Every year, our mission at the Bayan Hassaballa Foundation is to give grants to those organizations that make a difference in the lives of kids with cancer and A-T. This year, we are very excited to announce a $2000 grant to the A-T Children's Project.
It was founded by Brad Margus, father of two children with A-T, and the organization has done so much to help those kids afflicted with A-T, as well as their families. They helped us tremendously with Bayan. They recently sent our Executive Director Reem a letter about an exciting new discovery:
Dear Reem,
In the 24 years that I’ve been seeking support for our battle to treat A-T, I’ve always erred on the side of caution in describing what we hoped to achieve… maybe a slight improvement in a day-to day task like swallowing, or a small enhancement to some aspect of quality of life. But today, I’m bursting with excitement!
By applying a gene therapy approach called “antisense oligonucleotide” or “ASO” therapy, we may now have an opportunity to produce the functional ATM protein that is missing within the cells of children with A-T. I can’t be muted in my excitement today, because we may actually be able to stop the brutal disease process in at least one or two A-T kids this year!
Several ASO drugs have recently been approved by the FDA for other diseases that include Duchenne muscular dystrophy, spinal muscular atrophy and cytomegalovirus retinitis. Applying ASO therapy to A-T will involve making a drug that contains a short strand of RNA that will bind to the mutated site in the A-T child’s RNA so that the child’s cells avoid incorrect “RNA splicing” caused by the mutation. The drug will then be injected into the child’s spinal fluid so that it can reach the brain.
Initially, only the youngest A-T kids who have lost the fewest brain cells and who carry a certain type of mutation will be treated with this approach. In fact, a customized drug will be needed for each child. But despite these limitations, rescuing the first few children from this terrible disease will be the culmination of everything we have worked for. If we’re successful, we will then throw all of our resources and energy into addressing additional types of mutations and making the manufacturing faster and cheaper. Hopefully, we’ll eventually move onto many A-T patients, maybe even those as old as my A-T sons.
Since founding the A-T Children’s Project, we’ve focused primarily on treatments, with a cure in mind but always seeming so far away. And yet, here we stand, poised to leapfrog a symptomatic treatment and potentially give a full, normal, healthy lifetime to first a few, and then hopefully many more children born with A-T. I'm so grateful to you for previously supporting A-T research that has led us to this point.
To make this exciting next step happen, the A-T Children’s Project will need to spend $1.4 million over the next twelve months. Please, please consider joining Vicki and me in making a special donation and designating it for this initiative.
All the best,
Brad
Brad Margus
Volunteer Chairman and Founder
We are super excited about this development! Thus, we decided to support this wonderful organization with a large grant this year, to help them achieve something that was once thought to be unachievable.
And all this is because of you, our Bayan's Angels, who have generously supported us and our charity. We could not have done it without you. Thank you.