Stem cells, the source of all cells in the body, have the intriguing ability both to self-renew and transform themselves into specialized cell types. For this reason, they hold great therapeutic promise.
Project A.L.S.™ has pioneered the use of stem cells in ALS research since 1998.
Initially, it was hoped that we could effectively treat ALS with stem cell transplants, at first, by injecting stem cells into mice with ALS. Although Project A.L.S scientists at Johns Hopkins University showed a functional recovery after rats with an ALS-like disorder received stem cell injections (read the press release), transplantation has proved challenging.
While stem cell transplants remain a long-term goal, Project A.L.S.™ scientists are now using stem cells to create models of human ALS and to more accurately test for ALS drugs.
In 2006, Project A.L.S.™ opened the Jenifer Estess Laboratory for Stem Cell Research, the world’s only privately funded lab to focus exclusively on ALS and stem cells. Scientists at the lab have worked with many different types of human stem cells, utilizing a transcription protocol to turn them into functional motor neurons.
More recently, Project A.L.S.™-funded scientists at Harvard, Columbia, and the Estess Lab created the first ALS-specific stem cells, also known as iPS cells (watch the video). iPS cells gives scientists an unprecedented opportunity to study ALS in affected human motor neurons and other patient-derived brain cells. Project A.L.S.™ has derived patient-specific stem cells from over 50 patients and is currently using them to screen drugs.