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Gene Therapy

In what could be a science fiction scenario, Dvora Beeri became MUSC's first patient to undergo brain surgery March 29 as part of a clinical trial to see the effectiveness of CERE-110, a new type of gene therapy treatment for patients with Alzheimer's disease.

By Dawn Brazell
Public Relatins

Jacobo E. Mintzer M.D., co-principal investigator with neurosurgeon Istvan Takacs, M.D., said Beeri is the first in the state and the 19th in the world to have the procedure done as part of a Phase 2, double-blind study involving patients with mild to moderate Alzheimer's disease.

"We're skeptically optimistic. We're opening a new door that we don't know where it's going to lead us, but we're opening it locally in a way that puts us on par with the top centers in the world," Mintzer said.

Dr. Jacobo Mintzer

MUSC is one of 10 leading centers across the U.S. that has been selected to be a part of the study, which is being conducted in collaboration with the Alzheimer's Disease Cooperative Study (ADCS). Mintzer, who is director of the Department of Neurosciences a Division of Translational Research, said the trial is a sign of the success of ADCS, a national research consortium funded by the National Institute on Aging that conducts multi-center clinical trials.

Given the prevalence and devastation of Alzheimer's disease, this work required a network of sites with state-of-the-art capabilities where any new treatment or approach could quickly and effectively be put in the pipeline and tested, Mintzer said. It was noticed that true innovation wasn't necessarily coming from pharmaceutical companies but from small biotech companies who oftentimes did not have the resources to move the science forward.

The ADCS selected the 30 top centers in the country to form a network to aid these small companies and implement clinical trials on a national level to move this science forward faster through its grant system. Of those centers, 10 were selected for this study.

Gene therapy involves nerve growth factor being injected into a critical area of the brain called the Nucleus Basalis of Meynert. Medical illustration by Emma Vought

The science being promoted in this case studies how best to get NGF or nerve growth factor to a critical area of the brain called the Nucleus Basalis of Meynert (NBM), a brain region where cell degeneration occurs in Alzheimer's disease. CERE-110 is composed of an adeno-associated viral vector carrying the gene for NGF, a naturally occurring protein that maintains the survival of nerve cells in the brain. 

CERE-110 is surgically injected into the NBM, where it is hoped the delivery of NGF gene using a "virus" will allow NGF to replicate itself through the patient's own cell machinery and stop the progression of the disease or even potentially cause improvements.

"We have to get it in the right place in a way the cell can incorporate it," said Mintzer. "We need to get the cell to generate it by itself on an ongoing process. The only way you can do that is by injecting new DNA into a cell."

Beth Safrit, left, and Dvora Beeri

Beth Safrit, nurse practitioner III and clinical director of the Department of Neurosciences Alzheimer's Research and Clinical Programs, will oversee the monitoring of the five to 10 patients who will be participating in the trial. The patients are followed for 24 months.

"It's the only thing out there that can offer any true hope of improving," she said of the study, which accepts patients who have only a mild level of Alzheimer's. "If these patients can stabilize themselves or maybe even get a little bit better with this one-time treatment, because that little virus is producing for the rest of their lives, it's an amazing thing. If it proves to be effective, it's going to change their lives forever."

Mintzer said one of the main challenges is pushing the science forward, while managing people's expectations. It will be years before a therapy can be developed should the therapy prove effective. Having begun his research into Alzheimer's disease in 1981, Mintzer said he's seen impressive changes from when it was difficult just trying to convince people that such a disease existed.

"Now we have come to a moment where we are inserting new genetic material into cells."

It's been a complex process to bring the study to MUSC, with more than 400 people having to be trained in the MUSC family, from the pharmacists and doctors involved to the person cleaning the OR. Safrit holds up a thick file folder of all the employees who had to be checked off. Though they received no extra pay, they were willing to do the training.

"They were enthralled with what we were accomplishing here," she said. "They were happy to be a part of it."

The other critical part that enables the study to proceed are the patients who are willing to take the risk of a double-blind study, which means they will have their head shaved and may go through a "sham" surgery where they don't receive the gene therapy. The patients and researchers won't know until the end of the two-year study who received the real treatment, although eligible patients who received the sham surgery will be offered the therapy at the end if they want it.

Mintzer said it takes a very strong commitment from the person. It takes a commitment from MUSC as well.

"A few years ago, this appeared to be science fiction. I'm excited not only about this particular study but about the possibilities it opens for us scientifically. It lays the groundwork for gene therapy for other organs. There is a path that has been laid out, and that puts MUSC clearly on the forefront for this type of research and eventually, this type of therapy."

MUSC has one of the few neuroscience programs in the country to have neurosurgery working with neurology and Alzheimer's specialists, which is not a common event, he said.

"This project requires a very close collaboration between neurosurgery and the dementia specialists. We have both together at MUSC. The core of our MUSC mission is to bring to South Carolina what would not be available if the resources of a university of this caliber were not here."

For more information about the clinical trial, call 740-1592, ext. 14 or visit

Alzheimer's patients hope gene therapy study works

Avri Beeri knows his research.

He can tell you everything there is to know about Alzheimer's disease—about the myriad of clinical trials, the vitamins and supplements thought to slow the process, and how he helped his wife to be part of an elite group of only 50 people who will be in Phase 2 of the CERE-110 clinical trial. He can tell you what it's like to live with a spouse who suffers from a disease that requires him to be watching with "10 eyes all the time what is going on."

He smiles lovingly at Dvora, his wife of 44 years, who has returned to MUSC for a check up following her brain surgery March 29, where she may have received gene therapy as MUSC's first patient to enroll in the trial. Their hope is that this therapy will stop and possibly even improve her condition.

Avri and Dvora Beeri

Sporting a new cap to cover her closely-cropped hair, Dvora smiles at her husband. "I just need to remember," she said of her decision to enter in a double-blind clinical study that required her to have a portion of her head shaved for a treatment she may or may not have received when she underwent surgery.

Beeri nods at his wife's response. He needs to know he did everything he could to help her do just that.

He's the memory keeper for both of them now. Beeri met his wife when he went to take music lessons from her brother. "He invited me to come home with him, saying 'I will teach you some lessons.' I never learned how to play the guitar, but I learned her," he said, smiling.

Beeri found out his wife had Alzheimer's disease four years ago, when his life researching the disease began. He reads everything he can trying to find out what can break the cycle and stop the disease. "I'm constantly Googling all over the world to find out what study she could go on."

When he ran across the nerve growth factor research, he knew he wanted to get her into a study. On a wait list at Duke University, the couple decided to move forward with doing the clinical trial at MUSC, where they had participated in a previous study.

"I was following it very closely to see where it was going to be, and when I found out it was going to be here, I thought, 'Good, I know these people.'"

Beeri said they've been to MUSC so much, his car comes on auto-pilot now. The decision to participate in the trial was simple for him, but not as much for Dvora. "She was the one who had to be drilled," he said of the holes that surgeons have to make for the patient to receive the gene therapy. "I told her, 'If I were the one who had it, I would do it.' If anything could stop it, it's worth trying it even with all the pain."

Consulting with their internist in Charlotte, Beeri said he was relieved it took the doctor 10 seconds to advise to do it. "We talked to him together. He said he would do it for himself."

Dealing with the disease takes patience. He advises other caregivers to know how fast the research changes and how much more there is to know about how to help those who suffer from the disease. If his wife had the "sham" surgery, he wants her to get the gene therapy should the trial show promising results.

Beeri knows it might not work, but still thinks it was worth doing.

"It will help for the future, even if it doesn't help us right now."



Friday, April 29, 2011

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