![]() However other research suggested that GDNF was even more effective at protecting motor neurons, the nerve cells destroyed by ALS.Īrmed with the knowledge of GDNF’s ability to protect motor neurons, Svendsen and his team developed an experimental stem cell-based therapy that they hoped would treat patients with the sporadic form of ALS. Svendsen was studying how an important growth factor in the brain called Glial Cell Line-Derived Neurotrophic Factor or GDNF could be used to protect dopamine neurons in order to treat Parkinson’s patients. The transition from Parkinson’s to ALS was not without a scientific reason however. “Being normal one day, and then becoming rapidly paralyzed was hard to see.” “I chose to work on ALS mainly because of the effects it has on ALS families,” explained Svendsen. Unlike ALS, Parkinson’s patients have a longer life expectancy and more treatment options that alleviate symptoms of the disease, making their quality of life far better than ALS patients.Ĭlive Svendsen, PhD, Director, Regenerative Medicine Institute. Before that, he studied Parkinson’s disease, a long-term neurodegenerative disorder that affects movement, balance and speech. Svendsen, who moved to Cedars-Sinai in Los Angeles to head the Cedars-Sinai Board of Governors Regenerative Medicine Institute in 2010, has worked on ALS for the past 15 years. “Around 10% of ALS cases are genetic, and we know some of the genes involved, but 90% of cases are sporadic.” He explained that this black box makes it difficult for scientists to know where to start when trying to develop treatments for sporadic ALS cases that have no drug targets. “The problem with ALS is we don’t know the cause,” he said. However, developing a therapeutic strategy was challenging to Svendsen. Given this poor prognosis, making ALS the focus of his research career was an easy decision. However, they only slow disease progression in some ALS patients by a few months and there are no effective treatments that stop or cure the disease. Currently there are two FDA-approved ALS drugs in the US – riluzole and a new drug called edaravone (Radicava). ![]() Denervation, or loss of nerves, causes muscle weakness and atrophy, leaving patients unable to control their own bodies. ALS destroys the nerve cells that send signals from the brain and spinal cord to the muscles that control movement. On the receiving end was Clive Svendsen, PhD, then a scientist at the University of Wisconsin-Madison, determined to understand how stem cells could help patients like Jeff.Īlso known as Lou Gehrig’s disease, ALS is a rapid, aggressive neurodegenerative disease with a two to four-year life expectancy. ![]() The person asking the question was Jeff Kaufman, a Wisconsin man in his 40s completely paralyzed by amyotrophic lateral sclerosis (ALS). The sentence appeared slowly on a computer screen, each character separated by a pause while its author searched for the next character using a device controlled by his eye muscle. Photo of Clive Svendsen (top left) and Jan & Jeff Kaufman ![]()
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