NsGene seeks to bring brain repair technology into clinical practice

Device can deliver biologics to rejuvenate brain cells damaged by Parkinson's disease

Rupert Whiteley

Denice Spero, Ph.D., left, and Dr. Lars Wahlberg, discuss plans to bring their brain repair technology into clinical use.

By Richard Asinof
Posted 9/22/13
As many as 1 million Americans have been stricken with Parkinson’s disease, and approximately 60,000 Americans are diagnosed each year. The direct and indirect costs for Parkinson’s disease are estimated to be about $25 billion a year.

The potential breakthrough of a brain repair device to deliver biologics to help to repair and rejuvenate brain cells by a Rhode Island-based firm would help to further propel Providence and Rhode Island as a national hub for brain research and treatment of neurological disorders.
Spero, as co-founder of R.I. BioScience Leaders, a group of about 25 CEOs, was successful in the first foray in getting the R.I. General Assembly to budget a modest $500,000 in matching grants for SBIR award winners.

As more collaborative initiatives development across hospital and university boundaries – such as the new statewide autism registry – the question emerges: what kinds of economic incentives and investments can the state of Rhode Island provide in the form of matching grants to help win federal research awards?
Since 1997, Massachusetts has been published its own Index of the Massachusetts Innovation Economy, in an effort to create a fact-based method to benchmark the performance of the state’s knowledge economy against other states and nations.
Rhode Island currently lacks a similar instrument to benchmark its research and innovation enterprise, in order to help the state make better fact-based decisions about future economic development initiatives. Creating such an index, based upon the Massachusetts model, should become a priority of the state’s economic development agency.

PROVIDENCE – Linda Ronstadt is one of about 1 million Americans who are struggling with the devastating effects of Parkinson’s disease. The crippling neurological disorder has rendered her unable to sing.

When Ronstadt revealed her condition recently, news of the high-profile celebrity’s plight spread rapidly across the social media platforms, creating an emotional wave of support, concern, and questions about what, if anything, can be done to find a medical solution?

These are questions that many families, coping with loved ones who are suffering from the hellish ravages of Parkinson’s, have often asked.

Now NsGene, a startup company here in Rhode Island, could finally provide some answers.

The firm has a promising new therapeutic product that may be able to help regenerate some of the neurons damaged by the disease, with the potential for repair and rejuvenation.

NsGene has developed an implantable brain repair device that can deliver biologics to localized parts of the brain in order to treat neurological disorders such as Parkinson’s or Alzheimer’s disease.

In the treatment of Parkinson’s, for instance, NsGene’s brain repair device, what company founder Dr. Lars Wahlberg calls a “bio-reactor,” can secrete a specific biologic product into the surrounding brain through a semi-permeable hollow fiber membrane to target and repair the neurons damaged by the disease.

“We go in and target the dopaminergic neurons that are sick and dying from Parkinson’s disease, and we deliver a protein growth factor to the nerve cells that basically puts them back into a rejuvenated state,” Wahlberg explained. “It’s a regenerative type of treatment.

Clinical trials are still about a year away here in the United States, with the plan to work to implant the brain repair devices in Parkinson’s patients next year at Rhode Island Hospital, according to Wahlberg. “The FDA has a very clear road map in terms of what we need to be able to see to register the drug,” he said. NsGene’s work is being financed in part through a $700,000 grant from the Michael J. Fox Foundation.

“We’ll try to work with as early-stage patients as possible, in order to treat those nerve cells that are in the process of dying,” Wahlberg said. These neurons, he continued, “have been dysfunctional for a while before they disappear, and they are the ones that we’re targeting.”

The patients targeted for clinical trials will be in the first few years of their disease. “Because it is an implantable product, we need patients who are relatively healthy, that don’t have surgical risks,” Wahlberg said. The device is manufactured to last at least one year, but it may last as long as three or five years, according to Wahlberg.

A Team Approach
Wahlberg has recruited Denice Spero, Ph.D., formerly the co-director of the Institute for Immunology and Informatics, known as i-Cubed, co-founder of the R.I. BioScience Leaders, and former vice president of drug discovery at Boehringer Ingelheim Pharmaceuticals, Inc., to help NsGene secure the needed financial foundation.

Both Spero and Wahlberg have worn many different hats in their careers, and they have found it easy and enjoyable to work together as collaborators.

“At this point, we’re not selling a product, we’re selling a concept that has good data and the belief that we can make a difference,” Wahlberg said.

“I’ve been on the other side of the table,” Spero added, listening to the presentations from startup firms to a pharmaceutical company. “We have different networks on science side and business side and we’re trying to leverage those together.”

The business strategy moving forward, Spero told ConvergenceRI, is three-pronged. Find partnerships with other companies, bring in money through grants, and seek out private funding and donations, an approach that’s not traditional for biotech companies.

“I really believe we can do this, because the diseases we are working on are so miserable,” Spero said. “If a patient gets Alzheimer’s or Parkinson’s disease, the drugs that are out there only temporarily relieve symptoms. And the disease marches on and patients have a pretty horrible life.”

Spero said she believes that you can appeal to people and their sense of goodness, because they want to see these kinds of therapeutics come to the market to help people.

What Rhode Island offers
From a product development perspective, Rhode Island has several attractive features, according to Wahlberg.

“We have collaborations and facility-use agreements with Brown University, and we’re able to use resources there,” he explained. “We also have clinic resources at Rhode Island Hospital’s departments of neurosurgery and neurology. We are building relationships with Dr. Joseph Friedman, chief of the division of movement disorders in the department of Neurology at Brown University. We have relationships with Dr. John Robson and Dr. Rees Cosgrove at the Prince Neurosciences Institute. They all have a keen interest in supporting regenerative medical approaches to brain disorders.”

People in Rhode Island are more approachable, Wahlberg continued. “It’s a smaller place, it hasn’t matured as much as Cambridge and the Greater Boston area,” he said.

Spero saw Rhode Island’s sense of size and of being a neighborhood a big asset.. “That kind of closeness that happens in Rhode Island may be a little bit rare [to find] in other places. One of the things we love about Rhode Island is that it’s easy to talk with people. If you want to talk to a legislator, you can. Or the treasurer, you can. Or the head of any department in the hospital, it’s easy to do,” she said.

By contrast, Spero continued, “If you wanted to talk to someone who is in senior management at the Brigham [Brigham and Women’s Hospital in Boston]. It’s going to be difficult to get in there, and there’s going to be a list of 10 people in front of you.”

Being a risk-taker
The place where NsGene resides right now is on the cusp, according to Spero and Wahlberg. “We’re somewhere in between academia, and the translation of the technology into the hospital world, and then out into the pharma industry,” Wahlberg said. “We are in the place where we are taking early science and translating it into the clinic, creating value in early clinical trials, and then seeking to partner with the Pfizers of the world.”

The infrastructure of functional neurosurgery is being built, Wahlberg continued, with a large number of patients being treated with deep brain stimulation. “We have the imaging, we have the instrumentation,” he said.

The next step, he continued, is to understand the biology of the disease. “We’re going to go in and try to change the biology of these disorders. I think that it’s going to be an exciting thing, to profoundly change the course of medicine and treatments for neurological disorders,” he said.

Risk-taking is a big part of the enterprise of being a startup, Spero and Wahlberg agreed. NsGene is not part of the university, and it doesn’t have the backing of a particular institution. “You are carving out a new area. People should realize it’s not all fun and games starting a new company. You’re putting your head on the line.”


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