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Overcoming Challenges in Cell & Gene Therapy Trials

Explore Cell Therapy podcast season 2, episode 6 

This two-part "Explore Cell Therapy" podcast episode features a panel discussion from Advanced Therapies Week 2023, a conference focused on advancing cell and gene therapy. In a session sponsored by NMDP BioTherapies® and moderated by Chris McClain, MBA, Heather Stefanski, MD, PhD, Erin Leckrone, MBA, and Gabbi Giammarino, BS, discuss the current state of the cell and gene therapy industry to ramp up clinical trials and expand access to care.

Listen as they examine what's currently happening in the industry, the biggest challenges facing clinical trial design and management, and what to look for in a research partner to ensure trial success and advance patient outcomes. You'll gain insights into the future of cell and gene therapies and the role both hematology/oncology practices and transplant centers play in advancing clinical trials and access to these emerging treatments.

Key takeaways from this podcast

  1. Study design must be implementable and reasonable for patients, as well as for the hematology/oncology practices or transplant centers where enrollment is taking place. This means considering what is being measured against the trial goals and thinking strategically about how to best alleviate site and patient burden.
  2. Choosing the right sites and the right patients for cell and gene therapy trials is critical to the success of the trial and patient safety. While the long-term goal is for CAR-T and other therapies to move to community practices and outpatient settings, centers with the ability to manage acute toxicities are necessary, in addition to targeting patients with a low probability of suffering from toxicity. 
  3. Slow recruitment, enrollment, and lack of diversity in clinical trials is an ongoing problem in the field. Strong relationships between enrollment sites and research sponsors, open communication, and mindful protocol design can all help recruitment go more smoothly.
  4. The cost of cell and gene therapy clinical trials can be difficult to predict, and efforts need to be made to better plan for and reduce these costs. When considerations for reimbursement of standard of care procedures versus trial procedures aren't well accounted for early in the protocol planning, costs can end up falling back on the site or the patient. 
  5. Close collaboration with research sponsors early and often is key to avoid challenges in trial implementation, enrollment, and cost. This means making sure expectations are clear from the start, limiting late-stage trial amendments, and being overly communicative at all stages of the process.

This episode's expert guests 


Heather Stefanski, MD, PhD

Vice President, Medical Services

NMDP

 

 

 

 

 

Dr. Stefanski joined NMDP in May, 2021 as Vice President, Medical Services. Prior to her arrival, Dr. Stefanski was an associate professor of pediatrics and transplant physician at the University of Minnesota and was actively involved in clinical care and research for children with life-threatening blood and immune system disorders. One of her areas of focus is the use of cord blood units in stem cell transplants and other cellular therapy. She is board certified in both pediatrics and pediatric hematology-oncology.


Erin Leckrone, MBA

Senior Director, Clinical Trials

NMDP

CIBMTR® (Center for International Blood and Marrow Transplant Research®)

 

 

 

Erin Leckrone is the Senior Director of Clinical Trials at NMDP. She manages of the activities of the CIBMTR CRO Services, including the Survey Research Group, and oversees the administration of the Clinical Trials Advisory Committee. Prior to joining NMDP, she spent almost 14 years at Boston Scientific in Clinical Operations and served 8 years as a Medic in the Army National Guard.


Gabbi Giammarino, BS 

Blood and Marrow Transplant Clinical Research Coordinator

Moffitt Cancer Center

 

 

 

 

 

Gabbi Giammarino is a graduate from the University of South Florida with a degree in Biological Health Science. She is currently working as a Blood and Marrow Transplant Clinical Research Coordinator with experience in both adult and pediatric patient populations. Over the previous two years, she has worked on various trial designs and phases with concentrations not only in transplant, but in secondary complications such as GVHD.


Chris McClain, MBA (moderator)

Senior Vice President, Sales and Business Development

NMDP BioTherapies

 

 

 

 

 

 

Chris McClain is the Senior Vice President of Sales and Business Development for NMDP BioTherapies. His team engages with cell and gene therapy companies developing innovative, life-saving therapies that can leverage the cell sourcing, cell therapy supply chain, research support and outcomes tracking capabilities of the NMDP. Before joining NDMP BioTherapies, Chris was co-Founder of Nora Therapeutics, Inc., a venture-backed biopharmaceutical company developing novel therapies in areas of significant unmet medical need in reproductive health. 

Access all "Explore Cell Therapy" episodes 

Listen to "Explore Cell Therapy" on Spotify

Read articles or access trials discussed in this episode 

Resources for physicians 

NDMP BioTherapies
Explore NDMP BioTherapies, dedicated to accelerating cell and gene therapy development and optimizing cell collection, donor identification, and supply chain management.

HCT Consultation Timing Guidelines
The HCT consultation timing guidelines from the NMDP and ASTCT identify appropriate referral timing for allogeneic or autologous HCT based on a patient's disease characteristics

Transplant Center Directory for hematology/oncology physician
This searchable database provides details on the transplant centers in the NMDP Network, such as center contact information, number of transplants by cell source, patient survival information and more.

Evolution of HCT Self-Paced Learning Course
This five-part self-paced learning course will help you better understand the current state of the science for HCT and cell therapy by following the evolution of HCT and addressing common misconceptions. Part 2: Eligibility in Transplant contains a section specific to CAR-T therapies.

Resources for your patients 

NMDP Jason Carter Clinical Trials Search and Support Program
The Clinical Trials Search and Support Program helps patients find and join clinical trials through one-on-one support, an easy-to-navigate website, and financial assistance to help patients pay for travel costs to participate in a clinical trial.

NDMP Patient Support Center
The NMDP Patient Support Center provides support, information and resources for transplant patients, caregivers and families. All our programs and resources are free.