Gene therapy now offers a path to a cure. Not just for Batten Disease, but for hundreds of other rare diseases. This is a new kind of science—a one-time fix of the genetic code. No daily pills or injections to simply keep symptoms under control. Gene therapy targets the source of the problem: the broken genetic code. We all have broken codes, but for some, like Mila, the location of her broken code causes a devastating disease. Gene therapy has shown to stop and even reverse diseases like Batten.
STEPS TOward THE TRIAL
Below are the steps that our world-renowned team of scientists and doctors are working on now.
1. PRE-CLINICAL RESEARCH
In the research phase, the drug is tested in the mice for safety and efficacy. The drug involves the correct digital code for the disease-causing gene packaged into a benign tested virus. Data is collected to move into the human trial. Dr Steven Gray’s prestigious lab at the University of North Carolina is conducting all pre-clinical work.
2. DRUG PRODUCTION
The drug, or benign viral vector, is translated from mice to humans and produced under strenuous guidelines. Precision and safety are at the core of this process given the vector will be injected into the cerebrospinal fluid of the children in the trial.
3. TOXICITY STUDY
A portion of the drug being produced for the trial is injected into rats and monkeys under FDA-approved conditions.The goal is to ensure that the product is safe for administration into children during the trial.
4. TRIAL DESIGN
A dedicated team of doctors design the clinical protocol that will layout in detail how the trial will be conducted. The doctors work hand-in-hand with the pre-clinical team of scientists to come up with how the procedure will unfold, how patient follow-up will be handled, and so on.
THE CLINICAL TRIAL
Our trial will be a gene therapy trial direct to the brain. This will involve the viral vector carrying the correct genetic code being injected one time into the cerebrospinal fluid of children with Mila's type of Batten. We are in the process of signing a contract with a world-renowned hospital who has also generously offered to pay for the clinical aspect of our trial.
How expensive is gene therapy? $4 million. It costs money to save lives. Have a look at the breakdown of the costs of our trial work.
We need your help now
Mila has lost her vision and much of her ability to walk and talk. But she is a fighter. We need your help now to start a new clinical trial and give children like Mila a second chance at life.
Mila's Miracle Foundation, Inc. is a 501(c)(3) nonprofit organization. All donations are tax-deductible.