Federal Funding of Stem Cell Research: Past, Present and Future

WORLD STEM CELL REPORT 2009 GENETICS POLICY INSTITUTE 501c3

2010 WORLD STEM CELL SUMMIT • DETROIT, MI • OCTOBER 4-6, 2010 WWW.WORLDSTEMCELLSUMMIT.COM • WWW.GENPOL.ORG22

STEM CELLS, REGENERATIVE MEDICINE, AND SOCIETY

n March 9, 2009 President Barack Obamasigned an Executive Order expandingfederal funding for research using

embryonic stem cells in a White House signingceremony packed with legislators, scientists, andpeople who have suffered from a range ofdiseases and medical conditions that couldbenefit from the research. The President’sexecutive order was a major milestone in the 10-year effort by those patients, scientists,academics, and others who have argued insupport of a broader federal stem cell policy.1

In some ways, the most telling aspect of the ceremony was thatwhen President Obama signed the Executive Order, numerousmembers of Congress were in attendance. Their presence signaled asignificant shift in public opinion regarding the use of federal fundingfor embryonic stem cell research; further, it demonstrated theattitudinal changes regarding stem cell science that have taken placesince 1998, when embryonic stem cells were first derived.

Today, for many Americans, stem cell science is viewed aspotentially beneficial – not just for people suffering from disease –but in maintaining the country’s prominence and preeminence inscientific leadership. In a remarkably short timeframe, stem cellscience has made the unusual transformation from a non-issue to apolitical hot button – and to an issue that transcends politics.

BackgroundEmbryonic stem cells were first discovered in 1998 by Dr. Jamie

Thomson of the University of Wisconsin and Dr. John Gearhart ofthe Johns Hopkins University.2, 3 While widely applauded for itspotential in the scientific community, their discoveries very quicklyset off a firestorm affecting researchers, ethicists, politicians and, mostimportantly, patients in the United States (US) and around theworld. In fact, the first series of congressional hearings on the sciencefollowed quickly, beginning in January 1999, when the SenateCommittee on Health, Education, Labor, and Pensions convenedsessions involving a series of top researchers and ethicists.4

What became clear at the outset was that most US policymakersdid not have an understanding of stem cell research and thereforewere uncomfortable with the concept. Almost immediately, thesource of embryonic stem cells became a key issue in the discussionand debate and initially there were broad misunderstandings aboutthe origin of stem cells – with many assuming the issue to be

Oassociated with abortion.

At the time, relatively few legislators had a clear understandingof the reality; the stem cells at the center of the issue were cells derivedfrom embryos created for in vitro fertilization (IVF) procedures. Infact, the cells in question were not going to be used and were slatedto be discarded.

More often than not, the creation of embryonic stem cells waserroneously linked to abortion, and the corresponding political,moral, and religious issues that it entailed. To compound theproblem, there were a handful of legislators and others that mighthave understood that abortion was not tied to stem cells, but did nothave a detailed understanding of or were uncomfortable with the IVFprocess itself. As a result, the immediate political reaction to the 1998Thomson/Gearhart discoveries leaned toward a ban on the research.

The Role of Patient AdvocacyIn response to initial fears and negative reactions (often based on

misunderstanding), an organized effort was launched on the part ofpatient advocacy groups to delay any potential federal ban onembryonic stem cell research. The aim was to provide education onthe role of stem cells, the science, and their potential for therapies andcures for leading conditions. In addition, time was needed to enablediscussion and debate related to surrounding ethical and religiousimplications. This effort started with Patient’s Cure, originally formedby Dan Perry, who was the Executive Director of the Alliance forAging Research. Patient’s Cure’s major focus was to slow themomentum of any amendments that might surface in Congressrestricting federal funding and support for stem cell research.

Patient advocacy in the area of stem cell research emerged amongthe campaign promises made by then Presidential candidate GeorgeW. Bush, who pledged to impose a ban on federal funding forembryonic stem cell research. Once elected, it was clear that a largerscale and more comprehensive effort would be required to preservefederal funding for the research – if not the ability to conduct stemcell research itself.

Formation of the Coalition for theAdvancement of Medical Research (CAMR)

The result was the creation of the Coalition for theAdvancement of Medical Research (CAMR). Today, CAMRcomprises more than 100 nationally recognized patient organizations,universities, scientific societies, and foundations, and conductsadvocacy and education outreach focused on developing bettertreatments and cures for people with life-threatening illnesses anddisorders.

Federal Funding of Stem CellResearch: Past, Present and Future By Lawrence A. Soler, J.D.

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actually usable for research, because they didn’t exist or had othertechnical problems. The result was that scientific progress wasdelayed.

With a President opposed to expanding funding for stem cellresearch and a Congress increasingly supportive, the remaining yearsof the two terms of the Bush presidency were essentially seven yearsof stalemate. The science became an issue in the 2004 Presidentialelection – but with the re-election of President Bush, there was nomovement in either direction. But as a result of continued advocacyactivities by the research community, CAMR, and others, Congresstwice introduced legislation and passed bills, with strong support,that would overturn the 2001 policy and expand the fundingavailable for embryonic stem cell research. Twice, however, these billswere vetoed by the President, which Congress was unable to overridebecause it lacked the two-thirds vote to do so. Progress in stem cellresearch was increasingly limited as federal support became moretechnologically slowed.

Recent Policy Changes and Claims to Pavethe Way for Stem Cell Research

The issue again bubbled to the top of the political consciousnessduring the 2008 Presidential campaign, but with little of theprominence or vehemence that had typified previous politicaloutbreaks – owing in large part to stem cells no longer being thepolarizing political issue that they had been earlier in the decade.Candidate Barack Obama noted early and often in his campaign thatamong his first acts, if elected, would be to expand federal funding forembryonic stem cell science. Senator McCain also was on record insupport of the research.

Candidate Obama became President Obama, and true to hisword, acted to get the research back on track, with his historicExecutive Order, which brings us full circle to the beginning of thisarticle.

Current Policy Changes and Controversies:Questions for Consideration

But does President Obama’s March Executive Order signal thatthe public policy controversies surrounding embryonic stem cellresearch are over, once and for all?

Perhaps. But perhaps not.In July 2009, the National Institutes of Health issued its final

guidelines governing federal funding for embryonic stem cellresearch, after a 90 day public comment period on its initialguidelines. JDRF and others in the research community haveapplauded the guidelines, particularly for including a provision underwhich existing stem cell lines derived in an ethically-responsiblemanner would be eligible for federally funded research. Thisprovision will ensure that a process is in place so researchers can buildon the stem cell advancements made to date and accelerate researchon cell lines with the greatest potential to facilitate treatment of thedisease.

So where does that leave us? After 10 years of uncertainty, thescientific community finally has clear direction and support for asubstantially expanded platform for embryonic stem cell research.

In 2001, Tim Leshan of the American Society for Cell Biologyand I created CAMR to bring together patients, scientists, academia,industry and others to pool resources and build public support forembryonic stem cell research. Building on the efforts of Patient’sCure, CAMR provided a voice for the widespread interests that sawthe potential of embryonic stem cells to better understand a range ofdiseases, from diabetes to spinal cord injuries, and perhaps even oneday create therapies to alleviate their suffering. Most important,CAMR provided a rallying point for not only people impacted bydiseases that stem cells could possibly help, but for the medicalresearch community, which – understandably, given that it was sonew – did not have a unified voice vis a vis stem cell research at thetime.

During the initial months, CAMR operated with frantic effort.First and foremost, its advocacy activities focused on attempting tomake it difficult for the Administration to quickly implement anexecutive order that would simply stop embryonic stem cell research– a quite likely outcome at the time. These efforts were combinedwith a larger strategy to educate policymakers and the Americanpeople about embryonic stem cell research, with a particular focus onthe relationship between stem cell research and IVF, and the fact thatstem cell research is not related to the abortion issue but moreanalogous to organ donation.

As expected, scientific journals and research publications focusedintently on stem cells. But in just a few short months, stem cellsbecame one of the top general media issues as well. The issue wasfeatured regularly on the front pages of the nation’s largestnewspapers, and was a regular topic on cable television news andopinion programming. And the just-exploding blogosphere took upthe call, and stem cells were a regular topic for political and news-related websites. As more was explained about the research – what itwas, and particularly, what it was not – support for it and oppositionto the President’s potential banning of funding for the researchgradually began to grow.

Transparency around the issue, as it turned out, mattered.Ultimately, President Bush, in his first nationally televised address tothe nation, did not ban embryonic stem cell research or federalfunding for it; drawing a political, rather than a scientific, distinction,he allowed limited funding for embryonic stem cell science – just forthose stem cell lines that had been created prior to his address onAugust 9, 2001. Any chance of changing the policy ended onSeptember 11, 2001. The front page of the New York Times on thatday included a story on stem cell research policy, but given the eventsof that day, the issue was driven from the headlines – for a while.

While seen by many at the time as the best possible outcomegiven the circumstances, the President’s decision unfortunatelycreated an extended holding pattern for embryonic stem cell research.While not restricting its expansion, per se, limiting federal funding tothe lines existing before August 9, 2001 made it significantly moredifficult for stem cell research to expand at the rate one might haveexpected, given the promise of the science. Within a matter of years,it became clear that the stem cell lines actually available for federalfunding were far fewer than those that were meant to be included. Asit turns out, only 21 of the approximately 70 lines that were initiallypromised by the Administration for federal research dollars were



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Necessary “Next Steps”As positive as the Executive Order is for moving stem cell

research forward, the possibility always exists that it could beoverturned by a future president who opposes the science. Anotherpresident could simply issue a new Executive Order that returns tothe Bush Administration restrictions – or worse.

As such, a critical next step is to pass legislation in both theHouse and Senate, codifying federal funding for stem cell researchthat can be signed by President Obama. Such legislation would makeit extremely difficult for research to be stopped by a future president.In fact, once legislation is approved, the only way a futureAdministration could reverse it would be for a new law to be passedthrough the House and Senate and signed by the President. Given thestrong public support that has grown for embryonic stem cell researchover the years, that seems extremely unlikely.

Based on the previous two bills that were introduced during thepast decade, there would most likely be enough votes to approve a billfunding this research. But the legislative process includes pitfalls thatalways need to be considered, such as anti-research amendments thatcould be proposed that may not be adequately explained in acongressional debate and could lead to limitations on other types ofresearch.

ConclusionMoving forward, it remains important for patients, scientists,

universities, physicians, and others to continue to build supportamong policymakers so that an embryonic stem cell bill can bepassed.

Since the discovery of embryonic stem cells a little more than adecade ago, we have seen dramatic shifts in public perception of andpolitical support for the research. Clearly, both are at a high pointtoday. But now and throughout the darkest days of the past decade,there has been a constant – the people with diseases that might oneday benefit from this science. Our focus should be on them – and onmoving this science forward quickly, effectively, and ethically.

That is undeniably positive for researchers and patients alike. Butthere will still be some limitations. For example, the President’sexecutive order and the NIH guidelines do not include researchinvolving stem cells created through somatic cell nuclear transfer(SCNT) or parthenogenesis – alternatives to the “traditional” methodof creating embryonic stem cells that could potentially speed theprogress of utilizing these cells for research or replacement purposes.

Today the advantages of where the scientific community standssurely outweigh the disadvantages of the past. In fact, compared withjust a few years ago, there are a number of advantages in movingforward, both scientific and attitudinal. For example, researcherstoday have the freedom to effectively work on many additional stemcell lines, this creates cost efficiencies, opening the door for disease-specific science, and encouraging collaboration among researchersfrom different organizations, with different backgrounds, and withdifferent aims. All this is a major step forward for research incombating a wide range of diseases and medical issues.

Just as important, a more transparent and positive policy towardsfederal funding for stem cell research means that young scientists willnow begin to see a future in the field, and add to the talent pool ofresearchers working towards cures and treatments. Before, withlimited federal support and the constant threat of a possible overallban on stem cell research, only the most optimistic young scientistswould have considered stem cell research as a growth industry. Theincreased talent devoted to the field will, undoubtedly, have amultiplier effect on the pace of progress in stem cells in the near andlonger-term future.

That impact will be felt on the institutional level, as well. Untilnow, more than a few universities have been very conservative indetermining whether to allow private embryonic stem cell research –science using non-federally approved stem cell lines – to beconducted in university buildings that receive federal indirect costsreimbursement. The fear was that given the intricate accounting andprocedural issues surrounding research funding, non-approvedresearch conducted in buildings supported by federal funds, even ifthe accounting was done exactly right, could in some way jeopardizethe university’s federal funding eligibility. The broadening of thepolicy should substantially alleviate many of these concerns. But weare not done yet.



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References1. Remarks of President Barack Obama – As Prepared for Delivery,

March 9, 2009:http://www.whitehouse.gov/the_press_office/Remarks-of-the-President-As-Prepared-for-Delivery-Signing-of-Stem-Cell-Executive-Order-and-Scientific-Integrity-Presidential-Memorandum/

2. Thomson J, Itskovitz-Eldor J, Shapiro S, Waknitz M, Swiergiel J,Marshall V, Jones J (1998). Embryonic stem cell lines derived fromhuman blastocysts. Science 282 (5391): 1145-7(http://www.sciencemag.org/cgi/content/full/282/5391/1145); M.J.

3. M.J. Shamblott, J. Axelman, S. Wang, E.M. Bugg, J.W. Littlefield,P.F. Donovan, P.D. Blumenthal, G.R. Huggins, J.D. Gearhart.Derivation of Pluripotent Stem Cells from Cultured HumanPrimordial Germ Cells. Proceedings of the National Academy ofSciences U.S.A. 95

4. Melton, D. Testimony on Stem Cell Research before the SenateLabor-HHS Appropriations Subcommittee, January 10, 1999:http://www.mcb.harvard.edu/Melton/testimony.html.

5. Perry, D. (2000). Patients’ Voices: The Powerful Sound in the StemCell Debate. Science, 287, p. 1423:http://www.sciencemag.org/cgi/content/abstract/287/5457/1423?hits=20&RESULTFORMAT=&titleabstract=report+patient&maxtoshow=&andorexacttitle=or&HITS=20&

6. President George W. Bush’s address on stem cell research, August9, 2001:http://archives.cnn.com/2001/ALLPOLITICS/08/09/bush.transcript/.

7. National Institutes of Health Guidelines on Human Stem CellResearch, http://stemcells.nih.gov/policy/2009guidelines.htm.

Lawrence (Larry) A. Soler is ExecutiveVice President, Government Relations &Operations for the Juvenile DiabetesResearch Foundation, his fourth positionat the organization since joining it in1998.

In January, 2009, Mr. Soler wasappointed to his most recent position which added oversight ofinformation technology, international development, and legal issuesto his previous areas of responsibility, government relations and theorganization’s Artificial Pancreas Project.

During his time at JDRF, Mr. Soler has been responsible for leadingthe effort to secure $1.4 billion in type 1 diabetes research fundingthrough the congressionally mandated Special Diabetes Program. Inaddition, Mr. Soler has a been a leader in embryonic stem cell policy,creating and chairing the Coalition for the Advancement of MedicalResearch, a coalition of one hundred national organizations whosupport embryonic stem cell research. In this role, he oversaw thenational campaign which successfully convinced President Bush toreverse a campaign commitment to ban the research. He also was

instrumental in building public support for the issue over the years,culminating in President Obama issuing an executive order to allowmore funding for the research. JDRF has received recognition andhonors for its government relations program from the New YorkTimes, Wall Street Journal, Time Magazine, Research!America,National Journal among others.

Mr. Soler has also played a leading role in establishing JDRF’sArtificial Pancreas Project, a research initiative that combines workin regulatory and reimbursement policy, physician and patientacceptance, and JDRF/industry research partnerships to acceleratethe development of a closed loop artificial pancreas. JDRF-ledresearch related to this project was named a top 10 medical newsstory in 2008 by ABC News. Prior to joining JDRF in 1998, heworked for eight years in federal relations positions at the Associationof American Universities, focusing on education, health andimmigration issues.

Mr. Soler received a B.A. with honors from Clark University andhis J.D. from George Washington University. He is a member of theMaryland and D.C. Bar Associations.

Lawrence A. Soler, J.D.



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or many years, scientists, patient advocates,entrepreneurs, and politicians have claimedthat regenerative medicine holds enormous

promise. In some cases, this promise has alreadybeen realized as companies have commercializedregenerative therapies that have successfullytreated diseases and disabilities. Other products,while not yet approved for marketing, have shownsuccess in academic labs or in clinical trials.

Moreover, the potential of regenerative medicine to lead to curesand treatments for diseases and disabilities such as diabetes,Parkinson’s disease, ALS and spinal cord injury is the principal reasonhuman embryonic stem cell research has received widespread politicaland popular support for the past several years.

Regenerative Medicine Technologies: New Opportunities for Improving Healthcare

Successes to date represent just the tip of the iceberg.Regenerative medicine technologies represent the opportunity to notonly respond to currently unmet medical needs, but also to providebetter care to patients in a cost effective manner. Thus, regenerativemedicine could dramatically change the way health care is providedin the years ahead.

To reach that objective, however, will require a partnershipbetween academia, industry, and the government. Specifically, thefederal government needs to adopt policies that will facilitate andsupport the development of regenerative medicine. For example, thegovernment should support a research agenda – including both basicand translational research. In addition, we need a predictable andscience-based regulatory approach that provides an appropriatepathway for product approval. Moreover, a market-basedreimbursement system that rewards innovation is essential. Theregenerative medicine community (in this article, the term“regenerative medicine community” means academic researchers,

Ftherapeutic and diagnostic companies, investors, and patientadvocacy groups that will benefit from regenerative medicinetechnologies) should advocate for these policies.

Just as importantly, it should participate in the currentcongressional debates about health care reform and comparativeeffectiveness research. The decisions being made over the next severalmonths on these topics will impact the development of regenerativemedicine.

While there has been congressional support for funding of stemcell research for the past several years, the regenerative medicinecommunity needs to seek broader gains from Congress that willcreate a policy environment conducive to long-term success. Thatmeans funding for basic research and translational research, as well asappropriate regulatory and reimbursement policies.

How well the regenerative medicine community engages policymakers in Congress, at the Food and Drug Administration (FDA),CMS and private insurers, and other government agencies, will bekey to whether this technology will lead to cures and treatments forpatients.

To be most effective, the regenerative medicine communityneeds a specific advocacy effort. In this paper, I describe a newadvocacy organization – the Alliance for Regenerative Medicine –that will fulfill this mission.

What is the Alliance for RegenerativeMedicine (“the Alliance”)?

The Alliance is a non-profit organization whose mission is toeducate key policy makers about the potential for regenerativemedicine, and to advocate for public policies – funding, regulatory,reimbursement and others – that will facilitate advances in the field.It will be operated by Holland & Knight, LLP, a law firm withexpertise in health care and government relations with offices inWashington, DC and many other cities and Adjuvant GlobalAdvisors, a business development and commercial strategy consultingfirm based in Bethesda, MD.

The Alliance defines “regenerative medicine” as the National

The Alliance for RegenerativeMedicine: Securing FavorableResearch, Regulatory andReimbursement Policies toAdvance Important TechnologiesBy Michael J. Werner, Esq.


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priorities for research topic areas to be addressed by ARRA funds.Some of these topics – including use of biomarkers for treatment ofvarious cancers – are clearly relevant to regenerative medicine.

Thus, by educating federal policy makers about regenerativemedicine, the Alliance will help ensure that federal comparativeeffectiveness research is done appropriately. For example, the NIHhas issued proposals for comparative effectiveness research inregenerative medicine. The Alliance will engage the agency to discussresearch priorities for grants in this area.

Moreover, lawmakers are currently debating whether the resultsof this research should be used only to improve clinical decisionmaking or can also be used as a basis for coverage and paymentdecisions by Medicare and private insurers. The Alliance will engagein that debate as well.

The Regenerative Medicine AdvocacyAgenda

In addition to the broader health care reform, medical research,and comparative effectiveness research debates, the regenerativemedicine community should advocate on issues specific to facilitatingresearch and commercialization of regenerative technologies.

If these technologies are going to meet their potential and helppatients with diseases and disabilities, we need a comprehensivestrategy to make sure public policies are enacted that facilitatecontinued success. Specifically, our nation must adapt:

• Research funding policies that support necessary research inuniversities as well as in private companies;

• A responsible and predictable regulatory policy to ensure thatsafe and effective products reach patients as soon as possible;and

• A market-based reimbursement system that rewardsinnovation and will ensure patient access to new products.

The Alliance will develop policies and an advocacy strategy in allthese areas.

Research AgendaThe federal government plays a critical role in supporting

scientific research. While NIH funding of human embryonic stemcell research has been controversial (though still funded, albeit in alimited way), the agency has been supporting other regenerativemedicine and tissue engineering research for many years. In fact, theagency has provided hundreds of millions of dollars for regenerativemedicine projects. In addition, the Department of Defense (DoD)launched the Armed Forces Institute for Regenerative Medicine(AFIRM) in 2008. This program will provide up to $250 million forregenerative medicine research that focuses on conditions faced bymilitary personnel.

As an organization that represents the regenerative medicinecommunity, the Alliance will have the necessary expertise to workwith NIH, DoD and other agencies to identify and recommendresearch priorities, as well as to advocate for funding andprogrammatic support from Congress. The Alliance will also work toidentify and advocate for new funding sources from Congress andrelevant agencies.

Institutes of Health (NIH) does: the process of creating living,functional tissue to repair or replace tissue or organ function lost dueto age, disease, damage or congenital defect. It includes regeneratingdamaged tissues and organs in the body by stimulating previouslyirreparable organs to heal themselves, using stem cells to grow cells,tissues and organs to repair damaged or destroyed cells and tissue thebody cannot heal by itself, and potentially solving the problem of theshortage of organs available for donation compared to the number ofpatients that require life-saving organ transplantation.

The Alliance is the first advocacy organization exclusivelydedicated to representing the regenerative medicine community witha focus on product development. Its membership will consist ofbiotechnology and pharmaceutical companies, university-basedresearch organizations, independent research organizations, venturecapital firms, patient advocacy organizations, and serviceorganizations that share the goal of advancing cell-based therapies,biomaterials and tissue engineering products to the marketplace.

An incorporated and non-profit organization, the Alliance willact as the political and policy advocate of the regenerative medicinecommunity. It will be a source of information about the field ofregenerative medicine for policy makers , the media, and the generalpublic. The Alliance will provide a single, credible voice to representthe regenerative medicine community. It will educate policy makers– in Congress, at FDA, CMS, National Institutes of Health (NIH),and elsewhere – about the key healthcare policy issues affectingregenerative medicine.

Now is The Time for Creation of the AllianceThe time is right for creation of the Alliance. Scientifically,

regenerative medicine continues to demonstrate its promise. Almostevery day a new breakthrough is announced. In addition, there is atrack record of success as several regenerative medicine products areon the market and have demonstrated their effectiveness.

It is an opportune time from a political and policy perspective aswell. The Congress and Obama Administration are currentlydebating several relevant health care issues. Our nation is in themidst of a national debate about health care reform and politicalleaders are grappling with the details of designing a new health caresystem. To this point, much of that debate has focused on healthinsurance and taxes/financing issues.

However, this debate should not just be about providing healthinsurance; it also provides an opportunity to enact policies that willimprove Americans’ health. The Alliance can play an important roleas Congress continues these deliberations to communicate theimportance of federal policies that support innovation andcommercialization of new technologies that are key to regenerativemedicine.

Another important ongoing debate surrounds government-sponsored comparative effectiveness research. The AmericanRecovery and Reinvestment Act (ARRA or the so-called “economicstimulus bill”) provided over $1 billion for comparative effectivenessresearch. Funds are divided between the NIH, AHRQ and theSecretary of Health and Human Services (HHS); some grantproposals have already been issued by the agencies. In addition, theInstitute of Medicine (IOM) recently issued its recommended



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Documents

Federal Funding of Stem Cell Research: Past, Present and Future