Last week I attended the NHLBI meeting in Washington DC. This meeting involves HLBI public representatives and provides an overview of NHLBI's overall interest.
1. Presentation by Center for Alternative Medicine (CAM). This is complementary and alternative medicine. They spend 121M/year and support approximately 300 projects. They evaluate modalities that are held out to the public as hopeful but actually may not be worthwhile. Recent findings include a) Tai Chi - reduces stress and improves defense mechanisms (antibody response to shingles virus for instance). b) evaluating Chinese herbal formula that may be helpfu for peanut allergies c) Evaluating Ginkgo for dementia d) accupuncture thought to increase rate of IVF pregnancy. They will be launching a website Time to Talk (NCAM). They discussed the fact that there is no simple way to tell hype from hope, that rigorous trials are the only way to tell the difference. They have significant concerns that drug/drug and drug/herbal combinations represent substantial concern and recommend that patients/family members tell their physicians everything they are taking/giving. Discussion also centered around companies using the word 'natural' and CAM physicians discussed that this word "natural' does not mean there are any guarantees of safety and/or no concerns of hazards. Product integrity has to do with 1. stability of the produce, 2. contaminants 3. composition. Keep in mind all supplements and nutraceuticals are labeled as 'foods' by FDA and often have considerable variation in terms of composition.
2. Losartin and Marfan. Losartin is currently in trial for Marfan syndrome. The trial is current recruiting patients (need 600/have 200 patients recruited). The Marfan community are using the Pediatric Heart Network, an NHLBI established and supported clinical trial network. For the trial, the dose of Losartin is 1.4mg/kg once/day. While, there is no patient data available, I did find the mouse data quite interesting. Marfan is a autosomal dominant genetic disorder of the connective tissue. Patients with Marfan are typically tall stature and have a predisposition to cardiovascular abnormalities, specifically those affecting the heart valves and aorta. Slides of the Marfan mice showed the enlarged aorta. In the very young mice, Losartin was able to restore the aorta to normal size and showed improvement in skeletal muscle. In older mice, Losartin seemed to have less effect, perhaps a stabilizing effect rather then improvement. While we cannot extrapolate this to DMD, it does give us something to think about. For those families who have tried Losartin or are considering Losartin, it just might be worth putting in the back of your head, that timing is critical (and I think we all agree, timing of interventions is a critical factor), that if there is benefit, earlier may have be more significant effect. I guess the next question is 'what is early' and I don't have that answer. You would need to do a risk/benefit analysis with your doctor. Losartin has been used for more than 20 years, has a very low side effect profile. Young is ... diagnosis? This is a discussion for you and your son's physician(s).
EndDuchenne for every boy/young man at every stage...