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Arkadaşlar dün engelli sitelerini dolaşırken kök hücre tedavisinin müsküler distrofi hastalarında da uygulandığını okudum:clap: Hemen ilgili doktora translate çevirisi ile ingilizce bir mail attım. Cevap verdi ama translate türkçeye çevirmeye yetmiyor. Rica etsem çevirir misiniz? Benim için çok önemli bir konu
Mr. Cossu dedi ki:Dear Furkan,
Thank you for your message. We got good results by transplanting normal stem cells into dystrophic dogs, under a regime of immune suppression. We have published the characterization of the human cells that we hope to use in trials.
We plan a first clinical trial in patients, but still do not know exactly when (hopefully beginning of next year).
Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early in 2011 and will know how it works in the end of the same year.
If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.
Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.
With best wishes,