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【medical-news】杜克大学主持研究新的痛风治疗方法

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A new drug designed to normalize levels of uric acid in the blood appears to be safe and tolerable and may help patients with the painful arthritic condition known as gout better cope with their disease, say researchers at Duke University Medical Center.

The results of a phase II clinical trial show that the drug, an enzyme called pegloticase, lowered uric acid in blood to target levels within just a few hours in a majority of the participants.

"We were delighted to see this response, because all of the patients in our trial had already tried all the existing treatments for gout, and nothing was helping them," said John Sundy, MD, a rheumatologist at Duke and the lead author of the study.

During treatment most of the patients continued to have gout flares and a majority developed non-neutralizing antibodies to the drug, which shortened the half-life of the drug in the bloodstreams of some patients.

The study is online and will appear in the September issue of the journal Arthritis & Rheumatism.

Often incorrectly depicted as just a disease of overindulgent old men, gout usually takes people by surprise, says Sundy. "The typical patient is a male, in his 40s or 50s, who wakes up one morning with a searing pain in one of his big toes."

Gout arises when excess uric acid builds up in the joints. Uric acid is a natural byproduct of DNA metabolism and it is normally excreted in urine. But when that process is too slow, the level of uric acid in the blood rises and the excess turns into crystals, triggering severe inflammation in joints.

In some patients, that can lead to the accumulation of painful knots of crystallized uric acid that can be the size of golf balls (called tophi) that form around the joints and tendons. In extreme cases, gout can leave patients wheelchair-bound and unable to handle basic day to day tasks like cooking or buttoning clothes.

It is estimated that roughly five million people in the U.S. suffer from gout, and while most of them are men, Sundy notes that the prevalence of gout is higher than that of rheumatoid arthritis in women over age 60.

Current standard of care includes the use of the drugs allopurinol and probenecid. "But about five percent of patients will stop allopurinol because of side effects and it is often not effective at the most commonly prescribed dose," said Sundy. "We're looking at about 50,000 people who need some new options. We haven't seen any new treatments for the underlying cause of gout in 40 years."

Pegloticase was discovered after nearly two decades of laboratory research at Duke under the direction of Michael Hershfield, MD. Hershfield, who is not involved in the clinical study of pegloticase, says it helps get rid of excess uric acid by converting it into a substance that is more easily eliminated.

Sundy, along with colleagues in other medical centers and at Savient Pharmaceuticals, the company that is developing pegloticase, studied the use of the drug in 41 patients who were randomized to one of four treatment groups. Participants took either four or eight milligrams every two weeks, or eight or 12 milligrams every four weeks, for a 12 or 14 week period. The treatment is an infusion that takes about two hours.

Researchers found that pegloticase normalized uric acid levels within six hours for participants in all dosage groups, and those levels were sustained throughout the treatment period in the two groups at the higher dosage levels. The most effective dose was found to be eight milligrams every two weeks.

During the treatment, 88 percent of the patients experienced gout flares, which can happen for a while when uric acid levels are lowered. Some of the patients also experienced mild to moderate side effects, including reactions to the infusion and joint pain.

"The generally accepted goal of therapy is to reduce serum urate concentrations to less than six milligrams per deciliter, and we found that pegloticase can do that very, very quickly," says Sundy. "Perhaps what is most important is that it did this in patients who had run out of therapeutic options."

Sundy says more studies need to be conducted to confirm and expand the findings, but says he is encouraged by the results. Results of a phase III study of pegloticase are slated for presentation of the annual meeting of the American College of Rheumatology in October.

Co-authors of the study include Michael Becker, MD, from the University of Chicago Pritzer School of Medicine; Herbert Baraf, MD, Arthritis and Rheumatism Associates, Wheaton, MD; Andre Barkhuisen, MD, Oregon Health Sciences University; Larry Moreland, MD, University of Alabama at Birmingham; and William Huang, PhD; Royce Waltrip II, MD; Allan Maroli, PhD; and Zeb Horowitz, MD; all from Savient Pharmaceuticals.

Drs. Huang, Waltrip, Maroli and Horowitz own stock or stock options in Savient Pharmaceuticals and all have their names on pending patents for use of the drug.

http://www.mphtimes.com/us/index.php?option=com_content&view=article&id=566:-gout-duke-leads-study-of-new-treatment-&catid=120:gout&Itemid=122

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2008-09-09 04:56

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A new drug designed to normalize levels of uric acid in the blood appears to be safe and tolerable and may help patients with the painful arthritic condition known as gout better cope with their disease, say researchers at Duke University Medical Center.
杜克大学医学中心的研究人员称，一种降血尿酸的新药已被证实安全、可耐受，并对痛风患者可能有助益。

The results of a phase II clinical trial show that the drug, an enzyme called pegloticase, lowered uric acid in blood to target levels within just a few hours in a majority of the participants.
这种叫pegloticase的新药其实是一种酶。 II期临床研究结果显示，它能在几小时内将大多数受试者的尿酸降至目标水平
"We were delighted to see this response, because all of the patients in our trial had already tried all the existing treatments for gout, and nothing was helping them," said John Sundy, MD, a rheumatologist at Duke and the lead author of the study.
本研究的首席专家、杜克大学的风湿病医生John Sunday说，“这项研究中的患者之前都接受过各种痛风治疗但无效。我们很高兴看到新药对他们起了作用。”

During treatment most of the patients continued to have gout flares and a majority developed non-neutralizing antibodies to the drug, which shortened the half-life of the drug in the bloodstreams of some patients.
治疗期间大多数患者仍有痛风复发，并且大多数对药物产生非抑制性抗体，导致部分患者血药半衰期缩短。
The study is online and will appear in the September issue of the journal Arthritis & Rheumatism.
该研究结果已经在网上公布，并将刊登在9月份的《风湿与关节炎》杂志

Often incorrectly depicted as just a disease of overindulgent old men, gout usually takes people by surprise, says Sundy. "The typical patient is a male, in his 40s or 50s, who wakes up one morning with a searing pain in one of his big toes."
Sundy说，人们常误以为痛风是过度放纵的老年病，可事实却让人大吃一惊。“典型的痛风患者是40-50岁的男性，一夜醒来发现大脚趾烧灼般疼痛。”
Gout arises when excess uric acid builds up in the joints. Uric acid is a natural byproduct of DNA metabolism and it is normally excreted in urine. But when that process is too slow, the level of uric acid in the blood rises and the excess turns into crystals, triggering severe inflammation in joints.
当多余的尿酸沉积在关节就发生了痛风。尿酸是DNA代谢的正常副产物，通过尿液排泄。但如果这个过程过慢，血尿酸浓度就会升高并形成结晶，引发关节的严重炎症反应。
In some patients, that can lead to the accumulation of painful knots of crystallized uric acid that can be the size of golf balls (called tophi) that form around the joints and tendons. In extreme cases, gout can leave patients wheelchair-bound and unable to handle basic day to day tasks like cooking or buttoning clothes.
在部分患者体内，尿酸结晶还会在关节和肌腱周围形成痛性结节，尺寸可大到高尔夫球大小（又叫结节瘤）。在一些极严重的病例中，痛风可以导致患者生活不能自理，不能完成最基本的日常任务比如做饭或扣扣子，要依赖轮椅生活。
It is estimated that roughly five million people in the U.S. suffer from gout, and while most of them are men, Sundy notes that the prevalence of gout is higher than that of rheumatoid arthritis in women over age 60.
粗略估计，全美有500万痛风患者，其中大多数是男性。但Sundy指出，在60岁以上的女性中，痛风的患病率高于风湿性关节炎。

Current standard of care includes the use of the drugs allopurinol and probenecid. "But about five percent of patients will stop allopurinol because of side effects and it is often not effective at the most commonly prescribed dose," said Sundy. "We're looking at about 50,000 people who need some new options. We haven't seen any new treatments for the underlying cause of gout in 40 years."
目前治疗痛风的标准用药是别嘌醇和丙磺舒。“但大约5%的患者会停用别嘌醇，原因是该药的副作用和常用处方剂量的疗效不佳。”Sundy说，“我们的研究着眼于那5万需要新疗法的人。在最近40年里，我们还未见到任何针对痛风根本病因的新治疗。”
Pegloticase was discovered after nearly two decades of laboratory research at Duke under the direction of Michael Hershfield, MD. Hershfield, who is not involved in the clinical study of pegloticase, says it helps get rid of excess uric acid by converting it into a substance that is more easily eliminated.
在Michael Hershfield医生的带领下，杜克大学经过近20年的实验研究终于发现了Pegloticase。 Hershfield本人并不参与该药的临床试验，她介绍说pegloticase可以将多余的尿酸转化成一种更容易清除的的物质，从而控制尿酸水平。

Sundy, along with colleagues in other medical centers and at Savient Pharmaceuticals, the company that is developing pegloticase, studied the use of the drug in 41 patients who were randomized to one of four treatment groups. Participants took either four or eight milligrams every two weeks, or eight or 12 milligrams every four weeks, for a 12 or 14 week period. The treatment is an infusion that takes about two hours.
Sundy和其他医疗中心的同事，以及现正研发pegloticase的Savient制药一起进行了该药的临床试验。41名患者被随机编入4个治疗组中的一组（有点费解，为什么不是randomized to four treatment groups呢？）。四组用药分别为4mg、8mg，每两周一次；或8mg、12mg，每四周一次，疗程为12到14周。用药方式为静脉注射，持续2小时。
Researchers found that pegloticase normalized uric acid levels within six hours for participants in all dosage groups, and those levels were sustained throughout the treatment period in the two groups at the higher dosage levels. The most effective dose was found to be eight milligrams every two weeks.
研究人员发现，所有治疗组的尿酸水平在用药后6小时内均回复正常，其中高剂量组的尿酸浓度在治疗全程始终保持稳定。最有效的剂量是8mg，每2周一次。
During the treatment, 88 percent of the patients experienced gout flares, which can happen for a while when uric acid levels are lowered. Some of the patients also experienced mild to moderate side effects, including reactions to the infusion and joint pain.
治疗期间，88%的患者痛风复发，这可以在尿酸水平下降时暂时出现。还有些患者出现轻到中度副作用，包括注射反应和关节痛。
"The generally accepted goal of therapy is to reduce serum urate concentrations to less than six milligrams per deciliter, and we found that pegloticase can do that very, very quickly," says Sundy. "Perhaps what is most important is that it did this in patients who had run out of therapeutic options."
Sundy说：“痛风治疗的总体目标是将血尿酸浓度降至6mg/dl以下，pegloticase能够非常迅速地达到这个目标，更重要的是，这是在那些已经没有其他治疗选择的患者身上起效的。”
Sundy says more studies need to be conducted to confirm and expand the findings, but says he is encouraged by the results. Results of a phase III study of pegloticase are slated for presentation of the annual meeting of the American College of Rheumatology in October.
Sundy表示，尽管还需要更多的研究去证实和扩充这个结果，但他仍受到鼓舞。pegloticase的III期研究结果被安排10月举行的美国风湿学会年会上报告陈述。

Co-authors of the study include Michael Becker, MD, from the University of Chicago Pritzer School of Medicine; Herbert Baraf, MD, Arthritis and Rheumatism Associates, Wheaton, MD; Andre Barkhuisen, MD, Oregon Health Sciences University; Larry Moreland, MD, University of Alabama at Birmingham; and William Huang, PhD; Royce Waltrip II, MD; Allan Maroli, PhD; and Zeb Horowitz, MD; all from Savient Pharmaceuticals.
本研究的其他参与人员包括芝加哥大学Pritzer医学院的Michael Becker, Herbert Baraf（ARS会员）和 Wheaton医生; 俄勒冈健康科学大学的Andre Barkhuisen医生; 阿拉巴马和伯明翰大学的Larry Moreland医生; Savient 制药的William Huang博士，Royce Waltrip II医生，Allan Maroli博士和Zeb Horowitz医生.
Drs. Huang, Waltrip, Maroli and Horowitz own stock or stock options in Savient Pharmaceuticals and all have their names on pending patents for use of the drug.
Huang, Waltrip, Maroli 和Horowitz拥有Savient制药的股份或股票期权，并将同时拥有该药使用的专利权，专利目前正在申请中。

http://www.mphtimes.com/us/index.php?option=com_content&view=article&id=566:-gout-duke-leads-study-of-new-treatment-&catid=120:gout&Itemid=122

2008-09-10 10:29

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杜克大学医学中心的研究人员称，一种降血尿酸的新药已被证实安全、可耐受，并对痛风患者可能有助益。这种叫pegloticase的新药其实是一种酶。 II期临床研究结果显示，它能在几小时内将大多数受试者的尿酸降至目标水平。
本研究的首席专家、杜克大学的风湿病医生John Sunday说，“这项研究中的患者之前都接受过各种痛风治疗但无效。我们很高兴看到新药对他们起了作用。”
治疗期间大多数患者仍有痛风复发，并且大多数对药物产生非抑制性抗体，导致部分患者血药半衰期缩短。
该研究结果已经在网上公布，并将刊登在9月份的《风湿与关节炎》杂志
Sundy说，人们常误以为痛风是过度放纵的老年病，可事实却让人大吃一惊。“典型的痛风患者是40-50岁的男性，一夜醒来发现大脚趾烧灼般疼痛。”
当多余的尿酸沉积在关节就发生了痛风。尿酸是DNA代谢的正常副产物，通过尿液排泄。但如果这个过程过慢，血尿酸浓度就会升高并形成结晶，引发关节的严重炎症反应。在部分患者体内，尿酸结晶还会在关节和肌腱周围形成痛性结节，尺寸可大到高尔夫球大小（又叫结节瘤）。在一些极严重的病例中，痛风可以导致患者生活不能自理，不能完成最基本的日常任务比如做饭或扣扣子，要依赖轮椅生活。粗略估计，全美有500万痛风患者，其中大多数是男性。但Sundy指出，在60岁以上的女性中，痛风的患病率高于风湿性关节炎。

目前治疗痛风的标准用药是别嘌醇和丙磺舒。“但大约5%的患者会停用别嘌醇，原因是该药的副作用和常用处方剂量的疗效不佳。”Sundy说，“我们的研究着眼于那5万需要新疗法的人。在最近40年里，我们还未见到任何针对痛风根本病因的新治疗。”
在Michael Hershfield医生的带领下，杜克大学经过近20年的实验研究终于发现了Pegloticase。 Hershfield本人并不参与该药的临床试验，她介绍说pegloticase可以将多余的尿酸转化成一种更容易清除的的物质，从而控制尿酸水平。
Sundy和其他医疗中心的同事，以及现正研发pegloticase的Savient制药一起进行了该药的临床试验。41名患者被随机编入4个治疗组中的一组（有点费解，为什么不是randomized to four treatment groups呢？）。四组用药分别为4mg、8mg，每两周一次；或8mg、12mg，每四周一次，疗程为12到14周。用药方式为静脉注射，持续2小时。
研究人员发现，所有治疗组的尿酸水平在用药后6小时内均回复正常，其中高剂量组的尿酸浓度在治疗全程始终保持稳定。最有效的剂量是8mg，每2周一次。
治疗期间，88%的患者痛风复发，这可以在尿酸水平下降时暂时出现。还有些患者出现轻到中度副作用，包括注射反应和关节痛。
Sundy说：“痛风治疗的总体目标是将血尿酸浓度降至6mg/dl以下，pegloticase能够非常迅速地达到这个目标，更重要的是，这是在那些已经没有其他治疗选择的患者身上起效的。”
Sundy表示，尽管还需要更多的研究去证实和扩充这个结果，但他仍受到鼓舞。pegloticase的III期研究结果被安排10月举行的美国风湿学会年会上报告陈述。
本研究的其他参与人员包括芝加哥大学Pritzer医学院的Michael Becker, Herbert Baraf（ARS会员）和 Wheaton医生; 俄勒冈健康科学大学的Andre Barkhuisen医生; 阿拉巴马和伯明翰大学的Larry Moreland医生; Savient 制药的William Huang博士，Royce Waltrip II医生，Allan Maroli博士和Zeb Horowitz医生.Huang, Waltrip, Maroli 和Horowitz拥有Savient制药的股份或股票期权，并将同时拥有该药使用的专利权，专利目前正在申请中。

http://www.mphtimes.com/us/index.php?option=com_content&view=article&id=566:-gout-duke-leads-study-of-new-treatment-&catid=120:gout&Itemid=122

2008-09-10 10:33

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