Industry Update: Clinical trials

Written by Dusko ILIC

Latest clinical trial developments compiled from 01 — 30 June 2016

Latest clinical trials in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions from 01 — 30 June 2016, scheduled to be published in Volume 11 Issue7 of Regenerative Medicine.


Cellectis (France; announced that the first patient has been treated in the Phase I study of UCART19 in pediatric acute B lymphoblastic leukemia at the University College of London. This UCART19 clinical trial is sponsored by Servier (France; in close collaboration with Pfizer (NY, USA; The pediatric Phase I is an open label, non-comparative, monocenter study to evaluate the safety and ability of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19 positive B-cell acute lymphoblastic leukemia ahead of planned allogeneic hematopoietic stem cell transplantation. Cellectis will receive a milestone payment from Servier of an undisclosed amount. The company has also recently been selected as a 2016 World Economic Forum Technology Pioneer, based on its contribution to and impact on the global healthcare and life sciences industries via the development and therapeutic application of allogeneic CAR T-cell immunotherapies targeting cancer.


Celyad (Belgium, announced results for CHART-1 (Congestive Heart failure Cardiopoietic Regenerative Therapy), its European Phase III clinical trial for its lead cardiovascular disease product candidate, C-Cure®.The proprietary product consists of autologous bone marrow cells differentiated into cardiopoietic cells with a combination of cytokines and growth factors. CHART-1 trial is a prospective, controlled multi-center, randomized, double-blinded Phase III clinical trial comparing treatment with C-Cure® to a sham treatment. The trial recruited 271 evaluable patients with chronic advanced symptomatic heart failure in 12 countries in Europe and Israel. Although there was a positive trend across the complete trial population, no difference between treatment and control (sham procedure) was reached. However, for patients representing 60% of the overall study population and categorized by their End Diastolic Volume at inclusion, significance was met for the primary endpoint, and a statistical significant positive difference was seen in all individual elements of the composite primary endpoint (Mortality, Worsening Heart Failure Events, Quality of Life, 6 minutes Walking Test, End Systolic Volume and Ejection Fraction).


ImmunoCellular Therapeutics (CA, USA; announced that the first patient has been treated in the phase III trial of ICT-107, a patient-specific, dendritic cell-based immunotherapy targeting multiple tumor-associated antigens on glioblastoma stem cells. The phase III trial ( identifier: NCT02546102) is a randomized, double-blind, placebo-controlled study of 414 HLA-A2-positive subjects, which will be conducted at approximately 120 sites in the US, Canada and the EU. The primary endpoint in the trial is overall survival, whilst secondary endpoints include progression-free survival and safety. ImmunoCellular has also been honored with a US$19.9 million award from the governing Board of the California Institute for Regenerative Medicine (CIRM;, California’s stem cell agency, to implement the phase III registration trial.To support timely patient enrolment at participating sites across ten countries, ImmunoCellular has established agreements with the European Organization for Research and Treatment of Cancer (EORTC;, the Alliance for Clinical Trials in Oncology ( in the US, and the Canadian Brain Tumor Consortium (


Juno Therapeutics (WA, USA; announced that encouraging clinical data from JCAR015, a chimeric antigen receptor (CAR) T cell product candidate, support its strategic approach towards the commercialization of its first CAR T therapy. Updated results have been presented at 52nd Annual Meeting of the American Society for Clinical Oncology in Chicago. In a phase I clinical trial, 46 patients with relapse-remitting B cell acute lymphoblastic leukaemia received lympho-depleting chemotherapy followed by 19-28z CAR T cell infusion. Complete response and minimal residual disease-negative complete response rates were 91% and 71% in the minimal disease cohort, and 75% and 65% in the morphologic disease cohort, respectively. Additional information about this clinical trial is available in the abstract: and at (ID: NCT01044069).

Kite Pharma

Kite Pharma (CA, USA; delivered three oral presentations relating to its clinical programs at the 2016 European Hematology Association Annual Congress in Copenhagen, Denmark. Updated results from a phase I clinical trial ZUMA-1 ( identifier: NCT02348216) on the effect of treatment of 7 patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma with an anti-CD19 CAR T cells (KTEC19) therapy showed no adverse events after one-month post-dosing, with an overall response rate of 71% and a complete response rate of 57%. The promising results from a phase I/IIa study conducted as part of a Cooperative Research and Development Agreement between Kite and the National Cancer Institute evaluating anti-CD19 CAR T-cell therapy after low-dose chemotherapy in 22 patients with advanced lymphoma were also presented. Among 19 patients with diffuse large B-cell lymphoma of various subtypes, 8 demonstrated complete response, 5 partial response, 2 stable disease, and 4 progressive disease. A complete response was also noted in one patient with mantle cell lymphoma. Two patients with follicular lymphoma both obtained complete response. An additional oral presentation featuring data from the SCHOLAR1 study, a meta-analysis of outcomes in patients with chemorefractory diffuse large B-cell lymphoma, showed that response rates after chemotherapy and autologous stem cells transplantation ranged from 19% to 36% (complete responses from 2% to 18%), with a poor median survival, ranging from 4.6 months to 6.9 months.


The Riken Center for Developmental Biology in Kobe ( will resume a clinical study in which retinal tissues developed from iPSC will be transplanted to a person with an eye disease, in cooperation with Kyoto University and other medical institutes. Additional information on the study can be found at, the International Clinical Trials Registry Platform (ICTRP; under ID: JPRN-UMIN000011929, and at the Japan Primary Registries Network (JPRN; under ID: UMIN000011929.

San Bio

SanBio (CA, USA; has published the 12-month interim follow-up data from a two-year clinical trial of its SB623 stem cell treatment in patients with chronic motor deficits from ischemic stroke [1]. The trial was an open-label, single-arm dose escalation study of 18 patients. Patients had chronic motor deficits more than six months following their initial stroke. The group was split into three cohorts that each received different SB623 cell doses. SB623 cells, SanBio’s proprietary product, are modified allogeneic mesenchymal stem cells, derived from the bone marrow of healthy human adult donors. The data suggest the cell treatment was both well-tolerated and effective in promoting functional recovery in those living with motor impairments six months to five years following their stroke. Additional information about this clinical trial is available at (ID: NCT02448641).

Stem Cells

StemCells (CA, USA; has terminated the Phase II “Pathway” Study in spinal cord injury following an in-depth review of data from the study and after obtaining the concurrence of the study’s Interim Analysis Data Monitoring Committee. The Pathway Study was a single blind, randomized, controlled clinical trial investigating the use of StemCells’ proprietary human neural stem cells (HuCNS-SC) for the treatment of chronic spinal cord injuries. Patients eligible for the study had complete loss of motor control below the level of injury. Although the results showed overall improvement in patients treated with the Company’s proprietary cells, the magnitude of the effect and the perceived trend of the effect over time did not justify continuing the study or exploring the variability in the initial patient observations, given the financial resources available to the Company.


[1]Steinberg GK, Kondziolka D, Wechsler LR, Lunsford LD, Coburn ML, Billigen JB, Kim AS, Johnson JN, Bates D, King B, Case C, McGrogan M, Yankee EW, Schwartz NE. Clinical Outcomes of Transplanted Modified Bone Marrow-Derived Mesenchymal Stem Cells in Stroke: A Phase 1/2a Study. Stroke 2016;47(7):1817-24.