According to Fierce Biotech, what makes QurAlis fierce: There are currently four drugs approved by the U.S. FDA to treat ALS: Radicava, Rilutek, Tiglutik and Nuedexta, with Radicava’s 2017 FDA approval the most recent. But none of these drugs are curative, and all are limited in who they can help and by how much.
QurAlis wants to move beyond that. “ALS research been a minefield in the past,” said Roet, “ but that’s because we didn’t really understand what we were doing. We were trying to throw things up hill and just hoping something would hit.”But things are different now: “We’re really starting to understand this disease, and we are seeing there are different ALS patients and groups of patients, and these can be very different from other groups. What we now know is, these all each require a precision medicine approach.”These groups fall into three categories: motor system hyperactivity, toxic protein aggregates and cellular waste clearance.QurAlis aims to zero in on precision treatments by using the understanding of genetics in ALS, and new stem cell technology from Harvard University’s Kevin Eggan, Ph.D., who made the first stem cells from ALS patients and turned those into motor neurons.“That for me was the reason to start the company,” said Roet, “because we had the genetics giving us the understanding of the different groups that exist, but we couldn’t bridge those discoveries to the clinic until the discovery of this stem cell technology.”Roet worked on motor neuron projects with the labs of Clifford Woolf, M.D., Ph.D., co-director of the neuroscience program of the Harvard Stem Cell Institute, and Eggan, professor of stem cell and regenerative biology, who both helped found QurAlis, along with Roet and Jonathan Fleming, CEO of Eggan’s stem cell-focused Q-State Biosciences.Roet said animal models have been “horrible” in both neuro and ALS, but the stem cell tech offers a new tool to actually make neurons and use them at a larger scale to validate ALS targets, and also to select molecules. “We can now see these translate into the clinic,” Roet said.