Looking for stem cell treatments for Kernicterus (Cerebral Palsy/ANSD)

[SorcererXIII]

Hello, my name is Tom. My son, Alastair, is 9 months old, and he has a form of brain damage called Kernicterus that has symptoms much like cerebral palsy. It was caused by jaundice during his first week of life and left him with impaired motor function, auditory neuropathy (profound deafness), and assorted other neurological quirks.

Seven days ago I took him to Dr. Steenblock for a subcutaneous injection of 20 million allogeneic cord-blood derived stem cells. So far there has been no improvement. However, in that time, I found a research study where kernicterus was cured (or at least largely improved) in rats, here: http://www.ncbi.nlm.nih.gov/pubmed/26818600

This new study uses adipose derived stem cells injected intrathecally into the rats. So, I'm now looking for treatments like that. Having done more research on stem cell delivery methods, I just don't think the subcutaneous treatment my son already received is likely to ever work. But it's difficult since a lot of places (including Steenblock) don't do intrathecal injections due to possible complications, but since the only current empirical evidence is for that case, I feel like this is the best chance for my son.

 

[barbara]

There are some clinics doing intrathecal injections in Mexico, but I am not sure if they are treating children or not. Have you contacted any of the clinics there?

 

[SorcererXIII]

There are some clinics doing intrathecal injections in Mexico, but I am not sure if they are treating children or not. Have you contacted any of the clinics there?

Thanks for the suggestion...I'm (re)starting the process right now and only contacted 2 places in Mexico. Here is what I have:

• David Steenblock - CA - Uses allogeneic cord blood or adipose stem cells injected subcutaneously, won't do IV or intrathecal injections
• Norvastem - Tijuana - Cured Gordie Howe's stroke. Won't treat pediatric patients.
• Regen Center - Dominican Republic - Uses adipose or bone marrow stem cells injected with IV.
• StemCellGeneticMed - Lima/Cancun - Claims to have experience with kernicterus, but didn't reply with specifics. Administers neuron stem cells intrathecally. Treats infants as well. Is $35k.
• Stem Cell Institute - Panama. Uses cord tissue stem cells. Will not perform intrathecal injections on pediatric patients.
• Stemedix - Florida - Uses harvested adipose stem cells injected intravenously or intravascularly

Of these I've only contacted Norvastem, Steenblock, StemCellGeneticMed, and Stem Cell Institute so far.

 

[barbara]

What about World Stem Cells in Cancun?

 

[SorcererXIII]

What about World Stem Cells in Cancun?

Nope, wasn't on my radar, but thanks for bringing it to my attention! I just called them, here's what I gleaned:

• Stem cells from bone marrow of patient
• Both intravenous and lumbar puncture (intrathecal)
• 5 day treatment
• 1st day signing forms, patient evaluation, blood draw, stem cell stimulation
• 2nd day - same as first
• 3rd day - expand and culture stem cells for next day
• 4th day - 1st infusion done intravenously
• 5th day - 2nd infusion with lumbar puncture
• Cost is typically $13k - $18k

It's not adipose tissue, but, from what I have read, the main ingredient - mesenchymal cells - is the same for bone marrow (correct me if this is wrong). So this may be the winner.

 

[barbara]

Lots of doctors are using bone marrow now instead of adipose. I would also think it would be far easier for a child to have that done rather than liposuction. If the cells are cultured, then a good amount of msc's should be available for infusion.

 

[SorcererXIII]

Overall it seems the advantage of adipose is that it proliferates faster and is possible to get greater quantities of cells before they enter senescence (when their regenerative effects are lost). And cord tissue yields more cells than either, but most people (including my son) can't get autologous cord tissue.

Some random papers on the subject, about 10% of which I actually grasp:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2971538/
http://online.liebertpub.com/doi/full/10.1089/scd.2011.0722
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2031970/
http://www.bioinformant.com/adipose-and-bone-marrow-derived-hmscs-whats-the-difference/
http://stemcellres.biomedcentral.com/articles/10.1186/s13287-015-0066-5


One thing I've read (forgot the source) is the wound left behind by the mini lipo needs time to heal before re-injection, otherwise the stem cells will home in on the wound rather than where you want (in this case the brain).

In my son's case I'm pretty reluctant to take any of the already scant (for a baby) fat on his bones.

 

[barbara]

I am not a fan of lipo. I think for a child it might be very difficult.

 

[SorcererXIII]

Small update

I spoke with nurse Gina at Steenblock's office and she said that he does indeed offer intrathecal injections, but may not be willing to do so on a pediatric patient. We are going to discuss further at the 3-week mark, at which point she said we will have started seeing any benefits from the subcutaneous treatment (it has been 11 days).

 

[barbara]

Thanks for keeping us up to date on your search.

 

[SorcererXIII]

Update on World Stem Cells clinic

I had a productive conversation with one of the doctors from World Stem Cells Clinic.

He reiterated that they don't want to perform adipose stem cell treatments on such a young child. Also, he indicated that the bone marrow yield should be higher because adipose stem cells need to be separated out from the remainder of the fat tissue before they will proliferate, whereas bone marrow cells do nothing but proliferate. They inject the patient with something to increase the amount of stem cells the day (or is it two days?) before aspirating. He estimated that they could get 30-40cc of bone marrow from 1 or 2 punctures in the hip bone, which they later expand to 200-300 million cells. They then separate this into 3 parts, and apply 1 part intravenously and 1 part intrathecally. I didn't catch what they did with the remainder (he had an spanish accent) but I think it's unused or perhaps stored.

He said this will be his first kernicterus treatment (basically the case for all stem cell clinics I've talked to), but he put me in contact with the parent of child they treated for CP.

The total cost of treatment is confidential but was within the range mentioned before, and includes hotel and taxi.


As an aside, while trying to figure out how many stem cells are in a CC of bone marrow, I came across this, which leaves me with concerns about how much of the bone marrow is contaminated with regular blood...
https://www.researchgate.net/post/Amount_of_Mesenchymal_Stem_Cells_in_a_ml_of_Bone_Marrow_Aspirate11

Does anyone else analyze every last thing in such detail? I'm driving myself crazy.

 

[barbara]

You are making an important decision for your child, so of course you are wanting to know every last detail. I am very happy to hear that they would not consider doing adipose on a young child. A marrow extraction is much easier and less invasive.

The article and the comments are quite interesting, but I wouldn't dwell on the count as the cells will be expanded. The method they use to filter the marrow also can make a difference as to how many stem cells are lost in the process. Again, by expanding the cells this will help greatly to achieve the number they gave you.

It is probably G-CSF that is given beforehand. Whenever, I've had it, it was given a couple of days in advance. It made me feel great by the way.

https://en.wikipedia.org/wiki/Granulocyte_colony-stimulating_factor

I would ask the doctor if they store any of the unused cells. I think that is something you should know before committing to anything. If he's hard to understand, perhaps you could ask him to e-mail you the specifics.

 

[SorcererXIII]

Actually I wrote him about it and he explained that not only had he known about this but had actually experimented to find the best method, and that they can now get over 250 million mesenchymal cells in an hour, and that is before expansion.

The G-CSF sound really interesting. I wonder why this on its own isn't enough to get stem cells where they need to be?

You are making an important decision for your child, so of course you are wanting to know every last detail. I am very happy to hear that they would not consider doing adipose on a young child. A marrow extraction is much easier and less invasive.

The article and the comments are quite interesting, but I wouldn't dwell on the count as the cells will be expanded. The method they use to filter the marrow also can make a difference as to how many stem cells are lost in the process. Again, by expanding the cells this will help greatly to achieve the number they gave you.

It is probably G-CSF that is given beforehand. Whenever, I've had it, it was given a couple of days in advance. It made me feel great by the way.

https://en.wikipedia.org/wiki/Granulocyte_colony-stimulating_factor

I would ask the doctor if they store any of the unused cells. I think that is something you should know before committing to anything. If he's hard to understand, perhaps you could ask him to e-mail you the specifics.

 

[barbara]

That's excellent news to get that quantity before expansion. The G-CSF stimulates the marrow to produce more stem cells which can then be harvested for an even better yield. The doctor may be using something else, I just mentioned that I've had treatments where I was given G-CSF injections for several days prior. You should probably ask the doctor if it is G-CSF he uses.

 

[SorcererXIII]

Burton Feinerman

I just had a conversation with Dr. Burton Feinerman; we had started an e-mail conversation before but my last reply to him got lost and so we both sort of concluded the other had lost interest. But now that that's been rectified:

• Has treated children with Kernicterus, but it was some time ago
• The children improved at least to the point of being able to sit up
• He isolates neuron stem cells from allogeneic cord blood and injects it intrathecally
• Preferred location of treatment is Lima, Peru since the facility is more advanced and perform 2 injections over 3 days
• Cancun, Mexico is an alternative but they only perform 1 injection (so half the amount of stem cells). The cost is the same.
• Cost is $35k; financing is available.

I asked him about the kernicterus study where rats were treated with adipose mesenchymal cells (http://www.ncbi.nlm.nih.gov/pubmed/26818600); he said it was mostly wishful thinking that these stem cells can heal nerve damage. That seems to be in contrast to some other studies that indicated that adipose or bone marrow stem cells help due to the neurotrophic factors released (leaving me to wonder if just isolating neuronal stem cells would have as beneficial an effect), for example:

http://translational-medicine.biomedcentral.com/articles/10.1186/1479-5876-10-100
http://www.sciencedirect.com/science/article/pii/S0014488601978537
http://onlinelibrary.wiley.com/doi/10.1002/jnr.20494/abstract

It does seem a little weird to just outright dismiss these studies. Or perhaps, since you're doing a lumbar puncture anyway, why not just do both neuronal and mesenchymal cells at once?

Of course, he is the first doctor I've spoken to anywhere that has ever treated a patient with kernicterus before. I'm now left with some angst as to whether I should choose this over the World Stem Cells clinic treatment, seeing as how it's over twice the cost, but is possibly a more precise treatment for neuronal damage.

 

[barbara]

The decision has to be yours. I am a bit confused as to why the treatment in Cancun would be different if Dr. Feinerman is the one who oversees the treatment. It would make sense to me to have the same kind of protocol. Where were the other Kernicterus treated?

 

[SorcererXIII]

The decision has to be yours. I am a bit confused as to why the treatment in Cancun would be different if Dr. Feinerman is the one who oversees the treatment. It would make sense to me to have the same kind of protocol. Where were the other Kernicterus treated?

I am wondering the same thing. Maybe the treatment is patented and he licenses it out to them to use...so they are like the Harry Potter movies and he is like JK Rowling (probably bad analogy but you get the idea).

He seemed a little reticent to discuss the Kernicterus children...he said it was a long time ago, and that now he mostly treats degenerative disorders and cerebral palsy.

When we had our original exchange it ended immediately after I asked if he treated the children with stem cells and if they were improved, and he never responded, so I had actually taken that to mean they hadn't, and sort of forgot all about it. But when he just told me verbally over the phone that he had treated them and they had improved, I was surprised. Also little nervous because at first I couldn't remember which doctor he was (I contacted several) so I should have been more insistent.

 

[SorcererXIII]

We had our first day at WSCC today. The brand of GCSF is called "Neupogen". Today they took a blood count and gave him one subcutaneous dose based on his weight (I think it was like one mg/kg of weight) and tomorrow they check his blood again. If his leukocytes haven't increased enough they will give him another dose. Also tomorrow they will attach the IV connector for use later in the week. Wednesday they draw the marrow and use 1/3 for the IV, 1/3 for the lumbar puncture. The remainder they culture and reinject on Thursday via IV.

 

[barbara]

Thank you for your on the spot reporting! I know you must be excited and yet apprehensive at the same time. I've had Neupogen injections several times as I mentioned before. They were very effective. Is everything done at their clinic or do they also use the facilities of a hospital there? Is one doctor in charge of his treatment or is there a team?

Your child is certainly a pioneer since he's the first one they've treated with Kernicterus. The information that can be gained from his treatment will help others in the future too. I'm very proud of you both.

 

[SorcererXIII]

The clinic is upstairs from a fertility clinic and they share an operating room, but the rest is done in their clinic. The office seemed fairly small from the bits I could see. There were the usual certification notes on the wall albeit from the Mexican government. There was a clean room with a suited technician where I presume they do their cell processing. We were seen by two doctors and a nurse as well. They asked a lot of questions about Alastair's health and history but did not ask to see official medical records (though I have those up on Google Drive if they needed them). Judging by their website I think there is a separate anesthesiologist and possibly more, but I don't think I'll see them until Wednesday.