Letter from R.\'s parents
Diagnosis: Duchenne muscular dystrophy
This is a description of the events leading up to our meeting with you.
Since R. was young, we had noticed him having difficulty with walking and climbing stairs.
Approximately two years ago, we had taken him to his regular physician for a complete smack test with blood work. One of these tests concerned the CK amount. We inquired about the results and were told they were fine. Approximately one year later we were concerned about his progress and consulted another doctor who referred us to the Muscular Dystrophy clinic, at which time they performed the same tests which revealed the elevated CK enzyme. Further DNA testing revealed the deletion of the dystrophin gene. From there, I requested the prior smack results which also revealed an extremely high CK reading, which revealed that the doctor did not read the results correctly. Therefore lying to us that everything was all right.
From there, we dove heavily into researching this disease and the devastating effect it has on the victims.
We had traveled out to Memphis, Tennessee to look into a cell therapy procedure. Upon further investigation, we decided against this because of the makeup of the material being used and the way it was administered.
From there, after more investigation, we traveled to Ohio to investigate a gene therapy procedure. We concluded that from the phase of this study, it was still in it's infancy. Fortunately, we have a limited knowledge of genealogy and what was required to repair the DNA.
From all of the research we gathered, we believed that an fetal stem cell transplant could be beneficial.
After many, many hours we were lucky enough to contact a clinic called EmCell where we developed a dialog with two very caring professors. We explained our situation and they believed they could be of help. Before our first visit to EmCell, R. was on a fairly steady decline and we decided to go to visit Professor Karpenko and Professor Smikodub at the EmCell clinic.
Upon our arrival, we were greeted by a very friendly and knowledgeable man named Professor Karpenko. We had a very long conversation where he explained the procedure, which was to be administered to our son. It sounded very promising and very unintrusive.
The following day we began the treatments which included three IV treatments and approximately 8-10 myoblast injections. The procedure encompassed six days at the clinic. After the second day of treatments, we noticed a remission and improvement in our son's ability to bend his knees, run, sit up from the floor and also with his confidence level. This we attributed to our son feeling stronger, which our son also conveyed to us. Also, our son's reflexes and respiratory functions improved. All within the time of the treatments.
The Professors explained that, for approximately 10 days after the treatment, R. would experience a slight decline, which he did. After approximately 10 days, we noticed a steady increase, which leveled off and was stable for approximately 5 months.
Within these months we had regularly taken our son to several doctors including a neurologist and a BIODEX muscle strength testing machine which is also employed by NASA and a pulmonary function doctor. They have all recorded absolutely no decline for approximately four months after treatment. During these months we noticed our son was moving much better. Prior to the treatment, our son had a waddling in his run and walk, which was dramatically reduced. These results are still present.
After five months from the first treatment, we noticed a decline occurring which was explained to us by the Professors at the time of the first treatment. We were informed to make arrangements to return when the decline occurred. At this time, we returned to EmCell for the second round of treatments, which were similar to the first treatment. Also at this time, after the second day of treatments, we were amazed at our sons ability to do things he had not been capable of doing before. We have since returned approximately two weeks ago, and now we are observing a consistent increase in our sons ability.
At this time, we would like to add that the treatment and the experience of traveling to EmCell clinic and meeting the Professors have been very invigorating and pleasurable. We can really tell that the Professors heart and soul are in our son's treatment and we now refer to the Professors as our true friends. Their caring and dedication with our son has proved us beyond a doubt. It is really a shame that this procedure is not allowed to be performed in the United States. There are thousands of boys with Duchennes Muscular Dystrophy who could benefit from this treatment. As for our experiences, we have nothing but good to report to our friends and we look forward to the increasing strength our son is experiencing. We also thank God for meeting these people and giving us the strength and determination to overcome borders and stereotypes to seek help, wherever it may be. Myself and our families would strongly recommend this treatment with Duchennes Muscular Dystrophy.
We would also like to add that it will be a much happier Christmas this year, than last.
Thank you very much,
The R. Family