The Life Sciences Report: You wrote the book Inevitable Collision, which details your journey from being outside the scientific community to being at the center of stem cell therapy advocacy. In many ways, this book is about relationships with family, friends and God within the scientific and patient advocacy community. What led you to frame the story of stem cell research in this way?
Tory Williams: Inevitable Collision intertwines the process of research and funding with the experiences of TJ Atchison, the first patient enrolled in the Geron Corp. (GERN:NASDAQ) clinical trial that used human embryonic stem cell treatment to cure paralysis, and Dr. Hans Keirstead, a brilliant scientist integral to the work of overcoming paralysis.
The telling stems from my personal experience dealing with disease. My sister has battled cancer throughout her life, and I was diagnosed in 2010 with polycystic kidney disease (PKD). Then, TJ Atchison, the son of a good friend, was in a tragic car accident in 2010. TJ participated in the first clinical trial combining drugs and human embryonic stem cells to regenerate spinal cord tissue.
As TJ went through therapy, I realized this cutting-edge treatment needed a strong voice and patient advocacy to help rid it of the misconceptions surrounding it. Specifically, it needed someone who could communicate the benefits of stem cell therapy successfully in the conservative, religious South. The use of human embryonic stem cells is particularly sensitive here, but we have broken through many barriers and made tremendous strides in educating people on the power and importance of stem cell research.
TLSR: You aren't a scientist. You had to educate yourself about stem cell research, correct?
TW: I spent countless hours reading, researching and speaking with experts. I knew I needed to get in front of the researchers, specifically Dr. Keirstead, who developed the treatment used in TJ's trial. Roman Reed, president of the Roman Reed Foundation (which funds Dr. Keirstead and other researchers), and cofounder and chair of the Alabama Institute of Medicine (AIM), introduced me to Dr. Keirstead at the University of California Irvine (UCI). Roman and I had a several-hour meeting discussing the nuts and bolts of the Geron clinical trial, and the funding mechanisms that support the research. Through this network of relationships, I've built additional relationships with the leaders at the California Institute for Regenerative Medicine (CIRM). It has been remarkable to be part of this medical breakthrough in regenerative medicine.
TLSR: When I was reading Inevitable Collision, I was struck by the speed with which everything moved. In only four years you helped create TJ's Law (The TJ Atchison Spinal Cord Injury Research Act, funding stem cell research in Alabama), founded AIM, and also formed several spinoff companies. How did everything move so fast?
TW: Timing has been critical. We tried to maximize every opportunity, from speaking before city councils to addressing classrooms. Our No. 1 goal is educating people about the importance of stem cell research. But we also want to demonstrate tangible proof that what we're doing makes a difference. We are making great progress.
"The use of human embryonic stem cells is particularly sensitive in the South, but we have broken through many barriers."
In 2010, Alabama passed TJ's Law to fund spinal cord injury research. We received bipartisan support that awarded funding to the University of Alabama at Birmingham (UAB), which opened the door for me to work with researchers in our state. There's a need not only for funding spinal cord injury studies, but also for funding studies of cardiovascular disease, sickle cell anemia, diabetes and other chronic conditions. I thought it was unfortunate that there was no program to incentivize researchers to stay in Alabama and conduct stem cell research. That's when Roman and I decided to found AIM.
We launched AIM in 2013. Since then, we've raised over $2.2 million ($2.2M) from individuals. The state also added funds: Because of TJ's Law, UAB received $800,000 ($800K) it otherwise wouldn't have received.
AIM, a nonprofit organization, awards grants to investigators at research-based institutions in Alabama. We call for applications, which our review committee assesses using a double-blind approach to eliminate conflicts of interest or favoritism. In the past year, we awarded approximately $900K to UAB to support research. One program targets sickle cell anemia and may enter clinical trials within a few years. Another program targets cardiovascular disease. A third provides early seed funding for Dr. Kejin Hu's study of gene expression involving the PODXL gene, which he believes could transform to an embryonic state similar to that of induced pluripotent stem (IPS) cells. Only 10% of the funding for AIM stays with us for our overhead, salaries, consultants and fund-raising operations.
Stem cell researchers in Alabama are excited and optimistic about our program because it incentivizes them to stay in Alabama, so they don't have to relocate to another state or country in search of funding. That's our aim—to keep our researchers and their families here in Alabama.
AIM has also spun off three thriving companies: VivoStem LLC, WellPace Medical LLC, and Sacred Cells Research Partners LLC. It also has created local jobs. The economic impact will be amplified when we build a research plaza here in Birmingham. This will incentivize researchers to use our facilities to conduct experiments and to collaborate on various projects. We hope to move toward that goal later this year.
TLSR: It would be an embryonic stem cell research center?
TW: The research plaza wouldn't be limited to embryonic stem cells. Our nonprofit organization supports many types of stem cell research, including adult, embryonic and IPS cells. Our bylaws, however, state that we will not support any projects that use fetal cells.
TLSR: Let's talk about the patient advocacy community. Partnering conferences routinely emphasize the importance of patient advocacy foundations like yours. How does this type of foundation interact with the community?
TW: Roman and I have seen, firsthand, the challenges that patients and their families face in searching for better treatments or chronic care. We receive dozens of e-mails every week from patient advocates wanting to know where clinical trials are being offered, and so we recommend our great friend and brilliant researcher Dr. Paul Knoepfler's blog site as a tool for searching ongoing clinical trials using stem cell therapy.
Patient advocacy is just part of our program, though. Oftentimes, people come to us for inspiration. We've helped others who are considering starting similar programs in other states.
Social media, including Facebook and Twitter, as well as our own website, our book and our spinoff companies, have enabled us to publicize our information more effectively.
TLSR: From an investment perspective, what can foundations like yours offer startup companies?
TW: There are hundreds of great organizations that either fund projects independently or offer an application process similar to what we offer at AIM.
One of the most productive conferences I've attended is the annual World Stem Cell Summit, hosted by the Genetics Policy Institute. This year's summit—the eleventh—will be in Atlanta in December. When I spoke about the role of patient advocates in the biotech community last year at the Summit, there were many investors in the audience. Investors certainly are concerned about the potential returns on their investments when they consider funding a company or a study, but they also want to know there are dedicated people included on the company's leadership team. They notice our level of commitment and say it's astounding.
"AIM, a nonprofit organization, awards grants to investigators at research-based institutions in Alabama."
We've had to be really clever in developing a stable funding mechanism for our nonprofit organization. Our spinoff companies were created from necessity. Both Sacred Cells Research Partners and VivoStem have intellectual property (IP), and WellPace Medical has unique products and a dedicated staff, so we are on investors' radar. We're a young startup, so we have the time to prove ourselves to the industry as we expand operations for each of these companies.
TLSR: Let's talk more about these companies, all of which are spinoffs of AIM. Can you share the prospects for each?
TW: VivoStem, a for-profit company, is headquartered here in Birmingham, with an operation in Fremont, California, where Roman Reed lives. It will conduct stem cell research in collaboration with researchers globally. An incredible group of researchers are committed to various studies on spinal cord injury, cardiovascular disease and gene expression. These researchers have opened their arms to us, shared their advice and wisdom, and offered opportunities to collaborate on projects by combining our IP with their IP to create new drugs for regenerative medicine therapy.
We have especially strong relationships with researchers in California. They include Dr. Keirstead, who is president of NeoStem Oncology (NeoStem Inc. [NBS:NASDAQ]), and Dr. Jane Lebkowski, president of research and development at Asterias Biotherapeutics (AST:NYSE.MKT). Dr. Lebkowski, an advisor to our nonprofit organization, is a stem cell expert and has submitted numerous investigational new drug (IND) applications to the FDA, and received approvals as well. This knowledge and expertise of the clinical process is invaluable for our young organization. At the Gladstone Institutes, we work closely with Dr. Deepak Srivastava, the director, and the research lab of Dr. Shinya Yamanaka, who won the 2012 Nobel Prize in Physiology or Medicine for his discovery that adult stem cells can be reprogrammed to become pluripotent. We also have connections with researchers from the Harvard Stem Cell Institute, directed by Dr. Brock Reeve. He has advised us on many endeavors.
Roman and I launched Sacred Cells Research Partners in 2013. We have worked for more than two years to secure patented license agreements and worldwide exclusive rights to progenitor cell therapy.
Our first study, to build tissue for spinal cord injury research, is scheduled to begin in May. Dr. Aileen Anderson at UCI and Dr. Raj Singh, CEO of Vivo Biosciences Inc. (private) are co-principal investigators. The project uses Dr. Singh's HuBiogel platform technology, which is a matrix created from discarded human amniotic tissue that enables normal or diseased human cells to grow outside the body while functioning much like they do in vivo. Once we begin the study, topline results are expected in six to eight months.
Through Sacred Cells Research, we also would like to collaborate with other companies to advance treatment for diabetes and organ regeneration. Sacred Cells holds four global patents that have significant clinical value to this stem cell line. We've worked hard for two years to secure the necessary licenses, so it's exciting for us to be able now to put the study in motion.
TLSR: Tell me about WellPace Medical.
TW: WellPace Medical is our marketing and distribution company. It is headquartered in Birmingham and operated by Ron Williams, my husband, based upon his relationships with sales managers and sales representatives established during his 25 years with the medical device and equipment manufacturer Stryker Corporation (SYK:NYSE). WellPace will help place our products with physician groups across the U.S. and around the world.
Right now, Ron and the WellPace team are exhibiting at medical conferences and talking with physicians to build new relationships while maintaining longstanding relationships.
TLSR: Regenerative medicine finally is at the point of providing cures. There are about 60 therapies approved by the FDA. What reaction have you had from the biotech community since Inevitable Collision was published?
TW: We've had a tremendous response from the biotech community. We've been invited to speak at multiple conferences. Those opportunities helped us network with other not-for-profit organizations, as well as for-profits, in the biotech arena. People seem interested in learning from our experiences, and also in helping us improve and better position ourselves to penetrate various markets through our spinoff companies.
After Geron halted the clinical trial TJ participated in (for patients with complete cervical spinal cord injury), I noticed many people were discouraged and frustrated because the company halted the trial after so much time and money had been put into it. It had a negative effect on the biotech community.
"Embryonic stem cells are the gold standard simply because they can be used for anything."
However, in March 2014, hope returned to our community as Asterias announced that it was resuming the spinal cord injury clinical trial, and expanding it from 13 to 40 patients. This Phase 1/2a trial evaluates three escalating dosages, so we should see great results. People are excited again, not only for spinal cord injury research, but also for the whole field of stem cell research.
TLSR: What happened to the stem cell patients in the initial Geron trial?
TW: The five patients treated by Geron each received 2M cells. That's roughly 10% of what Geron believed was necessary to help them regain function. The trial was a safety study. It showed no adverse effects, even five years after the initial injection, and is considered a success.
TLSR: In the decade since embryonic stem cells have been studied, alternatives have emerged, including IPS cells. With all of these advances, is there still a strong case for using embryonic stem cells?
TW: Which stem cell type is best continues to be the most debated question in research labs around the world! Embryonic stem cells are the gold standard simply because they can be used for anything. They can be programmed to become any cell type in the human body—bone, skin, brain, etc. Embryonic stem cells are programmed by nature to build tissue, and even organs.
IPS stem cells, in contrast, lack this diversity. And, while research involving IPS cells shows promising data, it takes 10 to 12 years to develop a good stem cell line using this cell type. Then, the manufacturing costs are astounding. Therefore, researchers shy away from using IPS cells.
Manufacturing embryonic stem cells is relatively easy. They can be developed in abundance without the complexity involved in creating other cell types. Statistics from the National Institutes of Health (NIH) suggest that more than 500K embryos are discarded as medical waste every year. So we have an abundance of embryonic stem cells that can be harvested for this potentially lifesaving research.
TLSR: Regarding the NIH figures on embryo waste, do you mean blastocysts?
TW: Exactly. The early union of sperm and egg truly is a blastocyst—an undifferentiated cell. When people hear the word "embryo," they think "baby." When we are out talking to people, this is the first fact I try to mention. People need to understand the harvesting process and where these cells come from. Once people understand that these cells come from abandoned embryos at fertility clinics, and they are not being created specifically for research, it's almost an epiphany.
TLSR: Nearly every state has jumped on the biotech bandwagon, but it takes a life science infrastructure to support a biotech industry. Many states lack that. Alabama has researchers at UAB, and some spinoffs have occurred. What more is the community doing to help these companies grow and remain in Alabama?
TW: We are conducting an aggressive campaign for stem cell research here in Alabama, and I wish I had an army of people to help.
Our spinoffs are generating jobs, so we are demonstrating an economic impact. Job creation is what our legislators and constituents want to see. The state health advisor is supportive of our efforts and hopeful that our work will continue. Succeeding is, more or less, just a matter of being available, transparent and positive. Staying positive, even though it's a struggle.
TLSR: In the afterword of your book, Roman mentioned helping pass a research law in Australia. Are there plans for a federal research law in the U.S.?
TW: Once Roman and I accomplish our near-term goals for AIM and its spinoff companies, then certainly we would hope to be in position to launch a more national effort, and to partner with others who have that same goal.
TLSR: Thank you, Tory.
Tory Williams is a passionate stem cell advocate, author and cofounder of the Alabama Institute of Medicine (AIM), a nonprofit dedicated to funding regenerative research in the state of Alabama. She is also vice president and managing director of Sacred Cells Research Partners, a private company formed in 2013 to develop innovative treatments for chronic disease and injury through breakthrough cellular technologies and medical devices. Her nonfiction book Inevitable Collision puts a spotlight on the growing conversation surrounding stem cell research and regenerative medicine, and its implications for finding cures to debilitating conditions such as paralysis, diabetes, cancer, ALS and Parkinson's disease.
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