Practitioner’s Post: The Promise of Stem Cells
Dr. Sunjay Kaushal
During the Norwood operation, heart tissue taken from the right atrium, which would routinely be discarded post cannulation, is instead banked for future use on that same patient. The harvested heart tissue is then grown to duplicate the patient’s own stem cells for reintroduction to the patient’s heart. Once expanded, the cells are placed back into the heart during the Glenn/hemi-Fontan operation via injection into the coronary arteries just prior to coming off bypass. The infusion of stem cells takes less than 4-5 minutes to complete.
The purpose of Dr. Kaushal’s trial is to determine how effective these specific stem cells are in regenerating heart tissue and strengthening the heart muscle/function in single ventricle patients. Obviously, a major risk factor with HLHS patients is heart failure. The theory is: inject the heart with its own, younger stem cells and the heart will regain a large percentage of function. So, is this possible? Dr. Kaushal believes so. He believes that based on the adult studies performed to date, the trial is both safe and effective. In the adult-based study, heart function improved by 12%. For those of you who are familiar with the term “ejection fraction,” the EF improved by 12%. That is a staggering improvement. Not only did the study show effectiveness, but it illustrated that the stem cells continued to work over a year’s time. At 4 months, the heart function/EF improved by 4%, at 8 months, 8% and over a year’s time an overall improvement of 12%. Dr. Kaushal cautions that we must remember that this is a Phase I study, which only examines safety issues. Resident stem cells will need to be evaluated for efficacy in a larger Phase II study that will be appropriately powered to eventually determine their efficacy.
Researchers aren’t exactly sure. Dr. Kaushal will attempt to answer that question during his clinical trials with HLHS patients. The two thoughts include: 1) the new stem cells are secreting good proteins and therefore are improving function and/or 2) the new stem cells assist in preventing stress to the older cells. Either way, it’s promising news for parents of children with HLHS or any CCHD.
Imagine if surgeons and physicians can inject stem cells into the hearts of our children and dramatically improve heart function… We know we got the chills when thinking of the possibilities…
Dr. Kaushal’s trial is awaiting hospital approval. He’s also investigating the possibility of securing funding from both the public and private sectors so that parents can enroll their children free of charge.
Dr. Kaushal’s proposed clinical trial will enlist 30 HLHS patients and follow them over a year’s time, post-stem cell infusion at Stage 2 palliation. He will conduct a baseline MRI to measure function the morning of Stage 2. After Stage 2 and the injection/infusion of stem cells, he’ll perform repeat echocardiograms and MRIs at 6 and 12 months post-op to compare the heart’s performance and regeneration. No other tests are needed. Dr. Kaushal pointed out that the right heart (i.e. right ventricle) is “not normal” and very hard to assess on echo and therefore, the MRIs are needed to gain an accurate measurement.
Yes. Plain and simple. Dr. Kaushal’s hope is that, once approved, his trial will illustrate and prove the theory that stem cell therapy is safe and effective for single ventricle infants and children. If that is the case, if a child or adult with HLHS, “down the road” experiences dysfunction, they can receive stem cell injections from banked/frozen cells. The frozen stem cells, after thawing for 2 months, are just as potent as they were before freezing.
Data for stem cell therapy in infants and children living with HLHS is very scarce. Dr. Sano (who developed the Sano shunt for a modification to the Norwood) is conducting similar research in Japan but is using a different type of stem cell (still from heart tissue.) Dr. Sano is injecting cells into heart muscle at one month of age, shortly after the Norwoodprocedure.
Additionally, researchers in Germany have successfully used bone marrow cells in HLHS children. They’ve injected 10 CCHDers (not all HLHS) with autologous bone marrow stem cells and are seeing dramatic improvements in function. In fact, one child – an HLHSer in heart failure and listed for transplant – was injected with bone marrow cells while on the transplant list. The parents were willing to do anything to avoid transplant. And another lesson in asking many, many questions comes into play. They talked to doctors about their options and were told about the experimental bone marrow cell infusions. The parents agreed to the infusions and were ecstatic at its results. The child’s heart function improved significantly and subsequently, the child was removed from the transplant list. Based on his conversations with German researchers, Dr. Kaushal is gearing up for a similar study here in the United States using bone marrow cells for treating HLHS and dilated cardiomyopathy patients with depressed function.
Don’t panic. The body is full of stem cells that can be harvested. Dr. Kaushal emphasized that there are no current treatments for HLHS, or any CCHDs, utilizing umbilical cord blood stem cells, although there are current studies ongoing at Duke University regarding the use of umbilical cord blood therapy for HLHS patients. Dr. Kaushal recommends, if you have the resources to bank cord blood/tissue, go ahead and bank. Medicine evolves and you never know what options will be on the table 5, 10, 15 years from now.
Trust us when we tell you, Dr. Kaushal, we’re ALL cheering you on and sending all the positive energy we can muster. Our children’s lives depend on it.
Dr. Kaushal, with his patient, during a trip to Ecuador with the International Children's Heart Foundation
Watch Dr. Kaushal’s discussion on his clinical trial: http://www.umm.edu/media/video/kaushal-circ-study.htm
Sunjay Kaushal, M.D., Ph.D. is the Director of Pediatric Cardiac Surgery at the University of Maryland Medical Center. His special interests include pediatric CT surgery, neonatal heart surgery, fontan conversion, valve repair, adults with CHD and research with a focus on pediatric patients with HLHS and Cardiomyopathy. Learn more about Dr. Kaushal or contact him at: http://www.umm.edu/doctors/sunjay__kaushal.html#ixzz278rjvxNM