Leading scientist form several universities around the world and different scientific institutions are participating in joint research for finding a cure for AHC. They are joining forces in a single project — the AAV project. The aim of the project is the development of genetic medicine that will make the disease curable, and the lives of children with AHC and their families — normal. Scientists believe that gene therapy is the only way to develop a cure for AHC as has already been done for several other diseases.
For the project and the drug to be successful, studies go through several stages. The first is to develop a functioning copy of the gene, whose mutation causes the disease, and to put it in a virus to take that gene to the affected neurons. The next stage is pre-clinical research — the developed virus is tested on mice to observe the efficiency of the cure. The third stage is a toxicological study — before the cure can be tested to humans, it needs to be tested on rats a primates first. These tests aim to prove that the virus will only have a beneficial effect on children with AHC. Once this stage has been successfully completed, there are a number of steps to ensure the high quality of the cure. In the next stage of the project, specialists are required to draw up a protocol that will describe precisely on what principle the children will be selected, on which to test and how to closely monitor the body reactions to the virus.
When all stages of the project pass successfully, clinical tests are the next step. So far, two hospitals in the world specialize in the study of AHC and are candidates to host the first tests on people with the newly developed medicine.
For each stage, however, funds are needed. Parents and volunteers around the world are involved in awareness of the disease and fundraising for the project, which gives hope for all the patients with AHC and their families.
The project started in 2012 — the year, in which new hope rises for thousands of families around the globe who face the symptoms of AHC every day and are powerless to relieve the pain of their children.
Foundation's accounts remain open for all people and organizations willing to support the AAV project. The three foundations - Cure AHC, AHC Foundation and Hope for Annabel have united their efforts to fund the project.
If you want to support the AAV project, you can also do it by donating to the "Alternating Hemiplegia in the Childhood" Association — Bulgaria. For more information, visit our donation page.