Passage Bio released promising preliminary results from its phase 1/2 clinical trial evaluating an experimental treatment for FTD cases caused by a GRN genetic mutation (FTD-GRN).

The “upliFT-D” trial is evaluating PBFT02, a gene therapy that uses an engineered virus (known as a “vector”) to deliver an un-mutated copy of the GRN gene. FTD-GRN is caused by variations in this gene, which can result in an inability for the body to produce the protein progranulin. Low amounts of progranulin have been linked to hereditary FTD.

In Passage Bio’s clinical trial, PBFT02 resulted in a significant increase in progranulin in participants’ cerebrospinal fluid after 30 days, rising to levels that surpassed those in persons without FTD. Progranulin concentrations remained higher than baseline six months after being dosed.

The safety data of PBFT02 was also promising – while one participant experienced adverse effects consistent with an immune response, the trial team compensated by adjusting the steroid regimen given to participants to assist in immunosuppression. The gene therapy was generally well-tolerated in participants who received the adjusted treatment.

“We are excited to see these initial results from the upliFT-D trial,” said AFTD CEO Susan L-J Dickinson, MSGC. “Passage Bio’s encouraging early results provide much hope for families affected by FTD-GRN and reflect a growing momentum in the search for treatments for all forms of FTD.

“We look forward to seeing this momentum continue to build in 2024 — and to continue AFTD’s own efforts to hasten the development of effective FTD treatments,” she added.

Studies are actively seeking participants. In particular, people with FTD caused by GRN genetic mutations are actively needed for upliFT-D and other trials testing experimental, but possibly effective, treatments for FTD-GRN.

Are you interested in contributing to FTD research? Sign up for the FTD Disorders Registry, a secure electronic database that collects insights from persons diagnosed, care partners, and family members. Registrants can take part in survey research online and receive updates as FTD studies emerge for which they may be eligible to participate.