What I wish I would have known as a transplant center MD

A Q&A with Dr. Heather Stefanski, Vice President of Medical Services

September 24th, 2021

Heather Stefanski, MD, PhD, understands the unique challenges transplant centers face. Before joining the National Marrow Donor Program® (NMDP)/Be The Match® as the Vice President of Medical Services, she spent 27 years treating patients at the University of Minnesota (U of M) Division of Pediatric Blood and Marrow Transplant and Cellular Therapy.

In part 1 of our Q&A with Dr. Stefanski, she shares:

  • What she wishes she would have known and what surprised her about the NMDP/Be The Match that other transplant physicians should know
  • Her opinion on the greatest benefit of cord blood as a graft source
  • What the future holds for cord blood in transplant and emerging cell and gene therapies

When you joined the NMDP/Be The Match, what did you learn that you wish you would have known when you were practicing at the U of M?

There are two things that stand out to me. First, I wish I had known about the Cure-Ready cord blood units available through Search Strategy Advice, which are select, ethnically diverse units with high TNC/CD34+ counts. They’ve been typed using high-resolution NGS typing and have had release testing/potency testing completed, which eliminates the typical 14-day wait for testing results.

I used cord blood almost exclusively in my pediatric transplant practice unless a matched sibling was available, so I likely would have used Cure-Ready.

But that also brings me to the second thing I wish I would have known. I typically turned to cord because I could get it fast. I thought it took a minimum of two months to get an unrelated donor when, in fact, it can take as little as two to three weeks.

That may have changed my approach of automatically turning to cord if there wasn’t a sibling match.

What surprised you about the NMDP/Be The Match that might surprise other transplant centers, too?

I knew that the NMDP/Be The Match was involved with research, but I had no idea the breadth of the research and its ability ot quickly impact patients' lives. Heather Stefanski, MD, PHD, Vice President of Medical Services NMDP/Be The Match

I was surprised to learn that the NMDP/Be the Match was so much more than just helping to find a donor. There are the Cure-Ready cord blood units, Be The Match BioBank for cryopreservation, and the CIBMTR®. The NMDP/Be The Match also has many programs for patients, like the Patient Financial Assistance Program and the Patient Support Center.

As a transplant physician, I was unaware of just how much goes into the logistics of delivering the products to transplant centers. There are visa requirements. Weather impacts. The list goes on and on. While that wouldn’t have changed my practice, it would have given me a greater appreciation and understanding of the complexity of getting a donor and receiving that life-saving stem cell product.

Lastly, I knew that the NMDP/Be The Match was involved with research, but I had no idea the breadth of the research and its ability to quickly impact patients’ lives. Also, as physicians, we are very familiar with the CIBMTR, but we need reminders that the CIBMTR is a research partnership between the NMDP/Be The Match and Medical College of Wisconsin.

What drew you to join the NMDP/Be The Match after a 27-year career at the University of Minnesota?

I was looking for a new challenge and wanted to be able to impact patients on a national and global level. The NMDP/Be The Match has a very similar mission to my own, which is to improve the lives of transplant and cellular therapy patients.

It was very easy to get onboard with the organization’s mission of saving lives through cellular therapy. I’m excited to be in this role and appreciate the role NMDP/Be the Match has within the transplant community.

You mentioned you used cord blood almost exclusively as a pediatric transplant physician. What is the greatest benefit of cord blood as a graft source for transplant?

I would say the best thing about cord blood is that it does not typically cause severe acute graft-versus-host disease (GVHD) compared to other stem cell sources. If a patient that receives cord blood does have acute GVHD, it is most likely skin GVHD which is much easier to treat than GI GVHD.

In addition, you can get away with less of a match than you can with an unrelated or related donor, at least right now. That could change in the future as the NMDP/Be The Match is currently conducting the ACCESS clinical trial. That study will assess whether transplant using PBSC in adults or marrow in children from a mismatched unrelated donor in combination with PTCy is safe, feasible and results in a high likelihood of overall survival.

We know we can safely use mismatches in cord blood. If we can do the same with unrelated donor transplants, it would be phenomenal news for patients because it would greatly expand access to treatment with potentially fewer side effects.

What does the future look like for cord blood both as a graft source for transplant and for emerging cell and gene therapies?

A lot of research is underway in the cell and gene therapy industry that uses cord blood as a graft source. There's a lot of possibility with cord. It's an exciting time.

There is a lot of research underway in the cell and gene therapy industry that uses cord blood as a graft source. As a physician scientist at the University of Minnesota, I was able to make T-progenitors from cord blood. I’m hoping that work will eventually move forward to a clinical trial. Other investigators have been able to make T-regulatory cells, NK cells and even iPSC cells from cord blood, just to name a few possibilities.

We know some physicians are cautious about using cord blood for transplant because it does take longer to engraft and, for adults, a double cord is often necessary, so research like this could expand the use of cord blood in transplant in the future.

There’s a lot of possibility with cord. It’s an exciting time.

Watch for the second part of our interview with Dr. Stefanski where she’ll discuss cell and gene therapy, how it may impact the use of transplant in the future, and how the NMDP/Be The Match can support centers in delivering next-generation cell and gene therapies to patients.