Researching Ataxia Telangiectasia and finding out what is being discovered.
Some of it may be false and some may take the researchers to a dead end
One of the only things that helps me get through my day is
I have done some cutting and pasting and thought you may all enjoy reading what I have found so far.......
With the treatment for Ataxia Telangiectasia patients getting into full swing, it is very soon that the condition can be handled very well professionally.
Research is still going on in this respect.
EryDel S.p.A. (Urbino, Italy) said EryDex encapsulated in autologous erythrocytes met the primary endpoint in a Phase II trial in 22 patients with genetically-confirmed ataxia telangiectasia. The product significantly reduced the total International Cooperative Ataxia Rating Scale (ICARS) score from baseline to six months vs. controls (p=0.02). The controls were not disclosed. EryDex is a solution of dexamethasone delivered by encapsulation into autologous erythrocytes. EryDel plans to discuss the results with regulatory authorities and develop plans for additional trials that would lead to filing for worldwide regulatory approval. Ataxia telangiectasia is a progressive neurological autosomal recessive disorder
Allogeneic hematopoietic stem cell transplantation (HSCT) has not been a therapeutic option in ataxia telangiectasia (AT) due to overwhelming toxicity of conditioning in the context of the global DNA repair deficiency. Furthermore HSCT is unable to cure neurological involvement of AT.ncbi.gov
We are very excited to share this note that Brad Margus sent to over 400 researchers and clinicians:
Dear A-T researchers and clinicians:
Earlier this month, the A-T Children's Project joined others in sponsoring a meeting in India that included presentations about the ATM protein and related biological pathways. In addition, an excellent, clinically-oriented meeting has been planned by our English colleagues for this June that will focus on imaging studies, clinical scales, optimizing the management of the pulmonary, cancer and immune problems in patients, and therapeutic strategies such as steroids and read-through compounds.
I have now asked our A-T Children's Project staff to organize a third conference, for October of this year, that will focus solely on treatments for the neurological problems of ataxia-telangiectasia, sharing minimal overlap with these other meetings.We hope to bring together longstanding A-T researchers and clinicians as well as newly recruited experts in cerebellar function, movement disorders and drug discovery to prioritize treatments that can be tested in patients soon and to identify obstacles that may be preventing more rapid discovery and evaluation of additional therapies.
email from AT Children's Project.
Follow the link below and watch the 2 videos
Status copied from A-T Society, UK. Message from William our Chief Exec - Research is coming together! The ATW conference in India has just taken place; the A-T Society is organising a big Clinical Research Conference in Cambridge in June (21- 23rd, just before the family day); and now I gather that the AT Children's Project are looking to organise a third conference in the autumn on treatments for the neurological aspects of A-T. Things are really starting to move. Exciting times..!
The initial report on the Italian Dexamethasone trial led by Prof Luciana Chessa are out. In this trial, patients received the drug via a new technique where it is inserted directly into the red cells of the patients’ own blood. The good news is that there was a complete absence of the typical steroid side effects and there was a significant improvement in the neurological symptoms of some participants. However, there were also problems in getting the drug into the red-cells of some patients, which hadn’t been experienced with other conditions. The researchers, though, are positive they can resolve these problems and are hoping to organise a bigger trial in the near future. We will be studying the full results when published and working with them to assess the value and feasibility of setting up an arm of the trial in the UK.
AT Society in the UK.