Drug Repurposing
Also known as drug repositioning, is the process of identifying new therapeutic uses for existing drugs. This approach leverages the fact that many drugs, already approved or in development for one disease, may have potential to treat other conditions. Repurposing can accelerate the drug development timeline and reduce costs because it involves compounds that have already undergone significant testing for safety in humans.
Key Aspects of Drug Repurposing:
- Preclinical Insights: Researchers may discover that a drug has additional mechanisms of action that could benefit conditions unrelated to its original purpose. For example, sildenafil (Viagra), initially developed for hypertension, was repurposed for erectile dysfunction.
- Data Mining and Computational Methods: Databases of drug properties, genetic information, and disease pathways can be analyzed using artificial intelligence (AI) and bioinformatics to identify potential repurposing opportunities.
- Clinical Observation: Off-label use by clinicians may highlight unexpected benefits of a drug for conditions other than its approved indication. Off-label means your physician can prescribe a drug for use for another purpose other than its intended use, if medically appropriate.
- Screening and Testing: Drugs can be tested in new disease models, including in vitro (cell-based) and in vivo (animal-based) studies, to evaluate their efficacy. We have access to existing H-abc mice models.
- Combination Therapy: Some drugs are repurposed as part of combination therapies, where their mechanisms complement other drugs to treat complex diseases, such as cancer or infectious diseases.
Benefits of Drug Repurposing:
Faster Development: Since many drugs have already passed safety testing, repurposing can shorten the timeline to clinical trials.
Lower Costs: By avoiding the early stages of drug discovery and safety profiling, repurposing is less expensive than developing a drug from scratch.
Addressing Unmet Needs: Repurposed drugs can provide new treatments for rare or neglected diseases where traditional drug development is less financially viable.
The Foundation has identified and in negotiations with a drug repurposing AI-enabled therapeutics company that focuses on developing treatments for complex diseases by integrating patient data with advanced computational analyses. Their proprietary platform combines target and drug discovery, preclinical screening, and patient stratification to identify effective treatments efficiently. Their approach requires sampling of an affected patient and their same sex biological parent to gather RNA samples. Including the parent allows them to compare the affected patient with an unaffected parent and is critical to the analysis. This involves nasal swabbing of both to gather the samples over a short period of time, much like swabbing for covid virus, so its safe, non invasive and familiar to most of us. Anyone across the globe can participate as long as they have a mailing address. We need at least 20 patients (again along with their same sex biological parent which is a must), to participate to make this viable. The more the better! To be clear, this is not a cure, but a possible means of treatment.
Also, there are no guarantees we will be successful, but if we don’t try, we won’t know. Our hope is we identify an existing drug or compound that can be prescribed by and under the oversight of your physician to help as a means of treatment. And/or identify a drug candidate and modify for a new therapeutic use which can expedite clinical translation and provide near-term benefits to patients which will be a longer process. We will be involving key clinical advisors in this effort.
Once we identify patients who are willing to participate in the data collection, the Bio tech will manage the process from there. Process takes three months assuming the data is collected in expeditious manner. They will report back to the Foundation on the findings at which time we will share!
Please complete the form below if you want to participate, again first step is to secure at least 20 patients/parents to proceed. Assuming we proceed, we will host a session with the patient community and the Bio tech to further explain the process and next steps.