Tag Archives: stem cell research

CU Scientists’ Discovery Could Lead to New Cancer Treatment

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blood cancer

The University of Colorado’s Gates Center for Regenerative Medicine and Stem Cell Biology just announced a potentially game-changing discovery in stem cell research for blood cancers and a whole host of other diseases.

Yosef Refaeli and his research team have found a way to expand blood stem cells. This is big news because blood stem cells can help treat blood cancers like leukemia and lymphoma as well as inborn immunodeficiency diseases such as sickle-cell anemia and autoimmune diseases like multiple sclerosis. But up until now, treatment using blood stem cells has been limited by the number of cells a patient can produce. Hundreds of thousands of Americans could be affected by this discovery.

The research was supported in part by funding from the National Institutes of Health.

The goal now is to move the technology from the lab into clinical trials. Colorado-based biotech company Taiga Biotechnologies is in the process of setting up the trials.

The research was originally published in the academic journal PLOS ONE. Read the paper here.

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A Weekly Advocacy Message from Mary Woolley: First 100 Days

Dear Research Advocate,

With all the conversation about the debt ceiling and tax and entitlement reform, it may surprise you to know that an additional topic is on many minds. A wide majority of Americans, 72%, say the new Congress and the president should take action to expand medical research within the first 100 days of the new legislative session. See this and more in America Speaks, Volume 13, a compilation of national poll data providing insights into public sentiment on key research-related issues. See our press release and download the full Poll Data Summary. These polling results are designed to be used in your advocacy and outreach!

Among the growing number of issues that need to be resolved by the new Congress is the medical device tax, which could send research jobs overseas and shrink a critical segment of our innovation economy. In The Hill, Rep. Bob Latta (R-OH) writes about the consequences the tax could have on the medical device industry, including the possibility of a massive decline in R&D investment. As our economy recovers, policy makers must better incentivize R&D investment to keep our nation competitive and ensure that companies are continuing to invest in life-saving research.

More on the first 100 days: As you know, the sequestration deadline has been moved two months, with another delay possible, and there is talk of other cuts to discretionary spending. The delay is terribly frustrating for those planning research investment and sends a very negative message to young scientists planning a career, but it does buy us more time to make our case. The Washington Post published an op-ed by three Washington, DC, institutional members of Research!America that argues compellingly for such funding. Take action now and do two things — collaborate with your local colleagues to write an op-ed for a local publication and send an email to your representatives. Tailor the alert we provide to let them know how cuts could affect your institution and your community.

For those of you in Georgia, the appointment of Rep. Jack Kingston (R-GA) as chairman of the committee that allocates most of the federal funding for biomedical and health research funding presents an important advocacy opportunity. Research!America is helping to facilitate collective action by Georgia institutions, and we would welcome your participation. Please contact Max Bronstein, director of science policy, if you haven’t heard from us yet! Recently, I had the opportunity to discuss Rep. Kingston’s record and prospects for the 113th Congress with Randy Barrett of the ScienceInsider.

Great news! The U.S. Supreme Court issued a decision on behalf of patients and stem cell researchers, effectively bringing to a close the infamous Sherley v. Sebelius case that threatened federal funding for embryonic stem cell research. This decision marks a major victory for the stem cell research cause, but it is critical that all of us remain vigilant; actions at the state level could still curtail embryonic stem cell research. View our press statement on the decision and our updated resource page on stem cell research. We will be talking about the importance of stem cell research when we honor the California Institute for Regenerative Medicine (CIRM) at our upcoming Advocacy Awards dinner. See more about this March 13, 2013, event here.

Sincerely,

Mary Woolley

Research!America President and CEO Mary Woolley Applauds Supreme Court’s Dismissal of Embryonic Stem Cell Case

January 9, 2013

The U.S. Supreme Court’s dismissal of Sherley v. Sebelius, a case intended to block federal funding for scientists conducting embryonic stem cell research, is a victory for patients and the research community. This key decision will allow the continuation of federal funding from the National Institutes of Health, providing essential support for scientists to conduct lifesaving research. Embryonic stem cells, which can repair or replace damaged tissue and organs, have advanced research aimed at finding cures and therapies to treat a wide variety of diseases and disorders including vision impairment, spinal cord injuries, and multiple sclerosis.  Clinical trials have also shown promising therapeutic applications to help fight cancer, Parkinson’s disease, diabetes and other disabling illnesses. We applaud the ruling and will continue to support such innovative research that could save millions of lives.

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Statement from Research!America Chair John Edward Porter and CEO Mary Woolley on Passing of Senator Arlen Specter

We extend our deepest condolences to Senator Arlen Specter’s family, friends and colleagues as they mourn the passing of a loved and respected statesman and a true champion of medical research.  Specter’s leadership in generating critical support for medical and health research is a testament to his dedication to improving the health of all Americans and securing our position as a global leader in science and innovation.  As a congressional leader, Specter played a pivotal role in the doubling of the National Institutes of Health (NIH) budget over five years and two Administrations and in 2009 assured that funding for the NIH and other health agencies were included in the American Recovery and Reinvestment Act.  He was also a stalwart advocate for embryonic stem cell research and worked to secure U.S. funding for the Global Fund to Fight AIDS, Tuberculosis and Malaria.  Research!America was proud to honor Specter with our rarely-awarded Legacy Award in 2009 for his noteworthy contributions and the Edwin C. Whitehead Award for Medical Research Advocacy in 2000. His remarkable achievements as an advocate for scientific discovery will be long remembered and greatly appreciated by future generations.

Research!America Honors Trailblazers In Health Research Advocacy

Diane Rehm; John F. Crowley; Dr. John Mendelsohn; Dr. Mark Rosenberg;and California Institute for Regenerative Medicine to Receive 2013 Research!America Advocacy Awards

WASHINGTON-October 2, 2012Research!America’s 17th annual Advocacy Awards will convene leaders from government, academia, industry and health advocacy organizations to honor leading medical and health research advocates of our time. The event will take place on the evening of Wednesday, March 13, 2013, at the Andrew W. Mellon Auditorium in Washington, DC.

The 2013 Advocacy Award winners are Diane Rehm, author and host of WAMU 88.5 and NPR’s “The Diane Rehm Show;” John F. Crowley, patient advocate and inspirational entrepreneur, chairman and CEO of Amicus Therapeutics, Inc.; John Mendelsohn, M.D., director, Khalifa Institute for Personalized Cancer Therapy and former president at The University of Texas MD Anderson Cancer Center; Mark Rosenberg, M.D., president and CEO, The Task Force for Global Health; and California Institute for Regenerative Medicine (CIRM). A sixth Advocacy Award winner will be named by Research!America’s Board of Directors in December 2012.

“This year’s awardees embody the spirit of pioneers in advocacy, setting high standards for others to follow in achieving a greater awareness and appreciation for research to improve health,” said Mary Woolley, president and CEO of Research!America.”We applaud their leadership and tenacious dedication to informing and engaging the public.”

“The tireless efforts of these extraordinary individuals have brought hope to people affected by devastating diseases worldwide,” said Harry Johns, Research!America Board member, co-chair of the Advocacy Awards selection committee, and president and CEO of the Alzheimer’s Association  “Their collective achievements have greatly contributed to medical progress and innovation in the U.S. and abroad.”

Diane Rehm, author and host of WAMU 88.5 and NPR’s “The Diane Rehm Show,” will be honored with Research!America’s 2013 Isadore Rosenfeld Award for Impact on Public Opinion for emphasizing the value of research and increasing the level of awareness among policy makers and the public of medical conditions such as traumatic brain injuries, Parkinson’s disease, multiple sclerosis and Alzheimer’s disease. Her show reaches millions of listeners around the world, contributing significantly to public understanding of science and health.

Patient advocate and inspirational entrepreneur John F. Crowley, chairman and CEO of Amicus Therapeutics, Inc., will receive Research!America’s 2013 Gordon and Llura Gund Leadership Award for his outstanding efforts to accelerate new treatments for Pompe disease and other genetic diseases. His dedication led to the discovery of a treatment for Pompe disease, saving the lives of his children, Megan and Patrick. Crowley’s remarkable story and perseverance inspired the movie Extraordinary Measures starring Harrison Ford and Brendan Fraser.

John Mendelsohn, M.D., director of the Khalifa Institute for Personalized Cancer Therapy at The University of Texas MD Anderson Cancer Center, will receive Research!America’s 2013 Geoffrey Beene Builders of Science Award for pioneering translational research that has provided the foundation for targeted cancer therapies and for his advocacy for increasing our nation’s support for biomedical research. His leadership of MD Anderson during an expansive period of growth, as well as his own groundbreaking discoveries, have transformed cancer treatment, benefiting thousands of patients worldwide.

Mark Rosenberg, M.D., president and CEO of The Task Force for Global Health, will be honored with Research!America’s 2013 Raymond and Beverly Sackler Award for Sustained National Leadership for advancing injury prevention and road safety, reframing the concept that road traffic crashes are not accidents. His advocacy has increased funding for research and programmatic interventions for injury control and improved traffic safety not only in the U.S. but also in many developing countries.

The California Institute for Regenerative Medicine (CIRM) will receive Research!America’s 2013 Paul G. Rogers Distinguished Organization Advocacy Award for its role in accelerating stem cell research and the potential development of a new generation of promising therapies for previously untreatable human disorders. CIRM-funded discoveries have laid the foundation for a new industry in California and attracted top-level stem cell researchers to the state. Jonathan Thomas, PhD, JD, and chairman of CIRM’s Independent Citizens Oversight Committee, will accept the award on behalf of the organization.

The annual Research!America Advocacy Awards program was established by the Board of Directors in 1996 to honor outstanding advocates for medical, health and scientific research. Recognized individuals and organizations are those whose leadership efforts have been notably effective in advancing our nation’s commitment to research.

Research!America is the nation’s largest nonprofit public education and advocacy alliance working to make research to improve health a higher national priority. Founded in 1989, Research!America is supported by member organizations that represent the voices of 125 million Americans. Visit www.researchamerica.org.

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A Weekly Advocacy Message from Mary Woolley: Reading Between the Lines, part 2

Dear Research Advocate,

In last week’s letter, I highlighted research-related themes in the Republican National Platform. The good news included explicit support for basic and applied research and a pledge to make the R&D tax credit permanent. The bad news included strident criticism of FDA — such that support for adequate funding was unclear — and opposition to embryonic stem cell research. The Democratic platform asserts that Democrats will “double funding for key basic research agencies.” It also goes further than the Republican platform in improving the research and development tax credit and places a very strong emphasis on science education as critical to our innovation economy. And, it reiterates Democratic support for embryonic stem cell research.

Do platforms matter? Yes and no. Yes, in that the language comes from a broad base of each party’s membership. It lays out principles that we can ask policy makers to adhere to, and we can see how well those principles track with the polls we regularly commission. But also no — as a respected Nobel laureate reminded me in an insightful response to my last letter, it is a mistake to breathe easy based on the rhetoric in these platforms. Actions speak louder than words, and the fact is Republicans and Democrats alike supported the Budget Control Act (BCA), which not only applies across-the-board cuts to research spending but also tightly restrains annual growth in discretionary spending. That makes it difficult to envision any kind of “moonshot” for research or even a basic policy frame that truly promotes research and innovation. Despite what these platforms assert, policy makers have taken their eye off the ball. The public is not happy about that fact. Our new polling data shows that only 19% of likely voters believe elected officials are paying enough attention to combating disease. For more on this point, see my piece this week in The Hill’s Congress Blog. It ends with a call to action to engage the candidates — you can lead the way in doing just that among your network of colleagues, family and friends.

In case you missed it, a U.S. appeals court has upheld the legality of federal funding for embryonic stem cell research — a major victory for advocates and patients alike. See this recent ScienceInsider article to learn more about the ruling.

In past letters, I’ve written extensively about the grave threat that sequester poses to American research and innovation, and the news seems to be getting worse. According to the Alliance for a Stronger FDA, the user fees that FDA collects for review of drugs and devices may be subject to sequestration in addition to the funding provided through taxes. In effect, the FDA budget would be double-slashed with cuts totaling $294 million! Just imagine the havoc that these cuts would wreak on our nation’s ability to bring new, critical treatments to patients. With Congress reconvening next week, please remind lawmakers that they are playing with fire. Research is important. Innovation is important. Blind, across-the board funding cuts aren’t just an abdication of congressional responsibility, they are a divestment in medical and economic progress.

Sincerely,

Mary Woolley

A Weekly Advocacy Message from Mary Woolley: Reading between the Lines and then Taking Action

Dear Research Advocate,

As you know, the Republican Party Platform was unveiled Tuesday during the convention in Tampa. There are direct references to medical and health research and other statements that — if not explicit — definitely imply the need for such research. We can draw from both to enhance our advocacy efforts.

The following exemplifies the direct and indirect nature of the platform’s embrace of medical and health research:

“We support federal investment in health care delivery systems and solutions creating innovative means to provide greater, more cost-effective access to high quality health care. We also support federal investment in basic and applied biomedical research, especially the neuroscience research that may hold great potential for dealing with diseases and disorders such as autism, Alzheimer’s and Parkinson’s. If we are to make significant headway against breast and prostate cancer, diabetes and other killers, research must consider the special needs of formerly neglected groups.”

The platform explicitly supports federal funding for basic and applied medical research, and, if I am interpreting the text correctly, acknowledges the need to address health disparities as part of the nation’s research agenda. This statement also implies the need for health services research (HSR) to devise “solutions” that improve health care access, cost-effectiveness and quality. Unfortunately the House Labor-H appropriations bill precludes NIH funding for health economics research — a key subset of HSR — and virtually zeroes out the budget of the Agency for Healthcare Research and Quality, the main funder of HSR. The platform provides advocates fresh talking points as final appropriations decisions are made later this year.

The Republican platform also states: “Even expensive prevention is preferable to more costly treatment later on.” While the rest of the statement focuses on personal responsibility, research plays an undeniable role in effective prevention. Vaccines, the nicotine patch, successful drug and alcohol addiction treatment programs … all are grounded in research. Advocates can segue directly from the platform to the importance of prevention research at CDC and other agencies … and we should. Three other sections of the platform are noteworthy. It goes hard on the FDA, asserting that it needs significant reform. The platform does not mention funding, but there is a logical connection here. Patient groups, scientists, industry and FDA leaders themselves are all committed to strengthening the agency and are working hard to accomplish just that. Support for FDA reform cannot logically be decoupled from support for FDA funding, a point that must not get lost in the reform debate.

Second, the platform advocates making the R&D tax credit permanent. Bravo!  We should increase and make other improvements to the credit as well.

Finally, the platform opposes embryonic stem cell research. Not a surprise, but a disappointment.  Proponents must keep fighting this battle, drawing strength from the recent court victory in which President Obama’s executive order was once again upheld.

There is much to applaud in the Republican platform when it comes to federal support for both medical and health research. Let’s take that and run with it. In an article that appeared this week in Forbes, John Zogby discusses the results of our recent national poll. He focuses on the exceptional level of agreement between different demographic and ideological subsets of the American population on issues related to health and medical research. We see that reality reflected in many of the planks in the Republican platform. Indeed most of the results from our poll will not surprise you (except, perhaps, the fact that a majority of Americans of all stripes would pay a dollar more per week in taxes if they knew it was going toward medical research), but it’s a fact that most policy makers have not embraced medical progress as a goal worthy of mentioning in campaign speeches or on their campaign websites. Platforms aside, this gives Americans no basis by which to evaluate whether individual candidates will champion or chop research funding and no assurance that they will take medical innovation into account when evaluating policy decisions that could stimulate or stifle it. Your Candidates-Your Health is an important way that candidates can make their opinions known about medical and health research. Advocates can do their part by attending town halls, visiting campaign offices, writing op-eds and letters to the editor, and using these polling results to convince candidates that promoting medical progress should be one of their core missions.

We have our work cut out for us, but we will succeed if we do more than parse the rhetoric — we have to take action!

Sincerely,

Mary Woolley

A Weekly Advocacy Message from Mary Woolley: Romney adds Ryan and healthcare to election conversation

Dear Research Advocate,

With Rep. Paul Ryan joining the Romney ticket, health is back on the national agenda. Partisan politics aside, this conversation is overdue, since health is indeed an issue that will make or bankrupt us. Research has always figured prominently in the wellbeing of Americans and America – research brought an end to the polio epidemic, which could have bankrupted the nation in the 1950s, and research is the only answer to the scourge of Alzheimer’s that threatens health, quality of life and our national checkbook today. And that is just a starting point for the conversation I hope you are having with everyone who wants to talk about the election. Take the opportunity to bridge from health care to health research and remind Americans that research must be a higher priority. As Research!America Chair and former Congressman John Porter has said, “Priorities will be chosen, and money will be spent.” Let’s make sure health research is a top priority.

How much do we know about Rep. Paul Ryan’s position on our issues? One place to start is with Rep. Paul Ryan’s response to our Your Congress – Your Health questionnaire of 2007. In his responses, Rep. Ryan calls for increasing NIH funding and endorses the importance of STEM education, although not federal support for stem cell research. Obviously, the political and fiscal climate has shifted dramatically since 2007, and the “Ryan budget” passed earlier this year by the House could deprive discretionary programs of funding vital to research, (see my comment in Medpage Today).  

The case for research today is in fact stronger than it was five years ago. The Wall Street Journal has published an op-ed by two Nobel laureates, providing a clear and compelling case for the government’s role in fostering basic research — and including research in economics — yielding huge dividends for our health and economy as a whole. Dr. Peter Kohler, Vice Chancellor at the University of Arkansas for Medical Sciences has published a piece carrying an equally compelling message in the NWAonline and a terrific op-ed has appeared in the Press Democrat by Dr. Dennis Mangan, a former NIH program director now working as a science communication advisor in Santa Rosa, CA. It would be a privilege to work with you on your own op-ed or letter to the editor making the case for policies that promote continued medical progress.

In past letters, I’ve written about the sequester and its potential for gutting funding for health research. The Coalition for Health Funding, of which Research!America is a member, has released a grassroots toolkit to educate and equip advocates to fight the sequester. Please circulate these tools to your networks and make sure that we stand together against the sequester. Make it a point to engage with candidates while they are campaigning around the nation this month.

Sincerely,

Mary Woolley