Don't Forget About Dr. Lanza

Stem-cell biotech seeing strong growth

By Jonathan Saltzman Globe Staff,July 2, 2019, 5:57 p.m.

President Yoshitsugu Shitaka said the institute has the medical technology that has the potential to treat many diseases.(SUZANNE KREITER/GLOBE STAFF)

In February 2016, Japanese drug maker Astellas Pharma paid $379 million for a small Marlborough biotech that developed promising stem-cell therapies, including a potential one-time treatment for a leading cause of blindness.

The acquisition overcame stiff opposition from shareholders in the biotech, Ocata Therapeutics. Some of them believed Tokyo-based Astellas had undervalued Ocata. Others complained that technology pioneered by Robert Lanza, Ocata’s world-renowned chief scientific officer, would go to a foreign company.

Three years later, the biotech — now an Astellas subsidiary called the Astellas Institute for Regenerative Medicine — appears to be growing fast and making progress — although not enough to satisfy one former investor who opposed the deal and has a personal interest in cell therapy.

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The institute recently began a small early-stage clinical trial of embryonic stem cell-derived transplants for the dry form of macular degeneration, a major cause of blindness. It’s the first treatment developed by Ocata to be tested in patients.

The subsidiary’s workforce has roughly quintupled since the acquisition, from about 30 employees to 150, according to its top executives. The biotech had occupied a 30,000-square-foot building in Marlborough but now has two buildings totaling 60,000 square feet.

In January, all the employees will move to a 250,000-square-foot building being renovated in Westborough for $150 million. The new home will house drug research, development, and manufacturing, and serve as Astellas’ global hub for regenerative medicine. The workforce there is expected to grow to between 200 and 300 employees.

Behind the progress, says Yoshitsugu Shitaka, president of the institute, is an unwavering commitment to a medical technology he believes has the potential to treat many diseases, from eye disorders to lupus.

"We think that cell therapy is a very powerful approach to tackle intractable degenerative diseases,” Shitaka said in an interview in Cambridge, where he was joined by two other Astellas executives.

Astellas has 10 potential stem cell-derived therapies in its pipeline, including six originating from Ocata under the scientific leadership of Lanza. He produced hundreds of inventions, books, and scientific papers on stem-cell biology and tissue engineering. In 2014, he was named to Time magazine’s list of the 100 most influential people in the world. Lanza now serves as the institute’s chief scientific officer.

“It’s an exciting time for regenerative medicine,” he said in an e-mail. “After decades of basic research, the field is finally at a turning point.” The first of the institute’s stem-cell therapies to enter the clinic is a treatment for age-related macular degeneration. The disorder is the leading cause of vision loss, affecting more than 10 million Americans, according to the American Macular Degeneration Foundation.

The disease is marked by a deterioration of the central part of the retina, the inside back layer of the eye that records the images we see and sends them through the optic nerve from the eye to the brain. Age is a risk factor, says the foundation, with symptoms often appearing when people are in their 50s.

In the early phase of the disease, people may experience wavy or blurred vision. If the condition worsens, central vision may be entirely lost. About 10 to 15 percent of people afflicted with the disease have the “wet” form. Many receive injections of one of three approved drugs in their affected eye every few weeks. The injections may help slow the progression, preserve existing vision, and, if begun early enough, help patients recover some lost vision.

The “dry” form of the disease affects the remaining 85 to 90 percent of patients. But apart from vitamins and supplements, there are no approved treatments.

In April, the institute began testing one that involves transplanting retinal cells made from stem cells below the retina in the hopes of replacing dysfunctional cells and tissues.

So far, one patient in the early-stage clinical trial has received such a transplant. The institute hopes to administer the treatment to nine patients by the end of next year.

“By directing the body’s capacity to renew itself, regenerative medicine may fundamentally change the way we think about managing disease,” Shitaka recently wrote on an Astellas blog.

But not everyone was thrilled with Ocata’s acquisition by Astellas. Many shareholders argued that Ocata was worth more than the nearly 80 percent premium on the stock price Astellas offered, while some objected to the biotech’s potentially groundbreaking technology being sold to a foreign company.

The Japanese company wrangled with shareholders for three months and twice extended a tender offer for Ocata before Astellas accumulated more than 50 percent of outstanding shares to gain control of the biotech.

Among the former shareholders who remain unhappy is Edward Assad, of Monticello, Fla. Assad, 65, said he owned $5,000 to $10,000 worth of Ocata stock at one point and ultimately lost money in the acquisition.

Assad suffers from Parkinson’s disease. He said he had hoped a drug maker would have won approval of a stem-cell therapy to treat his condition by now and is disappointed by what he sees as a lack of progress.

“I’ve been following stem cells for years,” he said. “They just don’t seem to get anywhere with it.”

Jonathan Saltzman can be reached at jsaltzman@globe.com.