“For Alzheimer’s, there’s not that many treatments available despite hundreds of clinical trials over the past two decades and billions of dollars spent,” said Dr. Ali R. Rezai, a neurosurgeon at WVU who led the team of investigators that successfully performed a phase II trial using focused ultrasound to treat a patient with early stage Alzheimer’s.
The WVU team tested the innovative treatment in collaboration with INSIGHTEC, an Israeli medical technology company. Earlier this year, INSIGHTEC was approved by the U.S. Food and Drug Administration to begin a phase II clinical trial of the procedure and selected the WVU Rockefeller Neuroscience Institute as the first site in the United States for the trial.
Last summer, researchers at Sunnybrook Health Sciences Centre in Toronto reported the results of a phase I safety trial showing that they could reversibly open the blood-brain barrier in Alzheimer’s patients.
The phase II procedure involved the use of ultrasound waves focused through a specialized helmet with more than 1,000 probes targeting a precise spot in the brain, Rezai said, coupled with microscopic bubbles.
“And when we put a different frequency of ultrasound on the bubbles, they start osculating,” he said.
The reaction opens up the brain-blood barrier — a nearly impenetrable shield between the brain’s blood vessels and cells that make up brain tissue.
“It’s protected on one end for us to function, but also prevents larger molecules or chemotherapy or medications or anti-bodies or immune system cells or amino therapy or stem cells to get in,” he said.
In this case, the West Virginia team targeted the hippocampus and the memory and cognitive centers of the brain, which are impacted by plaques found in patients with Alzheimer’s.
“Plaques are these clusters of proteins that accumulate and block-up the brain’s connectivity,” he said. “In animal studies, it showed that these plaques are cleared with ultrasound technology.
The first patient, a person Rezai called a pioneer and hero, is West Virginia health-care worker and former WVU Children’s Hospital Neonatal Intensive Care Unit nurse Judi Polak.
“I think that, with Alzheimer’s there’s so much in the unknown. I’ve been with health science for a long time, and I understand that we need to be able to step forward and look into the future,” Polak said.
But getting to this point was a long journey beginning five years ago when she was first diagnosed with early-onset Alzheimer’s.
“That took me a while to deal with,” Polak said while sitting with her husband of 36 years, Mark Polak. “It was hard to say that I have Alzheimer’s. I didn’t want to be the person who felt sorry for myself and so we looked at clinical trials as a way to help not only me but other people, too.”
Early onset Alzheimer’s is an uncommon form of dementia that strikes people younger than age 65. Of all the people who have Alzheimer’s disease, according to research conducted by the Mayo Clinic, about 5 percent develop symptoms before age 65.
Judi Polak’s willingness to be the center of a study or research experiment in hopes of finding a cure for Alzheimer’s took an emotional toll, Mark Polak said, referring to a controlled drug-placebo trial at the University of Pittsburgh several years ago.
“Guess what, the drug didn’t work,” he said with contempt. “Just like every drug that has been tried doesn’t work.”
However, Judi Polak’s patience and persistence appears to have paid off. The procedure, which lasted three hours, safely and successfully opened her blood-brain barrier for a record 36 hours.
“It was opened longer than they expected,” Mark Polak said. “They were actually, I think both excited and scared. The team was ecstatic.”
One member of the team Mark Polak mentioned is Dr. Jeff Carpenter, a professor of neurology, neurosurgery and an interventional neuroradiologist at WVU.
“This is really step one,” Carpenter said of the successful trial. “This is to make sure it’s safe, and hopefully, we can decrease some of the big plaques in that part of the brain.”
Carpenter is what he jokingly called the “technical guy” on Rezai’s team with 18 years of experience working MRI technology and interventional radiology.
“It’s a combination of knowing MRI very well and also being used to actually treating patients,” Carpenter said. “This treatment marries MRI guidance with ultrasound targeting. “It really uses all the things I’ve been working with.”
Carpenter, a native of Fairmont, credited Rezai’s work and the leadership at WVU Medicine for supporting the research needed.
“It is really nice to be able to do this level of work this close to home,” he added.
The potential benefits of the first and subsequent treatments will take several years to fully evaluate, Rezai said. Two more similar procedures are scheduled for Judi Polak; one Tuesday and a final test in November.
“I am hopeful that focused ultrasound opening of the blood-brain barrier will prove to be a valuable treatment option for Judi Polak and other patients with early Alzheimer’s who are confronting the enormous challenges associated with the disease on a daily basis,” Rezai said.
Although Rezai stopped short of giving any immediate results from the first treatment, Polak said she noticed a change the next day.
“I think I could speak clearer and did not wait as long in answering questions,” she said. “Sometimes, in the past, things would leave my mind and I couldn’t remember things.”
“This is man on the moon stuff,” Mark Polak said of his wife’s success in the first trial. “Maybe we’re on to something.”