回复2楼 薛傳恽
回复2楼 薛傳恽:回复1楼 信主不白活
我买此药是原厂的。向厂方订购，对方邮寄过来的。但是药盒里的药单没用，因为那是介绍如何用于白血病的。我吃此药时，别的帕药，认知障碍药，还是在吃。是否因该如此也不知道。不过原来的感觉还是觉得如常。只是说话变好了。
现找到一篇比较详细的关于此药治帕症文章。哪位英文较好的，请帮忙翻译一下。我的精力不够了。
OCTOBER 17, 2015 – An FDA-approved drug for leukemia improved cognition, motor skills and non-motor function in patients with Parkinson’s disease and Lewy body dementia in a small clinical trial, say researchers at Georgetown University Medical Center (GUMC).
The drug, nilotinib (known as Tasigna® by Novartis) also led to statistically significant and encouraging changes in toxic proteins linked to disease progression.
The study’s findings were presented at Neuroscience 2015, the annual meeting of the Society for Neuroscience, in Chicago on Oct. 17.
Charbel Moussa, MD, PhD, who directs Georgetown’s Laboratory of Dementia and Parkinsonism, conducted the preclinical research that led to the discovery of nilotinib for the treatment of neurodegenerative diseases.
Walking Again
To conduct the clinical study, he partnered with Fernando Pagan, MD, a GUMC associate professor of neurology who directs the Movement Disorders Program at MedStar Georgetown University Hospital.
“To my knowledge, this study represents the first time a therapy appears to reverse – to a greater or lesser degree depending on stage of disease – cognitive and motor decline in patients with these neurodegenerative disorders,” says Pagan. “But it is critical to conduct larger and more comprehensive studies before determining the drug’s true impact.”
The investigators report that one individual confined to a wheelchair was able to walk again and that three others who could not speak were able to hold conversations.
Pagan notes there was no control group for comparison in the study, and that the drug was not compared with a placebo or other medications used to treat Parkinson’s.
Safety Test
But the researchers report that during use of the medication by the participants, production of dopamine increased in many patients, requiring doses of L-dopa and other dopamine-sparing drugs used to treat Parkinson’s to be lowered or stopped.
Stopping nilotinib treatment appears to lead to cognitive and motor decline despite reinstating L-dopa therapies.
The study’s primary objective was to test safety.
“The use of nilotinib in doses much smaller than are used to treat cancer, which is up to 800 mg daily, was well tolerated with no serious side effects,” Pagan explains. “The dose used in this study was 150 and 300 mg daily.”
The researchers also found that the drug penetrates the blood-brain barrier in amounts greater than dopamine drugs.
The observed efficacy in cognition, motor skills and non-motor function improvement (such as constipation) for many patients was the most dramatic result, Pagan notes.
“Study participants with earlier stage disease responded best, as did those diagnosed with Lewy body dementia, often described as a combination of Parkinson’s and Alzheimer’s diseases,” he says.
Life-Changing Improvement
Alan Hoffman, a professor emeritus of social science education at Georgia State University, was diagnosed with Parkinson’s disease in 1997. He says he participated in several clinical trials with no benefit until he enrolled in Pagan’s study.
“Before the nilotinib, I did almost nothing around the house,” he says. “Now, I empty the garbage, unload the dishwasher, load the washer and the dryer, set the table, even take responsibility for grilling.”
In the three weeks prior to enrolling in the study, Hoffman says he fell eight times, but he only fell once during six months on the study. His speech has improved, as has his thinking.
“My wife says it’s life-changing for her and for my children and grandchildren,” Hoffman says. “To say that nilotinib has made a change in our lives is a huge understatement.”
Translational Success
Moussa, an inventor on a Georgetown University patent application for nilotinib and other similar drugs for neurodegenerative diseases, notes that the research went to clinical trials only two years after his initial discovery.
He first set out to find approved cancer drugs that could penetrate the blood-brain barrier and turn on the “garbage disposal machinery” inside neurons to clear toxic intracellular proteins and prevent their accumulation within, or secretion outside of, brain cells.
“A lot of institutions talk about expediting the translation of research from the lab to the bedside, but it doesn’t happen quickly very often,” Moussa says. “This is a solid example of how that is possible and why it is so important.”
Patients Continue Treatment
Hoffman and other patients in the clinical trial can continue taking nilotinib as part of an expanded access study. Georgetown researchers are now planning larger clinical trials with nilotinib for patients with Parkinson’s and other similar diseases including Alzheimer’s disease, likely to begin in 2016.
The phase I study received philanthropic funding and was supported by the Georgetown-Howard Universities Center for Clinical and Translational Science.
Co-authors of the study represent the MedStar Georgetown Movement Disorders Program, GUMC’s Translational Neurotherapeutics Program and the Laboratory for Dementia and Parkinsonism, and the Georgetown-Howard Universities Center for Clinical and Translational Science Clinical Research Unit.
Nancy Robertson
Related Content
Georgetown University Medical Center and Howard University Receive $38.2 Million NIH Grant to Form Center for Clinical and Translational Science
July 14, 2010
In support of a critical partnership between Georgetown University Medical Center and Howard University, the National Institutes of Health (NIH) has awarded a $38.2 million Clinical and Translational Sciences Award to aid the universities’ effort to transform health care and preventive practices in communities through medical discoveries made in laboratories and clinical settings.
Learn More
FDSFSD
Developing a Regional Clinical Research Powerhouse
September 3, 2015
The Georgetown-Howard Universities Center for Clinical and Translational Science (GHUCCTS), is one of 62 clinical research hubs in the United States created by the National Center for Advancing Translational Sciences CTSA Program. The National Institutes of Health has renewed the CTSA grant, awarding GHUCCTS $27 million over five years.
Learn More
Fund a Fellow
Patients Drive Support of Parkinson’s Fellowship Program
March 2, 2015
Twelve years ago, Rick Schena, a United Airlines pilot, felt something amiss in the body he has always been so in tune with —the onset of tremors, some rigidity, and issues with processing vision. Six years ago he was diagnosed with Parkinson’s. This was devastating to the speed junkie who also races cars and go-karts.
Learn More
