A path forward, for one or for many
It’s still “rare disease day.” Every day is rare disease day for people living with rare diseases and for their family members and loved ones whose lives are impacted.
Last year on this day, I published an article titled, “It’s Monday. It’s still Rare Disease Day.” One of the key points was the notion that rare diseases aren’t rare. This reality is something that every active member of the rare disease community is painfully aware of; it is a key driver for why we started Rarebase. At Rarebase, we leverage the latest technology used in the most advanced biotechnology companies, and we put patients at the center of our approach. We can all agree that there are many rare diseases - 7,000 have been identified so far. Rather than attempting to address each rare disease one by one, Rarebase tackles this unsolved problem of rare disease drug discovery at large.
This is where our Function™ platform comes in. By partnering with many families and foundations each working on one rare disease, we are able to apply state-of-the-art technology to drug discovery for rare diseases that otherwise do not get attention from the pharmaceutical industry, and recruit a world-class team to tackle this collective challenge. This combination creates an opportunity to conduct biopharmaceutical quality drug discovery while prioritizing urgent patient community needs and timelines.
What would the biopharmaceutical industry look like if the main driver is to find treatments that help patients as quickly and safely as possible? I believe it would reflect a greater balance between developing new medicines and applying existing medicines wherever a significant patient benefit can be found.
Rarebase’s mission is to provide a path forward for anyone diagnosed with a disease that doesn’t have an effective or satisfying treatment yet.
Innovating and developing new medicine is a costly and time consuming effort. Don’t get me wrong, novel drug development is critical, but for many individual rare diseases, it is currently cost- and time-prohibitive. Setting up a clinical trial, even in a rare disease, can easily cost tens or hundreds of millions of dollars. No matter how sophisticated a drug discovery technology is, the drugs in development ultimately must prove effective in patients. Humanity doesn’t yet possess the technology to confidently predict whether a drug will be effective solely from preclinical experiments. This reality explains the high rate of failed clinical drug trials .
At Rarebase, we do spend our time and energy innovating new medicines like biotechnology companies are designed to do. But critically, we put equal time and effort into every partnership with research foundations and families searching for new applications for medicine that’s already being used for other diseases. There are around 4,000 FDA approved drugs on the market . Each drug has known safety profiles and has already gone through the costly and time consuming process of demonstrating efficacy in human clinical trials. If we can find new associations between these existing drugs and a rare disease of interest, we have the chance to apply existing drugs in novel ways to help our patients in the clinic much more quickly and cost effectively.
Finding new applications for existing drugs should be the first priority on our agenda, while we work on enabling the creation of new medicine in parallel.
I am personally benefiting from a repurposed drug. While I have a genetic tumor condition called NF2, I am currently using a drug (called Everolimus) that's on the market as an immunosuppressant for patients receiving an organ transplant. In that case, the drug prevents the patient's own immune system from rejecting a new organ. In my case, it has successfully stabilized the growth of my brain tumor for the last 2 years – with very manageable side effects. I am personally witnessing the power of drug repurposing, and we want to enable these opportunities for everyone. Finding new applications for existing drugs should be the first priority on our agenda, while we work on enabling the creation of new medicine in parallel.
Rarebase’s mission is to provide a path forward for anyone diagnosed with a disease that doesn’t have an effective or satisfying treatment yet. As patients, we don’t want to hear “there’s nothing we can do” or “just wait for the science to catch up.” Instead, we want to be in the driver’s seat. We want to be part of the solution, even if our chances for treatments are slim. Most importantly, we want to know that at the very least, we have maximized our efforts to apply the latest state of the art technology to improve our quality of life.
We want a path forward.