cystic fibrosis and criopreservation umbilical cord blood and tissue

[taafom]

We have a daughter of 10 months with cystic fibrosis (mutations F508del and N1303K). She was diagnosed at 5 months after 4 admissions.

We saved (criopreservation) the stem cells of tissue and blood of umbilical cord of our daughter, and we still saved the umbilical cord blood of our son, older (does not carry the defective gene, he is healthy).

Anyone know or can clarify me the following issues:

1. We can use the steam cells of tissue or cord blood of our daughter with healing perspectives?
2. And the brother’s umbilical cord blood?
3. Me and my husband we are 'A Rh positive' as my son, but my daughter is 'A Rh negative', there are any chance of incompatibility between the brothers?
4. We would also like to know specifically if the umbilical cord blood and stem cells can help my daughter?
5. you know some place / country where this type of treatment is being done? in my country doctors do not want to know these subjects.

Thank you in advance for your answer

a desperate and worried mother
tania

 

[Aboveallislove]

dear mom,
I am so sorry for all the worry CF brings. That first year is so very hard. We also had our son's umbilical cord blood/cells stored (not knowing he would later be diagnosised). I have checked and so far there is no treatment it can provide, but I am comforted to know it is there and may be years from now. But even more comforting is knowing how close the pharmaceutical companies are to getting some good meds out. Are you familiar with the new/upcoming drugs? If not, please post your little ones mutations and I'll see what I can share.

 

[Mallika]

Hi Tania,
Scientists are still taking baby steps on this subject.
Good news is that they have found that lung epithilium cells can be made of Hematopoetic stem cells available in Cord blood.
But the in the procedure to actually convert the Hematopoetic stem cells into Lung epithilium cells they are years(5-10) away.
They are also making slow progress on other parts of body like intestine and Pancreas.
Right now more research is directed towards diabetics and Arthritis where injection of stemcells into body is easy.
There are some legal and illegal stem cell applications
Here's some info on the legal stem cell applications
http://www.cysticfibrosisindia.com/cystic-fibrosis-and-stem-cells/
http://www.cysticfibrosisindia.com/mail-interview-with-dr-jaygopal-rajgopal-md-on-16-september-2014/

While the official research is a little slow. There also some unofficial and slightly illegal research happening around. check out

http://www.angelodistefano.com/adult-stem-cells-for-cf-overview/

I am not sure about the veracity of the above Angelo Di Stefano link and trying to verify it by all means.
Can not understand why such path breaking technology could not get FDA acceptance..
I am trying to get more details on it.

 

[LittleLab4CF]

We paid to have my niece's last baby's umbilical cord harvested, treated and quick frozen about fourteen years ago. It was a sensible decision in my opinion, then and still today. "Ryan" has a supply of personal stem cells. My decision to plunk down $3000 plus an annual storage fee of ~$160 may sound extravagant, and it may be. From my perspective as a scientist and cyto-geneticist, no greater present was imaginable. The lunatic fringe on both extremes of the stem cell debate lose traction when a person keeps their umbilical cord for the rich content of perfect and personal stem cells.

I don't believe that a large enough number of umbilical cords have been collected to encourage research on using this near perfect source of stem cells. These are versatile stem cells and any stem cell therapy would be easily adapted. Ryan has something genetic going on, he's undersized, and his physical development is lacking. Sort of like certain CF carriers, he's able to consume mass quantities of food and not increase his weight. He doesn't have CF and he's not a carrier. They suspect a group of growth related genetic diseases but there's no genetic testing for this. My point is stem cell research hasn't yielded any treatments or cures that could help your children currently. I'll do some research of what's available or may soon be. If I'm wrong or not current, I'll post under this topic. Fortunately your kids have stem cells adaptable to any and all stem cell discoveries.

Don't give up maintenance of the cryopreserved cords if you're able. Stem cells hold the realistic promise of growing new lungs or a heart, regrow a spinal cord, reconnecting paralyzed limbs and such. This is all being researched and many animal studies are quite advanced.

There's a stickler in a CFers stem cells, any stem from that person has CF genetics. Lungs grown from your daughter's cells or stem cells will be CF lungs complete with her mutations. This is a real hurdle for your daughter. A genetic moderator drug similar to Kalydeco would need to correct the stem cell's genetics before they will be any help.

A number of genetic moderators are in the final stages of trials including ∆F508. In the near future of CF medicine, Kalydeco like drugs are amongst the most promising treatment and arresting CF is a huge promise in a child as young as your daughter. This doesn't involve the umbilical cord, but I suggest you start looking into drug trials and getting onboard for the first public release of the most promising.

It was just a couple months ago that I paid Ryan's yearly storage and frankly I was a bit disappointed. I realized that the service is only a good idea if someone devotes research in the use of original stem cells. In fact research is going on and it's fast and furious. All the organs I mentioned and many more are being grown in animal studies. If anything new and substantial has been recently developed, chances are it's not happening in the U.S.. Most stem cell advances are from international companies who built labs in countries where work can be done in relative peace.

If anything remarkable in stem cell therapy and CF is out there, it should be known. Anybody?

Keep those well considered cords, they are going to be useful, I just don't know of anything currently being utilized.

Keep your hope alive,

LL

 

[taafom]

it is true, this first year has not been easy, 2014 is a year to forget!
The mutations of my daughter are: p.Phe508del (c.1521_1523delCTT ) and p.Asn 1303Lys ( c.C3909G )
Thanks by your answers and your help