I’m Alla, Artem’s Mom. Every single day I wake up and go to bed with one thought that pervades everything I do. My son is dying and I cannot do anything: I cannot breath for him, I cannot stop his pain, I cannot save my baby from the arms of death. This is a terrible, terrible feeling I live with every second of my life.
Ten long years my husband Norair and I could not conceive. When finally I got pregnant I was the happiest woman in the world. I couldn’t wait to see my baby for the first time. I could not wait to count his toes, to smell the first fluff on his head.
Artem was born healthy and strong (9.2 pounds, 20 inches long) with no symptoms of any condition. First months he slept and ate a lot like any child of his age. At 4 months checkup our pediatrician noticed that Artem could not concentrate on his favorite toy and ran an additional genetic test. The result came devastating — our baby was diagnosed with fatal disease called Canavan.
Canavan disease is a progressive, neurological disorder that begins in infancy and affects the brain. Artem lacks an essential enzyme, an important one, that causes toxic build-up in his brain. His motor skills have already been impacted. He cannot walk, sit, or stand on his own. As the condition progresses he will develop hearing loss, blindness, and total paralysis. If left untreated, the average life expectancy of children with Canavan is no more than 10 years old.
When we first learned that our baby inherited this condition we were in shock and paralyzed. However, the more I read about Canavan, the more I learned. Professor P. Leone and her group at the Department of Cell Biology & Neuroscience at Rowan University in New Jersey, USA have made major breakthroughs in the understanding of how this condition can be treated with gene therapy.
At the moment Artem is 19 months (he is going to turn 2 y.o. in June) and his brain still has plasticity. It means that we need to move fast and get this treatment as soon as possible. It is important to act Now. The therapeutic window for Canavan disease is up to 3 years of age.
Our #1 fundraising goal is $200,000 dollars to help bring gene therapy treatment out of the lab and available for children like Artem who have Canavan Disease.
We cannot watch our baby die, not when there is a treatment available that could save him. The $400, 000 will help to fund a clinical trial initiated by families with children afflicted with Canavan disease.
By supporting our fundraising efforts, you can help save our boy’s LIFE and be a part of the future of medical science.
Artem with his favorite person and big brother Sergey (below)
I love Artem more than anything else in life. He is my everything. My little hero, my sweetheart.
So many people heard us and reached out to help.
We feel so humble and blessed with all of your support.
The words cannot explain how deeply touched and grateful we feel.
Thank you for helping us save our baby! ❤️
Alla, Norair (Artyom’s Mom and Dad) and Sergey(12 y.o)
HOW YOUR MONEY CAN HELP:
1. Donate any amount: $5, $10, $50. Every little helps.
2. Email saveartem.com web link to all contacts you know
WHERE IS YOUR MONEY GOING?
Every dollar goes to the 501(c)(3) nonprofit organization, Rowan University Foundation. Rowan is funding this experimental treatment for small group of children including Artem. The scientists at Rowan developed a novel gene vector that has had remarkable results in the lab. At the moment we're funding the IND (Investigational New Drug) stage of the treatment development. If successful, the new gene therapy for Canavan disease will be available to all patients in need. 100% of the funds raised here will go toward covering manufacturing cost of new gene therapy at the GMP facility for Artem.
WHY THE TREATMENT IS SO EXPENSIVE?
In the USA the manufacturing costs of medicine for Rare Diseases usually extremely expensive and new drugs/procedures need to go through FDA approval as well. Currently there is no investor or manufacturer in the US to cut the costs for IND. That is why the cost of participation in the experimental treatment is so high.
WHAT HAPPENS NEXT
P. Leone and her team are working tirelessly to meet all FDA requirements to get the clinical trial up and running.
There are few important milestones needed to happen before Artem can receive his treatment:
• Final safety studies (accomplished)
• FDA approval to proceed with IND (accomplished)
• Manufacture GMP level drug production (in progress)
• Start IND
Video© Vlad Pervozvansky
Photos © Vera Smirnova
- Юлия Тарасевич
- Maria Guliaeva
- Elizaveta Kazantseva
- Yelena Bibik
- Natalia Ozkul
Fundraising team: Сanavan Research Foundation (3)
New Fairfield, CT