Wednesday, September 2, 2009

expanded clinical trials for EB

I'm not really sure what all of this means at this point, but one of my EB peeps (Thanks, Sara!) sent me this article this afternoon.

Stem Cell Therapy for Severe Forms of Epidermolysis Bullosa


This is a single institution trial to determine the effectiveness of stem cells from closely matched related and unrelated donors to replace missing or defective proteins responsible for severe forms of epidermolysis bullosa.

Eligibility Criteria
1. Patients aged < 25 years of age
2. Diagnosis of a severe form of EB characterized by collagen, laminin, integrin or plakin deficiency
3. Adequate organ function
4. HLA matched related or unrelated marrow donor or partially HLA matched unrelated umbilical cord blood donor

Ineligibility Criteria
1. Active infection
2. History of HIV infection
3. Evidence of squamous cell carcinoma

If you or your child may meet the above criteria, you may be eligible to come to the University of Minnesota for the proposed treatment.

Prior to evaluation and screening to confirm eligibility, the following information is requested:
1. Clinical summary from a treating physician, including history of prior surgeries, transfusions, skin grafts, medications, allergies, infections
2. DNA mutation report if available
3. Skin biopsy report (including immunofluorescent staining)
4. HLA typing on child or adult with severe EB and unaffected siblings (instructions will be provided by our team)

The screening visit will involve skin biopsies, blood tests, photographs, assessment of skin fragility, and genetic testing. In addition, the visit will entail review of the clinical history and detailed to review of what is known and unknown regarding the proposed treatment procedure using stem cell populations from an allogeneic donor.

It sounds like, to me, they are opening up the transplants to Junctional EB patients as well. AND they are not requiring a sibling match for this trial. Now, I'm not saying this is something we would pursue for Jonah any time soon (considering how young he is), but this is HOPE. And maybe by the time he IS old enough that we felt it would be safe enough, they'll KNOW that it works with Junctional and without an exact sibling match.

We are so thankful, and consider this a direct answer to all the prayer that is going up for Jonah and others like him. With each passing day, I feel, more and more, that Jonah WILL be healed this side of Heaven... whether that be a "snap His fingers" sort of miracle, a gradual toughening of his skin, or a bone marrow transplant someday in the future.

Praise God for what He is doing through these amazing doctors and researchers!

Please be in prayer for us as we make contact with the University of Minnesota folks and find out more about our options... Pray that it will be clear what we should do and that we will be wise in the decisions we make for Jonah (now or in the future).

He's doing great, by the way! The nutritionist said he is in the 50th percentile for both height and weight, and has gone from the less than tenth percentile for head size to the 25th. This is no small miracle, to be sure.

No matter what we find out about this trial, we know that God will use Jonah's life to bring Him glory, and we feel honored to be his parents.

So many times, when people ask me how Jonah's doing, I say something along the lines of, "For now, it's just blisters, and that's all we can really ask." But it's not all I can ask. I ask for and fully expect a miracle... whatever form that takes.

And please keep all the other EB kids in your prayers too. There are so many out there struggling with even more than Jonah. I hope they are all going to bed with a little more hope tonight too.


Anonymous said...

May God bless all who are struggling with this disease and those struggling to find a CURE.
God Bless Jonah and the Williams family.
Prayers Always,

RLR said...

Ooh, yay! I feel the same way about the whole 'peanut desensitization' that keeps popping up in the news. We aren't ready to be a part of that yet, but so look forward to the day that there is a treatment for peanut allergies.
Praying - and so thankful for this HOPE!

Summer said...

Wonderful news for you guys! What a great feeling!

Donna in NC said...

I believe if you check out his guy's site (his infant daughter has EB also), maybe it can give you some useful info. Looks like he's checking into the trial you're talking about. Hope this helps a little.

Amber Schmidt said...

Sometimes God's biggest blessings and miracles come in the form of people... people who care, people who research, doctors who heal... SO happy to hear that strides are being made!!!!

Laura A said...

Praise God for this, Patrice! Will pray for guidance as you talk with the people in MN. Keep us posted (I know you will!)

Love from TX!

Toni :O) said...

That truly is fabulous news and I continue to pray so hard for your sweet boy! I'm thrilled to hear he's doing so well..he just keeps getting cuter and cuter each day and I feel incredibly blessed that I get a sneak peek into his life and yours....thank you for sharing your sweet family with all of us. Gives me such hope to see him doing so well. Hugs to you all!

Katherine said...

Yea! Yea! What totally fabulous news! Any news about advances in EB research is incredible ... to believe that none of us knew anything about it a year ago seems so inconceivable to me now.

Janel Waters said...

I was SO excited when I saw Sara's post on FB! excited for you and Jonah and your family.

I hope that you find the answers that you are looking for and that you will know what choice to make for Jonah.

Please keep us updated!


Devon said...

Awesome awesome awesome!! That is great!!!

Lorraine-bo said...

Exciting to read about research that God may use for miracles in the future!

Praying the Lord makes clear to you & Matt what you are to do with this information now or in the future!

Rebekah said...

Well how old does Jonah have to be? In reading that I would just want to JUMP at the oportunity. I know you are though and its a process. Ill be checking in to see what happens with you and little Jonah

Just Breathe said...

It sounds very hopefull. I will pray for these studies to work and to work quickly so your little guy can get relief.

Shari said...

Yay! I am so glad that God is showing you that there is HOPE for Jonah's future. I will pray about this specifically, but be assured I pray for him daily.

*Mirage* said...

Praise God for HOPE!!!!! :) I hope they can find the cure!

Leah said...

I have been thinking about you a good bit lately. I just had a baby and every time I change her diaper or clothes, I think about how you are not able to do the same with Jonah. I can not imagine how difficult it is to take care of your precious boy when every move you make can cause issues. You are in my thoughts and prayers... I won't take what I have for granted, ever. Hang in there and thanks for sharing your story.

Anonymous said...

Wonderful news! Praying for a miracle for Jonah!

By the way, those were some beautiful words there at the end!

You are such a great mom...Jonah is blessed to have you and you are so blessed to have been chosen as his mother.

Anonymous said...

I am so excited about this news. Meg told me about it last night. Hopefully Leah will be able to get this done too. There really is hope and our prayers are being answered. Thanks for bringing attention to the other EB babies out there. Thank you God!!!

Lauren said...

saw this earlier today when sara posted it on facebook. so exciting to at least have the possibility later on.

thinking about you and praying for you guys. i miss you.

the-mommy-person said...

God bless you.

Janet said...

You know, I kind of wondered in the back of my mind if this would be an option at one point. Stem Cell research has come up with some wonderful cures. I hope, if not a cure, help. Anything that will make Jonah's life a little more comfortable. Please continue to keep us posted.

Anonymous said...

God is good...I will continue praying for your wisdom and a complete healing for your sweet boy.

Ginster said...

Please get him into the trial if you can!! I would not worry about his age unless, THEY tell you he is not allowed in. If he is allowed in, of course you would have to weigh the pros and cons before you decide to enter him , but just think if he could be cured sooner rather than later!!!!

Memphis Mama said...

I work at Vanderbilt University in AIDS research but you should check out This is a database that the NIH requires all Phase 2-4 trials be registered. You can search by condition or drug, ect. You would be able to find all trials that are open for EB and it would also tell you the different institutions that are currently recruiting for it. Good luck!

Memphis Mama said...

Anonymous said...

What exciting news! I will be in prayer for your family as well as the doctors in MN, for wisdom in knowing if this would potentially work for Jonah, and lots of clear guidance from God.

This past weekend I had the opportunity to see Beth Moore speak at a conference, and she talked about her prayer being for her family not that God would protect them from all pain and suffering, but that he would use them for glory and how glory is the ultimate honor/goal for us here in on earth. Jonah is definitely bringing glory to God every day, and for that you should be such a proud mama!

Donna in NC said...

I left you a comment last night, but I forgot to include the link I was trying to tell you about. This guy has some real encouraging things to say that can help us all. But he is looking into doing this clinical trial for his infant daughter. Maybe you will find some info on his site that can help you.

Anonymous said...

I found your blog through another blog. I wanted to tell you that I am truly inspired by your strength. You have a beautiful little boy and I will be praying for him and your family. What wonderful news of hope. God bless you and your family.

Jasmine said...

This is very encouraging news. It makes me happy to hear this! I am hoping for Jonah!

raymumme said...

Dear Williams Family,

My thoughts and prayers go out to you and yours.

I know you do not know me but I would like to try and help by sharing information that can truly make a difference. We have been able to help 1000,s of people with 100's of different conditions by releasing you own adult stem cells from your bone marrow, it is a natural process, it is the way the body normally repairs itself. The product is call StemEnhance we have multiply published peer review studies which prove this phenomenon. Two capsules release 3.5 million additional stem cells that is an increase of 25%.

God Bless,

Ray Mumme

Jeanie Baird said...

Hi Patrice,
I've followed your journey all year and can't wait to happen upon you at Target or ChickFilA. It's bound to happen eventually! :) But I wanted to tell you that I work at Wake's med school in the Office of Research. If you ever have questions about a clinical trial, please feel free to email me.

Anonymous said...

I don't begin to know the steps God is placing before Jonah for his complete healing, but I wanted to let you know that Lydia had an unrelated donor umbilical cord match transplant when she was only 8 months old...for leukemia NOT EB. All this to say, her body did amazingly well through it. It was unbelievably hard not knowing what the next minute would look like and she is still recovering from the affects of the transplant, but VERY well. One day at a time for you and your sweet Jonah.

Lori in MN said...

If you need a place to stay we would happily host your family in the Twin Cities.

Wanda Wilkinson said...

God answers prayers in many different ways. Amazing what the prospects of HOPE can do. Keep us informed

Wonderful hope, that is all we need to continue to believe in miracles. As I have said before, I truly believe that Jonah is going to be healed this side of Heaven.

I love me some Jonah...Praying

queenmari said...

this is awesome news about the research on eb. answer to prayers. we'll be keeping you, and the other eb families out there, in our prayers as you explore this new area.

i wasn't even aware of this disease until i happened upon your blog. i was recovering from spetic shock and on bedrest (really i had to learn to walk again) anyway, some how i hit a link to your blog. so glad i did. i've become very fond of you all.

blessings, mari

Sandy said...

This is such encouraging information! It is an honor to talk with God about Jonah's needs. I too believe we WILL SEE A MIRACLE.
Sandy in King

Anonymous said...

Wow Patrice, I look at all the comments , and I am amazed how God is using so many peaople with so many gifts ,like links in a chain to find a cure and bring hope to so many dealing with EB. God still is in the miricle buisness, working thru others!!!I am crying tears of joy tonight for my sweet Jonah.
praying for healing
Love Ya Gina

Monogram Girl said...

studies like these make me wonder why anyone would oppose and hinder stem cell research! there is such promise in it!

The Greene's said...

I love checking in on Jonah and your family and am excited about this potential opportunity for you!

I live about 20 min from the U of MN and if you happen to come here to check it out or participate I would love to help out in any way you need while here :)

Prayers and Blessings for your family!