Neurotech USA

In the age of advanced medicine, it is amazing that some of the most debilitating retinal diseases such as retinitis pigmentosa (RP) and atrophic age-related macular degeneration have experienced virtually no progress to date. Now, one Rhode Island company has the technology and the talent to turn around this stalemate.

Neurotech USA, a privately-held biotech company located in Lincoln, has developed a pharmacological product to treat various chronic retinal diseases. The company's main product, NT-501, applies the company's Encapsulated Cell Technology (ECT) which allows for a consistent flow of state-of-the-art retinal medication to the back of the eye.

"There are no cures some of the most serious eye diseases," says Neurotech USA President and CEO Ted Danse. "Doctors have spent the past 50 years treating eye infections and inflammations with eye drops. These are basically front-of-the-eye diseases. We are working on a novel approach to treat more complicated back-of-the-eye diseases."

This approach involves a 25-minute out-patient procedure to surgically insert an implant called NT-501. At 1mm in diameter and 6mm long, NT-501 is a semi-permeable hollow fiber membrane that contains human retinal cells that have been engineered to produce a therapeutic agent called ciliary neurotrophic factor (CNTF) that is released and diffused throughout the back of the eye. CNTF was licensed in from local pharmaceutical company Amgen, and has demonstrated significant retinal protection in pre-clinical studies.

NT-501 implants are placed in the back of a patient's eye, releasing CNTF for up to 18 months. Because the capsule material is porous, the protein is released safely into the eye, but immune cells that would typically attack a foreign object in the body are too big to get into NT-501.

"From a technical aspect, it is unprecedented to deliver large molecule protein medication consistently in the body for up to 18 months," says Danse. "The product is constantly supplying these proteins to the eye."

Neurotech USA has already completed a successful Phase I trial of NT-501 with the National Eye Institute (NEI) in Bethesda, Maryland. NEI tested the safety and tolerability of NT-501 on 10 patients with late-stage retinitis pigmentosa (RP) for a six-month period.

Not only was the product considered tolerable and safe by these patients, but some patients actually experienced improvement in their visual acuity score. "Of the seven patients in the study with visual acuity that was able to be evaluated, three had some improvement in their vision," says Danse. "This was very encouraging because the deterioration of the patients' vision was so far advanced."

The next state of Neurotech USA's clinical development will evaluate the safety and efficacy of the implant on patients with late RP, early RP and dry AMD.

Developing this one innovative medical device took about 10 to 15 years, says Dr. Weng Tao, Neurotech USA's Chief Scientific Officer. Tao's work on ECT pre-dates the company's acquisition of the technology in 2000. The ECT technology actually originated at Brown University, in 1989 became part of the technology employed by one of the first biotech companies in Rhode Island to branch out on its own — CytoTherapeutics. Neurotech S.A., located in Paris, acquired the technology from CytoTherapeutics and established Neurotech USA.

"The technology was started at Brown, but it got stuck in the mud," says Danse. "There was a lack of funding and support. Now it is getting on track."

If Neurotech USA is successful with NT-501, numerous companies could be spun out in Rhode Island, according to Danse. "Rhode Island hasn't had a big, home-grown success in biotech yet," he says. "We are hoping to achieve that kind of success."