Recent approved therapies for AD have been in the form of monoclonal antibodies targeting the accumulated end-products of neuron self-maintenance. We report our updated results testing the safety of using Wnt-expressing stem cells injected directly into the ventricles of the brain. 

Patients were screened for age less than 80 years, FAST stages 4 or 5, MMSE between 11 and 20, as well as exhibiting brain amyloid PET and CSF AD markers. These two cohorts were the first two of three cohorts of escalating autologous stem cell dosages (2 million, 5 million and 10 million cells.) The 6 patients underwent: liposuction, reservoir implantation, and a single injection following cell expansion and selection based on Wnt expression (the test product). The patients were injected a median of 53 days (range: 43-62) after initial lipoaspirate, and observed overnight. Vital signs (VS) and clinical examinations were performed per protocol. MRI, cognitive testing and Amyloid PET imaging were performed at specific intervals.

The test product injection process required median of 8 minutes to perform, without anesthetic. This was well-tolerated with no reported adverse events in all 6 patients, including immediate or remote headache. CSF analysis in the 6 patients showed a decrease in p-Tau from a median of 61.9 pg/ml (range: 46.9 to 152.8) to 29.8 (range 13.2 to 66.6) at 12 weeks. Amyloid PET scan centiloid scores decreased from 125.4 (range: 53.33 - 155.47) to 95.8 (range: 55.8 to 168.1) at 12 weeks. ADAS-Cog scores improved from a pre-injection median of 58.8 (range 40-69) to 50 (range: 37-69).

This Phase 1 trial of intracerebroventricular injection of Wnt-expressing, adipose-derived stem cells proved well-tolerated and safe. Phase 2 will ensue for AD, MS-P, ALS and CTE.