Alan Hoffman, shown with his wife, Nancy, at their home in Dumfries, Va., found
ID: 3164740 • Letter: A
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Alan Hoffman, shown with his wife, Nancy, at their home in Dumfries, Va., found that his Parkinson's symptoms improved when he took a cancer drug A drug that's already approved for treating leukemia appears to dramatically reduce symptoms in people who have Parkinson's disease with dementia, or a related condition called Lewy body dementia. A pilot study of 12 patients given small doses of nilotinib found that movement and mental function improved in all of the 11 people who completed the six-month trial, researchers reported Saturday at the Society for Neuroscience meeting in Chicago. And for several patients the improvements were dramatic, says Fernando Pagan, an author of the study and director of the Movement Disorders Program at Georgetown University Medical Center. One woman regained the ability to feed herself, one man was able to stop using a walker, and three previously nonverbal patients began speaking again, Pagan says After 25 years in Parkinson's disease research, this is the most excited I've ever been,' Pagan says. If the drug's effectiveness is confirmed in larger, placebo-controlled studies, nilotinib could become the first treatment to interrupt a process that kills brain cells in Parkinson's and other neurodegenerative diseases, including Alzheimers Hoffman was diagnosed with Parkinson's in 1997. At first, he had trouble moving his arms. Over time, walking became more difficult and his speech became slurred. And by 2007, the disease had begun to affect his thinking I knew l'd dropped off in my ability to read, ' Hoffman says. "People would keep giving me books and I'd have read the first chapter of about 10 of them. I had no ability to focus on it He had more and more difficulty making sense, ' Nancy Hoffman says. He also became less active, less able to have conversations, and eventually stopped doing even household chores, she says But after a few weeks on nilotinb. Hoffman improved in every way;"his wife says. THe began loading the dishwasher. loading the clothes in the dryer, things he had not done in a long time. Even more surprising, Hoffman's scores on cognitive tests began to improve. At home, Nancy Hoffman says her husband was making sense again and regained his ability to focus. "He actually read the David McCullough book on the Wright brothers and started reading the paper from beginning to end, she says The idea of using nilotinib to treat people like Alan Hoffman came from Charbel Moussa, an assistant professo of neurology at Georgetown University and an author of the studyExplanation / Answer
Q1) Due to parkinson's disease Mr. Hoffman was experiencing symptoms of -
Mr. Hoffman were having symptoms of trouble in moving his arms, diffiulty in walking, slurred speech, reduce in his cognitive processes like thinking, reading ability, making sense, concentration or focus . He stopped doing house hold works , became less active , due to Parkinson's disease.
Q2)How Mr.hoffman responded to Nilotinib?
When Mr. Hoffman took Nilotinib He improved in many ways , he started doing his household activities like loading a drayer, a dishwasher, his cognitive abilities also improved , he was able to focus more and started reading his reading abilities improved.
Q3) Explain what Dr.Mouss's reasoning was for thinking Nilotinib might be effective in patients with parkinson and lewy body dementia?
According to Moussa people who have Parkinson's disease with dementia or Lewy body dementia, toxic proteins get build up in their certain brain cells, and gradually kill them. Moussa thought nilotinib might be able to reverse this process.
Moussu's reasoning behind this was Nilotinib activates a system in cells which works like a garbage disposal , it helps in clearing unwanted proteins. Moussa showed that when cancer cells were exposed to Nilotinib they die but brain cells become healthier.
Q4) Describe the two studies and the findings that were done by Dr.Moussa.
In the first experiment
Moussa in his lab did an experiment taking brain cells in a Petri dish,and introduced nilotinib to it, he found by a very little amount of the drug , the proteins which were neurotoxic were cleared.
In the second experiment-
Moussa and his team gave the drug to transgenic mice which were almost completely paralyzed from Parkinson's disease.But the treatment saved the animals, It allowed them to move like the healthy mice. this proved Nilotinib helps in improving from parkinson's diseases.
The findings of the study of Dr. Moussa-
The pilot study was designed to determine whether Nilotinib was safe for Parkinson's patients and to determine the amout of drug from the capsules they were taking was reaching their brains.
The study found that levels of toxic proteins in blood and spinal fluid decreased when patients start taking nilotinib. Also, the tests showed that the symptoms of Parkinson's disease like tremor and freezing also decreased. And during the study patients were able to use lower doses of Parkinson's drugs,this suggested that the brain cells which produce dopamine were also working better.
Q5) What were the drawbacks of the drug Nicotinib?
The drawbacks of Nilotinib are it's cost effectiveness, they are very costly, and the patients have to take them lifelong , else there condition will worsen.It may cause high blood pressure and high blood glucose, it may cause abnormal heart rhythm called prolongation of QT interval. Prolongation of the QT interval may lead to sudden death.
Q6) What happened to mr.Hoffman once he stopped taking the new drug?
Once Mr. hoffman stopped taking the new drug his conditions started worsening he has more problem in his speech, in his cognition and faced more problem with mobility.
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