Conference provides critical connections between clinical projects and investors

Having a mission like CIRM’s, which calls on us to develop therapies for unmet medical needs, clearly means we cannot sit back and marvel at all the great projects we have in the pipeline. We have to deliver commercial products available to all patients in need. And that cannot be done without additional investors.

The Alliance for Regenerative Medicine (ARM) takes that maxim seriously as well. The international advocacy organization, of which CIRM was a founding member five years ago, will host its third annual RegenMed Investor Day in New York City next Wednesday March 25.
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During the full-day event 32 companies will present their progress to a wide array of investors. Traditional venture capital investors will be represented alongside investors from institutions and multinational pharmaceutical giants.

The day will be rounded out with three panel discussions and two fireside chats with market research analysts, company CEOs and leading clinicians. The fireside chat during lunch will feature CIRM President and CEO Dr. C. Randall Mills who will talk about public-private partnerships making joint investments to bring therapies to patients, and how the revised work plan we call CIRM 2.0 will make it easier for companies to work together with CIRM to advance promising therapies.

Getting just the eleven projects CIRM is funding in clinical trials today through to commercial products will require a broad mix of funding partnerships. With our portfolio and that of the industry as a whole growing rapidly, conferences like this one are critical.

What…exactly…do you do? How 12 year olds helped me learn how to talk about science

Jackie Ward in her lab at UC San Diego

Jackie Ward in her lab at UC San Diego

Jackie Ward is a graduate student at the University of California, San Diego (UCSD), and received a training grant from CIRM while studying for her PhD. At UCSD Jackie uses stem cells as a model to study rare neurodegenerative diseases in the lab of Albert La Spada. Her work as a PhD student focuses on a rare form of inherited neurodegeneration called spinocerebellar ataxia. From time to time Jackie shares her experiences with us. Here’s her latest.

One of the many questions I get over my annual trek home during the holidays is “What…exactly…do you do?” This is usually couched somewhere between “have you learned to surf yet?” and “how’s the weather?” In the past, I preferred to talk about my surfing skills (very minimal) and the sunshine (always amazing, thanks San Diego), more than what I do every day. It’s amazing how this seemingly innocuous question can be the most difficult to answer. Because we’re used to presenting our work in lecture formats or lengthy scientific papers, summing it up in three sentences of non-jargon can be difficult. A similar thought was outlined recently at UCSD, by the actor and science advocate Alan Alda. The title of his presentation, “Getting the Public Past a Blind Date with Science,” highlighted the uncomfortable feelings many people have towards science. Like any relationship, sustained communication and trust is necessary for success. Unfortunately, on many scientific issues, that relationship has suffered. As a PhD student, I am constantly surrounded by my peers—other scientists who know exactly what I mean when I use terms like “reprogramming” or “retinal photoreceptor.” While these scientist-to-scientist conversations are vital to our work, we often forget that it is equally, or perhaps more, important to have conversations with people who have no idea what we do. As any CIRM- or NIH-funded lab is well aware, a significant portion of our funding comes from taxpayer dollars. It’s these “investors” to whom we ultimately report back. This conversation is challenging. Not only do we have to change our language, we have to remember what it was like to not know everything we do now. The best practice I’ve gotten in this regard is talking to kids. Seventh graders seem to be less afraid to ask you questions or call you out on something that doesn’t make sense to them. (Now that I think about it, it might be beneficial to include some 13-year-olds on our grant review panels.) My graduate program allows students to fulfill their teaching requirement by doing science outreach activities. I chose to do this with the Salk Institute’s mobile science lab, where real scientists are connected to local middle schools to discuss their jobs and lead hands-on science labs. I didn’t realize how valuable this experience was until it started to become easier for me to answer the “what do you do” question. I changed the words I use. I replaced the word “reprogram” with “rewind” and “retinal photoreceptor” with “eye cell.” Unexpectedly, I think this practice helped me become a better communicator when I talk to other scientists now too. I try not to assume a certain level of knowledge with anybody. While I still love talking about pretending to surf and gloating about the weather, I’ve become more fond of the “what do you do” question. I hope to only improve with time. It’ll be my small contribution for getting science to that second date.

Strong ARMing regenerative medicine; bold thoughts on a bright future

It’s a time-honored tradition for the President of the United States to begin his State of the Union speech by saying “The state of our union is strong.” Well, Ed Lanphier, the incoming Chairman of the Alliance for Regenerative Medicine (ARM) – the industry trade group – took a leaf out of that book in kicking off the annual “State of the Industry Briefing” in San Francisco yesterday. He said the state of the industry is not just strong, but getting stronger all the time.

ARM_State_of_the_Industry_Briefing_2015_And he had the facts to back him up. In monetary terms alone he said the regenerative medicine field raised $6.3 billion in 2014, compared to $2.3 billion in 2013.

He pointed to the growing number of partnerships and alliances between big pharmaceutical businesses and smaller biotech and cell therapy companies as a sign that deep pocket investors recognize the potential in the field, saying “Big Pharma sees the value of these outcomes and the maturation of these pipelines.”

Lanphier also highlighted the more than 375 clinical trials that were underway last year, and the fact that more than 60 regenerative medicine products have been approved.

But he also pointed out that the field as a whole faces some big challenges in the coming years. One of the most pressing could be pricing. He cited criticisms that exploded over medicines like Gilead’s hepatitis C treatment Sovaldi because of its $1,000-a-day price tag. Lanphier warned that regenerative medicine could face similar criticisms when some of its therapies are finally approved, because they are likely to be very expensive (at least to start with). He said we need to start thinking now how to talk to patients and the public in general about this, so they understand why these treatments are so expensive, but may be cheaper in the long run if they cure rather than just treat disease.

As if to reinforce that message the first panel discussion in the briefing focused on the gene therapy and genome-editing field. Panel members talked about the high expectations for this field in the 1990’s but that it took decades of work before we finally started to see those early hopes turn into reality.

Jeffrey Walsh, the COO of bluebird bio talked about: “The excitement about gene therapy in the early days… and then having to survive the 15-20 years after that in the very challenging days for gene therapy.”

Katrine Bosley, the CEO of Editas Medicine, says those challenges have not gone away and that the field will have to address some big issues in the future. Among those are working with regulatory agencies such as the Food and Drug Administration (FDA) to win approval for completely new ways of treating disease. Another is anticipating the kinds of ethical issues they will have to address in using these techniques to alter genes.

Questions about the regulatory process also popped up in the second panel, which focused more on advanced therapy and drug development. Paul Laikind of ViaCyte (whose clinical trial in type 1 diabetes we are funding) highlighted those challenges saying: “Making the cells the way you want is not rocket science; but doing it in a way that meets regulatory requirements is rocket science.”

Paul Wotton, the President and CEO of Ocata Therapeutics (formerly called ACT) echoed those sentiments:

“We are pioneering things here and it’s the pioneers who often end up with arrows in their back, so you really have to spend a lot of time working with the FDA and other regulatory bodies to make sure you are having all the right conversations ahead of time.”

But while everyone freely acknowledged there are challenging times ahead, the mood was still very positive, perhaps best summed up by C. Randal Mills, the President of CEO of CIRM and moderator of the panel, when he said:

“I find it remarkable where we are in this space today – with this number of cutting edge companies in clinical trials. Stem cell therapy is becoming a reality, it’s no longer a place where only a foolish few dare to go in; it’s a reality. There is a change in the practice of medicine that is coming and we are all fortunate to be a part of it.”

Stem Cell Stories that Caught Your Eye: The Most Popular Stem Cellar Stories of 2014

2014 marked an extraordinary year for regenerative medicine and for CIRM. We welcomed a new president, several of our research programs have moved into clinical trials—and our goal of accelerating treatments for patients in need is within our grasp.

As we look back we’d like to revisit The Stem Cellar’s ten most popular stories of 2014. We hope you enjoyed reading them as much as we did reporting them. And from all of us here at the Stem Cell Agency we wish you a Happy Holidays and New Year.

10. UCSD Team Launches CIRM-Funded Trial to Test Safety of New Leukemia Drug

9. Creating a Genetic Model for Autism, with a Little Help from the Tooth Fairy

8. A Tumor’s Trojan Horse: CIRM Researchers Build Nanoparticles to Infiltrate Hard-to-Reach Tumors

7. CIRM funded therapy for type 1 diabetes gets FDA approval for clinical trial

6. New Videos: Living with Crohn’s Disease and Working Towards a Stem Cell Therapy

5. Creativity Program Students Reach New Heights with Stem Cell-Themed Rendition of “Let it Go”

4. Scientists Reach Yet Another Milestone towards Treating Type 1 Diabetes

3. Meet the Stem Cell Agency President C. Randal Mills

2. Truth or Consequences: how to spot a liar and what to do once you catch them

1. UCLA team cures infants of often-fatal “bubble baby” disease by inserting gene in their stem cells; sickle cell disease is next target

December ICOC Board Meeting to Begin Soon

The December ICOC Board Meeting begins this morning in Berkeley, CA.

The complete agenda can be found here. Dude to inclement weather our Spotlight on Disease has been canceled.

For those not able to attend, you are welcome to dial in:

To join the event as an attendee
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[Members of the Public will be invited to provide testimony before or during consideration of each item. Makers of public comments are asked to limit their testimony to three (3) minutes.]

Stem cells and professional sports: a call for more science and less speculation

In the world of professional sports, teams invest tens of millions of dollars in players. Those players are under intense pressure to show a return on that investment for the team, and that means playing as hard as possible for as long as possible. So it’s no surprise that players facing serious injuries will often turn to any treatment that might get them back in the game.

image courtesy Scientific American

image courtesy Scientific American

A new study published last week in 2014 World Stem Cell Report (we blogged about it here) highlighted how far some players will go to keep playing, saying at least 12 NFL players have undergone unproven stem cell treatments in the last five years. A session at the recent World Stem Cell Summit in San Antonio, Texas showed that football is not unique, that this is a trend in all professional sports.

Dr. Shane Shapiro, an orthopedic surgeon at the Mayo Clinic, says it was an article in the New York Times in 2009 about two of the NFL players named in the World Stem Cell Report that led him to becoming interested in stem cells. The article focused on two members of the Pittsburgh Steelers team who were able to overcome injuries and play in the Super Bowl after undergoing stem cell treatment, although there was no direct evidence the stem cells caused the improvement.

“The next day, the day after the article appeared, I had multiple patients in my office with copies of the New York Times asking if I could perform the same procedure on them.”

Dr. Shapiro had experienced what has since become one of the driving factors behind many people seeking stem cell therapies, even ones that are unproven; the media reports high profile athletes getting a treatment that seems to work leading many non-athletes to want the same.

“This is not just about high profile athletes it’s also about older patients, weekend warriors and all those with degenerative joint disease, which affects around 50 million Americans. Currently for a lot of these degenerative conditions we don’t have many good non- surgical options, basically physical therapy, gentle pain relievers or steroid injections. That’s it. We have to get somewhere where we have options to slow down this trend, to slow down the progression of these injuries and problems.”

Shapiro says one of the most popular stem cell-based approaches in sports medicine today is the use of plasma rich platelets or PRP. The idea behind it makes sense, at least in theory. Blood contains platelets that contain growth factors that have been shown to help tissue heal. So injecting a patient’s platelets into the injury site might speed recovery and, because it’s the patient’s own platelets, the treatment probably won’t cause any immune response or prove to be harmful.

That’s the theory. The problem is few well-designed clinical trials have been done to see if that’s actually the case. Shapiro talked about one relatively small, non-randomized study that used PRP and in a 14-month follow-up found that 83% of patients reported feeling satisfied with their pain relief. However, 84% of this group did not have any visible improved appearance on ultrasound.

He is now in the process of carrying out a clinical trial, approved by the Food and Drug Administration (FDA), using bone marrow aspirate concentrate (BMAC) cells harvested from the patient’s own bone marrow. Because those cells secrete growth factors such as cytokines and chemokines they hope they may have anti-inflammatory and regenerative properties. The cells will be injected into 25 patients, all of whom have arthritic knees. They hope to have results next year.

Dr. Paul Saenz is a sports medicine specialist and the team physician for the San Antonio Spurs, the current National Basketball Association champions. He says that sports teams are frequently criticized for allowing players to undergo unproven stem cell treatments but he says it’s unrealistic to expect teams to do clinical studies to see if these therapies work, that’s not their area of expertise. But he also says team physicians are very careful in what they are willing to try.

“As fervent as we are to help bring an athlete back to form, we are equally fervent in our desire not to harm a $10 million athlete. Sports physicians are very conservative and for them stem cells are never the first thing they try, they are options when other approaches have failed.”

Saenz said while there are not enough double blind, randomized controlled clinical trials he has seen many individual cases, anecdotal evidence, where the use of stem cells has made a big difference. He talked about one basketball player, a 13-year NBA veteran, who was experiencing pain and mobility problems with his knee. He put the player on a biologic regimen and performed a PRP procedure on the knee.

“What we saw over the next few years was decreased pain, and a dramatic decrease in his reliance on non-steroidal anti inflammatory drugs. We saw improved MRI findings, improved athletic performance with more time on court, more baskets and more rebounds.”

But Saenz acknowledges that for the field to advance anecdotal stories like this are not enough, well-designed clinical trials are needed. He says right now there is too much guesswork in treatments, that there is not even any agreement on best practices or standardized treatment protocols.

Dr. Shapiro says for too long the use of stem cells in sports medicine has been the realm of individual physicians or medical groups. That has to change:

“If we are ever to move forward on this it has to be opened up to the scientific community, we have to do the work, do the studies, complete the analysis, open it up to our peers, report it in a reputable journal. If we want to treat the 50 million Americans who need this kind of therapy we need to go through the FDA approval process. We can’t just continue to treat the one patient a month who can afford to pay for all this themselves. “

World Stem Cell Summit: The environment stem cells find themselves in after transplant really matters

On Friday’s closing day of the 2014 World Stem Cell Summit a panel of three researchers working on neurodegenerative diseases drove home the importance of paying attention to the environment that surrounds stem cells after transplant.

world-stem-cells-summit-2014

CIRM grantee Evan Snyder from the Sanford-Burnham Institute noted that most of the neurologic diseases people are looking at are conditions associated with aging and the cellular makeup of the brain changes as we get older, adding that most of the diseases result from chronic states that have existed over many years. He contrasted this against mouse models of the disease, which usually involve artificially recreating the disease and treating shortly after the injury happens.

“In stem cell therapies there is a dialogue between the transplanted cells and the recipient. The host influences the fate of the stem cells.”

He noted that the patients we will be treating have generally had long-term degeneration and asked if we might be able to develop drugs that effect the environment where the stem cells will be placed so that it mimics more closely the environment found in the animal model in the acute phase, that is right after injury.

One aspect of the environment in the brain in most patients with neurodegeneration is chronic inflammation. Another CIRM grantee on the panel, Jeanne Loring of the Scripps Research Institute, discussed a project her team hopes will take advantage of the inflammation that occurs in Alzheimer’s disease. They are loading nerve stem cells with an enzyme that can degrade the plaque that accumulates in nerves in the disease. Because stem cells home to inflammation, they hypothesize that the stem cells will be drawn to deliver their cargo to the nerves with the worst plaque.

The third panelist, Erzi Kokovay of the University of Texas Health Science Center in San Antonio, described the changes in the brain as we age in a bit more detail. She described infiltration of cells called microglia that researchers will need to take into account when they plan to transplant stem cells in the brain.

While on the surface this all may sound like another road block to getting to the stem cell cures we all want, the presentation actually made me optimistic that we are starting to learn enough about the field that we are more likely to get it right when we start to treat some of these devastating brain diseases.

Don Gibbons

Stem Cell Stories that Caught our Eye: Stem Cell Summit Roundup, Spinal Cords in a Dish and Stem Cell Tourism in the NFL

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

Success at the World Stem Cell Summit. This week some of the biggest names in regenerative medicine descended upon San Antonio, Texas for the annual summit. Along with researchers from the world’s top universities, institutions and companies were members of CIRM, including CIRM President and CEO C. Randall Mills.

We’ve been publishing top highlights from the Summit all week here on the Stem Cellar. There’s also been detailed coverage in the local San Antonio press, including the local ABC station. And if you’d like to find out more about this year’s conference, be sure to visit @WSCSummit and #WSC14 on Twitter.

Scientists have found a way to grow spinal cords from embryonic stem cells in a petri dish. [Credit: Abigail Tucker/ MRC Centre for Developmental Neurobiology/ Wellcome Images.]

Scientists have found a way to grow spinal cords from embryonic stem cells in a petri dish. [Credit: Abigail Tucker/ MRC Centre for Developmental Neurobiology/ Wellcome Images.]

Growing Spinal Cords in the Lab. Tissue engineering, the process of using stem cells to build new tissues and organs, has been the Holy Grail for regenerative medicine. And while there has been some progress with engineering some organs, others—especially the spinal cord—have proven far more difficult. This is because the biodegradable scaffolding cannot be made correctly to grow complex and intricately connected nerve cells.

But now, a research team in Germany has grown complete spinal cords in the lab, pointing to a new strategy for treating those with irreparable spinal cord injuries.

As reported in The Guardian this week, Andrea Meinhardt of the Dresden University of Technology and her colleagues worked around the problem of scaffolding by employing a new method called self-directed morphogenesis, first developed by the late Yoshiki Sasai. According to The Guardian‘s Mo Costandi:

“Self-directed morphogenesis is a method for growing embryonic stem cells in a three-dimensional suspension. Cells grown in this way can, when fed the right combination of signaling molecules, go through the motions of development and organize themselves to form complex tissues such as eyes, glands and bits of brain.”

While preliminary, this research offers immense promise towards the ultimate goal: reversing the devastating effects of spinal cord injuries.

Stem Cells and the NFL. Despite the best efforts of experts, stem cell tourism continues to proliferate. A new study published this week in 2014 World Stem Cell Report (a special supplement to Stem Cells and Development) describes the latest example of people seeking unproven stem cell treatments: this time in the NFL.

New research from Rice University is suggesting that some NFL players are seeking out unproven stem cell treatments—oftentimes traveling abroad without fully understanding the risks. This poses serious problems not only for players but also for the NFL as a whole. As Co-lead author Kirsten Matthews elaborated in a news release:

“With the rise of new and unproven stem cell treatments, the NFL faces a daunting task of trying to better understand and regulate the use of these therapies in order to protect the health of its players.”

Specifically, 12 NFL players are known to have received unproven treatments at some point during the last five years, including star quarterback Peyton Manning who we’ve blogged about before The authors caution that high-profile players broadcasting that they are receiving these unproven therapies could influence regular patients who are also desperate for cures.

In order to fix this growing problem, the authors recommend the NFL review and investigate these unproven stem cell treatments with the help of an independent committee of medical professionals. Finally, they suggest that the NFL could support stem cell research here in the United States—so that proven, effective stem cell-based treatments could more quickly enter the clinic.