Health

HEALTH • Spring 2017 17 Exploring treatment Stargardt's macular degeneration (SMD) An inherited disorder of the retina causing vision loss in childhood or adolescence, usually pro- gressing to 20/200 vision but not complete blind- ness. Affects 1 in 8,000 to 10,000 people. Dry age-related macular degeneration (AMD) Gradual breakdown in light-sensitive cells in the macula, usually starting at age 50 or older. Affects around 1 in 2,000 people. Retinitis pigmentosa A group of genetic disorders that involve a breakdown and loss of cells in the retina, typically beginning in childhood and eventually leading to a near-total loss of sight. Affects around 1 in 4,000 people. Glaucoma A group of diseases that damage the optic nerve, resulting in vision loss and blindness. Second leading cause of blindness in the world. Lupus Potentially fatal disease affecting an estimated 240,000 Americans, mostly women. Believed to be an autoimmune disease, affecting many parts of the body including joints, skin, kidneys, heart, lungs, blood vessels and brain. Crohn's disease A chronic disease of the gastrointestinal tract affecting as many as 780,000 Americans. Symptoms including diarrhea, bleeding, loss of appetite, cramps, weight loss, and fatigue. Sources: U.S. National Institutes of Health National Eye Institute; U.S. National Library of Medicine Genetics Home Reference; Glaucoma.org, Crohn's & Colitis Foundation AIRM is working on therapies to address a range of medical conditions 1994 Advanced Cell Technology Inc. established 1999 Chief Science Officer Robert Lanza joins ACT 2010 Company receives FDA approval for the first human clinical trial using embryonic stem cells to treat retinal diseases CEO William Caldwell IV dies and is suc- AIRM timeline ceeded by Gary Rabin January 2014 ACT agrees to pay $4.1 million to settle SEC claims that it sold billions of shares of unregistered penny stocks between 2008 and 2009; Rabin leaves the company after fail- ing to report stock sales between 2011 and 2013 October 2014 ACT Phase I/II clinical trial results for Stargardt's macular degeneration (SMD) and dry age-related macular degeneration (AMD) published November 2014 ACT changes its name to Ocata Therapeutics September 2015 Ocata enrolls the first patient in Phase II dry age-related macular degeneration trial February 2016 Astellas Pharma Inc. acquires Ocata for $384 million; becomes AIRM tified us as one of the leaders in regenerative medicine," Lanza said. "So they're leaving it to us, with our experience, the scientific back- ground, to decide what's feasible." Lanza has high expectations for feasibility, starting with finally commercializing the therapy for vision problems that the company's been working on for years. The treatment targets two forms of vision loss that currently have no cure, Stargardt's macular degeneration (SMD) and dry age-related macular degeneration (AMD). So far, Lanza said, 38 people have received treat- ment for the conditions through clinical trials, and all but two have had their conditions stabi- lize or improve. One patient whose story stuck with him was a cowboy from Kansas who had been blind in one eye has had his vision improve to 20/40. "He can now ride his horses again," Lanza said. "It makes me feel good to know that (we're) helping people. People can now go to the airport on their own, read their watch, use a computer." Beyond treating those conditions, though, AIRM has bigger plans. Lanza said cell lines the company has been working with have been shown to restore vision in totally blind animals suffering from a disease called retinitis pigmentosa, and to restore function in the eyes of mice with glaucoma. Other applications Other cell lines show promise for treating dis- orders of the immune system including lupus and Crohn's disease. In 2015, AIRM tested one such therapy on six pet dogs with the canine equivalent of Crohn's who were patients at the Cummings Veterinary Medical Center at Tufts University. A paper published at the time report- ed that all the dogs showed improvement, and only two relapsed. Lanza said Astellas is also thinking bigger. He said he and other research leaders are now col- laborating to put resources into therapies for the most deadly ailments—such as kidney, heart, and liver diseases. "We put together a dream team," he said. While he works with researchers all over the world, Lanza said he's happy to be doing it from Marlborough. For scientists in the Boston area, MetroWest can be either a great place to live or an easy commute from the city, going against the flow of traffic. And the vibrant Boston-area biotech research world is appealing to new hires from other countries and other parts of the U.S., he said. "You have a lot of the top universities here," Lanza said. The proximity to big research institutions is helpful not just in recruiting but also because it allows for more interactions with leaders in the field. It even helps with basic logistics, Lanza said: if AIRM wants to get cell samples from Boston, someone can just drive them over rath- er than worrying about shipping. Today, AIRM has reached the maximum capacity of its current building in Marlborough, and it will be expanding as it continues to add to its headcount, Lanza said. "We've already hired some of the top scien- tists in the field, and we plan to continue expanding here in MetroWest," he said. H Astellas Institute for Regenerative Medicine (AIRM), Marlborough

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