Senators Support Regenerative Medicine Promotion Act of 2014

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Whitefish, MT / April 28, 2014 / Last month, the regenerative medicine industry received important support that could facilitate a road map to accelerate the discovery of new therapies for some of the world's most hard-to-treat and cost-intensive diseases and conditions. Senators Mark Kirk (R-IL) and Barbara Boxer (D-CA) introduced the Regenerative Medicine Promotion Act of 2014 in the U.S. Senate seeking to establish a national strategy to support regenerative medicines and a Regenerative Medicine Coordinating Council.

Less than one month later, the regenerative medicine industry was seemingly dealt a blow when Dr. Mahendra Rao stepped down from his position as director of the National Institute of Health's Center for Regenerative Medicine (CRM). Just days later, the CRM website was taken down. Dr. Rao reportedly was frustrated with the action of the NIH to approve funding for the advancement of projects. CRM was established in 2010 to create a central hub for NIH stem cell research and allocated a budget of $52 million over seven years to develop therapies using induced pluripotent stem cells (iPSCs), adult stem cells that are reprogrammed back to an embryonic, or naive, state. However, only one clinical trial application, that of Kapil Bharti of the National Eye Institute for age-related macular degeneration, had been approved during Dr. Rao's time as head of the agency.

It's noteworthy that Dr. Rao is now serving on advisory boards for three companies focused on stem cell research: Cesca Therapeutics (KOOL), Stemedica and Q Therapeutics, as well as recently accepting the position of VP of Regenerative Medicine at the New York Stem Cell Foundation, a non-profit organization the provides funding for stem cell research.

What some may have viewed as a sign of weakness in government support towards regenerative medicine could very well one day be seen as the catalyst for a watershed moment in stem cell development in the U.S. Consider that the U.S. certainly does not want to fall behind as a biotechnology leader; the country is far more comfortable thinking of itself as the global leader in the development of innovative new technologies.

Other countries are not waiting on the U.S. to take action to maintain its longstanding leadership position. Japan has already implemented new initiatives in a bid to safely expedite stem cell products to commercialization, including providing $1.1 billion in funding to support iPSC and other forms of stem cell research, as well as implementing an accelerated regulatory framework that will enable qualified regenerative medicine therapies to be conditionally approved and commercialized years earlier than previous standards. The United Kingdom is launching its Early Access to Medicines Scheme, a program that will throw a lifeline to seriously ill patients by allowing them to use groundbreaking new drugs years in advance of when the drugs would potentially make it to market using the traditional regulatory approach. Recently, the European Medicines Agency announced its own pilot program designed to enable earlier conditional approval and marketing of new drugs that show potential to address areas of unmet medical need before late-stage trials are completed.

With those things in mind, the happenings at CRM should ring alarms in Washington and highlight the need for a national strategy in the U.S. to advance stem cell research as one of the most promising life sciences today.

Senators Kirk and Boxer putting aside any political grandstanding to join together and lead bipartisan initiatives is exactly what is needed today to shepherd reformation in NIH regenerative medicine policy. NIH spokeswoman Renate Myles recently told BioPharma-Reporter.com that the NIH is holding an expert workshop on May 5 to help "prioritize a number of requirements that the larger community has articulated through white papers over the past two years," with the NIH using that information to establish a set of goals for the CRM.

On that point, CRM is not shutting down. It is articulating and redefining its direction. Advocacy for change supported by Senators Kirk and Boxer comes at an auspicious moment with Rao's resignation putting an exclamation point on the need for efficient, expanded and forward-thinking legislation. Further, the Regenerative Medicine Promotion Act of 2014 is being supported by leading organizations, such as the Alliance for Regenerative Medicine, the ALS Association, the Juvenile Diabetes Research Foundation, the National MS Society, the National Stem Cell Foundation, the Parkinson's Action Network and many more.

"Introduction of legislation into both the House and the Senate with bipartisan support represents a watershed moment," Geoff MacKay, Chairman of the Alliance for Regenerative Medicine, and President and CEO of Organogenesis Inc., remarked in a phone interview. "It illustrates the growing appreciation that the U.S. faces continued and substantial challenges on the healthcare front; challenges that could be solved through the development of new regenerative medicine technologies that improve medical outcomes for patients, enhance their quality of life, and reduce overall healthcare costs."

The need to accelerate development and create greater access to new stem cell therapeutics is particularly close to Senator Kirk and a personal mission that he is championing with visionary leadership. Not only did the Senator lose his father to pulmonary fibrosis, but in January 2012, he suffered a significant ischemic stroke, a potentially deadly occurrence resulting from an obstructed blood vessel in the brain. Like most stroke victims, Senator Kirk became acutely aware of the limited treatment options available.

There is only one FDA-approved treatment for an ischemic stroke, tissue plasminogen activator (tPA), which must be administered within three hours of the stroke to improve chances of recovery. Athersys, Inc. (ATHX) is aiming to reshape stroke treatment by expanding the window of treatment opportunity to days, rather than hours. The company's allogeneic cell therapy product branded MultiStem(R) is nearing completion of enrollment in a Phase 2 trial for ischemic stroke patients, with results expected to be reported later this year. If successful, the trial could help redefine stroke medical care, and represent a transformational point for the both the company and the field.

Similarly, there is no effective standard of care for idiopathic pulmonary fibrosis, a progressive and fatal restrictive lung disease, but research suggests that use of mesenchymal stem cells (commonly referred to as MSC's) may provide a therapeutic benefit to IPF patients. Through its recent acquisition of the Osiris MSC-based regenerative medicine franchise, Mesoblast is evaluating Prochymal, an IV-administered formulation of mesenchymal stem cells, in a Phase 2 trial of patients with chronic obstructive pulmonary disease (COPD), the fourth-leading cause of death in the U.S.

Point being that there is hope on the horizon for innovative therapies to bridge significant gaps of unmet medical need through regenerative medicine. Not only for stroke and lung disease patients, but also for a plethora of diseases and conditions affecting millions of Americans who suffer from neurodegenerative diseases, diabetes, spinal cord injuries, etc.

Senator Kirk knows first hand how intensive recovery from a major stroke can be and what sort of internal fortitude it takes to not give up in the face of a dearth of approved therapeutics. Following brain surgery to reduce inflammation, the Senator spent nearly a full year in rehabilitation before making a triumphant return to Washington, where he was cheered by members from both sides of Congress and onlookers as he climbed the 45 steps of the Capitol Building one-by-one. Senator Kirk, who still uses a wheelchair to get back and forth to the Capitol, told CBS Chicago last month that more than two years since his stroke, he goes through six hours of physical therapy per week to try and increase his mobility. It's this type of tenacity and respect from peers that Senator Kirk is bringing to the table to spearhead efforts to see the Regenerative Medicine Promotion Act of 2014 enacted into law.

There may not be a better motivator than personal experience to champion humanitarian efforts, but the leadership of Senator Kirk, as well as Senator Boxer, is multi-faceted as it can deliver a considerable financial benefit as well. In a joint statement announcing the introduction of the Regenerative Medicine Promotion Act, Senators Kirk and Boxer cited a published study that "estimated that regenerative medicine could save the U.S. $250 billion per year in direct costs associated with late-stage Parkinson's disease, new cases of spinal cord injury, heart failure, stroke, and insulin-dependent diabetes." Given that the U.S. is more than $17 trillion in debt, $250 billion in annual savings could certainly help. Furthermore, the potential indirect, long-term benefits of regenerative medicine could help keep people healthier and more productive contributors to the economy.

Michael Werner, the Executive Director and a founder of the Alliance for Regenerative Medicine and partner at the Washington, D.C-based law firm Holland and Knight LLP, says, "Regenerative medicine products have already demonstrated the ability to treat complications from diabetes, heal wounds, and address cartilage repair. But that's just the tip of the iceberg. These products hold the promise of addressing the diseases -- heart disease, stroke, and Parkinson's -- that impose a tremendous social and economic cost on families and our country. The Regenerative Medicine Promotion Act launches the national strategy the US needs to ensure these products fulfill that promise."

All of the posturing and rhetoric on Capitol Hill for the last decade over stem cells has been a double-edged sword that has been defeatist in many ways, but the debate has also cemented some cornerstones of the framework for rapid advancement. The industry has long passed the tipping point where embryonic stem cell research is the only option and legislation has removed that from the argument. On March 9, 2009, President Barack Obama issued Executive Order 13505, rescinding law set in place by President G.W. Bush that restricted funding on scientific research involving human stem cells, expanding the scope of stem cell lines that can be researched and charging NIH with the task of developing new guidelines. In 2011, an April Appellate Court upheld a ruling permitting the government funding stem cell research. In January 2013, the Supreme Court refused to hear a final appeal in the case of Sherley v. Sebelius, which challenged the legality of NIH funding for embryonic stem cell research, effectively ending the years-long court battle. These actions set precedents and affirmed the government's commitment to advance regenerative medicine.

To quote President Obama on the enactment of Executive Order 13505,

"Today, more than ever before, science holds the key to our survival as a planet and our security and prosperity as a nation. It's time we once again put science at the top of our agenda and worked to restore America's place as the world leader in science in technology."

The days of political wrangling, pomposity and inaction need to be replaced with unity, dedicated effort and strength to meet the goals of the Presidential order. The leadership of Senators Boxer and Kirk should help realize the transformation and potential impact of regenerative medicine for the good of the country, the people afflicted and those who care about them, and the national economy.

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