Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Alliance must steer clear of hESC debate in pursuing regenerative medicine bill.

This has been a busy month in stem cell research. A Tokyo University of Science team published data in PLOS ONE showing how it used mesenchymal and epidermal stem cells from mouse embryos to grow a set of fully-formed teeth in mice. Korea’s Food and Drug Administration approved for clinical use Hearticellgram-AMI, an adult stem cell-based heart attack treatment developed by FCB-Pharmicell.

Now, a coalition of stem cell companies, research institutions, patient advocates, and government-created agencies wants to see more such activity in the U.S. The nonprofit Alliance for Regenerative Medicine hopes to accomplish that in part through the Regenerative Medicine Promotion Act of 2011 (HR 1862), which was introduced in May.

Rep. Brian P. Bilbray (R-CA), whose district includes the biotech hub of South San Francisco, has won support for the bill from 10 House co-sponsors: Reps. G. K. Butterfield (D-NC), Michael E. Capuano (D-MA), Diana DeGette (D-CO), Charles W. Dent (R-PA), Anna G. Eshoo (D-CA), Marcia L. Fudge (D-OH), Jim Gerlach (R-PA), Rush D. Holt (D-NJ), James R. Langevin (D-RI), and Steven C. LaTourette (R-OH).

That adds up to seven Democratic and three Republican co-sponsors. While smaller in number, the Republicans are more important since the GOP controls House leadership. Whatever their party, the co-sponsors have positioned themselves as staunch supporters of stem cell research and/or the broader biotechnology industry.

Calling on Both Parties

Biotechs are among supporters of Bilbray’s HR 1036, a business tax-relief measure titled the Job Creation and Innovation Investment Act. Capuano was named 2010–2011 legislator of the year by BIO; DeGette and Holt won the honor in 2006. Fudge secured $2.5 million in federal funds during federal FY 2010 for Cleveland-based Arteriocyte Medical Systems, a biotech with cell therapies based on adult marrow, peripheral cord blood, and cartilage stem cells.

Dent is lead Republican co-sponsor and Langevin among co-sponsors of a separate DeGette bill re-introduced on June 29 that would codify into law President Obama’s 2009 executive order allowing federal funding for human embryonic stem cell (hESC) research. Eshoo and Gerlach have voted to lift federal curbs on funding hESC research, though, the Regenerative Medicine Promotion Act doesn’t specify whether adult or embryonic stem cells should be used in regenerative medicine.

“Our bill neither calls out nor cuts out any particular technology,” Michael J. Werner, co-founder and executive director of the Alliance for Regenerative Medicine, told GEN. “It’s about doing an assessment of what’s happening, it’s about setting priorities and figuring out how the research budget should go. It’s about figuring out the obstacles to commercialization and ways to remove those barriers.

“We’ve found bicameral, bipartisan support for the technology, and we expect that to build as time goes on,” Werner added. “People recognize that it’s a broad technology, that there are already some products on the market, and that it’s got enormous potential.”

Proposals to Legislation

HR 1862 plans to create three grant programs, using funds now being spent for stem cell research:

  • NIH academic-industry collaboration grants: One program would fund nonprofits or higher education institutions conducting basic or preclinical stem cell research if it is partly funded by a private company. The other would fund R&D and require an IND or IDE application within four years.
  • NIH private companies grants: It will provide money to support product development through the Cures Acceleration Network created within NIH last year. Grants will support basic research, preclinical studies, and clinical trials for pre- and IND/IDE applications.
  • FDA grants for regulatory research: It will authorize funds for projects by public-private partnerships to foster development of a clear, predictable regulatory pathway toward speedy approval of products. The Act authorizes FDA to conduct regulatory research to help in the approval process.

The regenerative medicine bill encourages but does not set aside any money for the funding of stem cell research: “We recognize the fiscal times,” Werner noted. Funding will come around, supporters hope, when Washington finally gets its fiscal house in order. That won’t start until President Obama and Congressional leaders can agree on a spending measure that will move the nation away from annual trillion-dollar deficits.

“We’re trying to create some programs that will use existing dollars very wisely and really target them to the projects that have commercial potential. We want to use that money to actually get those projects into the clinic to lead to the filing of an IND at FDA,” Werner said.

The bill also calls for creating a Regenerative Medicine Coordinating Council, a public-private entity within the U.S. Department of Health and Human Services. The council would devise a national strategy intended to promote stem cell research as well as translate that into new drugs, biological products, medical devices, and biomaterials. The council is also expected to identify and recommend policies to overcome barriers in R&D as well as specify research priorities. Additionally, the council would issue an annual report on the state of regenerative medicine.

While the council sounds like another layer of bureaucracy, Werner asserted that it would help cut red tape for stem cell companies by bringing them together with patient advocacy groups and the numerous federal agencies involved in regenerative medicine. In addition to obvious agencies like NIH and FDA, they include the Department of Defense and the National Institute of Standards and Technology.

Alliance members hope to see a Senate companion bill introduced later this year. Just when that will happen and who will introduce it are not yet known. Werner believes prospects for the bill are “pretty good” and will continue to improve as Congress focuses more on getting new drugs out to patients as well as addressing the economic and social burden of disease.

Economic Impact v.Morality

For that to happen, the alliance and its Congressional supporters will have to keep the bill from getting bogged down in the moral debate over hESC research. That’s easier said than done in today’s hyper-political Congress, with a presidential election just a year away. NIH’s ability to continue funding hESC research depends on the outcome of Sherley et al. v. Sebelius et al. In April the Court of Appeals for the District of Columbia Circuit vacated U.S. District Court Judge Royce Lamberth’s decision granting a preliminary injunction on all federal funding for hESC research.

To its credit, the alliance has a broad membership—74 members are listed on its website. They include big pharma mainstays, smaller stem cell companies, investment firms specializing in science start-ups, private research institutions, and publicly created agencies like the California Institute for Regenerative Medicine (CIRM). CIRM was created after California voters in 2004 approved Proposition 71, authorizing the state to sell $3 billion in general obligation bonds to fund stem cell work. State officials then spent more than three years in court, battling opponents seeking to scuttle the bond act.

Earlier this year, CIRM issued an economic impact report that sought to show just what return on investment California taxpayers will get for the $3 billion borrowed under Proposition 71. The agency said that the first $1.1 billion it spent generated $200 million in new tax revenue and 25,000 “job-years” through 2014. “Job-years” are aggregate numbers calculated by adding up each year’s number of jobs generated by a multiyear project. The tax gain exceeds the approximately $50 million in debt service that must be paid on the bonds, Art Torres, vice-chairman of CIRM’s governing board, the Independent Citizens Oversight Commission (ICOC), told GEN.

CIRM’s job and tax numbers were swelled by the construction of stem cell research facilities across California. The ICOC approved $271 million in grants to 12 institutions in 2008. The 12, in turn, committed an additional $560 million from charitable donations and their internal reserves. CIRM plans a follow-up study detailing how many jobs were created by each lab facility whose construction the agency helped fund, Torres said. At its June 23 meeting, ICOC unanimously voted to support HR 1862.

The Alliance for Regenerative Medicine foresees someday releasing a similar report showing the economic effects of regenerative medicine R&D nationwide. “There really is a need for better information about what’s going on in the field and its impact,” Werner said.

Focus on Advancing the Technology

It will be hard for the alliance and other stem cell research supporters to build support for HR 1862 or their broader enterprise by citing glowing economic numbers. CIRM’s economic report has yet to generate support among California taxpayers for another stem-cell bond, something the agency would like to see happen when the current one runs out.

Indeed in recent weeks, CIRM has endured criticism over the six-figure salaries it pays several top executives as well as the newly elected chair of its governing board, Torres admitted. CIRM has countered that the two board leaders devote considerable time to their duties and that it must retain talent by staying competitive with higher-paying academic and private research institutions.

A better path toward getting HR 1862 approved would be to lay out the progress generated by recent stem cell research and spell out how the bill will aid in filling the gaps its agencies identify in R&D and translation. The alliance and other stem cell advocates should watch for efforts to combine the bill with DeGette’s measure and resist them, because that bill will just get trapped by the same-old debate over hESCs.

Alex Philippidis is senior news editor at Genetic Engineering & Biotechnology News.

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