Cell Therapies
There is no denying that cell therapy has the potential to be one of the most powerful therapeutic options available. Cell therapy can take several forms and serve many purposes including altering normal cell response, stimulating native signaling cascades, performing missing metabolic functions, restoring lost tissue, or changing the normal course of repair into true regeneration. Designing and implementing a plan for commercialization takes many paths, each with their own set of biological, technical and regulatory considerations.
WEDNESDAY, AUGUST 13
7:30 Breakfast Technology Workshop (Sponsorship Available)
HARNESSING CELLS AS THERAPIES
8:30 Chairperson’s Remarks
Nancy L. Parenteau, Ph.D., Co-founder and Managing Member, Parenteau BioConsultants, LLC
8:35 The Clinical Application of Corneal Epithelial Stem Cells: From Bench to Bedside
Sajjad Ahmad, Ph.D., Clinical Lecturer, North East England Stem Cell Institute
The cornea at the front of the eye is covered by an epithelium, which is renewed by stem cells located at the corneal periphery, in a region known as the limbus. These so-called limbal stem cells are deficient in the painful and blinding disease of limbal stem cell deficiency. In this talk, the successful animal-free culture and transplantation of limbal stem cells for limbal stem cell deficiency will be described. In addition, we have developed a protocol for differentiating human embryonic stem cells into the corneal epithelial lineage and the results from this will be discussed.
9:05 Use of Fluorescent Quantum Dot Bioconjugates for Cellular Imaging and Monitoring of Bone Marrow Cell Homing into the Rat Myocardium in Myocardiopathy Model
Nasser Aghdami, M.D., Ph.D., Immunology, Royan Institute
In some experimental settings, transplantation of stem or progenitor cells after myocardial infarction reduces scar formation and fibrosis, and preserves cardiac function, but the biodistribution of the therapeutic cells after delivery and the specific mechanism by which therapeutic cells contribute to functional improvement remain to be investigated. So using the peptide-based reagent QTracker to label rat and human Stem cells with QDs and evaluate the utility of QDs for imaging stem cell therapy in heart diseases model. Next evaluation of QD™ potential for imaging stem cells in vitro and in vivo.
9:35 Developing Stem Cell-Based Therapies for Cancer
Khalid Shah, Ph.D., Massachusetts General Hospital, Harvard Medical School
Novel therapeutic agents are emerging as important treatment options for cancer. However, inefficient delivery and the lack of non-invasive methods to follow delivery, pharmacokinetics and efficacy has been a limitation in elucidating their effects in vivo. Neural stem cell (NSC) homing to tumors in the brain represents an attractive modality for onsite-delivery of therapeutic molecules to tumors. We have recently shown that engineered human NSC expressing novel secretable anti-angiogenic and pro-apoptotic proteins have anti-tumor effects in culture. Using a highly malignant human glioma-model and employing real-time-imaging, we show that secreted therapeutic proteins are continuously delivered by NSC, target both the tumor cells and the vascular-component of gliomas and have profound anti-tumor effects. These studies demonstrate the strength of employing engineered stem cells and real time imaging of multiple events in preclinical-therapeutic tumor models and form the basis for developing novel cell based therapies for cancer.
10:05 Technology Spotlight (Sponsorship Available)
10:20 Networking Coffee Break, Poster and Exhibit Viewing
11:00 Engineering T-Cells for Cancer Gene Therapy
Robert Hawkins, Ph.D., Professor, Cancer Research and Director of Medical Oncology, University of Manchester
Advanced cancers evade immune recognition and suppress immune responsiveness. Adoptive cell therapy from tumour infiltrating lymphocytes can be effective in melanoma but for many other cancers tumour specific T-cells are rarely found. Genetically engineering T-cells to express receptors based on T-cell receptors or antibodies recognising tumour associated antigens is a simple and effective approach to producing large numbers of tumour targeted T-cells. Pre-clinical models demonstrate the efficacy of the approach and the value of pre-conditioning lymphodepleting chemotherapy to enhance surivival of the engineered T-cells. Trials testing these approaches are commencing targeting gastrointestinal and B cell malignancies. The EU ATTACK project (www.ATTACK-cancer.org) is focused on further development of this approach.
11:30 Adipose-Derived Stem and Regenerative Cells Improve Lameness and Pain in Dogs with Osteoarthritis: A Summary of Results from Three Clinical Trials
Robert Harman, D.V.M., CEO, Vet-Stem
Since 2003, veterinarians have treated more than 2,500 horses with adipose-derived stem and regenerative cells for orthopedic injuries. In 2006, Vet-Stem began three clinical studies to evaluate the use of this cell therapy in dogs with osteoarthritis (OA). The interim results are presented here. The first study evaluated cell therapy in OA of the canine elbow (non-blinded prospective study). A second randomized, prospective, blinded, placebo-controlled study evaluated cell therapy in OA of the canine hip. The last study, a non-blinded, prospective study, examined cell therapy in end-stage, chronic, post-surgical OA in the knee of the dog. The clinical outcome measures included lameness, pain, range of motion, and functional disability scores. All three studies demonstrated highly statistically significant improvements in dogs treated with cell therapy when compared to historical baseline values. In addition, the prospective, blinded hip study showed a statistical improvement in treated versus placebo-treated dogs. Safety, dosing, and statistical outcomes will be presented.
12:00 pm Luncheon Technology Workshop (Sponsorship Available) or Lunch on Your Own
1:15 Session Break
REGULATORY AND CLINICAL STRATEGIES FOR COMMERCIALIZATION
1:30 Chairperson’s Remarks
Lee Buckler, Business Development & Marcom Management Consultant, Progenitor Cell Therapy LLC
1:35 Developing a Strong Scientific and Commercial Strategy for Your Cell-Based Product
Nancy L. Parenteau, Ph.D., Co-founder and Managing Member, Parenteau BioConsultants, LLC
Cell therapy was first envisioned as a way to replace lost, damaged or deficient tissues. With experience, we have learned that in many ways cell therapy acts through its interaction with the body rather than its incorporation into functional tissue. This has opened the door to the possibility of using cells, particularly stem cells, as therapeutics rather than transplants. However, the potential offered by cell transplantation still remains. Regardless of the product approach, the true clinical potential of cell-based therapies will lie in achieving success in both realms. Although seemingly separate in mode of function, those two modes of action are actually intimately linked. This talk will discuss the similarities and differences in strategic challenges involved in the development of a cell-based therapy as a therapeutic and as a transplant, highlighting their points of intersection.
2:05 Engineering hESC to Create a Cell Therapy to Treat Insulin Requiring Diabetics: Promises and Challenges
Anne Bang, Ph.D., Associate Director, Stem Cell Technologies, Novocell Inc.
Of paramount importance for the development of cell therapies to treat diabetes is the production of sufficient numbers of pancreatic endocrine cells that function similarly to primary islets. We have developed a differentiation process that converts human embryonic stem cells (hESC) to endocrine cells capable of synthesizing the pancreatic hormones insulin, glucagon, somatostatin, pancreatic polypeptide and ghrelin. The hESC-derived insulin-expressing cells produce insulin in response to glucose in an in vivo animal model. Production of these hESC-derived endocrine cells represents a critical step in the development of a renewable source of cells for diabetes cell therapy. The therapeutic application of hESC-derived endocrine cells will require demonstrating that these cells are stable and show an appropriate safety profile in preclinical studies.
2:35 Therapeutic Dosage and Scale-Up Considerations
John E. Davies, Ph.D., Tissue Regeneration Therapeutics Inc.
3:05 Networking Refreshment Break, Last Chance for Poster and Exhibit Viewing
3:35 Stem Cell Therapy for Ischemic Brain Injury: Strategic Considerations
Robert Mays, Ph.D., Senior Director, Regenerative Medicine, Athersys, Inc.
Clinical development for cell therapy of stroke and related brain injuries is complicated by the high failure rate of neuroprotective drugs. Have pre-clinical data established a therapeutic paradigm for cell therapy distinct from drug pharmacokinetics to provide confidence to the investment community and support clinical studies? Our translational experience with adult stem cell development for stroke and neonatal hypoxic ischemia will be presented with an emphasis on Regulatory experience and financial support avenues to move through the clinic.
4:05 Commercial Manufacturing of Autologous Cell Therapies at Genzyme Biosurgery
Leslie Wolfe, Vice President, Genzyme Discovery, Genzyme Biosurgery
This talk will outline the production and quality control elements for the manufacturing of autologous cell therapies, specifically Carticel and MACI (Matrix-Assisted Autologous Chrondrocyte Implantation). Both of these products are used for the repair of cartilage defects in the knee by isolation and expansion of chrondrocytes from a patient’s healthy cartilage biopsy. Carticel was the first cell therapy product to receive a Biologics License from the FDA. Consideration on how cell culturing is handled in a GMP environment and providing service to patients and surgeons, will be described.
4:35 Panel of Experts Discussion
Business Models for Commercialization
Moderator: Lee Buckler
Panelists: Dawn Applegate, Bob Harman, Robert Mays, Nancy Parenteau, Leslie Wolfe
5:30 Close of Conference