Stem cell therapy: Has it helped Max?

"If it couldn't hurt and it might help, we'll try it." That's pretty much been our motto since Max's birth, and it's why we did stem cell therapy back in August. As far as we could tell, it wasn't going to harm Max, and there was potential for it to do some good. So we got the infusion at Duke with with Dr. Joanne Kurtzberg, using stem cells from cord blood we'd banked at Max's birth. We only had enough for one shot.

Lately, some of you have been asking whether the progress we've seen in Max is from the stem cells. My answer is, I don't know. Nobody does. Duke didn't do an MRI before the infusion or request a comparative one after, since conventional MRIs aren't sensitive enough to show differences in the brain of a child with cp. However, I've heard Duke is going to be doing another study where they'll be trying out a new type of MRI that is more sensitive.

We've hit the six-month mark, the time when changes might kick in, although they can also show up before...or not at all. In recent months, I've seen small but marked improvements in Max. He is more spontaneously using both hands. He is trying to speak more words. His overall cognition seems improved—he's always been alert, but now he seems even more so. I have not found any data that stem cell therapy can trigger an obsession with the color purple.

As Dr. K says, "I'm glad Max continues to improve, for whatever the reason."

Exactly.

Here's a recent CBS video about a newborn who got an infusion at Duke University. I continue to have tremendous hopes for the potential of stem cell therapy to help our children, and the groundbreaking work Dr. Kurtzberg is doing.

"M-A-X spells MAX!" says Max. A stem cell miracle? Who knows....

This is the week we're due to e-mail an update to Dr. Kurtzberg, the wonderful Duke University doctor who did Max's stem cell transplant in August. It's been four months, and while she said that if we were to see results they would happen at around the six-month mark, she wanted us to get in touch in December.

I haven't seen any major transformations in Max since the infusion other than his sudden passion for the color purple, not a known benefit. I feel like I've maybe seen some improvement in cognition and alertness, though it's impossible to say whether that's the stem cells doing their thing. It was always going to be a crap shoot: We'd never know whether Max's progress would be due to the stem cells, due to his own natural development, or a combination of both.

You know what?

I don't care.

I'll take whatever progress he makes, for whatever reason. I'm not concerned with the hows or the whys; all the proof I need is in his smile, in his eyes, and in the sound of his voice saying "M-A-X!"

Lots and lots of info on stem cell therapy

Those are Max's stem cells, above. Aren't they just beautiful?

As you can imagine, I had a ton of questions for the doctor who's spearheading Duke University's stem cell program: Joanne Kurtzberg, MD, Professor of Pediatrics and Pathology and Director of Duke University's Pediatric Blood and Marrow Transplant Program. She is a brilliant, progressive-minded doctor and a person who manages to be both matter-of-fact yet warm at the same time. She first started researching stem cell therapy for kids with leukemia back in the 80s. Since then, she's treated more than 1600 kids with life-threatening cancer, metabolic and blood disorders, using donor stem cells, and 120 kids who had neurological conditions such as hydrocephalus, brain injuries from falls or drowning and cerebral palsy, using their cord blood. The stem cells are injected into the arm or leg, but it's possible that someday, they will be injected into the brain. It is still unknown where the best injection spot is.

Right now, Dr. Kurtzberg is only using stem cells from cord blood to treat cerebral palsy because the risks of using ones from a donor, even a sibling, are too high. In order to make donor stem cells "engraft" (or take) a child needs to receive chemo, a process that takes two months and can be dangerous. Since cerebral palsy is not a life-threatening condition, the doctor says, the risks outweigh any potential benefits (unlike, say, leukemia).

Dr. K told me that stem cell transplants have helped kids with cancer and other diseases. She and her team were able to do a biopsy on the brain of a child who died 10 months after getting a stem cell transplant. It was her progressive neurological condition that ultimately killed her. In fact, when they looked at her brain, they could see that 40 percent of the cells had reached there. With kids who have cp, the results are less clear-cut; reports come in from parents, but they may not be the most objective source. There is a lot of misinformation out there. Remember that video of Dallas Hextell I mentioned a couple of weeks ago, a video that showed he was able to walk after receiving a stem cell infusionl? That video and story were misrepresentative; Dr. K said Dallas was walking before he got the stem cell therapy. But she says that she has heard of kids being better able to use their limbs, and improvements in speech.

Results can start showing up around six months after the infusion. I'll be e-mailing Dr. K every few months to tell her how Max is doing, and we'll go back and see her in a year. Before we go home today we'll be visiting a pediatric neurologist at Duke U., who will assess Max and then see how much he may change by next year.

Dr. K believes that stem cells are best administered as soon as possible after a brain injury has occurred. Two other stem cell infusions happened yesterday, and one of them was for a six-week-old infant. I didn't feel comfortable asking what had happened, but I was thrilled for that child. I only wish, as I've said before, I could have done this sooner for Max.

For now, treatment is still considered experimental and unproven (more on that below), but with help from champions like Dr. Kurtzberg, perhaps in the near future we will get proof that it works. Once that exists, more hospitals around the country would start doing this. And that would be tremendous. As you know, there is no cure for cerebral palsy. Not yet, anyway.

A few more questions Dr. Kurtzberg answered for me:

Can you give a basic explanation of how stem cell therapy is supposed to be effective?
"We believe that stem cells can travel to the brain and then help repair damage caused by stroke, low oxygen or other vascular problems. The cells may work in two ways, first by releasing hormones that calm down inflammation and also attract cells from the brain to heal damaged areas, and second, by providing stem cells to contribute to the repair."

What are your hopes for the state of stem cell therapy five years from now? Ten years from now?
"I hope that we first learn if stem cell therapy works and also the best way to use it. We need to know what are the best cells to use, what is the best place to give them and what dose is most effective. We also need to better understand when children with cp sustain their brain injury and how to detect it as early as possible. I believe that stem cell therapy will help children with brain injuries but I also believe that the earlier it is given, the more effective it will be. I think that it may be possible, in the future, to scan babies in utero or at birth to determine whether they have an injury, and then to treat them within the
first few days to weeks of life."

What advice do you have for parents who did not bank their child's cord blood at birth?
"First, these parents need to know that we don't know if cells help yet. While it's very hard to wait, that's what they should do. I can promise that if we find that cells do help, we'll next find a way to safely use donor cells for children who didn't have their own cord blood cells stored."

Can you give any information on the upcoming trial?
"The upcoming trial will enroll 120 children with spastic cp between the ages of six months to six years. They will be tested with an MRI, neurodevelopmental testing and a neurological examination before treatment. Children will randomly receive their own cord blood cells or a placebo for their first treatment. One year later, they will be tested again and will cross over and receive the treatment they did not receive the first year. They will be studied again one year later. The treatment will be blinded, which means that no one will know which treatment each child received first and second until the end of the study."

If you are interested in finding out more information on getting your child into the trial, which they hope to kick off this fall, you can contact the Director of Clinical Operations, June Allison (the woman is a saint) at allis006@mc.duke.edu.

Max gets his stem cell infusion

Stem cell therapy for cerebral palsy: Does it work? We're hopefully at the beginning process of finding out. I'm sitting here at Duke University Children's Health Center writing this as Max is watching Toy Story and vegging out. The stem cell infusion is done; it took all of nine minutes. The infusion started at 12:56 p.m., and ended at 1:05 p.m. It's such a crazy-short amount of time for all that anticipation; one nurse here described it as being like Christmas morning.

Max did amazingly, from the second we stepped into the day hospital at Duke at 10:30 this morning. The pediatric staff is wonderful.


A nurse found him a truck.


Mary Margaret, a sweet woman who works for a North Carolina program called Arts for Life—which works with hospital patients—did painting with him.


Max showed off his painting to everyone who passed by.


Next, Max got down with the music therapist, Tray.

Then all of a sudden, a team of people showed up at the door: It was time for his infusion. Dr. Joanne Kurtzberg was there, assorted nurses and a camera crew, who were taping her for a TV segment. They asked our permission to tape the infusion and I said yes; I am all about spreading awareness of stem cell therapy, cerebral palsy, Max's cuteness, you name it.

Max only cried when they put the infusion needle into his foot. And then the drip started. I found out that there were 190,000,000 of Max's stem cells in the infusion bag, which sounded pretty damn good until I learned that a billion is not uncommon. Max had a relatively low number of stem cells harvested at birth; the amount gotten depends on a variety of factors, including the ability of the person doing the job to snag every last stem cell possible. So, we didn't exactly luck out in that department, though we were very lucky we had banked his cord blood at all.


After the infusion, he was back to his usual happy camper self. We'd been told his breath might have the scent of creamed corn, and sure enough, Max smelled like a Thanksgiving side dish.

I'd been a little worried about how Max would react to being in a hospital; he was a trooper. I was a little worried how I'd do, too. I'm sure many of you know just how hospital settings can conjure up painful memories. But I was fine. I am now able to say things like "an intern in the NICU noticed his leg twitching, and that's when we were first aware something was really wrong" and "the doctors said he'd had a large bilateral stroke" without shedding a tear. But one thing that got to me was seeing other kids at the day hospital undergoing treatment. There were a lot of kids with cancer. A sweet little three-year-old girl was so puffed up from chemo, and I felt sad for her and for the hell her parents were going through.

I've learned lots about stem cell therapy, which I'm excited to share with all of you. But first, here's a snippet of the infusion. It was pretty uneventful—and that's a good thing. SWIM, you stem cells, SWIM!!!



March 2 2010 update: Stem-cell therapy: Has it helped Max?

A letter to Max: I do not regret your disabilities

Dear Max,

It's the night before your stem cell infusion at Duke University.

We've both come light years away from the week after your birth when you were in the NICU and I sat at the kitchen table in our house, our too-empty house, and desperately Googled "treatments for brain damage." I was terrified of what lay in store for you. That's when I first learned about stem cell therapy. I couldn't find a single doctor in the country who was doing it. There was one I got on the phone who said he would be willing if we got special dispensation from The Food & Drug Administration for a life-or-death situation. Then it became clear that you were going to make it; it was less clear you were going to thrive.

We lost track of the stem cell therapy, caught up in a whirlwind of doctor visits, other alternative treatments, Early Intervention and finding private therapists to work with you. You made progress on your own timeline, proving the NICU doctors wrong along with the so-called specialist who said your future looked "ominous." And then, this spring, I learned about the Duke University stem cell program from Kate, an incredible woman who somehow found this blog and who keeps a journal about her son. Kate, there are no words strong enough to express how grateful I am. I am also eternally thankful to my friend Wendy, who's watching Sabrina while we're away and who told me to bank your cord blood at birth. "You never know," she said. So true, so true.

Max, if you are someday reading this blog, I need you to understand something very important. Your dad and I are not doing this because you are lacking in our eyes. It's not because we want you to be like other kids. We are doing this only because it might make life easier for you in some way. We try our hardest to give you the best life possible, but if there is a chance you might be able to use both hands a little easier, to eat a little easier, to speak a little easier, to think a little easier, then we need to go for it.

Back when I first started talking with other moms of kids with special needs, I remember one in particular telling me that she would never trade her child for a "typical" kid. I thought she was in deep, deep denial. But now I understand. I look at you and I see only an amazing kid, not a disabled one.

You are a boy who is full of enthusiasm and sweetness and brightness and good humor and all sorts of wonderfulness. You are fascinated by cars, planes, trains, garbage trucks, tow trucks, dump trucks and basically anything that has wheels (last month, you were able to steer a toy tractor by yourself for the very first time); you can play baseball and enjoy watching it, too; you can count to ten, speak a bunch of words and recognize some in writing; you can down seemingly endless amounts of chocolate ice-cream and birthday cake; you get a wicked kick out of pulling your sister's hair; you adore rain, but not the thunder; you're obsessed with the movie Madagascar, Curious George books and, tragically, the TV show Spongebob Squarepants; you like room service and demand it when we travel; you give the most delicious slurpy kisses; you adore computer games; you enjoy all sorts of music and sometimes break into this crazy dance move that kills me every time; you have a happy squeal that makes strangers smile and a giggle that makes the world laugh with you. I am not exaggerating when I say that you charm everyone who meets you. Even grumpy people.

On this day, in the seventh summer of your life, I want you to know that I love you infinitely. I could not possibly love you any more if the stem cell therapy were to work miracles. And if it doesn't do a thing I will, of course, love you just the same.

I love you as you are, Max.

OK, this sign I can't ignore

I blogged the other day about seeing butterflies flitting around our backyard and wondering whether they were some sort of sign, but not quite believing it.

This morning, as I was packing for our trip to Duke University, I went to hang up a cotton top of mine that's been hanging on the back of the bedroom door for weeks now. I happened to glance at the label.

See?

Hmmm....

We arrived safely, the only hazard was when Max decided to push the luggage cart all over the lobby of the hotel we're staying in and nearly castrated some poor guy. Tomorrow, we head over to Duke at 8:15. Max is going to have his vital signs taken, get weighed and measured and have some blood work done. Then we'll have free time, we're planning on visiting Frankie's Fun Park, which sounds like...fun. Oh, and we're definitely trying some good ol' North Carolina BBQ, too. Yes, we are trying to cram lots of fun in before the stem cell infusion. Max deserves to have a good time. Come to think of it, so do we.

Not-so-wordless Wednesday

Thanks for the comments on yesterday's post about dumb stuff I waste money on; they helped me realize that I am far, far worse than I'd even thought. Like Insomniac Mummy and Debbie from Finding Normal, I am a sucker for getting the kids toys. Like Lianna, I buy Max bananas as if he were a primate or something, and then half of them end up rotting. Like Meredith from Templin Family, I stock up on Band-Aids, of the Princess and Cars variety. Like Tiff, I hoard photo frames. And like Dianne, when I'm fond of a t-shirt, I get it in several colors.

I hope you will all be here to comfort me when I someday spend my last cent on bananas or Band-Aids.

In other news, I still haven't given in to Max's repeated requests to have our car towed just so he can see it on a tow truck. If any of you happen to own a tow truck and are willing to drop by, just say the word.

Sabrina, who's four, continues to think she is the adult in our relationship. Dave called my cell last night as I was headed home from work, he'd taken the kids for ice-cream. "Tell Sabrina she can't eat Max's ice-cream," I said. "Mommy says you can't eat Max's ice-cream," Dave told Sabrina. "Mommy's just kidding," Sabrina informed him.

Last week I told her that Max is going to go to a special doctor soon (for stem cell therapy), and she'll be getting to go to a sleepover at my friend Wendy's house. She didn't ask too many questions, other than whether Max will be getting on a plane and could she bring her Ariel doll to the sleepover.

I know I said I wasn't going to get my hopes up too high, but they're inching up there. We've been getting encouraging e-mails from people as far away as Turkey and Australia who've gotten stem therapy for themselves or their kids. One reader, Lorraine, sent me this video about a boy I'd heard of, Dallas Hextell, who got a stem cell infusion at Duke (that's where we're going). It's incredible to watch. But Dallas is so young, it's hard to tell whether or not it was the stem cell infusion or natural development that helped him progress. See for yourself.

Last, words of gratitude for Eunice Kennedy Shriver, an amazing lady who founded the Special Olympics in 1968. Her advocacy for people with special needs helped bring them into mainstream America. And for that, I owe her. May she rest in peace.

Photo by Alistair Hall

Stem cell transplant update!

We have a date: Tuesday, August 25. We'll be flying to Raleigh on Sunday, getting Max checked out on Monday at Duke, then the infusion will happen Tuesday, with a followup with a neurologist on Wednesday.

The amazing Jenn, Jude's mom, recently mentioned stem cell therapy research from Stanford School of Medicine on her blog that shows how promising it can be, check it out. Still, as you know, I am not getting my hopes up too high, a defense mechanism I've learned over the years.

I haven't yet started talking about this with Max or Sabrina, but I'm going to be straight up: I'll tell Max he's going on a trip to the doctor, and I'll tell Sabrina that she's going to stay with our friends while Max is there. My friend Hedy pointed out that Sabrina shouldn't be too disappointed missing a trip to the doctor, but you just never know with her—she gets jealous when we give Max any extra attention. Yesterday I was showing Max in the mirror that he'd lost another tooth and she kept shoving her face in front of it to look at herself. She is so four.

I'm not getting my hopes up too high about the stem cell transplant. But I am getting awfully excited.

Here's to a weekend filled with good things for all of us!


iStock graphic/Paul Rosado

Max is getting stem cell therapy (and other news on slithery stuff)

So, a brief recap on where things stood with stem cell therapy: Weeks ago, I contacted the people at Duke University doing the trial. The place where we'd stored Max's cord blood sent a sample over to Duke. Then we had to get a few vials of blood drawn from Max and mail those, so they could make sure that it all matched.

I got word today: Everything is good to go. The infusion could happen in the next couple of months, I'll know by the end of this week.

I can't believe it. I can't believe it. I can't believe it.

Like I've said, I'm not hoping for miracles here. I'd be grateful for even a little improvement. But, bottom line, I am not expecting anything, this is all still so experimental.

In other news, I walked in from work tonight to find this on the kitchen counter:



In case you can't tell, that's a bunch of caterpillars crawling in a glass jar. We bought the kids a Live Butterfly Garden; you send away a pre-addressed postcard that comes with the kit to order the caterpillars. Once they develop into chrysalides (within seven to ten days), you move them into the mini habitat.

Sounds like a good learning experience, right? It is. Only thing, I forgot how much I HATE caterpillars. When I was a kid, we spent our summers in the country. One year, I must have been ten or so, there was a gypsy moth caterpillar infestation. I still remember seeing the bulging nests in the trees filled with ominous blackness—ultimately, they killed the trees—and then, eventually, caterpillars underfoot everywhere.

Creepy.

I am definitely going to have to find a new place for them besides the kitchen counter.

A world of possibilities

This weekend, the future seemed filled with possibilities.

Here's Max, playing with his new tennis racket, and Sabrina, showing off her mastery of hissy fits.

Lately, Max is into all sorts of new things—baseball, tennis, swimming. As he's become more sure-footed and has gained better trunk control, he's more willing to try sports. Grasping things is still a major challenge, even with his left hand (the more functional one) but hopefully, better hand control will also come along. One thing I have learned over the years is that, when it comes to Max, there is no time limit for accomplishments. In toddlerhood, when everything seemed laser-focused on developmental this and developmental that, it was a hard thing to accept. But now, I know that we have time.

Last week, I told you there's a possibility we might be able to get Max a stem-cell infusion. Here's what Max's pediatric neurologist e-mailed me when I asked him about the program at Duke University:

"I am quite aware of the program and have had some kids go down to participate. I have not recommended it to anyone yet because I am waiting for results in the kids I treat. I have not seen anything bad, and perhaps a bit of good. So, if they judge his cord blood 'eligible' there would not appear to be any harm in trying it, but there are not any clear parameters that I have seen to judge the effectiveness of the treatment."

Of course, I e-mailed back right away to ask what he meant by "a bit of good," and this is what he said:

"As for the good, a bit more attention and focus, and a slight improvement in motor capacity, but perhaps it is too early. If you look at the cases that have been reviewed in the media, perhaps there is more to be expected with time. The numbers are not sufficient yet, however."

No matter, we are ready and willing to try this. This week, Duke is mailing us a kit to get some blood samples from Max, they need to make sure it's still a good match with the cord blood we've banked.

In case you are wondering, the procedure would cost around $10,000. I haven't yet called the insurance company, I highly doubt they will cover this as it is experimental and, as you know, I have battled them to get them to pay just for speech therapy (and still am).

Dave remembered that when he called the cord blood bank after Max was born, he was told that the sample they had gotten was excellent, filled with viable stem cells.

Yes, the world seemed filled with possibilities this weekend. What possibilities have you been dreaming of for your own child lately?

Stem cell therapy for cerebral palsy: Max might get it!

After hearing from Kate (THANK YOU, KATE!) that Duke University has a stem-cell injection clinical trial, I got in touch with them. Today, I learned that they would be willing to consider Max as a candidate.

Back when I was pregnant, my lovable worrywort of a friend, Wendy, told me she'd done cord-blood banking for her baby. I figured if Wendy did it, I should, as I am also a worrywort (and just as lovable, I hope). So I signed up with Cryo-cell, one of a handful of cord-blood banks out there. There was an initial fee (can't recall how much); storage costs us $95 a year per kid (we did it for Sabrina, too).

I never imagined that we might actually need the cord blood. Never imagined that my baby could have a stroke at birth. Yet that's what happened, and I have a beautiful little boy with cerebral palsy. There are no miracle cures, but there is this possibility.

In a nutshell, Duke isn't sure cord blood is helping out kids with CP but, based on work they've done in general with genetic diseases of the brain and also animal studies, they believe it can help. The injections of cord blood are not risky, and while Max might not like getting an IV, he will deal. I've been Googling around, and it seems that some kids with CP who've gotten the cord blood injections have shown improvement.

I plan to run all of this by our pediatric neurologist. To me, this seems to fall into the camp of treatments that can't hurt and might help, and Dave and I fall firmly in that camp. Here's an article from Duke on what they've been doing.

The next steps are to get a copy of the report on Max's cord blood from Cryo-cell for Duke to review, to make sure there are enough cells and that the unit was frozen properly. If this works out, we'll have Max's blood drawn to make sure it matches up well with the banked cord blood. Then that would get shipped to Duke, and then we'll have to ship ourselves to Duke.

My deepest wish is that a stem-cell infusion could help bring along Max's speech. Although if it could also enable him to better use his hands then, well....

I don't want to fantasize too much here. Who knows, for one reason or another, Max may not be a candidate for an injection.

But, heck, I am going off to sleep now and letting myself dream.


Photo by nushuz