Whenever I hear the words "MD Breakthrough', my heart skips a beat. Is it really? Often, it is in reference to a study in the mdx mouse. We can cure mice. But people are not mice... just ask Victor Dubowitz. And when the 'breakthrough' talks about a young man with Duchenne, it feels like we are all standing at Attention!

Over the last couple of days, many of you have seen a news story that recently aired about a young man named Ryan. I wanted to share with you my thoughts about this story. For those of you who have not seen this piece, check out the story by following this link: http://www.kake.com/home/headlines/69538137.html.

I have watched the video of Ryan (by Jemelle Holopirek) several times and admit to some skepticism. To be quite honest, I have a lot of skepticism. My own sons were in the myoblast transfer trials of long ago, receiving some extraordinary number of donor myoblasts in their major muscles. It did not work, not at all. There was a moment when I thought I saw something, when Chris and Patrick did something or said something that seemed to suggest improvement. But there was nothing. And I used the same words "feels better," "seems stronger," etc. All generalities that cannot be measured.

I admit to some bias and for that reason, I wanted to get some answers. I have called the 800 number for Dr. Neil H. Riordan, the specialist featured in the story who is working from Costa Rica, and I am waiting on a return call. The number is answered in the US, and the person answering promised he would relay the message and have them get back to me.

Conceptually, stem cells make sense. The goal would be to deliver sufficient numbers of stem cells, to all of the muscles in the body, or at least the major muscle groups and make sure they integrate with existing muscle cells. The would potentially replace what is lost and if all that works, one might expect, over time, to see improvement.

But - and there is always a "but" - I have any number of questions.

We know this is difficult. Myoblast transfer trials were done in the late 80s and 90s. Jacques Tremblay continues with his myoblast transfer trials in Canda, now moved to limb delivery. Cossu (Italy) is moving toward limb delivery with mesioangioblasts. Gillian Butler Browne is working on stem cells. Lee Sweeney has just requested a small amount of money from us to expand some of his stem cell work (adult-derived stem cells). Barriers to stem cell delivery have been discussed in a variety of forums over the years. For Duchenne, and many other conditions, stem cells are suggested to be the Holy Grail, but it just has not been as easy as hoped for. Some years ago, we sponsored Marie-Therese Little at the Fred Hutchinson Center in Seattle, working on stem cells in the dog model. The barriers included identifying the specific type of stem cell, engineering it to ensure it would make muscle, delivery and rejection. And what does the immune suppression protocol look like? Loads of questions and progress for sure, but not magic.

The video describes three treatments over a year and a half with 46 shots into every major muscle group that resulted in a muscle biopsy with "100% dystrophin levels."

As a community we have to ask the difficult questions. Some of them include:
* What muscle did they biopsy? How was the level assessed?
* What was Ryan's functional ability before the transplant? How has he improved? His wonderful friend Clint (thank heaven for friends like Clint!) described 'neck and trunk stronger' and 'balance better' and 'gained 30 lb.' On the video, Ryan moved only his hands/forearms. And increased weight may not signifiy increased muscle mass and circumference of a specific muscle does not mean increased strength.
* Muscle biopsy - who did the staining? Quantification of dystrophin?
* Transplanted cells - cells from Ryan's sister and donor umbilical cords. What is the immune suppression protocol? Any concerns regarding graft vs host rejection?
* Function: What tests pre- and post- were done to document benefit?

It seems to me Dr. Riordan would do well by convening a meeting with researchers and physicians, including leading experts in the field like Cossu, Gillian Butler-Browne, Terry Partridge, Lee Sweeney, Jerry Mendell, and others. He could then describe his research, procedure(s), data, and outcomes in detail answering a wide range of questions.

I hope I do not sound overly negative. But I have been through this with a wide range of treatments and promises made to our community. And my frustration is not with Dr. Riordan. I hope, just like all of you, that his work is the "breakthrough" this report claims it to be. I just wish the media would understand that words such as "breakthrough" should be used with caution because the reality is often different from the message, and it is their responsibility to ask questions and get substantiated answers before rushing on air.

I will try to contact Dr. Riordan again and look forward to talking to him and getting more answers for all of us. As always, I will share any information I get.

Warm regards,

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Comment by amit gupta on February 3, 2011 at 4:40pm
Pat, do you think your comment "Cossu would like to move into trial, but to date, has not been able for a variety of reasons" and also Tremblay's admitted failure to do the same for the last 5 years is indicative of govt's restrictive policies more than anything else (which is probably what Calos was hinting at)?
Comment by Lisa Mancin on February 3, 2011 at 4:19pm

Pat, Thank you for the update on Ryan.  I am assuming that the tests were mainly done to determine whether Ryan has Beckers or Duchenne's.  If the tests show that he has Beckers, where will that leave further research from PPMD?

Also, there has been alot of research already done regarding delivery, distribution and immune response.  Stem cells are already being used (in this country) for many other diseases.  These issues have already been successfully addressed.  It would seem that, in order to save years of research, we could utilize the information that has already been proven.

Comment by Pat Furlong on February 3, 2011 at 3:00pm
Cossu would like to move into trial, but to date, has not been able for a variety of reasons.
Comment by cheryl cliff on February 3, 2011 at 2:53pm
I understand Pat.  Armit- now I understand what you are saying...too bad she isn't expecting to see Cossu begin trials.  I thought Debra Miller @CureDuchenne was funding this.  I had read his trials were delayed for lack of funding or something, although he did enroll enough patients for it.  Perhaps D.Miller can further expand on Cossu's status. 

Comment by Pat Furlong on February 3, 2011 at 2:47pm

HI all, I was not commenting on any specific person trial or planned trial, rather on the concept of stem cell research and existing barriers.



Comment by amit gupta on February 3, 2011 at 1:29pm
cheryl, i think what she meant was that Cossu will not succeed in starting trials.
Comment by cheryl cliff on February 3, 2011 at 1:12pm
Thanks Amit!  Does sound like she (Michele Calos) was saying her opinion is just what Pat said.  Thanks Pat and good news about Ryan's testing!!  Soon we will know more. 
Comment by amit gupta on February 3, 2011 at 12:19pm

Cheryl, she was specifically talking about Cossu's attempts at trials.

Comment by Pat Furlong on February 3, 2011 at 12:18pm
Hello Everyone, The genetic testing on Ryan and family are almost complete. We are waiting on one last test to be completed.
And it is very true, this is 'early' research.
There are inherent problems with stem cell research that include delivery, distribution and immune response. These are major barriers. Several groups are meeting to discuss the immune response in DMD and we will focus on this aspect during the annual conference in Baltimore.
Comment by cheryl cliff on February 3, 2011 at 11:50am
So Amit when Michele Calos/Stanford said "it is early" was it your perception that she was talking generally about stem cell research regarding duchenne?  Meaning, is it her opinion that stem cell science is in the earliest stages of research and not ready for clinical trials yet?

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