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Different forms of cell transplantation therapy are being tested in models of ALS. While the approach offers hope to patients with ALS, much still needs to be learned in the laboratory before it is ready for human trials. Nevertheless, clinics across the world offer various types of open label cell transplantation therapy for high fees. We report a woman who received an injection into each frontal lobe in Beijing, China. Her ALS progressed at a more rapid rate after the procedure and she suffered disabling side-effects. Clinics that give experimental and potentially harmful treatments outside the construct of well-designed clinical trials put patients at risk and do a disservice to the ALS community. [ABSTRACT FROM AUTHOR]

Objectives: The prognosis for adults with relapsed/refractory (R/R) B-cell precursor acute lymphoblastic leukemia (BCP-ALL) is poor. Blinatumomab is a CD3/CD19-directed BiTE® (bispecific T-cell engager) molecule approved globally for the treatment of BCP-ALL in adults and children. This multicenter open-label single-arm China registrational study evaluated the safety, efficacy, and pharmacokinetics of blinatumomab in Chinese adults with Philadelphia chromosome-negative (Ph-) R/R BCP-ALL (NCT03476239)., Methods: Patients aged ≥ 18 years were treated with up to 5 cycles of blinatumomab. The primary objective was to evaluate the hematological response rate (complete remission/complete remission with partial hematological recovery [CR/CRh]) within 2 cycles of blinatumomab., Results: At the interim analysis (April 12, 2019), 90 patients (median age 31.5 years [range: 18-74]; 53.3% female; 77.8% with bone marrow blasts ≥ 50% at study entry) were enrolled at 23 study centers in China and had received blinatumomab. As of data cutoff, 43 patients (47.8%) continued the study. The CR/CRh rate within 2 cycles of blinatumomab was 45.6% (41/90 [CR, 37; CRh, 4]; 95% CI: 35.0-56.4). Median overall survival was 9.2 months (95% CI: 6.5-11.7); median relapse-free survival was 4.3 months (95% CI: 3.2-9.4). Mean serum concentration at steady-state and systemic clearance of blinatumomab in Chinese patients were within the range reported in adults from global clinical trials. No new safety risks were identified in Chinese patients., Conclusions: The efficacy and safety of blinatumomab in these heavily pre-treated Chinese patients with Ph- R/R BCP-ALL is comparable to that for patients within global clinical trials.