NIH starts new center for Rare Diseases, HemaQuest starts phase 2 drug trials, NHS wrote official guidelines for sickle cell patients and new research on how sickle cell protects us from malaria. Catch your sickle cell news updates here.
The NIH, an international consortium of patient advocacy groups, and research funders have vowed to discover 200 new therapies for rare diseases by 2020. They are first focusing on 5 rare diseases, and fortunately for us, sickle cell is one of them. The National Institutes of Health is opening a center in the fall to translate research findings in genetics to usable therapies. They plan to re-examine existing research in the hopes that this will provide a key to finding the solution to these rare diseases. Hopefully they will be able to achieve their objectives. Read more on the story HERE and HERE.
HemaQuest has enrolled the first sickle cell patient in the phase 2 of their multi-center randomized clinical trial. The trial will be conducted in US, Canada, Lebanon, Egypt and Jamaica and will enroll about 50 patients for the drug HQK-1001. HQK-1001 is a medication that stimulates the production of alpha-feta-proteins and red blood cell production in patients with sickle cell and thallesemia. We first heard about it in December last year when they were approved by the FDA to begin the clinical trials. Preliminary results should be out by late 2011 with a final report out in 2012. Read the press release HERE.
In UCLA, a new medication has entered phase 3 trials. The drug (Yet Un-named) has been developed since 1992, and will be available for compassionate use in Kenya within a year. Dr. Niihara (primary researcher of the drug), said that it is an L-glutamine medicine that ameliorates effects of sickle cell by reducing oxidation in red blood cells so they do not deform. There isn’t much news about it, and I’m surprised that they are doing phase 3 trials in Kenya vs. the US…but I’m sure we will hear more soon. Read more HERE.
In the UK, the National Health Service has written a set of guidelines for sickle cell patients. The Royal College of Nursing accredited Competence Framework: Caring for people with sickle cell disease and thalassaemia syndromes, details what nurses need to know if they are looking after patients with these conditions.
Nurse advisor at the Royal College of Nursing, Rose Gallagher, said: “This project is designed to better equip nurses to deal with sickle cell and thalassaemia. This effort has never been more urgent. Despite the fact that sickle cell is now the most common serious genetic disease in England, we still see tragedies arising from ignorance and misunderstandings. Nurses are in a unique position to prevent these.”
I was super stoked to read this news. I think that this a huge step in ending medical ignorance in regards to sickle cell. Now if only there was a way to make these guidelines mandatory to all medical professionals all over the world….*sigh* Read more HERE.
The biggest news this week was that a research team in Portugal has identified that exact mechanism that sickle cell gene/trait protects against malaria. We already knew that sickle cell trait/gene prevents fatal cases of malaria…but how does this happen? Well, it is super scientific for even me to understand, so Here is the Link for you brainiacs;)
This rounds up the latest sickle cell news and research. See you next time.