SAN FRANCISCO – Regenerative medicine is coming of age, and not a day too soon to address an expected 80 percent increase in individuals older than 65 expected by 2030, from 40 million to more than 70 million in the U.S. alone.

The same population shift is expected to occur in Europe and may be even more pronounced in Asia, which has experienced lower growth over the past two decades.

Aging populations will bring with them enormous increases in cardiovascular disease, neurological conditions, inflammatory and immune disorders and renal disease, prompting an explosion in health care interventions and related costs.

"The reality is that we don't have the resources to deal with this problem," said Gil Van Bokkelen, CEO of Athersys Inc., of Cleveland, and chairman of the Alliance for Regenerative Medicine.

That is, unless regenerative medicine can ride to the rescue as part of the biopharma solution, Van Bokkelen and a panel of specialty biotechs and pharmas told attendees at Biotech Showcase 2012, held at the same time as the 2012 J.P. Morgan Healthcare Conference in San Francisco.

The Alliance was formed two years ago to improve networking among large and small biopharmas, private and public companies, nonprofits and advocacy groups seeking to advance regenerative medicine therapies in order to "reduce the Mt. Everest of health care" into a manageable beast, Van Bokkelen said.

The clinical opportunities are broad and deep. In the cardiovascular arena, more patients survive heart attacks, but progress to congestive heart failure – a greater long-term clinical and financial burden. Millions of individuals live with vascular diseases such as critical limb ischemia, but endure amputations. Each year, more than 2 million people in the U.S., Europe and Japan, combined, suffer a stroke, and many require long-term care. The list goes on and on, Van Bokkelen said, from neurodegenerative diseases to traumatic brain injury, rheumatoid arthritis to multiple sclerosis – and diabetes, almost a class in itself.

Regenerative medicine firms are seeking "to innovate our way out of these problems," he said. "None of us believe that all of the approaches will be successful and, frankly, they don't need to be. The reality is that if just a few of them are successful, they will have a transformational impact on clinical medicine as we know it."

The range of investigative efforts encompasses not just cell-based approaches but also tissue engineering, medical devices and the interface between small molecules and cell therapy. Although the number of marketed products is small, especially in comparison with conventional biotech compounds, the field now boasts tens of thousands of research citations exploring the biology of regenerative medicine as well as a growing list of programs – more than 20,000 in cell therapy, alone, according to NeoStem Inc. Chairman and CEO Robin Smith – that have advanced to clinical trials.

And the space is attracting the interest of pharmas, as evidenced by last year's blockbuster acquisition of Genzyme Corp. by Sanofi-Aventis SA, of Paris, and the last-minute nab of Advanced BioHealing Inc. (ADH), by Shire plc, of Dublin, Ireland. (See BioWorld Today, Feb. 17, 2011, and May 19, 2011.)

"When you're a pharma looking at this field, one of the most fundamental decisions to make is whether to build capabilities, buy capabilities, invest externally, partner, wait or watch and see," said Jay Siegel, chief biotechnology officer and head of pharmaceutical global regulatory affairs for New Brunswick, N.J.-based Johnson & Johnson. "Our strategy is a combination of all of those activities."

Access to public markets and partnerships represent two important keys to propelling regenerative medicine, said Silviu Itescu, CEO of Melbourne, Australian-based Mesoblast Ltd. Regenerative medicine requires "serious financial commitments," Itescu said. "This is not about being able to spend $10 million or $15 million, if we're really serious about putting regenerative medicine on the map."

Mesoblast is one of the few regenerative medicine companies to present around the corner at the J.P. Morgan conference.

In 2010, Mesoblast inked a $350 million strategic alliance with Cephalon Inc. (now part of Teva Pharmaceutical Industries Ltd.) to develop and commercialize its mesenchymal precursor cell (MPC) therapeutics for hematopoietic stem cell transplantation in cancer patients, as well as degenerative conditions of the central nervous and cardiovascular systems, including congestive heart failure. The deal could potentially exceed $1 billion in milestone payments. (See BioWorld Today, Dec. 9, 2010.)

The cash influx helped Mesoblast move its stem cell technology forward rapidly, Itescu said. Last May, the company reported that its allogeneic adult stem cell product, Revascor, significantly reduced cardiovascular deaths in a randomized, placebo-controlled Phase II trial. The company plans to begin a large Phase III in congestive heart failure in the near future, according to Itescu.

Aastrom Biosciences Inc., of Ann Arbor, Mich., expects to launch its Phase III REVIVE-CLI study of ixmyelocel-T, its expanded multicellular therapy, this quarter in patients with critical limb ischemia, CEO Tim Mayleben told conference attendees. In November, the company reported Phase II data at the American Heart Association Scientific Sessions in Orlando.

In one indicator of how far regenerative therapies have progressed, "several years ago, you would not have found a cell therapy company presenting real Phase II data – placebo-controlled industry-standard clinical trials – at a major medical meeting," Mayleben said. "For us to be able to present at that venue speaks to the regenerative medicine industry's development over the past few years."

In fact, some regenerative medicine executives insist the discipline is not a subset but the next iteration of biotechnology.

"With regenerative medicine, we're not treating the symptoms of these diseases," Mayleben said. "We're actually changing the course of the disease, and we've seen that in the results of our Phase II clinical trials."

Dean Tozer, senior vice president of corporate development at ADH, agreed, noting that the company often struggled to find "like-minded partners" during its formative years.

"We didn't fit into pharma, we didn't fit into device and we didn't fit into biotech," he said. "We always believed that regen med was going to be an entirely new field. We built a company to accomplish that, and I think that's why we were successful."

Unlike traditional big pharmas and some biotechs, many regenerative medicine companies are developing platforms that can be developed across multiple diseases.

"The ability to cross-reference common manufacturing platforms and safety data and then apply that to accelerate therapies into midstage clinical development and beyond is very powerful from an investor perspective," Van Bokkelen observed. "It means you can develop these therapies more efficiently and cost-effectively."

And regenerative medicine companies are making swift progress in their ability to capture and report compelling clinical data. For example, Aldagen Inc., of Durham, N.C., has an ongoing Phase II trial of ALD-401, a stem cell population derived from a patient's own bone marrow, for the treatment of stroke – an indication that has seen its share of failures with small molecules and other approaches.

"This is an area where cell therapy has a unique opportunity, because stroke is a multifactorial problem," said Ed Field, Aldagen's chief operating officer.

The 100-patient, randomized, double-blind study was the largest of its kind initiated for cell therapy study in stroke when it was approved by the FDA last January, according to Field.

"We need to do studies the right way in cell therapy, and the randomized double-blind method is the standard," he said. The size and design of the study "allow us to look at an endpoint that the FDA will accept for approval of a stroke therapy. We can actually power a Phase III appropriately from the 100-patient Phase II study. We're about 12 months away from having a major Phase II data set in a very large unmet need for cell therapy."

Regenerative medicine companies face the same regulatory hurdles cited by other biotechs, including those attending J.P. Morgan. (See BioWorld Today, Jan. 11, 2012.)

Members of the Alliance are seeking to keep the lines of communication open with the agency through frequent conversations "about common concerns," Van Bokkelen said. "Our experience with the FDA has been a very, very positive one. The FDA is anxious to learn from us, just as we are anxious to learn from them, about how we can work together to establish a more transparent regulatory framework."