Print
Email
Print Edition Stories
Courtesy photo
Funding drugs for rare diseases
MHT's Julie Donnelly: Life science startups finding investments
MHT's Marc Songini: Mass. bio firms prep for lifting of stem cell ban
Advanced Cell Technology Inc. last month filed an FDA application to use human embryonic stem cells to treat an untreatable disease that is the most common cause of juvenile blindness. But a little more than a year earlier, the Worcester company was teetering on the brink of extinction.
According to ACT chief scientific officer Robert Lanza, the journey from a once-high-profile biotech focused on cloning, to basic services being shut off, to a rebirth as one of the few companies to have filed for clinical trials of a stem-cell-based treatment, has been a tough one. “Obviously we had a very rough financial past here, and I think that was reflective of many stem cells companies — and I think it is a pretty good lesson,” Lanza said.
ACT’s path started in Worcester in 1994, and early on, the company made a name for itself in the growing field of cloning large mammals using its proprietary nuclear transfer technique. In 2006, ACT gambled on a move to California to take advantage of that state’s much-touted $3 billion state-backed funding plan for stem cell research. Almost immediately, however, the funding was locked up in court battles and almost none of that funding has been released to date.
By early 2008, ACT, which had kept its research operations in Worcester, had no products in clinical trials — and it was clear that California’s much-touted funds were not going to be doled out anytime soon. In the meantime, back in the Bay State, Gov. Deval Patrick had floated a proposal for a $1 billion life sciences investment bill, so ACT — which had ended 2007 with a $15.9 million loss on just $650,000 in revenue — moved its headquarters back to Worcester.
But by the summer of 2008, ACT had all but run out of money and was barely clinging to life.
“When your telephones are turned off and you can’t get water and pipettes, it’s not good,” Lanza said of those days. “And it doesn’t hurt to get a paycheck.”
ACT had the benefit of years of research and intellectual property to leverage, and it did so in August 2008, licensing embryonic stem cell technology to Embryome Sciences Inc. in a deal that could be worth up to $1.25 million, but brought in just $250,000 initially. ACT followed that with $1 million in bond sales to Irish investment firm Transition Holdings Ltd.
At the end of 2008, ACT signed a deal with Korea-based CHA Biotech Co. Ltd. to form a development company called Stem Cell & Regenerative Medicine International. The joint venture uses ACT’s hemangioblast cell technology to develop human blood cells. It is majority-owned by CHA.
That led ACT to focus on using its stem cell expertise to treat macular-degeneration diseases that cause blindness, and last month the recovering company filed the IND with the U.S. Food and Drug Administration to begin a consolidated Phase 1 and 2 trial to treat patients with Stargardt’s Macular Dystrophy.
Few companies can come back from being as close to the brink as ACT was, said Douglas Fambrough, general partner at Oxford Bioscience Partners. There is a flipside to the coin of hanging in there in tough times, however, Fambrough points out.
“There’s a fine line between impressive stick-to-it-iveness and a fool chasing a dream,” he said. “Biotech, more than most places, is a market for dreams. They are good, positive dreams, but a lot of times what people are selling is dreams, some of which will come true and most of which won’t.”
Lanza and the staff at ACT are committed to their dream of using the vast potential of stem cells to help fight difficult diseases. “We have a team of dedicated scientists who have spent their lives on this, and they believe in this,” he said. “To walk away from that would have been irresponsible.”
Comments
If you are commenting using a Facebook account, your profile information may be displayed with your comment depending on your privacy settings. By leaving the 'Post to Facebook' box selected, your comment will be published to your Facebook profile in addition to the space below.
RSS Feeds
