. Andrew Feigin, a Huntington's researcher at the Feinstein Institute of Medical Research in Manhasset, part of the North Shore-Long Island Jewish Health System.
Feigin, who studied the drug in the clinical trial, added that tetrabenazine "has been around for many years and in other countries, and we knew that it improved chorea."
"It was a matter of a company being interested in it" and ultimately taking steps to get it to market, Feigin said.
Tetrabenazine is produced by Prestwick Pharmaceuticals in Washington, D.C.
Doctors have been aware of tetrabenazine since the late 1950s and have used it to treat other movement disorders. Regulators in Europe and Canada approved tetrabenazine in the 1990s for Huntington's chorea.
In the U.S., advocates for patients say, major pharmaceutical companies show little interest in developing treatments for orphan diseases because they do not have millions of patients who can drive profits.
"It's just wonderful to have a drug approved by the FDA," said Dr. Fred Marshall of the University of Rochester, who led the clinical trials. "It's also important symbolically for the patients and their families. They've suffered for so long. The big victory here is putting the drug on the map."
He emphasized that tetrabenazine is not perfect, and too high a dose can lead to depression and suicidal thoughts and exacerbate chorea.
Huntington's disease was first described in 1872 by Dr. George Huntington, of Columbia University, who studied an East Hampton family affected by the disease over several generations." "Federal drug regulators Friday approved a medication to treat a major symptom of Huntington's disease, marking the first ti... more -
Parkinson's, Lou Gehrig's and Huntington's diseases: great news in ...
Creatine is the white powder athletes use in juice, water or capsules, from the health stores, for endurance.
In scientific studies conducted at leading universities, these Creatine based compounds have been shown to significantly reduce the specific symptoms and/or to significantly delay the progression of these diseases. The company presently has compounds in Phase II or III clinical trials for the treatment of ALS, Huntington's, and Parkinson's disease.
A novel compound called C2-8 could prove a valuable lead in the drug discovery process. Researchers (from left) Jonathan Fox, Vanita Chopra, Aleksey Kazantsev, and Steven Hersch
Steven Hersch, M.D., Ph.D.
Harvard Medical School and Massachusetts General Hospital
Alliances:
http://www.avicenagroup.com/about_us/research_alliances...
Participating clinicians:
http://www.avicenagroup.com/about_us/participating_clin... Creatine is the white powder athletes use in juice, water or capsules, from the health stores, for endurance. ... more -
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"A woman who suffers from a disease which causes balance problems was thrown into a cell - because police mistakenly thought she was drunk." "A woman who suffers from a disease which causes balance problems was thrown into a cell - because police mistakenly thought sh... more
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Huntington's Disease Phase 3 trials successful...where's the news covera...
"We are very enthusiastic about the study results of HD-02 to date and excited to be advancing it into a Phase III trial", stated lead investigator Dr. Steven Hersch. "This Phase III study will evaluate whether HD-02 can slow the progression of Huntington's disease, and pending positive results, may provide a much needed treatment option to Huntington's patients."
Also with this are treatments for Parkinson's and ALS. "We are very enthusiastic about the study results of HD-02 to date and excited to be advancing it into a Phase III trial", s... more -
Hats Off to Dr. Hersch...More Evidence Creatine is an Effective Treatment for Hunt...
Creatine is a harmless nutritional supplement used by athletes.
It has just been found that it STOPS the progression of "untreatable and always fatal" Huntington's Disease..
Yes you heard right!
First; this study answered very important questions regarding creatine uptake into brain, and documented pharmacologic dosing levels. This is important because brain penetration is absolutely necessary for any (successful) Huntington�s drug. It can be argued that this type of drug/brain penetration testing should be required of any drug candidate prior to entering expensive phase III trials in Huntingtons.
Second; the authors used MRS, a radiologic technique reviewed previously at Lighthouse, ( NAA - biomarker for HD?) to measure creatine brain levels. MRS technique may prove useful as a sensitive measure of disease progression in the future. And sensitive measures of progression will shorten the time needed for clinical trials.
Third, they reported increased levels of 8OH2dG in the blood of Huntingtons people that was decreased with creatine use. Earlier reports had described elevation of this chemical in blood, urine and brain tissue of Huntington's mice. This chemical is a measure of DNA oxidative injury, and is elevated in other neurodegenerative diseases as well. The authors showed that creatine decreased the level of this marker in Huntingtons people, suggesting that creatine also decreased oxidative injury. They further speculate that 8OH2dG is a promising biomarker for studying drug response in HD.
This is a scientifically elegant study with many practical (and promising) results for HD people. This is the kind of trial we�d like to see more of.
-- LaVonne Veatch Goodman, M.D.
Creatine in Huntington disease is safe, tolerable, bioavailable in brain and reduces serum 8OH2'dG.
Authors: S. M. Hersch, MD, PhD, S. Gevorkian, MSc, K. Marder, MD, MPH, C. Moskowitz, MS, A. Feigin, MD, M. Cox, MS, RN, P. Como, PhD, C. Zimmerman, RN, M. Lin, MD, L. Zhang, MD, A. M. Ulug, PhD, M. F. Beal, MD, W. Matson, PhD, M. Bogdanov, E. Ebbel, A. Zaleta, BA, Y. Kaneko, BA, B. Jenkins, PhD, N. Hevelone, MPH, H. Zhang, MS, H. Yu, MPH, D. Schoenfeld, PhD, R. Ferrante, PhD, MSc and H. D. Rosas, MD, MS Creatine is a harmless nutritional supplement used by athletes. ... more
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Huntington's Disease
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Huntington's Disease
