LBA-04\u2003INTRADETRUSOR INJECTION OF ADULT MUSCLE-DERIVED CELLS FOR THE TREATMENT OF UNDERACTIVE BLADDER: INTERIM REPORT ON SAFETY AND EFFICACY (NCT02463448)

Abstract

INTRODUCTION AND OBJECTIVES: INTRODUCTION & OBJECTIVE: Underactive bladder (UAB) affects >15% of the population yet no consensus treatment beside catheterization. This interim report is the first regulatory approved clinical trial of a two-year prospective open label study assessing the safety and efficacy of intradetrusor injected autologous muscle-derived cells (AMDC) treatment for UAB. METHODS: Approved by Beaumont IRB; 20 non-neurogenic UAB were enrolled and 15 have received treatment thus far. Baseline evaluation included history, physical, voiding diary, multichannel urodynamic testing and endoscopy to confirm no obstruction. Approximately 150 mg of quadriceps femoris muscle was collected using a spirotome 8-gauge needle, sent to Cook MyoSite (Pittsburgh, PA), processed and upon reaching a final concentration of 125 million AMDC, patient received 30 intradetrusor injections of 0.5 mL delivered to the bladder, for a total of 15 mL. performed utilizing a flexible cystoscope under direct vision using topical local anesthesia. Follow-up assessments included adverse events and efficacy at 1, 3, 6 & 12-month post-injection. Optional second injection was offered at the end of 6 months visit. RESULTS: 20 patients biopsied, 15 received one injection, and 12 a second injection. Age range from 41-82, median 65 years old. 15 males and 5 females, etiology BPH post TURP in 7 and idiopathic in 13. Bladder function at baseline: CIC only, 10, voiding only, 1 and mixed CIC/voiding, 9. Table 1 is the result on 15 subjects to date based on participants Global Response Assessment (GRA) collaborated by the voiding diary report, showing a gradual increase in the number of responders with regards to bladder function responsiveness over time, 27%, 31%, 58% & 80% at 1, 3, 6 & 12-mo follow-up visit respectively. No serious procedure or treatment-related AEs occurred. No AEs related to AMDC product were reported. All biopsy and injection-related AEs were expected complications, and either self-resolved or were easily treated. CONCLUSIONS: Interim analysis up to 12 months revealed encouraging improvement in bladder function and safety with intradetrusor AMDC for UAB. Cellular therapy may be a promising novel treatment for UAB and multicenter controlled trials needed. Table. No title available. Source of Funding: Robert B. & Ann Aikens Center for Neurourology Research