Sanath Ramesh, a software engineer, created the nonprofit Open Treatments after his son was born with an extremely rare condition without any viable treatment options. Raghav, now two and a half, has a mutated GPX4 gene, a mutation shared by only nine people around the world. In an effort to find treatments for his son’s condition and other rare diseases, Sanath launched his nonprofit and software platform. It is intended to give patients and their caregivers the opportunity to operate just as biotechnology or pharmaceutical companies do.
Open Treatment
Open Treatment allows patient groups to take charge and play more of a role in drug development, which will hopefully lead to quicker drug development and more treatments for rare diseases. While the company is still growing and evolving, Sanath’s goal is for 10,000 patient groups to join the platform.
Upon joining, these groups must make an account before being able to utilize the platform’s features. These include:
- Locating experts
- Creating clinical trials
- Finding literature/prior knowledge
- Fundraising
To give a better example, if a patient group wanted to conduct a clinical trial using mouse models, they could locate a researcher, find out if they have access to the correct mouse models, discover the cost of the endeavor, and get an estimate of how long the process will take. Beyond access to the mice and other necessary tools, the platform also provides locations for research and legal agreements.
The platform is currently free to all, as it operates off of the generosity of medical professionals and other companies. This may change in the future, and users may be required to pay a small fee in order to compensate those helping.
Looking Forward
Open Treatment is not the first organization to take on this goal. The n-lorem Foundation and Rare Genomics Institute both have similar goals. The former develops oligonucleotide drugs without a price tag, while the latter sequences genomes of patients. Cure CMD is another company, and they focus on treatment for inherited muscular disorders. Where Open Treatment is unique is in its user-friendliness. As the rare disease landscape can be very confusing for patients and caregivers alike, this is very important.
In terms of his son’s treatment, Sanath has found a number of drugs that have improved his quality of life and raised a whopping $200,000. Unfortunately, none of these treatments have been able to address the neurological symptoms Raghav faces. The next effort will be a gene therapy, which will be developed through Open Treatments.
Read more about their story and Open Treatments here.