Stem Cells: Q&A with Randall Mills, Osiris Therapeutics
Although stem cell research has sparked controversy for years, Osiris Therapeutics has quietly been flying under the radar for a decade with a revolutionary secret. The company is in the final stages of developing Prochymal, widely expected to be the first-ever FDA approved stem cell drug. With a pre Biologics License Application (pre-BLA) meeting already under their belt, and an FDA OK for Prochymal use in adult Graft vs. Host Disease (GvHD) patients, Osiris is steadily marching toward the finish line.
According to President and CEO Randall Mills, with about 300 patents issued worldwide and a contract with the US Department of Defense for Acute Radiation Syndrome treatments, “there’s nobody else in the ballpark of where we are right now.” And that’s not to mention Osiris’ three other indications currently in pivotal trials.
Before joining Osiris, Mills was an executive officer of Regeneration Technologies, a company that deals with tissue transplantation. When he joined Osiris in 2004, Mills couldn’t believe the potential of stem cell technology. Four years later, he’s just as enthusiastic about the progress Osiris is making. Pharm Exec sat down with Mills to discuss what’s next for the stem cell industry, and how Osiris demonstrated that it’s possible to turn interesting science into a lucrative business.
What is the promise of stem cells?
How do you get stem cells, and have you had to work with any third parties to regulate your research?
We get our stem cells from bone marrow of healthy young adults between the ages of 18 and 30. Bone marrow contains a number of different cells, but we use only the mesenchymal stem cell, or the MSC. Once we collect them, we’re able to expand the cells to produce 10,000 doses of our drug. The process by which we grow stem cells is a fairly standard GMP compliant biotechnology and manufacturing process.
We don’t get involved in the harvesting of the bone marrow. Many companies collect bone marrow, so we can literally buy it off the shelf. It’s inexpensive, noncontroversial, and we can get as much as we need. Because we mass produce the product at low costs, we’re able to transform this technology from interesting science into a business.
Your new drug, Prochymal, can be used to combat heart attacks, diabetes and nuclear terrorism. How do you develop a platform drug that can be used for such varying problems?
Because MSCs naturally have a broad range of things they can respond to, our job is to package them as something that will satisfy the FDA. We need scientifically sound clinical trials that show a significant benefit in a particular patient population.
Is patenting intellectual property the same in this area as in other areas?
We have what’s called a “composition of matter” patent around the mesenchymal stem cell itself, but we also have patents on how you manufacture cells for intravenous infusion, how you formulate a product like this, and how you test a product for lot release.
What’s happening in stem cell work now as compared to 2004?
The FDA recently gave us expanded access approval. We can now treat children 17 years of age and younger with our product. In 2004, it was unthinkable that a stem cell product could be routinely used in the treatment of any patient population, let alone children.
When I came to Osiris in 2004, Osiris was what I called Osiris University. It was highly academic, brilliant people doing great science, but there was no commercial focus. That’s changed nicely over the last four years because companies have been rewarded in the share price of their overall value or market cap. The really good news is that the change ultimately helped patients. To date, we’ve treated more than 20,000 patients with Osteocel.
Who would you consider your closest competitor to date?
Because this is such a new area, does the infrastructure need to be developed, perhaps even outside your company?
But I don’t think the FDA had as much work to do from a regulatory standpoint as the companies preparing to be regulated. The FDA actually has reasonable regulations in place for stem cell therapy, but the industry had to come to grips with the requirements. FDA expects us to demonstrate that our products are safe and effective. That’s the standard we are held to. It’s the same standard that every drug is held to.
FDA has borne the brunt of the tremendous disparity between interesting preclinical scientific discoveries and what it actually means to translate a product into something that’s usable everyday.
When do you expect to hear from FDA?
When do you plan to submit your BLA?
What do you see as your biggest challenges in the next year?
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