Trial of Fibroblast Growth Factor-1 Shows Safety, Suggests Efficacy

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by Lindsey Shapiro, PhD |

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Multiple blood vessels surround a profile image of the human brain.

A first study in patients of an intranasal-delivered  therapy for Parkinson’s disease is now complete, and early findings support the treatment being safe and leading to improvements in motor function, Zhittya Genesis Medical, the therapy’s developer, announced.

Zhittya is offering a first look at the trial data via a free Zoom webinar set for June 23 at 3 p.m. PT (6 p.m. ET). Registration is available online.

“Please join us for our Zoom webinar on June 23 where you can view this videotaped data with your own eyes and also hear from the test subjects themselves,” Daniel Montano, Zhittya’s CEO, said in a press release.

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“We are still in the early phases of discovering what is the best dose, the best duration of treatment, etc., however, I believe we are on a great start to address and resolve Parkinson’s disease issues for millions of people,” Montano added.

The release did not detail the number of Parkinson’s patients involved, other than to say it was a small study, or where the trial took place.

Evidence has suggested that Parkinson’s may arise from disrupted blood flow in small blood vessels surrounding the brain’s dopamine-producing nerve cells — those that are progressively lost in the neurodegenerative disorder. As a result, these nerve cells don’t receive enough nourishment and die off, the company stated.

Zhittya’s compound is a proprietary version of fibroblast growth factor-1 (FGF-1), a protein that is able to stimulate blood vessel growth, called angiogenesis, in damaged or oxygen-deprived tissues. Its aim is to restore blood flow, allowing nerve cells to be regenerated. FGF-1 is administered directly into the brain via the nasal cavity using a proprietary intranasal device, Zhittya states.

The company has reported that studies in monkeys showed “genuine disease modifying effect as it [the investigational treatment] successfully regenerated new dopamine neurons in the brain.”

Intranasal delivery of FGF-1 “is incredibly simple and non-invasive and avoids the use of central catheters (picc lines) [for intravenous delivery] that had been originally envisioned,” said Jack Jacobs, PhD, president and chief scientific officer at Zhittya.

Earlier this year, Zhittya announced it would launch an open-label (meaning no placebo group) “compassionate use” pilot trial to test the therapy’s safety and efficacy, administered using the intranasal device, in people with mild-to-moderately severe Parkinson’s.

It is not clear if results to be reported in the Zoom call concern this pilot trial.

Dosing in the trial began on May 3 and was completed by May 30, Zhittya stated in the release, with patients receiving two escalating doses of intranasal FGF-1. No adverse events have been reported in those treated, it added, and an analysis of videos taken before and after treatment indicated signs of motor skill improvement over the first month.

“Although this first study was mainly geared to test the safety of intranasally-administered FGF-1, we were pleased to see evidence of patient improvement, both on physical examinations and filming of motor skill functions, as well as testing with the standardized Parkinson’s Disease Questionnaire,” Jacobs said.

Participants will return for follow-up sessions at 60 and 90 days (about two and three months) to be monitored for side effects and further changes in motor function.

Zhittya also announced that trial findings will be added to a request filed with the U.S. Food and Drug Administration, with plans to open a clinical trial of FGF-1 in the country within two years.

A Phase 1 clinical trial of the investigational therapy was cleared to start in Mexico in 2020; its status is not known.