An unusual business model that encourages transparency is trying to beat a rare and deadly childhood cancer

M4K Pharma, or Medicines4Kids, has an unusual business model. Owned by the Canadian charity Agora Open Science Trust, M4K wants to find a treatment for a rare pediatric cancer through totally open science. While allowing for patient confidentiality, M4K’s data will be freely available to any scientist who is interested.

“They want to get a compound into the clinic as quickly as possible,” said Sue Cramp, Group Leader in Chemistry at Charles River’s Harlow, UK site. “Their view is that [open science] dramatically lowers the time and cost, because you are not restricted by patents or keeping things secret.”

The organization is focused first on diffuse intrinsic pontine glioma (DIPG), a rare brain cancer found in children. Currently there are no treatments for DIPG, and the condition is always fatal. Through partnerships with companies like Charles River, M4K will strive to find a treatment as quickly and efficiently as possible.

M4K’s current funding comes from government grants and donations, with Charles River and other companies performing pro bono research to help deliver drug candidates. For example, either using available resource in the lab or through additional volunteered time, Charles River chemists design and synthesize compounds alongside other M4K partners, like the Ontario Institute for Cancer Research (OICR). All of Charles River’s contributions to M4K are overseen by Project Leader Cramp as a Charles River Corporate Social Responsibility Project.

Some Charles River employees also provide consultancy services to M4K. These consultants will provide drug discovery and development expertise to help M4K achieve their objectives as quickly as possible.

“They are planning on using exclusivities granted during the regulatory process to prevent generics coming in at [the clinical] stage,” Cramp said. “Particularly if it is an orphan disease, you can get extra exclusivity time. Patents would provide protection in the pre-clinical phase to prevent others from pursuing similar chemical targets but this is not consistent with the open science approach.”

The organization is still working on building a database, but for now the data they have generated is available through their recorded meetings. Anyone can view the meetings via their website, blog or on Twitter. They also include slide decks of their current research.

Plans are already in place for a second venture, which will be called Medicines 4 Neurodegeneration (M4ND Pharma). This new project will focus on genetic drug targets for Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS), though the details still need to be worked out.

Cramp acknowledges that while the open science approach may not be feasible for all diseases, the model lends itself well to orphan diseases. Compiling all the work in one place can help overcome the double issues of lack of funds and lack of research that plague most rare diseases. The model also allows organizations like Charles River to contribute whatever time and resources they can to helping future patients.

You can learn more about the M4K Pharma project by attending this oral presentation at AACR on Tuesday, April 2 from 3-5 pm. at Room B206Georgia World CC.