The Cancer Center team hosted a celebration on Thursday, December 15, 2022 to thank their team for reaching a significant milestone: our 1,000th stem cell transplant. This accomplishment is the culmination of years of dedication, hard work, and innovation to make this treatment more accessible, safer, and effective for a variety of complex blood diseases.
In a recent interview, Medical Director of the Stem Cell Transplantation Program, Karen Ballen, MD, shares how far this program has come and what’s next for the future of this lifesaving treatment.
Karen Ballen, MD
What conditions can be treated with stem cell transplant?
Ballen: Most of the patients that we treat have blood cancers like leukemia, lymphoma, and myeloma. Transplant is curative for many of these blood cancers. Stem cell transplant is also used for other blood diseases, such as aplastic anemia, and sickle cell anemia. In children, stem cell transplant is curative for some genetic diseases. It’s also being studied for the treatment of autoimmune diseases, such as scleroderma and multiple sclerosis.
How has this treatment changed since our program began in 2012?
Ballen: One of the things that I find so exciting about this field is all of the advances that help our patients. My personal research has been focused on finding a donor for every patient. Ten years ago, many patients could not have a transplant because they didn’t have a matched donor. You’re most likely to find a donor who has your same ancestry. Because the National Marrow Donor Program is composed mostly of donors of Northern European background, it is difficult for patients who are of African, Latinx, Asian, and Eastern European descent to find matched donors.
We’ve really worked on this in the transplant field over the last 10 years so that everyone has a donor. There are several different options for patients right now. One option is umbilical cord blood, which is blood that’s normally thrown out when someone has a baby, but that can also be used for transplant. The good news about that is because the cells, from an immune standpoint, are little naïve baby ones, they do not have to be perfectly matched to the patient.
Another advance is something that we call haploidentical or haplo transplant, which means a half transplant. Most people will have a half-match stem cell donor in their family because parents and children will be a half-match. Then the third option, which is new really in the past year or two, is a donor from the transplant registry who is not a perfect match. Those patients are treated on a clinical trial because it’s new, but the results are also very promising. I’m really happy to say in 2022 we have not had to deny anyone a transplant because we could not find a donor. We’ve been able to find a donor for everyone.
The second big advance we’ve made is preventing and treating infection, such as fungal infection, that used to be fatal for transplant patients. Now, often they can be prevented — or if they do occur — can be treated with medication. Our program worked very closely with our team in infectious disease because infection can be a big problem in our patients. It’s nice to have that collaboration and know that there are so many new drugs that are available.
The third thing that I want to mention is drugs to prevent and treat something that we call graft-versus-host disease, which is an immune problem that occurs because the immune system of the patient and the donor are different. That can be very mild, but it can also be serious. So there are several new medications and strategies to prevent or treat this complication.
How has UVA been involved in advancing this treatment?
Ballen: All three of those processes I mentioned have been advanced through clinical trials and the work of our team here at UVA. We have participated in national trials and trials of our own to advance efforts to find donors for all patients, reduce the risk of infection, and reduce and treat graft-versus-host disease. We are also working on improving the diet of transplant patients and strategies to prevent relapse.
What’s in store for the future of the Stem Cell Transplantation Program?
Ballen: I think the future is bright, both for our program and for the field as a whole. One of the things that I enjoy about working in this field is that we can see advances that are done in the laboratory, readily translated, to help a patient within a few years and, in some cases, within even a few months. That’s really dramatic.
One of the new treatments are CAR T-cells, which stands for chimeric antigen receptor T-cells. That’s a new way of doing transplant and using the patient’s own immune system to help fight their cancers. That’s now FDA approved for certain types of blood cancers, and is in clinical trial for some others as well.
The UVA Stem Cell Program team is looking to advance CAR T-cell therapy, transplanting patients with non-cancers, such as sickle cell anemia, and autoimmune diseases. We are also doing research to try to prevent relapse of cancer after transplant by giving low-dose treatment after transplant — what we call maintenance — to try to prevent relapse. I think this is going to be the wave of the future in transplants. And I’m happy to say that UVA is leading that wave, and we have some exciting clinical trials in that area.
How has our team helped make this program successful?
Ballen: As anyone knows who cares for these patients, this is a very tough journey for patients and their families. And it can be months, sometimes even years, for patients to start to feel well and get back to their normal activities again. We are so fortunate to have such a dedicated team that is with the patient and their family every step of the journey, even after the patient returns to their own home and to the care of their local oncology team. We’re always still there for the patient. I think patients, their families and their doctors know that, and that is one reason why our team and our program has been so successful.
There are so many people involved that the patients never meet. I often tell patients that these are the people who are most important in their care. It’s the people who clean their rooms, make their food, obtain insurance approval, and read their pathology slides and their X rays. I really do want to give a shoutout to all members of the team and to let people know that, even if you never meet the patients, they are so appreciative of everyone’s efforts. So on behalf of the patients, I want to thank our entire team for their hard work and dedication, which has saved so many lives.
Article by Holly Ford
Photos by Kay Taylor
Filed Under: Clinical