Monday, September 28, 2009

NIH Gives 115 Research Awards to Encourage Innovation

Supported in part by the American Recovery and Reinvestment Act, the National Institutes of Health (NIH) announced last week that it is awarding $348 million to encourage investigators to explore bold ideas that have the potential to catapult fields forward and speed the translation of research into improved health.

The range of awards is granted under three innovation-based research programs: the NIH Director’s Transformative R01 (T-R01) Awards, the Pioneer Awards, and the New Innovator Awards.

The NIH Common Fund encourages collaboration and supports a series of exceptionally high impact, trans-NIH programs known collectively as the NIH Roadmap for Medical Research. The Roadmap is a series of initiatives designed to pursue major opportunities and gaps in biomedical research that no single NIH institute could tackle alone, but that the agency as a whole can address to make the biggest impact possible on the progress of medical research.

These award programs are part of the NIH Roadmap, funded through the Common Fund and managed by the NIH Office of the Director and the National Institute of General Medical Sciences.

"The appeal of the Pioneer, New Innovator, and the T-R01 programs, is that investigators are encouraged to challenge the status quo with innovative ideas, while being given the necessary resources to test them," said NIH Director Francis S. Collins, M.D., Ph.D. "The fact that we continue to receive such strong proposals for funding through the programs reflects the wealth of creative ideas in science today."

Since no budget cap is imposed and preliminary results are not required for submission, scientists are free to propose new, bold ideas. They also have the flexibility to work in teams if the complexity of the research problem demands it.

The NIH expects to make competing awards of $30 million to T-R01 awardees, $13.5 million to Pioneer awardees, and approximately $131 million to New Innovators in Fiscal Year 2009. The total funding provided to this competing cohort over a five-year period is estimated to be $348 million.

This year's awards make the largest number of Pioneer and New Innovator awards in the programs’ history. Investigators funded via the 2004 cohort, the first year of the Pioneer Awards, have completed their projects. Details on their progress is available here.

Several of this year's awards were given to scientists researching neurodegenerative diseases and neurological disorders. For descriptions of the 2009 New Innovator Award recipients’ research plans, click here. For descriptions of the 2009 T-R01 recipients' research plans, click here. Information on the Pioneer Award is at, including information on this year's awardees, is available here.

by Meghan Kallman

Tuesday, September 22, 2009

Neuralstem, Inc., receives FDA approval for Phase 1 ALS clinical trial

Neuralstem, Inc., a Maryland-based biotech company, announced yesterday that the FDA had approved its application to conduct a Phase I safety trial of its stem-cell based approach to treat ALS (read the Reuters press release here).

The FDA’s approval makes Neuralstem the first company to commence a stem cell trial for ALS. The trial will examine the safety of Neuralstem's cells and the surgical procedures required for delivering injections of these stem cells directly into the human spinal cord. The FDA's approval represents a significant step toward realizing the promise of stem cells for helping damaged neural cells in humans.

This trial will be led by Dr. Eva L. Feldman, M.D., Ph.D., Director of the University of Michigan Health System ALS Clinic and the Program for Neurology Research & Discovery, and Dr. Jonathan Glass, Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center. Both researchers are renowned for their study and treatment of ALS patients.

Neuralstem expects to conduct the trial at Emory University with Dr. Jonathan Glass, M.D., as site Principal Investigator (PI) and with Dr. Nicholas Boulis, M. D. performing the neurosurgery. The overall PI for the ALS trial program will be Dr. Eva Feldman, M.D., Ph.D.

The 18 ALS patients participating in the trial will be treated through a series of spinal injections of Neuralstem’s patented human neural stem cells. This first trial will primarily evaluate safety of the cells and the surgical procedure. The FDA has approved the first stage of the trial, which will consist of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come 24 months later.

Neuralstem focuses on treatments for major central nervous system diseases, including ALS. Pre-clinical work has shown Neuralstem's cells to extend the life of rats with ALS (as reported in the journal Transplantation, October 16, 2006, in collaboration with Johns Hopkins University researchers), and also reversed paralysis in rats with Ischemic Spastic Paraplegia, (as reported in the journal Neuroscience, June 29, 2007, in collaboration with researchers at University of California, San Diego).

Prize4Life congratulates Neuralstem for its impressive work, and is excited to see the FDA take this important step towards actualizing stem cells as a potentially promising therapy.

Prize4Life also congratulates Israel-based biotech company BrainStorm Cell Therapeutics, who recently-secured funding to complete pre-clinical stem cell trials for ALS. A competitor for Prize4Life’s Avi Kremer ALS Treatment Prize, BrainStorm expects to begin Phase I clinical trials in early 2010. BrainStorm’s new funding includes both a prestigious grant from the Israeli government’s Office of the Chief Scientist, as well as private investment.

This FDA approval indicates a major change in the ALS landscape, making it easier for subsequent researchers to gain FDA approval and to move forward with testing other stem cell therapies. Prize4Life is thrilled on behalf of PALS, for whom Neuralstem’s discovery may signal an advance towards lifesaving treatments. Congratulations to all!

by Meghan Kallman

Tuesday, September 15, 2009

AIDS Video Ad Aired--Why Not "Sarah's Story"?

In a publicity push similar to that of “Sarah’s Story”—which Prize4Life blogged on several weeks ago—German public health organization Regenbogen will air a provocative TV ad next week. Hitler, Saddam Hussein, and Josef Stalin figure into a series of highly sexualized video ads as part of a shock campaign promoting World AIDS Day. The provocative commercials, which end with the tagline "AIDS is a mass murderer", aim to scare young people into using condoms by associating the deadly disease with political dictators, reported ABC News (The Telegraph also covered the story).

The ad opens in a darkened bedroom with a man and a woman in bed. The man is a look-a-like of the German dictator. The tagline reads: "AIDS is a mass murderer - Protect yourself!"

The project is the brainchild of German charity, Regenbogen, whose website reads: "Up until now 28 million people have died. And every day there are 5,000 new cases. Which is why AIDS is one of the most effective mass murderers in history."

Some HIV organizations have distanced themselves from the ad, complaining—in a familiar argument—that shock tactics are ineffectual. Some agencies claim also that the tactics serve only to stigmatize the infected and to guilt women especially.

However, the campaign’s producers see the fuss—and the fact that the videos are being watched thousands of times on YouTube—as a sign that their plan is working.

Dr. Amir Afkhami, instructor of psychiatry and behavior sciences and Global Health at the George Washington University, supports the initiatives. "It's effective because it raises awareness of the risk factors...This issue has come up among activists in the U.S. and there have been arguments that there needs to be more shock value," he said.

A 2003 study of by Darren Dahl, published in the Journal of Advertising Research found that shock tactics in AIDS ads significantly increase "attention and retention" of the message. Other studies, including one from Ohio State University, show that appearing to peoples’ fears are "powerful persuasive devices."

"It should encourage conversation about disclosure [of HIV status]," said David S. Novak, a senior public health strategist for Online Buddies Inc. "What is terrible is holding a secret and the secret disease inside of them." Afkhami notes that "The real failure on the part of health care advocates and social advertising has been raising awareness."

Prize4Life is perplexed by the apparent double standard here. These ads—which in some ways are more controversial than the “Sarah’s Story” campaign—have encountered less negative reactions, and have been allowed to remain on the air. The press has commented rationally and academically on the use of shock tactics that Regenbogen employed. Graphic ads warning against the dangers of drunk driving, drug use, and texting while driving, are all considered appropriate fodder for public television.

Given that Regenbogen’s ads have not been removed for their ‘disturbing’ features, we wonder what, exactly, differentiates the shock tactics employed here with the ones used in filming "Sarah’s Story". Is it because HIV/AIDS is well-known, and ALS is not? Is an ALS shock campaign less palatable because ALS' cause is largely unknown, whereas preventative measures are effective in controlling HIV infection?

Whatever the case, we consider it telling that this series of ads, while it has received its fair share of buzz, was allowed to remain public, while “Sarah’s Story” was banned. We support Regenbogen’s initiative, and we believe that “Sarah’s Story” should join it on the airwaves.

As always, Prize4Life believes that sanitized presentations of illnesses are not effective or appropriate—that by ‘cleaning up’ images of HIV or ALS, we are able to ignore them, and that ‘clean’ publicity detracts attention from diseases that could be used to find a cure.

Like ALS, HIV/AIDS is brutal; Prize4Life believes that awareness can help lead to a cure, and that the pain inflicted by disease progression is not ‘too shocking’ to be public, especially in comparison to other things that are publicly aired without controversy (rape, violence, etc).

One commenter on the ABC story wrote, “I think the whole point of the ad is to shock and encourage a rethink of your actions. It's supposed to be disturbing. All the complaints prove it's effective. Nicy-nice ads don't work.”

We think that applies to ALS, too.

by Meghan Kallman

Wednesday, September 2, 2009

Translational Research

As with any advance in human health, scientific discoveries must be translated into practical applications. Scientific breakthroughs typically begin at “the bench” with basic research, and then progress to the clinical level, or the patient's “bedside.”

Researchers are increasingly aware that this “bench-to-bedside” approach to translational research must be a two-way street, an exchange of information. Basic scientists provide clinicians with new tools for use in patients, and clinical researchers make observations about the nature and progression of disease that often stimulate basic investigations.

However, barriers between clinical and basic research, along with the complexities involved in conducting clinical research, have traditionally made it hard to translate new knowledge to the clinic – and back again to the bench.

The National Institutes of Health (NIH) and numerous pharmaceutical companies have channeled billions of dollars into basic research, and have realized that their return on investments is lower than anticipated. Translational research often is seen as the missing component, though organizations dedicated to translational research do exist (such as the Center for Clinical and Translational Studies at the University of Texas).

Through the Clinical and Translational Science Award Program (CTSA), the NIH recently created a national consortium that includes 39 centers in 23 states with an annual funding commitment of $500 million by 2012. Though still young, the program was designed to shorten the time required to translate research results into therapies.

In an editorial in Science Magazine, 13 leaders of a diverse range of organizations wrote a collective editorial entitled “Translational Careers”. The editorial’s authors share a deep interest in clinical and translational research, and express a collective commitment to the development of an effective clinical and translational research workforce.

The editorial is positive about the potential of the CTSA program, but cautions against an overreliance on federal design and an under-reliance on support systems necessary for this kind of a program.

“People are the prerequisite for success,” they write. “We need an array of innovative investigators whose expertise spans all the disciplines of basic discovery and medical science. As a counterpoint to federal efforts, our private, nonprofit organizations have addressed the human capital need in robust ways, training and funding physicians and other clinical scientists, and piloting models for interdisciplinary graduate training involving biologists, physical and computational scientists and engineers, as well as a wide range of clinical and public health professionals."

The editorialists argue that beyond the rigorous research education essential for all scientists, translational scientists who will work at the frontiers of discovery and clinical science must possess a wide variety of practical and logistical skills.

"They must understand the processes by which discoveries turn into therapies, as well as the evolving role of private industry." the group writes. "They must navigate the regulatory environment surrounding human-subjects research, work in teams and share the rewards of their work, and defer financial rewards while spending years in extra training to gain this knowledge. Existing investigators must learn new skills, but we must also attract new people and facilitate productive interactions among them.

“If it fulfills its potential, translational research will lead to better health for people. But translation is not one-way; the insights gained at the bedside, and from clinical and population-based studies, will spawn hypotheses, enabling scientists to probe the mechanisms of disease in new ways and ultimately enriching basic biology. Therefore, strengthening the support systems for those who will accomplish this multidirectional translation can only be good for science,” the group finishes.

Prize4Life finds this thoughtful editorial particularly relevant to the ALS field. We fully support the CTSA initiative, and, in keeping with our mission and our current projects, we are also conscious of the need to create a vibrant and supportive arena in which researchers can work effectively and cures for diseases such as ALS can be developed.