Lisa Jarvis: Parkinson’s disease finally finds a source of hope

Two small studies published just now in Nature offer early but key validation that stem cell treatments for Parkinson s affection are viable They also are a step toward a future where stem cells can be used not just to treat but ideally to repair or prevent damage to the brain Getting there will take incredible coordination and a continued commitment to understanding the drivers of neurodegenerative diseases we can t fix what we don t know is broken The treatments one originally developed by a band at Memorial Sloan Kettering Cancer Center in New York and the other by researchers in Kyoto Japan are the culmination of decades of work to figure out how to turn stem cells into functional therapies for Parkinson s To be clear these stem cells are designed in a lab and are not the same as the dubious therapies sold in stem cell clinics none of which are FDA-approved Parkinson s disorder is marked by a loss of neurons that make dopamine a chemical messenger involved in movement and coordination By the time someone shows signs of the sickness such as a hand tremor or muscle stiffness they have already lost anywhere from to of those nerve cells in the part of the brain that controls movement Since the s researchers have imagined using stem cells to replace those lost neurons At last it seems they are figuring out the right set of cues to prompt stem cells to turn into dopamine-producing nerve cells Moreover these two experiments which together tested separate therapies across patients offered hints that those cells once implanted in the brain might work as intended The main goal of both studies was to ensure the stem cells were safe well tolerated and feasible as a therapeutic So far so good There was one small caveat Because the treatments were made with donor stem cells rather than the subject s own cells an off the shelf approach that could make them easier to commercialize participants initially had to take immunosuppressants to keep their bodies from rejecting the therapy and specific experienced mild to moderate side effects related to those drugs Even better the cells settled right into their ecosystem and seemed to be functional even after people stopped taking immunosuppressants Once implanted a relatively straightforward procedure where millions of cells are scrupulously distributed in a part of the brain the young nerve cells need to mature and form the right connections to their neighbors before they can start shipping out dopamine That process takes a large number of months but the hope is that once that infrastructure is in place these cells could be functional for a multitude of years perhaps even for the rest of a Parkinson s individual s life Using an imaging technique that lights up the endings of the nerve cells that make dopamine the researchers manifested that people continued to produce more of the neurotransmitter than before the transplant And both research groups also exposed promising but preliminary signs that the approach could improve motor manifestations and potentially quality of life for various patients Of lesson much more work is needed to prove these treatments work Researchers must affirm their safety in larger studies and better understand whether these cells remain functional for the long term and can make a meaningful difference in patients lives To that end BlueRock Therapeutics a subsidiary of Bayer AG that licensed Memorial Sloan Kettering s stem cell machinery related to Parkinson s has begun a Phase trial to test its medication in roughly people Multiple other earlier studies are underway to test other stem cell approaches in Parkinson s Eventually Parkinson s patients will have to decide if they even want these therapies In the years it has taken researchers to get to this promising stage better options of delivering dopamine precursors to the brain or treating the movement responses of Parkinson s using deep brain stimulation have emerged Regardless this is an significant advance perhaps even more so for the promise it holds for other brain diseases Proving that stem cells can be safely implanted in the brain is a step toward researchers ultimate dream of designing therapies that go beyond reactions and can literally fix the brain or even protect it from future damage This is a proof of concept that we can repair parts of the brain to give it new life and function which opens the door to other neurological disorders says Viviane Tabar a stem cell biologist and neurosurgeon at Memorial Sloan Kettering Cancer Center Designing those therapies is by now the easy part says Lorenz Studer director of MSK s Center for Stem Cell Biology Things are going to go much more briskly from an engineering perspective But Studer cautions understanding the right way to apply those tools in other words knowing what endorsement cell or nerve cell to deliver into the brain continues to be a challenge There s a huge amount of work ahead but this proof of concept in Parkinson s should be motivation to keep pushing both at the basic biology and at driving stem cell treatments forward Lisa Jarvis is a Bloomberg Opinion columnist covering biotech vitality care and the pharmaceutical industry Previously she was executive editor of Chemical Engineering News Related Articles Christopher Cokinos To dumbly go where no space budget has gone before Jonathan Levin Warren Buffett caps a career built on humility Parmy Olson OpenAI can t have its money both means Lisa Jarvis RFK Jr s measles strategy is leading the US down a dark path Erwin Chemerinsky Trump wants to topple the republic s last line of defense