Open Treatments founder Sanath Ramesh and his son Raghav Ramesh
Ramya Ramaswamy
Sanath Ramesh’s life took an unexpected turn 2.5 years ago when his son, Raghav Ramesh, was born unable to move his hands and legs. After he learned the cause was a defect in a gene called GPX4—and that just nine people worldwide have this disease—he scrambled to find experts and a treatment.
Although Sanath Ramesh is not there yet, the software engineer now hopes to speed the process for other families facing rare genetic diseases by creating a nonprofit organization and a software platform to lead them through the drug development process. Called Open Treatments, the site launching today will “supercharge” patient groups by giving them a road map that allows them to operate like biotech companies, says Ramesh, who works for Amazon in Seattle. “My vision is to have 10,000 [groups] on the platform building treatments for diseases.”
Others say there’s value in the idea. “It really breaks [drug development] down so the patient organizations are empowered,” says gene therapy researcher Jude Samulski of the University of North Carolina, Chapel Hill, an adviser to Open Treatments. Several rare disease nonprofits, contract research organizations, and software groups are collaborating with the project.
Groups can create an account and get help with an array of tasks: searching the literature on their disease, finding experts, raising funds, and setting up clinical trials. For example, a user can find out whether researchers have access to mice with the genetic mutation that causes a specific disease and, if not, get an idea of the cost of developing a new mouse model (which can be $40,000 to $45,000) and how long it will take (30 to 40 weeks).
There will be a list of places to order research mice, such as the Jackson Laboratory. Users can also get help with things like legal agreements that allow researchers to exchange biological materials that might be patented or have other intellectual property protections.
For now, Open Treatments’s services are free and will rely on academic scientists and companies who are volunteering their time. But the organization might eventually charge users a small fee and compensate experts, Ramesh says.
Many other projects have similar aims, such as the Rare Genomics Institute, which sequences patients’ genomes, and the n-lorem Foundation, which develops oligonucleotide drugs for free. But Open Treatments may be unique in being so user friendly, Samulski says. Currently, he says, he assigns a postdoc to families that come to him for help with rare diseases, because navigating the research process can be daunting and time consuming.
But Open Treatments could shave years off the drug development timeline, Samulski believes. The problem now, he says, is that “everyone is reinventing” that process. “It doesn’t matter who they are or how smart they are,” he says. Open Treatments could help by providing a standard checklist for progress.
One group test driving the new site is Cure CMD, which works on several inherited muscle diseases. Spokesperson Terry Selucky says although research on treatments is well underway for some of them, the group needs help with others. And, “We do want to make the process easier for other disease groups,” Selucky says.
As part of Sanath Ramesh’s own odyssey, he raised $200,000 and found several existing drugs that have improved Raghav Ramesh’s strength and skeletal abnormalities. But the drugs haven’t helped with his neurological symptoms. Sanath Ramesh is now working on a gene therapy through Open Treatments.
The project’s goals are “ambitious,” says Sharon Terry, CEO of the Genetic Alliance. She thinks the platform may work best for groups that already have researchers from academia and industry on their team. But “for advocates and/or parents without sophisticated partners, it might be less useful,” she says. Still, given the costs and long timelines for developing therapies, Terry says, “anything to advance that trajectory will be beneficial.”
Source: Science Mag