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Hello Pat,
Thanks so much. I look forward to the information!
Sincerely,
Lisa
Hello Lisa,
yes we do have the results of his testing. We have written up the information and plan to circulate it through our clinical advisors, Ryan and his family. I am hopeful we will post the information sometime this week.
Sincerely,
Pat
Hello Pat,
I wondered if you had found any answers to the questions you had mentioned on your February 3rd post, regarding Ryan's test results. I was interested to know if there was any new information from PPMD on the stem cell issue. Thanks so much!!
Amit, there are so many factors to include federal policies on stem cell research, the public's view of stem cell research and its implications, FDA regulations and other factors. Long ago in my career, I was involved part of the transplant team at OSU. We have come so far in whole organ transplant, but we had to learn so much over the last 35+ years and now it is routine... all except for the fact that we do a boatload of studies to determine compatability, use sophisticated immune protocols. The transplant teams are amazing and operate like a well rehearsed orchestra.
For Duchenne, we would need to identify the right type of cell. Cossu uses mesoangioblasts. Tremblay uses myoblasts. So, what is the 'right' cell? How will you deliver it? How will you insure that whatever the number of cells (probably billons) gets into muscle and into every area of the muscle and then every muscle of the body or at least major muscles? And if you are able to deliver to major muscles, what happens to the auxillary muscles. Will they hold up to the burden? How will you make sure that the cell you have delivered understands it is to make muscle rather than (for instance) a fat cell? And how will you prevent the immune system from recognizing that these cells you have delivered are not 'self' and send in specialized cells to get rid of them?
Duchenne is extraordinarily complex and I think we may not yet know some of the questions.
By the end of February we will have Ryan's data. I am hoping this will shed some light on some of these questions or maybe just add to our current list of questions.
Hi Again,
The tests are done to answer the following questions.
1. What is Ryan's diagnosis
2. Are his sister's stem cells evident in his muscle -i.e we arelooking for donor DNA in Ryan's muscle.
3. Are other members of the family carriers?
How will this data inform PPMD's research invesments in the future? Let's wait for the results and discuss.
And you are right, there is a growing body of research on delivery and immune response, currently the major delivery pathway is bone marrow, blood or nerve - targeted tissues as opposed to, for instance every muscle of the body.. There are immune protocols used in bone marrow transplantation and whole organ transplant are quite useful and serve as a basis for the discussion for work in DMD. The idea here is that whoever does a trial will need to thoughtfully consider an immune protocol and in DMD, this has been considerable discussion as it is anticipated that there will likely be significant variability due to degree of immune response to muscle degeneration and cell death. Keep in mind, the immune response kicks in in varying degrees, at various time points, individual variation (is considered why some do not respond to steroids) and recently Mendell and others suggest some boys may produce an immune response to revertant fibers. It is just not simple and there is a lot we do not know.
. So, the short answer is that the growing body of evidence in stem cell transplantation will enable Cossu and others to build upon the base of knowledge and apply it to Duchenne. But that this approach is still very early days in stem cell transplant for Duchenne. We have not yet delivered stem cells to a single muscle, confirmed that they are in place in sufficient numbers to have an effect, let alone delivery to a limb or the entire body.
It makes perfect sense theoretically, it is just that the devil is in the details.
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