Saturday, January 25, 2014

Riding the advocacy revolution: stem cell activists, the future of CIRM, and public awareness

If the promise of stem cells to treat a myriad of diseases – a potential outlined in my first of two reports on the sixth World Stem Cell Summit – is to be fulfilled, advocates and patient organizations must exercise effort and expertise in helping steer the research.

Starting with AIDS-related patient advocacy, a “revolution” has occurred over the past two decades in how patients have related to their doctors and the pharmaceutical industry, said Kevin McCormack, the senior director of Public Communications and Patient Advocate Outreach for the California Institute for Regenerative Medicine (CIRM). CIRM was one of the leading co-sponsors of the summit, held last month in San Diego.

“This is a really exciting time and really interesting time in medicine,” McCormack said, introducing a panel on patient advocacy and stem cell research on December 5. “There’s a lot of change going on. Part of it is due to all the advances that have been taking place, all the progress that is being made in many different fields, but obviously in stem cells in particular. That’s why we’re here.

“There’s also a change in the way we’re engaged. In the past it was a very paternalistic system, for want of a better word, where you went to the doctor, and the doctor said, ‘This is what you have,’ and then the doctor said, ‘This is how we’re going to treat you.’”

Now, said McCormack, that has changed. “Patients and patient advocates are really demanding more of a role, more of a voice, beginning with HIV-AIDS, where people just didn’t want to wait around ... to get the therapies,” he said. “And so they pushed and advocated and demanded in every part of the decision-making process.”

Beyond the key issue of Huntington’s disease advocacy, this article also addresses the future of the world-leading, public-bond-supported CIRM and the need for greater public education about the stem cell field, including safety concerns.

Show up, get involved

Judy Roberson, RN, one of California’s leading HD activists, led off the above-mentioned panel with a simple but crucial tenet of advocacy: “Show up for things. Show up for meetings like this. Join support groups and national organizations…. When you get involved, opportunities come your way.”

Such opportunities become available because major “decision-makers” usually attend scientific meetings such as the Huntington’s Study Group conference she keynoted in 2009, Roberson pointed out.

As a result, Roberson, then the president of the Northern California Chapter of the Huntington’s Disease Society of America (HDSA) and head of the Joseph P. Roberson Foundation (founded by and named for her HD-stricken brother-in-law), was invited by the Food and Drug Administration (FDA) to become an FDA HD advocate. The FDA oversees clinical trials for drugs and approves treatments. Roberson’s husband Tim died of HD at the age of 51.

“You’ll never get anything unless you ask for it,” Roberson continued, recounting how in the early 1990s she approached her neighbor, California State Senator Mike Thompson, to seek support for Huntington’s programs. Thompson, who headed the legislature’s powerful budget committee, helped allocate $1 million for HD programs over a period of five years. Thompson now serves in the U.S. Congress.

More recently, Roberson and other California HD advocates worked to help Drs. Vicki Wheelock and Jan Nolta of the University of California, Davis, secure a $19 million grant to conduct research towards a stem cell trial for HD, as mentioned in my previous article on the summit and detailed in a 2012 article.

You can watch the Stem Cell Summit advocacy panel, including Roberson’s speech, in the video below.

An eye-opening experience

For Katie Jackson, also of Sacramento and the vice president for Help4HD-International, a newly emerging patient and family support organization, the summit opened up a new world. Jackson’s husband was diagnosed with HD in 2006. The couple has two untested children with a 50-50 chance of inheriting the HD mutation.

“This summit has been so eye-opening and so amazing,” Jackson told me in an interview on December 5. “I thank the California Institute for Regenerative Medicine for sending me here….  From the second you wake up in the morning till the second you go to bed, you’re amongst these innovator-researchers that are just changing the world.”

Jackson said she was excited to be “part of the medicine that’s today, and that is regenerative medicine…. People are starting to understand that this is important stuff.”

The Stem Cell Summit left Jackson with a feeling of great hope, she said.

“There are a lot of people that are working hard to find therapies and possibly the cure,” she said. “Hopefully this is the last generation of Huntington’s disease.”

You can watch the entirety of our interview in the video below.

The center of the stem cell universe

As stated by outgoing CIRM President Dr. Alan Trounson in his keynote speech on December 4, numerous CIRM-supported projects are putting the drive towards stem cell treatments into “high gear.”

According to Jonathan Thomas, the chair of the Independent Citizens’ Oversight Committee (ICOC), CIRM’s governing board, the agency is currently funding research regarding 40 incurable diseases and conditions. That research is part of projects funded at 70 different California universities, research institutions, and biotech companies.

Critically, these efforts aim to get potential therapies into human clinical trials, the final, crucial step before the FDA can approve a drug.

CIRM, approved in a 2004 statewide vote on Proposition 71, has so far spent $1.85 billion of its mandated $3 billion budget, funded by state-issued bonds.

California was already “loaded” with research talent, Thomas observed in a CIRM public forum on December 3, “but the fact that CIRM is here has enabled the state to attract senior stem cell scientists from all over the world just to have the opportunity to get funded for their work.”

Thanks to CIRM, California has become “the center of the stem cell universe” and the “envy of the rest of the world because of what the voters have enabled us to do,” Thomas continued.

“No other state has been able to duplicate this,” he said. “There are smaller efforts that are funded either by annual appropriations by state legislatures or largely funded through philanthropic gifts or whatever. No place has the bonding authority we do.”

Keeping the research pipeline open

CIRM will fund its final round of projects by 2017. Actual administration of the grants will extend to about 2021, McCormack said.

“But obviously the key question here is: what about funding the research that’s already in the pipeline, about the kind of exciting progress that we’ve made that we don’t want to see wither and die, because if we go away, who’s going to fund it?” he said.

McCormack explained that continued support for the early and middle stages of a project enables the researchers to avoid the so-called “valley of death” – a lack of funds preventing the scientists from approaching large drug companies to promote the idea of the large, expensive, and sometimes lengthy Phase III clinical trials, the final stage before FDA approval.

“We’re looking at a number of different ways of getting new money – private funding, philanthropy, a number of different choices that we’ll be exploring,” McCormack said. “We’re not ready to go public yet with some of the things we’re looking at, but hopefully we’ll be able to find some way of continuing this research.”

“I hope with all my heart that we can go for a part 2 of Proposition 71,” said panel member Don Reed, a leader in the 2004 effort, who became active in the cause because of his son Roman’s paralysis resulting from a college football accident in 1994. “It’s going to be the greatest stem cell battle in the history of the world, and if we win, there’s just going to be unbelievable stuff that will happen…. Support it. Send letters to the editor. Fight every we you can.”

Roberson noted that Prop 71 originator Bob Klein’s comments at the Stem Cell Summit included plans for an “advocate boot camp” in support of CIRM. Klein served as the first chair of the ICOC from 2004-2011.

Panel member Alex Richmond, an advocate for treatment of children’s neurological disorders and the executive director of Children’s Neurobiological Solutions, observed that, as a public agency, CIRM cannot advocate for its own existence. “So it puts more pressure on organizations like ours and individuals like you, individual scientists that are out there, to be really as good an advocate as they can,” he said.

Public education critical

Researchers speaking at the CIRM public forum and presenting projects at the Stem Cell Summit both emphasized the need for greater public understanding of stem cell science.

In addition to reports on specific diseases such as Parkinson’s, AIDS, and cancer, the public forum provided easily understandable introductions to stem cell basics, focusing on such themes as adult stem cells, pluripotent stem cells (which can become any kind of cell type), and cell replacement therapy.

You can watch videos of all the public forum presentations, as well as a number of other panels and presentations of the summit, by visiting my 2013 World Stem Cell Summit video album, to which I've just added twelve more presentations.

To promote stem cell education, two researchers presented a poster titled “Developing an Easy-to-Understand Booklet for the General Public to Discuss Issues in Stem Cell Research.” Authored by Keiko Sato, Ph.D., and researcher Mie Samura of Kyoto University, Japan, the project seeks to correct misinformation and misunderstanding about the field.

“Misleading information released by the media is a routine problem for stem cell research stakeholders,” they stated on their poster, which refers to Japan but can be applied to the U.S. and other cultures. They cited the examples of embryonic stem cells, reported in some media outlets as being “created by destroying fertilized eggs intended to become babies.”

The book will appear in Japanese and also online in English.

Dr. Keiko Sato (right) and researcher Mie Samura at their poster about stem cell education (photo by Gene Veritas)

Their poster also warned about the “hype” surrounding unproven stem cell treatments, sometimes leading to “unexpected side effects” in patients who try them.

“Thus, the current situation has the potential to undermine the relations of mutual trust between researchers and the general public about stem cell research,” the authors wrote.

The poster “Stem Cell Clinics and the Internet,” by Dr. Ruairi Connolly and two collaborators from the National University of Ireland, further underscored the dangers of unproven treatments offered by online clinics.

“Despite a lack of appropriate accreditation, (such) clinics report major clinical improvements and the curative potential of treatment,” they wrote. “The provision of stem cell therapies in such an unregulated online environment is jeopardising the development of this nascent branch of medicine and offers a substantial risk to both the health of patients availing of these treatments and to the credibility of long term research in this domain.”

Dr. Ruairi Connolly with poster about unaccredited, online stem cell clinics (photo by Gene Veritas)

(Disclaimer: I received a stem cell summit scholarship from CIRM, which covered the cost of registration. CIRM officials did not in any way influence or control what I have written here.)

Wednesday, January 15, 2014

Game-changers in the fight against disease: a report from the World Stem Cell Summit

With a growing array of possibilities, stem cell treatments for diseases and other medical conditions hold the potential for a new era in human health.

That upbeat message – including a report on Huntington’s disease research – dominated the 2013 World Stem Summit, held in my home city of San Diego last December 4-6. I attended the sixth annual summit as an advocate for the Huntington’s Disease Society of America (HDSA).

“Over the next 20 years we need the brightest young minds using all the platforms of technology to drive creativity for solutions to defeat the problems of disease using stem cells,” said Alan Trounson, Ph.D., the outgoing president of the California Institute for Regenerative Medicine (CIRM), during his keynote address. “If we do that, I’m sure we’re going to be successful.” CIRM, a state-run research funding agency, is spending a voter-approved $3 billion by 2017 to explore stem-cell treatments for various diseases.

“All of us nationally and internationally involved in stem cell research firmly believe that some of these things will work out,” Jonathan Thomas, Ph.D., J.D., the chair of the CIRM oversight board, said at a CIRM public forum. “The great thing about this field is everything’s a game changer. So whatever any of these terrific scientists are able to get through to fruition will literally change the world when it comes to that particular disease or condition. Therein lies the promise of stem cell research.”

You can watch the Trounson and Thomas speeches, as well as other presentations, in my stem cell summit album by clicking here.

‘Cell sheets’ for eyes and hearts

From AIDS to cancer to urinary incontinence, researchers presented exciting advances in stem cell research and the efforts to improve people’s health.

In one of the most striking presentations, Teruo Okano, Ph.D., of Tokyo Women’s Medical University, demonstrated his “cell harvesting” technique to create “cell sheets” of particular kinds of tissue. He has transplanted these sheets onto diseased eyes, cancerous esophageal tissue, and damaged heart muscle.

The small number of patients receiving these experimental treatments has shown dramatic improvement.

Dr. Okano’s team is seeking to employ cell sheets in the treatment of conditions affecting the gums, lungs, liver, pancreas, cartilage, and the middle ear. They are currently seeking to develop a cell sheet-based tissue and organ factory to automate and standardize cell sheet production, aiming to minimize human error and expand the availability of these treatments.

You can watch Dr. Okano’s presentation in the video below.

Safe cells for an HD trial

On the same panel, Jan Nolta, Ph.D., the director of the Stem Cell Program and Institute for Regenerative Cures at the University of California, Davis, provided an overview of her lab’s work with mesenchymal stem cells (MSCs), which scientists primarily derive from the bone marrow.

“I’ve been working with these cells and have a love affair with them for over 25 years,” Dr. Nolta said. “What we do with them is to genetically engineer them…. They are in clinical trials. They are safe. And they have some really cool properties.”

The MSCs' abilities include restoring blood flow, preventing cell death, reducing inflammation, and keeping the immune system at bay during tissue remodeling (natural repair of tissue), Dr. Nolta explained. MSCs have been used with “statistically significant success” in clinical trials involving heart disease, orthopedics and spine fusion, cartilage repair, autoimmune diseases, Crohn’s disease, stroke, and arthritis, she added.

Dr. Nolta also provided an update on her lab’s projected clinical trial of MSCs to combat Huntington’s disease by using the cells to deliver a key growth factor, BDNF (brain-derived neurotrophic factor), to brain cells. In July 2012, CIRM granted her lab $19 million to support the project. (Click here to read more.

“We’ve just started the lead-in clinical trial,” Dr. Nolta told the summit audience. “We’ll observe patients for a year, before they would get the cell therapy. We’re recruiting patients at that trial now.”

You can watch Dr. Nolta’s presentation in the video below.

Becoming part of the fabric

Further confirmation of advances in the stem cell field came in the release of a report, Stem Cell Research: Trends and Perspectives on the Evolving International Landscape, which revealed that stem cell research is growing at more than twice the world average for research in general (7% versus 2.9%). The report further noted that about half of stem cell papers refer to “drug development” or “regenerative medicine,” further evidence of the field’s promise for developing treatments.

In the summit’s exhibit hall, scores of scientific posters demonstrated progress on numerous fronts, and displays by stem cell related companies, flanked by expert salespersons, showed how much stem cells are becoming part of the fabric of business.

At the booth for BioSpherix, sales representative Ray Gould explained to me how his company’s product Xvivo System, a small, modular GMP (good manufacturing practices) setup, provides an alternative (for a fraction of the cost) to the large, multi-million-dollar facilities put up by organizations for stem cell and other kinds of research.

Ray Gould of BioSpherix explains the use of the company's modular GMP system (photo by Gene Veritas).

Fulfilling the promise, understanding the odds

For me, even though I carry the deadly HD gene, the glow of hope from the summit has not worn off.

After the conference, as requested, I started receiving e-mails from the Genetics Policy Institute, which, along with CIRM, was one of the event’s six major sponsors. The messages update the latest developments in stem cell research.

Of the many items, two in particular struck me: a project by the Mayo Clinic to grow stem cells at the International Space Station as a pathway to treatments for stroke and the use of inkjet printing technology to print eye cells to potentially treat retinal disease and help cure blindness.

Although a number of news reports echoed the optimism of the summit (click here for one example), at least one carried the reminder that many attempts at developing stem cell treatments have failed.

Such failures are not surprising. In general, 90 percent of all clinical trials fail to produce a treatment. By their very nature, science and drug discover involve a long process of trial and error.

Stem cells capture our imagination because they come from our bodies. In this respect they differ from typical pharmaceutical agents such as vaccines and medicines, which involve introducing non-human agents into the body (such as dead viruses or chemicals). In addition, as the work of Dr. Nolta and others has demonstrated, stem cell research gives us a greater understanding of the function of the human body.

Stem cells comprise just one part of the toolkit for treating diseases. Despite the likelihood of a high failure rate in clinical trials, having them in the kit along with gene therapy and numerous other approaches increases the overall chances of discovering effective treatments, including Huntington's disease and other currently untreatable neurological disorders.

Next time: the stem cell summit, advocacy, and the future of CIRM.

(Disclaimer: I received a stem cell summit scholarship from CIRM, which covered the cost of registration. CIRM officials did not in any way influence or control what I have written here.)