‘Right To Try’ laws called ‘Right To Beg’ by Stem Cell Advocates

In recent years, ‘Right to Try’ laws have spread rapidly across the US, getting approved in 32 states, with at least three more states trying to pass their own versions.

The organization behind the laws says they serve a simple purpose:

‘Right To Try’ allows terminally ill Americans to try medicines that have passed Phase 1 of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves. ‘Right To Try’ expands access to potentially life-saving treatments years before patients would normally be able to access them.”

That certainly sounds like a worthy goal; one most people could get behind. And that’s what is happening. Most ‘Right To Try’ laws are passed with almost unanimous bi-partisan support at the state level.

Beth Roxland

Beth Roxland

But that’s not the view of Beth Roxland, an attorney and health policy advisor with an extensive history in both regenerative medicine and bioethics. At the recent World Stem Cell Summit Roxland said ‘Right To Try’ laws are deceptive:

“These are not patient friendly but are actually patient unfriendly and could do harm to patients. The problem is that they are pretending to do something that isn’t being done. It gives patients a sense that they can get access to a treatment, but they don’t have the rights they think they do. This is a right to ask, not a right to get.”

Roxland says the bills in all 32 states are almost all identical, and use the same cookie-cutter language from the Goldwater Institute – the libertarian organization that is promoting these laws. And she says these laws have one major flaw:

“There is no actual right provided in the bill. The only right is the right to try and save your life, “by requesting” from a manufacturer a chance to try the therapy. The manufacturer doesn’t have to do anything; they aren’t obliged to comply. The bills don’t help; they give people false hopes.”

Roxland says there isn’t one substantiated case where a pharmaceutical company has provided access to a therapy solely because of a ‘Right To Try’ law.

However, Starlee Coleman, the Vice President for Communications at the Goldwater Institute, says that’s not true. She says Dr. Ebrahim Delpassand, a cancer specialist in Texas, has testified before Congress that he has treated dozens of patients under his state’s ‘Right To Try’ law. You can see a video of Dr. Delpassand here.

Coleman says ;

“We think the promise of ‘Right To Try’ is self-evident. If one doctor alone can treat 80 patients in one fell swoop, but the FDA can only manage to get 1200 people through its expanded access program each year, we think the potential to help patients is significant.”

Other speakers at the panel presentation at the World Stem Cell Summit said these laws can at the very least play an important role in at least raising the issue of the need for people battling terminal illnesses to have access to experimental therapies. Roxland agreed it was important to have that conversation but she pointed out that what often gets lost in the conversation is that these laws can have hidden costs.

  • 13 states may withdraw hospice eligibility to people who gain access to an early or experimental intervention
  • 4 states may withdraw home care
  • 6 states say patients taking part in these therapies may lose their insurance
  • Several states allow insurers to deny coverage for conditions that may arise from patients getting access to these therapies
  • 30 states say the companies can charge the patients for access to these therapies

Roxland says the motives behind the ‘Right To Try’ laws may be worthy but the effect is misleading, and diverts attention from efforts to create the kind of reforms that would have real benefits for patients.

Here is a blog we wrote on the same topic last year.

Why Goldilocks could provide the answer to changing the way FDA regulates stem cells

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Panel on FDA regulation at World Stem Cell Summit

One of the hottest topics of the past year in regenerative medicine has been the discussion about the need for regulatory reform at the Food and Drug Administration (FDA) so it’s no surprise that topic was the subject of the first main panel discussion at the 2016 World Stem Cell Summit in West Palm Beach, Florida.

The panel, titled ‘FDA Oversight in Regenerative Medicine: What are the Options to Accelerating Translation’, kicked off with Celia Witten, Deputy Director of the Center for Biologics Evaluation and Research at the FDA. She laid out all the new steps that the agency is implementing to try and be more responsive to the needs of researchers and patients.

Perils facing pioneers

Martin McGlynn, the former CEO of StemCells Inc. was up next and he wasted little time listing the companies that had once been considered pioneers in the field only to fail for a variety of reasons. He said one of the big problems is that translational efforts, moving from a good idea to a clinical trial, take too long, saying 15 – 20 years is not unusual and that Big Pharma and strategic investors won’t invest until they see strong Phase 2 study results.

“We need to do great science and design and conduct great clinical trials to advance this field but we also have to come up with a sustainable business model to make this happen.”

A good start

He called the 21st Century Cures Act, which the US Senate approved yesterday, a good start but says many of the challenges won’t be helped by some of the new provisions:

“Many sponsors and companies don’t make it out of open label early studies, so the existence of an accelerated pathway or some of the other enabling tools included in the act will come too late for these groups.”

McGlynn warned that if we don’t take further steps, we risk falling behind the rest of the world where companies are buying up struggling US ventures:

“Many non-USA companies in Japan and China and Australia are quicker to recognize the value of many of the products and approaches that struggle here in the US.”

Too much, too little, just right

Marc Scheineson was the final speaker. He heads the food and drug law practice at Washington, DC law firm Alston & Bird and is a former Associate Commissioner for Legislative Affairs at the FDA. He began his presentation with what he said are the scariest words in the English language: “I‘m a lawyer from Washington D.C. and I’m here to help you.”

Scheineson says part of the problem is that the FDA was created long before cell therapy was possible and so it is struggling to fit its more traditional drug approval framework around stem cell therapies. As a result, this has led to completely separate regulatory processes for the transplantation of human organs and blood vessels, or for the use of whole blood or blood components.

He says it’s like the fable of Goldilocks and the Three Bears. Some of the regulation is too hard- resulting in a lengthy regulatory process that takes years to complete and costs billions of dollars – and some of the regulation is too soft allowing clinics to open up around the US offering unproven therapies. He says we need a Goldilocks approach that blends the two into regulations that are just right.

Time to take a second step

Scheineson agreed with McGlynn that the 21st Century Cures Act is a good start but it’s not enough.  He says it still relies heavily on the use of traditional criteria to regulate stem cells, and also leaves much of the interpretation of the Act to the discretion of the FDA.

“It’s a first step, an experiment to see if we can break the logjam and see if we can move things to an affordable BLA (The Biologics License Application is needed to be able to market a product once it’s approved by the FDA). But make no mistake, a cell therapy revolution is underway and I believe the FDA should seize the opportunity to promote innovation and not defensively protect the “status quo”.

 

 

The Critical Role of Patient Advocates in Accelerating Stem Cell Cures

At CIRM, our goal is to bring stem cell therapies to patients with unmet medical needs, and we do that by funding the most promising and innovative research in regenerative medicine. A critical component of this goal is to support our patient advocates and make sure that their voices are heard.

At this year’s World Stem Cell Summit, patient advocates from around the world, representing a breadth of diseases and disorders, came together to share their stories, goals, and needs with the larger scientific community.

One session that particularly stood out, was “Accelerating Cures: The Critical Role of Patient Advocates” on Day 3 of the conference. This panel featured key leaders in patient advocacy:

  • Don Reed, the “Grandfather of Stem Cell Research Advocacy”, Vice President of Public Policy at the Americans for Cures Foundation
  • Frances Saldaña, an advocate for Huntington’s disease (HD) and founder of HD-Care at UC Irvine, which is a support group to advance HD research and clinical care
  • Tory Williams, the Executive Director of the Alabama Institute of Medicine (AIM) which raises funds and awareness for stem cell treatments and cures of disease and injury and the author of “Inevitable Collision

The panel was moderated by our fearless leader and head of communications, Kevin McCormack. Each speaker shared their story about how they became a patient advocate and what they are currently doing to push the pace of stem cell research.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed described the heartbreaking story of his son Roman Reed, who suffered a severe spinal cord injury while playing football. Through Don and Roman’s relentless efforts, “Roman’s Law” was passed in 1999, which raised $17 million in California state funding for spinal cord injury research. Don was also a key instigator for the passage of Proposition 71, which gave $3 billion dollars to our agency to fund stem cell research. He continues to be a passionate advocate for stem cell research and spinal cord injury patients, and recently published a book called “Stem Cell Battles: Proposition 71 and Beyond” which you can read more about in our recent blog.

Next, Frances Saldana told a compelling story of raising a family of three beautiful children with a husband who had Huntington’s disease. Unaware of his condition when they were together, Frances’ world took a devastating turn when he died of HD, leaving her to question whether her children would face the same fate. Sadly, all three of Frances’s kids carried the HD mutation. Having to deal with the passing of her two daughters, and a son who is battling the end stages of this disease, Frances decided to share her experience with others and to create a support organization called HD-Care so that others wouldn’t have to face similar experiences alone. HD-Care is conducting an aggressive campaign to bring visibility to HD and supports cutting-edge research in the field including the work done by CIRM-grantee Dr. Leslie Thompson at UC Irvine.

Frances told the audience that her happiest moment since this all began was when her daughter Margie, already suffering from symptoms of HD, spoke at CIRM in 2007. She saw the Board and the scientists and thought, “somebody cares, and somebody will find a cure.” It was a new chapter for her, she explained, and she knew something good was going to happen.

Lastly, Tory Williams, introduced the Alabama Institute of Medicine, which is a non-profit organization that supports the stem cell community with education and public dialogue. She started the institute following both personal and family experiences with cancer and after TJ Atchinson, a close family friend, suffered a severe spinal cord injury. Along the way, she forged a close relationship with Roman Reed who helped her pass TJ’s law in 2013, which is an Alabama state law that promotes spinal cord injury research.

“The goal [of AIM],” said Williams, “is to make a difference in people’s lives affected by disease and injury by helping to advance medicine to eradicate these debilitating issues.”

Laurel Barchas, Student Society for Stem Cell Research

Laurel Barchas, Student Society for Stem Cell Research

When the session was opened up to questions, the atmosphere in the room turned electric. Patients and scientists stood up to tell their stories and asked hard questions. One question came from Laurel Barchas, one of the founders of the Student Society for Stem Cell Research, who asked how we as a society can advocate for mental illness and similar diseases where the symptoms are not visible and where patients are either embarrassed or hesitant to make their disease public. Another question was how emerging countries like Mexico who don’t have the same benefits and infrastructure as the US can promote and support patient advocacy.

The mood of the advocates was positive but measured. They know that new treatments and cures take time but they also pointed out that many people don’t have much time so we have to work as hard as we can to help them.

The panel ended with the consensus that the voices of patient advocates are invaluable, and that they will be the key to accelerating stem cell therapies into cures. Frances Saldaña urged other patient advocates that the key to progress is to be aggressive, and be unafraid to be out there. Don Reed concluded on a similar note with quote from Shakespeare’s Hamlet:

“Whether ’tis Nobler in the mind to suffer

The Slings and Arrows of outrageous Fortune,

Or to take Arms against a Sea of troubles,

And by opposing end them.”


Related links:

Why “Right to Try” laws are more feel good than do good

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L to R: Don Gibbons, CIRM; Jeanne Loring; Beth Roxland; Aaron Levine

In the last few years some 24 states have approved so-called “Right to Try” laws. These are intended to give terminally ill patients faster and easier access to experimental therapies. But a panel of experts at the World Stem Cell Summit in Atlanta today said they are more symbolic than anything and do little to actually help patients get much-needed therapies.

The Right to Try laws are modeled after a federal law that allows “compassionate use” of experimental medications and lets doctors prescribe investigational medicines being safely used in early stage clinical trials.

Beth Roxland, a bioethicist with Johnson & Johnson, says the name of the law is misleading:

“If you look at the actual text of these laws they only say you have the right to “ask” for these drugs. That right already exists in federal law but neither federal law nor these Right to Try laws say you have the right to access.”

Aaron Levine from Georgia Tech says it’s also misleading to assume that just because a state passes a Right to Try law that it has any legal impact. He says state laws don’t over rule the Food and Drug Administration’s (FDA) regulation of this area and so the federal government would still have the authority to stop this kind of access.

But Levine says these laws are interesting in that they are indicative of the growing determination of patients and patient advocates to work around obstacles to access and have a bigger say in their own care.

One of the audience members, William Decker from Baylor College of Medicine, says that in Texas a law was recently crafted saying that as long as a potential therapy had gone through a Phase 1 safety trial it should be offered to the public and the public should be able to pay for it.

“If you know how clinical trials work you know you can get almost any schlock through a Phase 1 trial and the kinds of things that you can get to the public without any idea if they work often turn out to not be very useful. We saw this as an avenue to promote fraud, and the last thing you should be doing to a dying patient is take their money or divert their attention away from something that might help them.”

Decker and his colleagues argued before the Texas Legislature that potential therapies should at least have to go through a Phase 2 trial to make sure they were not only safe but also showed some benefit for patients. In the end Texas lawmakers rejected the Phase 2 idea but did say patients could not be charged for the therapy, and there could be no compensation from insurers or anyone else for the manufacturer of the therapy.

He says removing the financial benefits and incentives pretty much ensured that no company would offer patients a therapy under this law.

Jeanne Loring, a CIRM grantee from the Scripps Research Institute, says that likely won’t stop other clinics in other states:

“Some stem cell clinics are using adipose (stem cells derived from fat) therapy as an option for every disease imaginable and I’m sure some will take advantage of these laws to say it gives them the right to offer these to patients and the patients will pay for them directly. “

Roxland says that may already be happening:

“I think there is some evidence on the stem cell side that companies have popped up in states that have these laws, to make it easier to offer their therapies to patients.”

The panel agreed that in most cases these laws don’t give patients any rights they don’t already have, but do give the appearance of making access easier. They said it’s feel-good legislation, allowing people to feel they are doing something without actually doing anything.

Aaron Levine said that while some companies may try to take advantage of these laws, the most serious ones won’t:

 “Almost any legitimate company that wants an FDA approved product wouldn’t want to take advantage of these laws. It could put their product at risk. Most companies that need to work with the FDA have no incentive to go this route.”

 

 

The bottom line: stem cell therapies will never be widely available if insurers won’t pay for them

The second session of the World Stem Cell Summit in Atlanta moved past all the promising science and right to the nitty-gritty of making cell-based therapies common. Four panelists reminded the audience that while they too are super excited by the potential for this field, unless folks developing therapies think about reimbursement early those therapies will not become a reality in routine clinical care.

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“Stem cell therapies seem unstoppable with seemingly limitless possibilities, but success requires early planning for reimbursement,” said moderator Michael Levinson, a lawyer and physician with the law firm Hogan Lovells.

Elizabeth Powers of the IMS Consulting group suggested the audience pay close attention to the cancer market.  She said insurers and other payers of health care services are tired of paying for “statistically significant” improvements in survival that only translate to a few weeks on average. She said payers are moving away from just whether a new therapy is different from prior therapies and want to be shown true value.

A further reminder to start the reimbursement process early came from panelist Deborah Dean of MiMedx.  She said the process of just applying for a reimbursement code takes two years and after that it can take months or more to then present your case to insurers to turn that code into actual payments.

During the question period there was a bit of potential good news attached to an industry trend I did not expect. The consolidation of insurers, with two major mergers on deck, could actually extend the average length of time a customer is with an insurer from between two to five years to between five to ten years. This may make insurers more willing to pay for a one-time curative therapy that is expensive but eliminates chronic therapy costs.

Call to Action by FDA at World Stem Cell Summit

Califf

FDA Deputy Commissioner Dr. Robert Califf talking at the World Stem Cell Summit

The World Stem Cell Summit annual conference in Atlanta kicked off today with a clarion call from Dr. Robert Califf, the Deputy Commissioner for the Food and Drug Administration. He told the audience:

“We want you to accelerate translation to produce safe and effective therapies that can be delivered reliably”

It was a message that everyone in the room, scientists and patient advocates, would love to be able to comply with. The question of course is how do you do that in a way that puts the emphasis on both speed, to get the therapies to patients who need them, and safety, so you don’t put those patients at risk.

That’s quite a challenge considering that, as panel moderator Julie Allickson of Wake Forest Institute for Regenerative Medicine said:

“the estimate now is it costs $2.4 billion and up to ten years to take something to the clinic.”

Even if that dollar amount is higher than many think it would take to bring a stem cell therapy to a clinical trial it is an indication of the challenge the field faces.

Califf, who has only been at the FDA for 8 months, says that regenerative medicine is:

“not the only field exploding with scientific knowledge and seeing a future that’s very different from what we see today so it’s exciting but also an enormous challenge for the FDA. One of the real eye openers for me is to be at the FDA and hear about drugs that have been on the market for 45 years and we’re still learning about them.”

He says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. “That’s an issue we struggle with every day.”

Califf was optimistic that the balance can be struck and progress can be made, but said that this can only truly be done if the patient is at the table as an active participant.

“Our national clinical research system is well intention but flawed. We need to have a new system that shares information right across the system and where patients are at the center. Patients should be driving the national research infrastructure. They are an essential part of change. It’s happening in Congress because they are hearing from constituents that this is what they want, a voice in the research being done that affects them.”

For the patients and patient advocates in the audience it was a welcome message. For years they have been calling for a louder voice in the research that affects them and their loved ones. Knowing they have a sympathetic ear in the FDA could be an encouraging sign that their voices are finally being heard.

We will be writing more as the conference unfolds so stay tuned!

 

 

 

A scientific conference we can all enjoy

Scientific conferences are fascinating events. You get a chance to mingle with some of the leading researchers and thinkers in the field, and to learn about the latest advances. But, to be honest, for those of us who don’t have a scientific background, they can also be a little bit intimidating.

This is sometimes how I feel at them.

Courtesy The New Yorker

Courtesy The New Yorker

That’s where the World Stem Cell Summit comes in. It’s an annual event that brings together researchers, companies, scientists and patient advocates to talk about the progress being made in stem cell research and to explore ways to advance the field even further, and faster by working together.

Changing the tone

The patient advocate role is a critical one here. It makes the voice of the patient a key element in every discussion and changes the tone of the event from talking about what is being done to or for patients, to what is being done with patients. It’s a small but tremendously important difference.

Dr. Evan Snyder, Director of the Stem Cells and Regenerative Medicine program at Sanford – Burnham Medical Research Institute captures that feel when he says:

“We’re looking forward to the valuable information-sharing opportunities and discussions that only occur when stem cell researchers, patient advocates, and representatives of many other stakeholder groups converge at the World Stem Cell Summit. Occasions like these help us advance our research on the basic biology of stem cells and spur the development of new, and more personalized, medical applications for this science.”

Because more than ten percent of those attending are patient advocates the talks are given at a level that someone without a science background can generally understand. The presentations are no less fascinating; they are just a lot more accessible.

Stephen Rose, the Chief Research Officer with the Foundation Fighting Blindness says it brings different groups together in a way other conferences usually don’t:

“Policy experts learned about researchers’ needs. Advocates learn about policy and legislation. It also brought ethical issues to the table, which is critical if we’re going to resolve them and keep the research moving forward.”

Researchers have a lot of opportunities throughout the year to meet with other scientists but patient advocates don’t, so the World Stem Cell Summit is a great chance for them to meet with their colleagues and counterparts from all over the US. It gives them a chance to share ideas, offer support and explore ways they can collaborate.

More than just a meeting

For many advocates who are focused on diseases that affect relatively small numbers of people these events are a great way to recharge their batteries and to remind themselves they are not alone in this fight.

If you are thinking about going to one conference this year, this is a great one to chose. This year the World Stem Cell Summit is being held December 10 – 12 in Atlanta, Georgia.

We’ll be there and we’d love to see you there too.

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.