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huntington [작성일 : 2016-07-13 16:07:42 ]   
제목 헌팅턴병 증상완화 신약관련 글 올립니다.

기존 tetrabenazine에서 개선 된 deutetrabenazine 에 대한 내용으로,

부작용이 다소 감소되었다고 하나 기존 테트라베나진과 비교 향상된 것인지는 좀 더 두고봐야할 것 같습니다. 테트라베나진과 성분이 거의 유사하여 기존 부작용들이 해결되지는 않았을 것 같은데 앞으로 좀더 개서되는 약들이 나오길 희망합니다.


아래 원문 첨부합니다.


New Drug Eases Huntington's Disease Symptoms - WebMd 7-7-2016

 

 

New Drug Eases Huntington's Disease Symptoms

 

http://www.webmd.com/brain/news/20160707/new-drug-eases-huntingtons-disease-

symptoms-study

 

Experimental med seems to have fewer side effects than current treatment,

researcher says

 

WebMD News from HealthDay

 

By Steven Reinberg HealthDay Reporter

 

THURSDAY, July 7, 2016 (HealthDay News) - An experimental drug may help

control the involuntary, sudden muscle movements associated with

Huntington's disease, with fewer side effects, according to the results of a

small trial.  "Deutetrabenazine is not yet FDA-approved, but assuming it

becomes available, practicing clinicians may have another choice for their

individual patients," said lead researcher Dr. Samuel Frank. He is a

neurologist and instructor at Harvard Medical School in Boston.

 

The involuntary movements associated with Huntington's disease are called

chorea. Huntington's disease is a fatal genetic disorder. It causes the

progressive destruction of nerve cells in the brain. Symptoms vary from

person to person. People who have Huntington's lose their physical and

mental abilities over 10 to 25 years. The disease has no cure, according to

the Huntington's Disease Society of America (HDSA). Every child with a

parent with Huntington's disease has a 50 percent chance of carrying the

faulty gene. Approximately 30,000 Americans have symptomatic Huntington's

disease. More than 200,000 are at risk of inheriting the disease, the HDSA

says.

 

The choice of drugs to treat chorea should be based on safety,

effictiveness, cost to patients and the best chance of compliance, Frank

said.  "The opportunity to possibly improve quality of life should be part

of the decision-making process as well," he said. "If approved by the FDA,

deutetrabenazine may provide another treatment option, making Huntington's

disease an increasingly treatable condition."

 

Funding for the trial was provided by the maker of the drug, Auspex

Pharmaceuticals, a subsidiary of Teva Pharmaceutical Industries, Ltd.

 

Currently, tetrabenazine

<http://www.webmd.com/drugs/drug-151522-tetrabenazine+oral.aspx>  (Xenazine

<http://www.webmd.com/drugs/drug-151569-xenazine+oral.aspx> ) is the only

drug approved by the U.S. Food and Drug Administration for treating chorea

in Huntington's disease, the study authors said. Although the drug is

effective, tetrabenazine often has to be taken three times a day and can

cause side effects, such as sedation, fatigue, anxiety or nausea, the

researchers said.

 

For the new study, Frank and his colleagues randomly assigned 90 patients

with Huntington's disease and chorea to receive either deutetrabenazine or a

placebo.

 

Over eight weeks, the dose of deutetrabenazine was increased to a level at

which it was most effective. That dose was maintained over four weeks, the

study authors said.

 

"We found that deutetrabenazine reduces chorea, the hallmark movement

disorder in Huntington disease," Frank said. "There was also an overall

improvement in participants' condition and improvement in a quality-of-life

measure. There was, however, no improvement in balance."  The rate of side

effects was similar for the drug and a placebo, which included depression,

anxiety and akathisia (a movement disorder), the researchers found.

 

The study findings were published July 5 in the Journal of the American

Medical Association. Frank said larger trials are underway to assess the

long-term safety and effectiveness of deutetrabenazine.

 

One neurologist said he would like to have seen a head-to-head comparison

between deutetrabenazine and tetrabenazine.  "I wish they had included a

tetrabenazine arm and a placebo arm," said Dr. Michael Geschwind. A

professor of neurology at the University of California, San Francisco, he

co-wrote an editorial accompanying the study results.

 

Such a trial is needed to see which drug is superior, Geschwind said. "But

for FDA approval, they only have to show it's better than placebo," he said.

Looking at this trial and another that compared tetrabenazine with a

placebo, Geschwind found that both drugs appeared to be equally effective.

However, deutetrabenazine had fewer side effects, he said.

 

"If a patient is doing well on tetrabenazine and tolerating it well, I

wouldn't consider changing it," Geschwind said. "But if I were starting a

new patient and the drugs were similar in cost, I'm probably going to go to

the new drug because out of the starting gate I'm probably going to have

less side effects."

 

===============================================

 

July 5, 2016, Vol 316, No. 1

Editorial | July 5, 2016 

 

Deutetrabenazine for Treatment of Chorea in Huntington Disease

 

JAMA. 2016;316(1):33-35. doi:10.1001/jama.2016.8011.

 

Authors: Michael D. Geschwind, MD, PhD1; Nick Paras, PhD1

[+-] Author Affiliations

<http://jama.jamanetwork.com/article.aspx?articleid=2531986>

 

In this issue of JAMA, the Huntington Study Group (HSG), First-HD study

investigators, reports findings from a randomized trial examining use of a

deuterated form of tetrabenazine, called deutetrabenazine, for treatment of

chorea in patients with Huntington disease.1

<http://jama.jamanetwork.com/article.aspx?articleid=2531986>  Tetrabenazine,

a vesicular monoamine transporter type 2 inhibitor that depletes monoamines

including dopamine, is used worldwide for the treatment of chorea and

dystonia. Tetrabenazine was approved by the US Food and Drug Administration

(FDA) for the treatment of chorea in Huntington disease, based on a prior

HSG study.2 <http://jama.jamanetwork.com/article.aspx?articleid=2531986>

 

First Page Review:

http://jama.jamanetwork.com/article.aspx?articleid=2531986

 

 

 

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