August 29, 2010

Post-Infusion Report

Bertrand receiving his stem cell infusion.

The past two weeks have been surprisingly easy for Bertrand, but tough on his mom and dad. Thanks to all of you who sent thoughts of love and support our way. We couldn't have done it without you. :) We apologize for the lack of an update following such a big event for Bertrand.

Bertrand underwent an autologous hematopoietic stem cell infusion on Tuesday, August 24 at Duke University. In other words, he was given stem cells from his very own umbilical cord blood collected at birth.

Umbilical cord blood consists primarily of red blood cells, white blood cells, platelets, plasma and there are a few hematopoietic stem cells in this mix (in Bertrand's case, a meager 160 million). When a child's cord blood is "banked", the processing center does its best to harvest and save the hematopoietic stem cells through centrifugation, which stratifies the cellular components. Dimethylsulfoxide (DMSO) is then slowly added as a cryoprotectant--a type of cellular antifreeze--and just the stem cells, with a few sneaky red blood cells and plasma, are frozen.

[NOTE: DMSO is the stuff which made Bertrand smell like creamed corn for over 24 hours after the infusion. It is also the reason why Bertrand's IV was first filled with benadryl and solu-medrol, a steroid. Around 1% of kids are allergic to DMSO and you don't find out until the infusion begins. To prevent an allergic reaction to DMSO from occurring during the infusion, patients are preventatively treated for allergic reaction. Which in Bertrand's case meant he slept through the entire thing!]

A bag full of hope: 160 million stem cells.

Why save just the stem cells***? They are the cells capable of differentiating into a diverse range of specialized cell types. And amazingly, they have a propensity to specialize and repair at an area of insult, such as an area of the brain damaged by oxygen depravation.

With Dr. Kurtzberg at the forefront, researchers have been studying this phenomena: that stem cells specialize when they reach areas of insult (injury) in the body--in particular the nervous system (spinal cord and brain). Our understanding of this mechanism is growing rapidly with advances in science, but there is a great deal left to learn, since it varies greatly by individual and injury. What this means, using cerebral palsy for example, is that two children with similar brain injuries can have vastly different outcomes.

Currently there are autologous stem cell infusion trials for cerebral palsy (now in phase 2 clinical trial), hydrocephalus and a general study. (Bertrand falls under the general study.) Dr. Kurtzberg at Duke University hopes that through the double blind trial now underway for cerebral palsy, we may soon learn more about the way autologous hematopoietic stem cells behave, and their effectiveness.

Even though study results haven't come out, one of Dr. K's pending studies speaks loudly to what results may look like. Researchers under Dr. K will be infusing young infants believed to have a predisposition for cerebral palsy (due to some kind of trauma etc. during gestation or birth) with autologous stem cells shortly after birth. Treating these infants proactively, if you will. Reading between the lines, I can see that researchers see a lot of promise in these stem cells and their behavior the earlier in development that they are infused.

Bertrand played and watched Elmo through IV placement and slept through the infusion! This was the easiest procedure he has ever had!

Back to Bertrand! Dr. K was very frank with us from the beginning that we should not expect much, or anything. She is still under the impression that Bertrand's case is an undetermined, inborn error of cellular metabolism and this is the cause of his ongoing brain damage. In such a case, Bertrand's infused stem cells would contain the same genetic error found in his existing cells, resulting in either no change or a temporary one. Bottom line, if we are going to see results, we will see them in three to six months. We won't know what form these results could take until/if we see them. Regardless, we have to email Bertrand's progress every 3 months and return for a follow-up exam at Duke in one year.

In April 2009 we made our first pilgrimage to see Dr. Kurtzberg at Duke - if she could diagnose and treat Bertrand's then assumed lysosomal storage disorder.
In September 2009 we returned to Dr. Kurtzberg as a follow-up and because Bertrand's neurological condition continued to deteriorate.
In August 2010, we simply infused Bertrand's with his umbilical cord stem cells because his condition had stabilized and it wouldn't hurt.


***With the prevalence of cancer in today's world it's shocking to me that all cord blood stem cells aren't systematically banked. When an individual with leukemia undergoes a bone marrow transplant, they are really receiving a stem cell transplant. The stem cells found in the bone marrow of a donor--hematopoietic stem cells, mesenchymal stem cells and endothelial stem cells--are transfused with the expectation that they'll go and take up residence where the patient's own bone marrow has been killed by radiation. However, the stem cells found in cord blood are considered superior to the ones found in adult bone marrow. Since the hematopoietic stem cells prevalent in cord are more primitive, when used in place of adult bone marrow stem cells, there is a lower incidence of Graft Versus Host Disease, easier HLA matching, and a host of other advantages. If you don't bank your child's cord blood PLEASE consider donating it to a public bank! Otherwise these life-saving stem cells are tossed away after birth as medical waste!


  1. Very interesting. I often wish we had banked Cody's cord blood but were naive parents expecting nothing but health. I've been reading a little about fetal stem cell treatment this week. I'm sure nothing will reverse his TS but anything that could fix some of the atrophy caused by his hydrocephalus would be amazing. I REALLY hope this transfusion helps Bertrand. I will anxiously await future posts of his progress. Take care


  2. You are so good at explaining medical information, Cristina. Thank you for this post - no apologies needed for the timing.

    I visited the regional cord blood bank here in my town last year. The manager of the bank gave me enough information to understand the complexity of handling the cells after birth - so complex that it reduces the amount collected even among those who donate.

    Most of the cells are used for bone marrow transplant, as you said, but I understood the cord blood cells are used for child transplants (mostly?).

    I am struck by the statement by Dr. K regarding infusing B with his own cells with genetic errors. This gives me pause and I will be thinking on this.

    I am a Bertrand-fan - regularly looking for news of him. Could you post some current photos of him in standing (for my presentation)? At the same time, don't worry about it as this would be low priority among all of Bertrand's needs (posting the photos, as standing is important).

    Thanks, Barbara