Session Details

Plenary Session 6 - Advances in Cell-Based Therapies for Pediatric and Adult Patients
Saturday, June 1, 2019 01:30 PM - 03:00 PM
Plenary Hall
Chair:
Donald G. Phinney PhD, The Scripps Research Institute, Scripps Florida, USA

This Plenary Session will highlight recent advances and future challenges in the development of efficacious cell-based therapies.  The first presentation by Dr. Eleuterio Lombardo will provide an overview of the development of darvadstrocel, the first allogeneic stem cell-based therapy approved by the European Medicines Agency for treatment of complex perianal fistulas in Crohn’s disease. The talk will also provide a perspective on the present challenges in the field of MSC-based cell therapies. The second presentation by Dr. William Sietsema will review data from clinical trials involving over 700 patients treated with CD34+ cell therapy for treatment of advanced cardiovascular disease. The talk will highlight results from several trials, discuss mechanisms by which CD34+ cells replenish the damaged microcirculation in ischemic tissue and preview continued efforts to advance CD34 cell therapy on several fronts.  The final presentation by Dr. Joanne Kurtzberg will discuss results of preclinical and IND enabling studies of MSC-based therapies for treating pediatric patients with cerebral palsy and autism. The talk will provide information on safety and potential mechanisms of action of MSCs in these patients.

Speakers:

Present and future challenges of allogeneic MSC-based therapies: A personal perspective
Eleuterio Lombardo PhD, TiGenix (Takeda Group), Spain

2018 was the year that TiGenix (now part of Takeda) received the marketing authorization approval of darvadstrocel, the first allogeneic stem cell therapy (based on allogeneic adipose derived mesenchymal stem/stromal cells) approved by the European Medicines Agency, for treatment of complex perianal fistulas in Crohn’s disease. During this talk, a review of TiGenix experience of success, as well as a personal perspective of the present and challenges to be faced in the near future in the field of MSC-based cell therapies will be presented.

Single Administration CD34 Cell Therapy Reverses Cardiovascular Disease: Evidence from 700 patients in Clinical Trials
William Sietsema, PhD , Caladrius Biosciences, USA

As the population ages and the acute mortality from cardiovascular disease decreases, a large population of patients is emerging who have symptomatic chronic ischemic cardiac and vascular disease, many of whom remain severely symptomatic despite exhausting conventional medical therapy and mechanical revascularization. Mounting evidence suggests that microvascular insufficiency plays a significant role in the pathophysiology of ischemia. Recognizing this problem, investigators have worked to understand the biology of the vascular system and harness this information to develop new treatments for patients with ischemic diseases.  With this end in mind, the science of therapeutic angiogenesis has been evolving for over two decades.

Preclinical studies provided evidence for safety and document the mechanisms by which CD34+ cells replenish the damaged microcirculation in ischemic tissue.

Clinical trials involving over 700 patients have been completed providing data supporting the feasibility, safety and efficacy of CD34+ cell therapy for treatment of advanced cardiovascular disease. In No Option Refractory Disabling Angina (NORDA), Critical Limb Ischemia, Congestive Heart Failure and Coronary Microvascular Dysfunction a single administration of autologous CD34 cells have been shown to result in long term improvement in function and mortality. Accordingly, the goal of ischemic tissue repair appears within reach.  CD34 cell therapy has entered a pivotal clinical trial for patients with critical limb ischemia in Japan, a US study in CMD us under way and a pivotal clinical trial in NORDA is scheduled to start in 2019.

The evolution of the strategy of CD34+ cell based ischemic tissue repair will require an ongoing dialogue between clinicians, scientists, regulators, industry and payors to take full advantage of advances in our understanding of the biology of these processes and thereby drive their appropriate application to patients who are disabled with their condition and have exhausted conventional medical and surgical therapies.

Emerging MSC Therapies in Pediatric Patients
Joanne Kurtzberg MD , Duke University Medical Center, USA

Results of preclinical and IND enabling studies will be presented to provide information about safety and potential mechanisms of action of MSCs in cerebral palsy and autism. Data from early phase human clinical trials for safety and efficacy in these diseases will be presented by the speaker in this session.