Practitioner’s Post: The Promise of Stem Cells

Dr. Sunjay Kaushal is a cardiothoracic surgeon with a passion for reconstructing the hearts of those battling critical congenital heart disease.  During reconstruction, Dr. Kaushal also looks at the pediatric heart with an innovative eye.  In talking to Dr. Kaushal, it’s clear that he’s dedicated not only to reconstructing the hearts of our babes, but also in finding ways to improve their lives for many years to come.

Dr. Sunjay Kaushal
Thank you, Dr. Kaushal, for taking time out of your busy days to share your research and trials with Sisters by Heart and our families.  There is so much hope in medical advancements and your continued work. 
A few years ago, Dr. Kaushal began studying the positive outcomes with stem cell therapies, in the adult population, following damage to the heart after ischemic events (i.e. heart attacks.)  He asked himself a simple question:  If stem cells are assisting in the regenerative process for adults, can we use similar therapies for children?  The short answer, he found, is yes.
Over the past year-plus, Dr. Kaushal preliminarily proved his theory – that infant heart tissue containing specific stem cells has the ability to: 1) survive outside of the body, 2) grow and multiply (yes, like Gremlins) and, 3) assist in recovery and strengthen heart function when injected into the heart.  How did he prove this theory, you ask?  He did it by testing harvested human heart tissue from 150 HLHS and CCHD patients and injecting them into animal models.  Armed with quantitative information and data, he presented to the FDA for approval of a new clinical trial which is slated to begin soon at University of Maryland Medical Center.
Essentially, the clinical trial looks like this: 

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.

Even more exciting… when Dr. Kaushal compared his preliminary results in children to that of an adult heart, he’s finding that the infant cell models outperform the adult models “3 to 1.”  Therefore, the younger the stem cell, the more potent and effective it is in repair and regeneration.
Why does it work? 

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. 

How will we know if the clinical trial works?

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. 

Can my older HLHS child or HLHS adult benefit?

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.

What do we know now?

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.

What if I didn’t bank my child’s cord blood/tissue?

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.

Dr. Kaushal and his team hold much enthusiasm and cautious optimism about their new clinical trial which, once approved to begin enrolling patients, could change the landscape of medical treatment for HLHS infants, children and even adults.

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:

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: