Friends this is an answer to so many prayers, there are no words to express our hearts just tears, tears and more tears. There is hope for our beautiful RCDP children. We are amazed at the goodness of God! My prayer tonight is that not one more child would be lost. We received this letter tonight from the Rhizo Kids Board of Directors. October 7, 2014 “Exciting” does not accurately describe the opportunity that has been offered to RhizoKids. As most of you know, for the past seven years we have been fighting and praying for a cure or a therapy for our beautiful children with RCDP. Dr. Nancy Braverman has dedicated her life to RCDP research, and the synthetic plasmalogen replacement that she has helped to develop has now gathered the attention of a drug company in Canada, that would like to bring it to a clinical trial next year in the fall of 2015. This trial will be open, free of charge, to any family with a child diagnosed with RCDP, in North America. The trial will consist of giving your child a weekly injection of the synthetic plasmalogen. The injection will be a subcutaneous (just under the skin) injection. Phase one will last six months and will be a test for safety and efficacy, which means that the injection site will be monitored to see if there is any reaction (SAFETY), and blood will be drawn periodically to test the plasminogen level and see if there is an increase (EFFICACY). This phase of the study will be monitored locally by your doctor. The next phase will be longer…a year or longer, and will be observing clinical changes in the children. During this phase, children will need to be seen in one of the designated monitoring sites that will be spread throughout the US. At this time, we know that Dr. Bober in Delaware will host one of the monitoring sites. The remaining two have yet to be determined. We believe that children will possibly need to be monitored every six months. We also realize that the travel expenses might be an issue for some families, so we are trying to work out a way that RhizoKids might be able to offer a supplement to participating families. There are many details that are being decided, as we are still VERY early in the planning stages. We understand that there are going to be many questions, so below you will find some of the questions that our Board has asked, and the answers that were supplied by Dr. Braverman. What kind of results are you seeing on the trials in PEX7 mice? “The company that I am working with to supplement purified, synthetic plasmalogens to our PEX7 deficient mice- is interested in starting a clinical trial late next year. We have data from the mice that short term (several weeks) , the purified plasmalogen recovers plasmalogen levels in the blood, lung and liver- but not yet in the heart, kidneys and brain. One thought is that longer studies will recover plasmalogens in other organs and we are doing these experiments now with the mice. However, it could be that we are stuck with the fact that getting plasmalogens into the brain may take years, or may not happen at all because the brain cannot take up plasmalogens from the blood. We will have to see. No one thinks we can correct what is there at birth- ie the rhizomelia and the cataracts, but since the brain lays down white matter over the first 2 years of life, and there are other complications in our kids, like lung disease, we would hope that these features could be improved. So we would hope that the infants enrolled would benefit at a developmental level, and older kids would benefit at a general health level, like less admissions for lung complications and possibly less deaths because of this. It is possible that the more milder RCDP patients might have the best results, but the study would include all patients-because it is such a rare disease and we need all patients for numbers.” Will international families be able to participate (providing they can set up a monitoring site)? “NO, BECAUSE THE COMPANY HAS TO GET AN IND ( INVESTIGATIONAL DRUG APPROVAL) THROUGH THE FDA AND HEALTH CANADA. SO, WE ARE SURE FOR NORTH AMERICAN PATIENTS, BUT THEY WILL NOT BE ABLE TO GO THROUGH THE LEGAL EXPENSES IN GETTING AN ADDITIONAL IND IN EUROPE”. The commitment that is being asked of RhizoKids is to: 1. Participate in the trial (if your child is still living) 2. Promote the trial and help with the recruiting process (Please remember, this is a study for patients with an RCDP diagnosis). At this time the company has asked for no financial commitment....they are in the process of seeking their own investors to join their company. However, they have asked that if we should know of any potential investors to please send them their contact information, etc. This trial will most likely be open to the first 40 RCDP patients to express their desire to participate. At this time we do not need a definite commitment from you, we only need to know if you are interested. Dr. Braverman has to provide the company with a number of interested persons so that they can determine if there will be enough participation to produce data for them to analyze. Also, if you choose to participate in this clinical trial, you will need to be registered in the Natural History Registry that Dr. Bober is maintaining for us at Nemours. Doctors will send their observations during the clinical trial to the registry, and this will be source of information collection for Dr. Braverman and the drug company as they analyze clinical changes in the children. Registration in the Natural History Registry is free. For more information and a registration packet, please contact We have ALL worked very hard to see a clinical trial come to fruition, so now the ball is in our court….is this what we want for our children? The RhizoKids Board has great confidence in Dr. Braverman and her decision to offer this trial to our families. Thank you for your consideration of this proposal, and for your support of RhizoKids. If you have further questions or need more information, please contact... Most Sincerely, RhizoKids International, Board of Directors RhizoKids Ohio
Posted on: Wed, 08 Oct 2014 03:53:09 +0000
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