Intradetrusor injection of adult muscle-derived cells for the treatment of underactive bladder: pilot study.

We conducted the first-regenerative medicine cellular therapy for underactive bladder (UAB) in an FDA-approved, compassionate-use IND trial to evaluate treatment safety and potential clinical efficacy of autologous muscle-derived stem cells (AMDC) on a patient with UAB. No study-related adverse events or side effects were reported. In the 1-year follow-up period, the subject denied any gross hematuria, urgency, frequency or infection. A reduction in maximum cystometric capacity from 844 to 663 mL was observed, and the patient was able to void small amounts but continues to require self-catheterization 1 year after AMDC injection. Intradetrusor injection of AMDC is safe, minimally invasive and a promising treatment option for the UAB.

Novel Therapy with Intravesical Liposomes for Ulcerative Interstitial Cystitis/Painful Bladder Syndrome.

OBJECTIVES: A Federal Drug Administration-approved, compassionate-use, investigational new drug single-subject trial was conducted to evaluate the safety and clinical outcomes of intravesical instillation of liposomes in a woman with ulcerative interstitial cystitis/painful bladder syndrome (IC/PBS). METHODS: After obtaining informed consent, the 48-year-old woman, diagnosed with ulcerative IC/PBS, received four weekly instillations of intravesical liposomes. Subsequently she was evaluated for 8 weeks post bladder instillation. RESULTS: No side effects or adverse events were reported during the 12 week study period. Voids per day decreased from a baseline of 18 voids per 24 h to 11.3 voids per 24 h at week 3, and 12.6 voids per 24 h at 8 weeks after final instillation. Urgency score also decreased from a pre-instillation mean of 1.75 (out of 10) to 1.07 8 weeks after the final instillation. Bladder ulcers noted by cystoscopy at baseline were absent at the 8 weeks post-treatment and no evidence of bladder inflammation was noted. CONCLUSION: Intravesical liposome instillation is minimally invasive and presents an appealing new treatment for IC/PBS. Prospective trials are needed to assess intravesical liposomes for IC/PBS.

Comparing functional status and community integration in severe penetrating and motor vehicle-related brain injuries.

OBJECTIVE: To examine the functional status of persons surviving a severe penetrating traumatic brain injury (TBI) resulting from a gunshot wound who require inpatient rehabilitation. DESIGN: Data were collected prospectively at 4 different time periods: rehabilitation admission and discharge and year 1 and year 2 postinjury. SETTING: Rehabilitation hospital within a Traumatic Brain Injury Model System. PARTICIPANTS: Forty-five persons with severe penetrating brain injury and 45 persons involved in a motor vehicle crash (MVC). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Disability Rating Scale (DRS), FIM instrument, and Community Integration Questionnaire (CIQ). RESULTS: Results indicated functional improvements for both the penetrating and motor vehicle severe TBI groups on the DRS and the FIM from rehabilitation admission to discharge. Follow-up data at 1 and 2 years postinjury revealed continued improvements on the DRS and FIM measures for both groups, with the greatest improvement in recovery during the first year. In addition, improved community reintegration emerged between 1 and 2 years postinjury for both groups, as measured by the CIQ. There was a small significant difference on the outcome measures between the 2 groups in the course of their recovery. CONCLUSIONS: Persons who survive severe penetrating brain injuries and who require inpatient rehabilitation may show continuing improvement in functioning over time. For persons receiving inpatient rehabilitation services, initial improvement is most likely to occur during the hospital stay and continue postinjury, with the largest improvement in the first year after injury. Community reintegration can also be expected over time. One can expect similar outcomes for individuals who sustain a severe penetrating brain injury and a severe brain injury ensuing from an MVC.