Moving beyond treatment to repairing the brain

LIFE SCIENCES
LIFE SCIENCES

NsGene Inc.’s innovative brain-repair therapy couldn’t have come at a better time, as one of the neurodegenerative diseases it would treat – Parkinson’s disease – has gotten a lot of press lately.
Parkinson’s disease is robbing singer Linda Ronstadt of her legendary singing voice. Entertainer Robin Williams had just been diagnosed with the condition before his recent suicide.
“If this therapy gets to a patient in the early stages of their condition,” said Denice Spero, NsGene’s vice president of strategic alliances, “it could make an exciting difference in their lives.”
NsGene’s brain-repair treatment device delivers therapeutic antibodies and proteins to the brain through a millimeter in diameter device which is surgically implanted behind the blood-brain barrier. A hollow fiber capsule at the end of a flexible tether contains human cells engineered to target the particular neuro-disease being treated, such as Alzheimer’s or Parkinson’s.
“It’s exciting,” Spero said of the company’s new therapy. “The technology holds so much promise for treating these conditions, and it is a different method than has been tried before.”
She said half of the patients participating in a Phase IB of the device’s first clinical trial at the Karolinska Institute in Sweden – which focused on treating Alzheimer’s disease – exhibited improvements in their cognitive abilities which correlated with biological readouts, and it showed the implants to be safe and well-tolerated.
She said the implants are placed where the nerves in the brain are dying. The proteins that the device delivers protect these nerves and restore them. The device needs no reservoir of medicine. The cells within the device produce the proteins which do the all work for a year or more before the implant needs replacing.
“We don’t need to make a drug,” Spero said. “The human cells in the device make the protein. The technology potentially solves the problem of how to get therapeutics behind the blood-brain barrier safely and in sufficient concentrations. Currently marketed drugs can help only the symptoms of these serious neurodegenerative disorders, but to date no drugs can slow down these diseases or cure them.”
Spero, who joined NsGene in 2013, said the company plans next to expand the Alzheimer’s study to 18 months. The company is primarily focusing on Parkinson’s disease at the Providence labs. Nsgene is also exploring the use of the device for treating epilepsy and hearing disorders.
“I’ve had three relatives die of Alzheimer’s,” she said. “It’s a cruel disease. If I can contribute my knowledge to help those with the condition, that’s what makes you want to go to work every day.”
Neurosurgeon Lars Wahlberg, president and CEO, one of the inventors of the technology at Brown University, founded NsGene in Denmark. He opened the Proovidence office in 2011.

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