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,
Pat

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Comment by clinton bailey on November 13, 2009 at 12:41am
I too am happy for Ryan and his family. I am saddened by hearing that there is skepticism in the success of a boy with Duchenne MD "not experiencing symptoms he used to experience". I have met Ryan and his parents...they have gone through amazing times with this disease as many on this site have. I however am waiting for it to happen to us, and like the rest of you, am very scared for my son. I see him get worse and the hope that a story like this brings to me is the greatest thing I know. Be it false hope or true hope, it is hope! I don't care if the benefits are that his symptoms are less severe; that is better than what we have. Heaven forbid that nothing should happen, how horrible would that be? Oh wait, not any worse at all, it is actually the exact same thing. Who knows, maybe this is just the answer to give a little more time to some boys that may not make it to a cure that could be just around the corner. Maybe not, but I am glad someone did something and I think it needs to make its way into normalcy.
Comment by Lisa Mancin on November 12, 2009 at 10:41pm
YEAH!!! To Ryan and his family for taking things into their own hands. I am so happy that the stem cell treatment was successful and that they are happy and hopeful for the future. Who in the world would want to take that away from him or his family??? I think that it is absolutely amazing that his dystrophin level increased into the normal level after the treatment. Shouldn't we all be jumping up and down with excitement at that result?? No matter how much skepticism and negativity you have, there is nothing else available that can produce those kind of results. The myoblast transfer procedure of long ago can not compare to the stem cells they are using today.

Let's face it, stem cells are here and now. They are not a "thing" of the distant future. Dr. Riordan is just one of many doctors who are doing stem cell treatments in other countries. In fact there are over 500 stem cell treatment centers worlwide. And there are many people with MD , as well as other diseases, who are seeking out their help and having successful results. Ryan just happens to be one who went public with his story, and look where it got him. It is just so sad that our country is so far behind on stem cell research because it relies on Big Pharma to solve all of its problems. Drugs do not cure diseases. Stem cells do.

Sure, you have to ask questions and do your own research. And I bet Ryan and his parents did. It is irresponible and quite frankly insane to call Dr. Riordan a criminal or to judge Ryan and his parents for their decision. Stem cells are not bad science, they are THE answer . Maybe it is time we catch up with the rest of the world and figure that out.
Comment by Kelvinsmom - Michelle on November 12, 2009 at 8:44pm
We happen to be heading to San Jose, Costa Rica on vacation in a few weeks. I doubt we'll have a chance to talk to the doctor (and of course, I would never, ever do anything without tons of tests, follow ups, etc.), but I'd love to see which muscles were tested for the dystrophin, etc. as well as the complete reports. Is the child on here that had the treatments? They say that 3 other kids have had it. I'm wondering, are any of them on here? Michelle
Comment by Rosanna Ouellette on November 11, 2009 at 9:11am
I agree that Dr. Riordan should meet with the other Duchenne researchers to share his information. Maybe all these great minds could take the information and make something out of it. Thanks Pat for sorting it out for us.
Comment by Anne Hancock on November 11, 2009 at 8:07am
With all the news of possible breakthroughs it is overwhelming on so many levels. I am so thankful for all you do Pat. I agree with your list of "difficult questions." As a parent of a 7 year old with DMD, I am vulnerable and at times desperate. However, in my heart of hearts I know there is a difference between good science and bad science. I look forward to hearing what you discover.
Comment by Lori Ware on November 10, 2009 at 11:28pm
Thanks for checking Pat...and thanks for always being there for us!
Comment by Jason Darienzo on November 10, 2009 at 10:49pm
As an adult with Duchene, im 30 this "Dr. Riordan is playing with these parents emotions and these patients lives. Hs actions are against common sense and scientific methods and research protocals. The MDX mouse is not the gold standard for dmd research. Almost all DMD studies use immuno-suppresant MDX mice. The best test mice are double knockout mice without dystophin or utophin because they have the closest physciology to human patients. There is one study i can think of that used double knock-out mdx mice without having their immune system disabled, I believe Dr. Jeffrey Chamberlain was involved. So we have little knowledge of how a Duchene patients immune system would react to dystropin again. The point is the question of immune response is fundamental if there is to be any breakthrough in DMD treatments. This "Dr. Riordan" is criminaly negligent to inject even water into anyone once, nevermind 46 injections of live human cells. Tht guy Ryan could be facing a deadly immune response with that weight gain. Your not being negative your protecting people from a predator. He may be lying totally and injecting saline. Check out what hes doing with proper research the right way but tell everyone this man is a criminal. Anyone that brings there kids there is needs counseling. There are great dedicated scientist working on stem cells and they will bring treatments to clinical trials. We dont need people playing games and wasting precious time and money. These families could have donated their airfare and hotel expenses to real scientists working for cures not fame.

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