Fujifilm is Expanding Its Focus to Regenerative Medicine

Fujifilm began as a photography company, but today is a well-known multinational imaging and information technology corporation. More recently, it’s expanded its focus (pun intended) on developing innovative technologies in the healthcare and regenerative medicine space.

The news that Fujifilm was expanding into regenerative medicine was surprising to some given the company’s expertise in areas unrelated to stem cell research, but with the acquisition of Cellular Dynamics International, a company from Madison, Wisconsin that specializes in large-scale manufacturing of human cells, and the revamping of Fujifilm’s Japan Tissue Engineering subsidiary, which is developing regenerative treatments for damaged skin and cartilage, Fujifilm has solidified its position as a competitive company that’s accelerating the pace of regenerative medicine to develop treatments for patients with unmet medical needs.

Mr. Ban

Mr. Toshikazu Ban

So what progress has Fujifilm made in regenerative medicine and what advancements are they making towards the clinic? You’ll find the answers to these burning questions in my interview with Mr. Toshikazu Ban, Corporate Vice President, General Manager of Regenerative Medicine Business Division at Fujifilm Corporation. Enjoy!

Q: Why did Fujifilm decide to enter the regenerative medicine space?

TB: At first glance, Fujifilm may seem an unlikely candidate to become a leader in regenerative medicine, yet its engagement in the healthcare industry goes back many decades. Founded in 1934, Fujifilm started offering X-ray film just two years later. By 1983, Fujifilm became the first in the world to offer a digital X-ray diagnostic imaging system.

Today, Fujifilm has been able to expand the use of its core fundamental technologies in cosmetics and supplements and pharmaceuticals. Combined, these have allowed Fujifilm to transform into a major healthcare company committed to prevention, diagnosis and treatment.

Unfortunately, there are still many diseases for which there are no effective treatments, and millions wait in hope of their discovery. Regenerative medicine treatment has the potential to cure diseases that cannot be cured by drugs. Fujifilm feels a sense of responsibility to apply its technology in a way that helps make promising treatments a reality.

Q: What advantages do you think Fujifilm has over other healthcare companies in regenerative medicine?

TB: Fujifilm’s advanced engineering technology provides tremendous possibilities in the regenerative medicine space.

The chief component in photographic film is gelatin, which is derived from collagen. Fujifilm has developed a human-type recombinant peptide which can be scaffolds for growing cells and restoring tissue.  The human-type recombinant peptide is non-animal based, has high cellular adhesiveness, is flexible, safe, biocompatible, biodegradable and bioabsorbable. Cells survive better when they are combined with our recombinant peptide because it holds the cells better and allows space in between so that oxygen and other critical growth factors can reach the cells.

Fujifilm also has two subsidiaries that provide synergies and efficiencies to be more competitive in the regenerative medicine field, Cellular Dynamics International, Inc., (FCDI), and Japan Tissue Engineering Co., Ltd. (J-TEC).

In 2015, FCDI announced the launch of a stem cell bank with funding from CIRM to create induced pluripotent stem (iPS) cell lines for each of 3,000 healthy and diseased volunteer donors across 11 common diseases and disorders to be made available through the CIRM human pluripotent stem cell (hPSC) Repository.

The lines available from the CIRM stem cell bank directly complement FCDI’s ability to provide differentiated cells corresponding to each of the iPSC lines, which will allow researchers to model the diseases represented, better understand disease progression, perform more targeted drug discovery, and ultimately lead to better treatments.

A lot of pharmaceutical companies use these cells to test for the screening and toxicity of new drug candidates. If iPS cells can improve the productivity including efficacy and safety, the technology can greatly reduce time and cost as well as the drop-out rate in clinical development.

In 2014, J-TEC became a consolidated Fujifilm Group subsidiary. J-TEC launched the first two regenerative medicine products to receive approval from the Japanese government (one product is used to treat severe burns, while the other is used to replace damaged cartilage in knees).

J-TEC Lab (Image courtesy of Fujifilm)

J-TEC Lab (Image courtesy of Fujifilm)

Q: Can you describe some of the stem cell therapies you’re developing for the clinic for major diseases?

TB: FCDI plans to start iPS cell therapy clinical studies in the U.S. for age related macular degeneration in the year 2017, and clinical studies for retinitis pigmentosa, Parkinson’s and heart failure around 2019.

In March 2015, Fujifilm announced it had developed diabetes therapies in animal tests. CellSaic is a three-dimensional mosaic structure that combines cells with a recombinant peptide (RCP) scaffold made from micro-sized petaloid pieces of the protein. In a study involving type 1 diabetic mice, we created a CellSaic of human mesenchymal stem cells and cells from pancreatic islets and transplanted them in the mice. The purpose of the study was to verify whether using the recombinant peptide as a scaffold would increase the survival rate of the transplanted cells compared with just transplanting the cells alone. We also wanted to demonstrate a reduction in blood glucose levels of the diabetic mice since the recombinant peptide was able to sustain the viability of the pancreatic islet cells.

The study showed that seven days after the transplantation, CellSaic had a significantly more prominent introduction of blood vessels, which provide passageways for nutrients, oxygen and waste product to get to, and away from, the cells.  In addition, 28 days after transplantation, the test group of diabetic mice with the recombinant peptide-based CellSaic scaffold saw blood glucose levels lowered to the level equivalent to that of the healthy mice. In contrast, the diabetic mice who received pancreatic islets alone showed no change in blood glucose levels. 

Q: When you move into clinical trials, do you anticipate US trial sites in parallel with those in Japan?

TB: FCDI plans to start clinical trials of iPS cell treatments in the US. J-TEC conducts clinical trials for autologous cultured corneal epithelium and plans to start clinical trials for allogeneic cultured dermis in Japan. Currently we plan to conduct these clinical trials where these companies are located. We may expand the clinical trials of the products to other countries in the future.

Q: Can you speak to Japan’s regulatory system for stem cell therapies and how this could give Fujifilm a leg up on developing stem cell treatments more rapidly?

TB: The go-to market conditions for regenerative medicine in Japan have become more favorable since the November 2014 implementation of the Pharmaceutical and Medical Device Law, which has significantly cut the time it takes to gain marketing approval in Japan and created more interest in this sector.

Within regenerative medicine, academic institutions have shown remarkable progress. The mission of the industry is to apply findings from academia to patients and deliver high-quality treatments at a reasonable cost.

Note: Technologies that pertain to Japan Tissue Engineering Co., Ltd. (J-TEC) are not approved for use in the US.

You can learn more about Fujifilm’s latest efforts to “make regenerative medicine a reality” by visiting its Innovation website.

Women in Bio on The Influential Paths of Great Visionary Leaders

Powerful women made powerful statements last week at the Women in Bio (WIB) Plenary Event during the 2016 BIO International Convention. A panel of influential women leaders discussed difficult yet critical topics, such as how to brand yourself as a woman in a male-dominated industry, the importance of side hustles, and how to close the gender gap. It was a dynamic and inspiring event that engaged both men and women in the audience in productive conversation about how we can all work together to support women in the life sciences industry.

The panel was moderated by Nicole Fisher, the Founder and CEO of HHR Strategies and Forbes Contributer, and the speakers included Renee Compton Ryan, VP of Venture Investments at Johnson & Johnson and Frances Colón, Deputy Science and Technology Adviser to Secretary of State John Kerry.

Frances Colon, Renee Ryan, Nicole Fisher.

Frances Colon, Renee Ryan, Nicole Fisher.

The panel was more of a fire-side chat with the three woman talking intimately at a small coffee table, first sharing stories about their career paths and the road blocks along the way, and then delving into the controversial topics that women in the life sciences face.

Career Paths of Influential Women

Nicole told her story about how she got into the healthcare space. She started by ghostwriting about healthcare, innovation, and politics for the Congressional Budget Office director. Her passion turned into an opportunity with Forbes where she now runs the Health Innovation and Policy page and eventually into her company HHR Strategies which focuses on healthcare and human rights.

Renee discussed how she started as an investment banker in healthcare and made an investment in a company that benefitted patients. This experience made her want to be a part of the solution for patients, which she described as “a calling we are all fortunate to have,” and ultimately brought her to her current position at J&J.

After completing a Ph.D. in developmental neurobiology, Frances switched gears and found her strengths and assets in science policy and communications. She wanted to bring science into international affairs and shared that her mission now is to “make science cool to political scientists and diplomats to the point where my job becomes irrelevant.”

Other Panel Highlights

Branding

Renee’s advice on branding was, “challenge yourself to know your brand, and revisit your brand”. Everyone builds a resume chronologically, but she forces herself to revisit her resume every two years. Her trick is to flip the resume over to the blank side and list all her skills but do it through a different lens so you can have perspective. This process helps her decide where she wants to grow and learn.

Having Side Hustles

Frances mentioned the importance of having “side hustles”. These are things that you are really passionate about that will also build on your strengths, raise your visibility and help you take your brand to the next level. She mentioned two side hustles in particular, a non-profit she founded that supports the Puerto Rican Diaspora Network and a group she organized called the Science Technology Table, which brings together government and the private sector to discuss trending topics in science, tech and innovation. Nicole chimed in and said that all three of her side hustles have turned into companies or big opportunities that have significantly advanced her career.

Closing the Gender Gap, No More Manels!

The panelists had much to say about closing the gender gap. Renee encouraged women in high-up positions to mentor other women that show promise and to be a hands-on mentor. She also said that everyone in the biotech and pharma industries should be studying the data to see why there are less women in the life sciences and what can be done about it.

Frances said that the gender policies at companies need to change, and that people at companies have to hold each other accountable and have the conversations that can create change. One of her key points that got a laugh from the crowd was getting rid of “manels”, or all men panels, which are prevalent at major conferences in the biotech and healthcare space. She also spoke about how we need to strive for 50/50 representation on boards and executive management.

What the audience had to say

The panel was a hit with the Women in Bio audience. Dr. Leah Makley, a WIB member and Founder and CSO of ViewPoint Therapeutics, had this to say about the event,

Leah Makley

Leah Makley

“The panelists shared candid wisdom from their own career trajectories, passions, and ‘side hustles’ that far surpassed the typical depth of career panels.  Moreover, I thought Nicole Fisher did an exceptional job of framing the conversation and asking provocative questions.”

She also spoke about the importance of the WIB community and the resources they offer:

“WIB is a supportive community of powerful, inspiring women. Both the members and the events tend to be action- and solution-oriented, and I’ve walked away from each event I’ve attended with new insights, perspectives, and energy. I’m so grateful that this resource exists.”

Marco Chacon

Marco Chacon

A moment that really stood out in my mind was a moving speech by Marco Chacon, Founder of Paragon Bioservices, and a WIB sponsor. Marco shared that he recently attended a meeting in Boston and listened in on a few diversity forums. He was appalled to hear the statistics on gender diversity in the executive suite and boards of directors in biotech and pharma. Passionately he said, “This has got to change, and to the degree that I can affect this in some way, I can assure you I will do so.”

Final Thoughts

Influential leaders like Nicole, Renee, Frances, and Marco and organizations like Women in Bio, are laying the groundwork for the career advancement of women in science. This event was a great reminder that the issues facing women in the life sciences industry can be addressed in the immediate future if we continue the conversation and challenge one another to create change.

BIO 2016: IMAGINE Curing Disease and Saving Lives Part 2

As promised, here is Part 2 of our blog coverage on the BIO International Convention currently ongoing in San Francisco. Here are a few more insights on the talks we attended and highlights of other coverage from top biotech journalists and media outlets.

Keynote with Dr. Bennet Omalu and Will Smith on “Concussion”

If you haven’t seen the movie Concussion, add it to your watch list right now. It’s certainly at the top of mine after listening to Nigerian-American doctor Bennet Omalu share his story about how he single-handedly changed the way the National Football League (NFL) and the world views concussions and brain science.

Will Smith and Dr. Bennet Omalu at #BIO2016

Will Smith and Dr. Bennet Omalu at #BIO2016

In this keynote address, Dr. Omalu sat down with actor Will Smith, who portrays Dr. Omalu in the movie, to discuss how knowledge and truth precipitates evolution. Because of his passion for seeking the truth, Omalu’s autopsy of former NFL player Mike Webster led to the first diagnosis of chronic traumatic encephalopathy (CTE). Omalu’s main message was that faith and science go hand in hand. “Faith searches for truth and science searches for truth. There is no end to truth.” He also emphasized that while the truth can be inconvenient, it’s worth pursuing because truth is empowering.

For Will Smith, portraying Dr. Omalu in Concussion, was both an honor and a duty. As a parent of a son who plays football, he was compelled to tell this story and share this knowledge with parents around the world. Smith was so motivated to take on Omalu’s character that he even watched Omalu conduct four autopsies so he could really understand both the man and the science behind CTE.

This dynamic conversation was the highlight of BIO, and you can read more details about it in this article by Eleena Korban of BIOtechNOW. 

Fireside chat with US FDA Commissioner Robert Califf

Robert Califf and Steve Usdin

Robert Califf and Steve Usdin

Robert Califf, the Commissioner of the US Food and Drug Administration, sat down with Steve Usdin, the Senior Editor with BioCentury, to discuss the most important topics facing the FDA right now. Here are some of his main points:

  • FDA will focus more on patient engagement. Califf said that patients should be involved from the beginning and not just be the recipients of the end product. He also touched on risk tolerance for patients and that it can vary based on disease. The FDA wants to engage patients, advocacy groups, and industry on this topic so that patients can make more educated decisions about their treatment options.
  • The cost of clinical trials is going up 3-4 times the consumer price index which is not sustainable. Califf suggested that we can use integrated health systems and already available data from electronic medical records and patient registries to reduce the costs of large clinical trials. He commented, “The question is, can you create a different playing field that would radically reduce the cost of clinical trials while actually getting us better data about what people really care about and solve their problems related to the use of our products. I think we are close to that point now.”
  • Califf mentioned the FDA’s role in President Obama’s Precision Medicine Initiative as a step towards radically accelerating the rate of drug development. The FDA is partnering with the NIH to create a cloud-based workspace where genetic information on disease can be stored, shared, and studied.
  • Lastly, Califf mentioned how the FDA is creating a virtual center of excellence for cancer research as part of the Cancer Moonshot Initiative. He said that the FDA needs to do a better job of collaborating across its different product centers and that drug devices and biologics will be brought together starting first in the oncology space, and then eventually rolled out to other disease areas. On the clinical side, they will focus on patient involvement and the needs of cancer patients.

More coverage on the FDA fireside chat from BIOtechNOW

 Final Thoughts

While BIO ends today, the partnerships, conversations, and innovation certainly will not. In just four short days, the vibrant and eager atmosphere of BIO has transformed this year’s theme of Imagination into one of hopeful reality. Curing disease and saving lives might not be in the immediate future, but after what I’ve seen at BIO, I’m confident that the groundwork has been laid out to accelerate us down this path.


Other #BIO2016 coverage

IMAGINE Curing Disease and Saving Lives: BIO 2016 Part 1

Did you hear that? It’s the sound of more than 15,000 people taking a collective breath. That’s because we are now at the halfway point of the 2016 BIO International Convention, the world’s largest biotechnology gathering with over 900 speakers, 180 company presentations, 19 education tracks, 6 super sessions, and 35,000 partnering meetings. Now that’s a lot of stuff!

While many at BIO are focused on partnering – establishing new and exciting relationships with other biotech and pharmaceutical companies to push their products forward – others come to BIO to learn about the latest in research, innovation, and healthcare in the biotechnology space.

With so much going on at once, it’s hard to choose where to spend your time. If you follow BIO on twitter using the hashtag #BIO2016, you’ll get a condensed version of the who, what, and how of BIO.

For those of you who are more partial to blogs, here’s a brief recap of the talks that we’ve attended so far:

Mitochondrial Disease Education Session

A panel of scientific experts and patient advocates gave an overview of mitochondrial diseases and the latest research efforts to develop therapies for mitochondrial disease patients. Phil Yeske of the United Mitochondrial Disease Foundation described his foundation as the largest funder of mitochondrial research next to the government. Their focus is on patient-centered therapeutic development and they’ve established a community registry of patients that makes patients the central stewards for research and clinical development.

The most moving part of this session was an impromptu speech by Liz Kennerley, a mitochondrial disease patient and advocate. She bravely spoke about the roller coaster of symptoms affecting all of the organs in her body and aptly described her daily experience by quoting Forest Gump, “Life is like a box of chocolates, you never know what you’re gonna get.” She ended with the powerful statement that patients are at the core of everything scientists do, and encouraged the panel to engage patients more often because they will tell you everything if you ask them the right questions.

Mitochondrial Disease Patient Liz Kennerley.

Mitochondrial Disease Patient Liz Kennerley speaks at BIO 2016.

Moving out of Stealth Mode: Biotech journalists offer real-world advice on working with media to tell your story

One of my favorite panels of the conference so far featured three biotech journalists, Christina Farr of Fast Company, Jeff Cranmer of BioCentury, and Alex Lash of Xconomy. It was a dynamic conversation about how biotech companies coming out of stealth mode can best pitch their story to the media. Take home points include:

  • When pitching to a journalist, make sure that you are honest about what you can and can’t say. Have a “BS committee” that can address the validity of your work and your research claims.
  • When pitching, journalists want to know what the problem is you’re trying to solve and how you are trying to solve it better than anyone else.
  • On press releases: Unless it’s a press release from a big name, journalists won’t read it. The panel said they would prefer a personalized email detailing a company’s background and stage of work. They would also consider reading a press release that included a short personalized email from the company CEO.
  • Most hated words used to describe research: “Revolutionary” “Game-changing” “Disruptive”.

    Biotech journalist panel with.

    Moderator Carin Canale-Theakston with biotech journalists Jeff Cranmer, Alex Lash, and Christina Farr

Fireside Chat with University of California President Janet Napolitano

In an intimate Fireside chat, Janet Napolitano described her passion for higher education and making a difference in students’ lives. In her new role as the President of the UC system, her main focus is on aligning the policies and initiatives between the UC campuses and promoting research and innovation that can be commercialized around the world.

When asked about how she values basic research compared to applied research, Napolitano responded,

UC President Janet Napolitano

UC President Janet Napolitano

“We want an atmosphere where basic research is supported and one where innovation and entrepreneurship is fostered through incubators and public/private partnerships. We need to make these a tangible reality.”

 

Napolitano also mentioned that the UC system needs support from the private sector and gave PrimeUC – a collaboration with Johnson & Johnson Innovation that is part of her innovation and entrepreneurship initiative – as an example of a step in the right direction. You can read more about PrimeUC in this Event Recap.

From Ebola to Zika, how can we go faster in a global emergency?

I was only able to sit in on part of this expert panel, but here is the gist of their conversation. The global number of human infectious diseases is rapidly increasing every year due to hard-to-control factors like overpopulation, deforestation, and global climate change.  As a result, we’ve had two global health emergencies in the past two years: Ebola and Zika. We were more prepared to deal with the Ebola epidemic because more treatments were already in development. Unfortunately, we weren’t as prepared for Zika as it wasn’t on the world’s radar as a serious disease until 2015.

Martin Friede of the World Health Organization (WHO) said we should take what we learned from the recent Ebola outbreak and apply it to the Zika threat. He said the WHO wants to plan ahead for future outbreaks and remove bottlenecks to health benefits. They want to predict what diseases might surface in the future and have products ready for approval by the time those diseases manifest.


That’s all for now, but be sure to read Part 2 of our BIO2016 coverage tomorrow on the Stem Cellar. We will give highlights from an entertaining and fascinating Keynote address with Dr. Bennet Omalu (the doctor who blew the whistle on concussion in the NFL) and Oscar-nominated actor Will Smith (who played Dr. Omalu in the movie “Concussion”) on “Knowledge precipitates Evolution”. I’ll also tell you about an eye-opening Fireside chat with the US Food and Drug Administration Commissioner Robert Califf, and much more!

Get your BIO on: Sneak Peak of the June 2016 BIO Convention in SF

Screen Shot 2016-06-01 at 8.43.36 AM

Summer is almost here and for scientists around the world, that means it’s time to flock to one of the world’s biggest biotech meetings, the BIO International Convention.

This year, BIO is hosted in the lovely city of San Francisco. From June 6-9th, over 15,000 biotechnology and pharma leaders, as well as other professionals, academics, and patients will congregate to learn, educate, and network.

There’s something for everyone at this convention. If you check out the BIO agenda, you’ll find a plethora of talks, events, education sessions, and fire side chats on almost any topic related to science and biotechnology that you can imagine. The hard part will be deciding what to attend in only four short days.

For those going to BIO this year, make sure to check out the myBIO event planning tool that’s free for attendees and allows you to browse events and create a personalized agenda. You can also set up a professional profile that will share your background and networking interests with others at BIO. With this nifty tool, you can search for scientists, companies, and speakers you might want to connect with during the convention. Think of all the potential networking opportunities right at your fingertips!

Will Smith (source)

Will Smith (source)

For those who can’t make it to BIO, don’t worry, we have you covered. CIRM will be at the convention blogging and live tweeting. Because our mission is to bring stem cell treatments to patients with unmet medical needs, the majority of our coverage will be on talks and sessions related to regenerative medicine and patient advocacy. However, there are definitely some sessions outside these areas that we won’t want to miss such as the Tuesday Keynote talk by Dr. Bennet Omalu – who helped reveal the extent of brain damage in the NFL – and actor Will Smith – who plays Dr. Omalu in the movie ‘Concussion’. Their join talk is called “Knowledge Precipitates Evolution.”

Here’s a sneak peak of some of the other talks and events that we think will be especially interesting:


Monday June 6th

Education Sessions on Brain Health and Mitochondrial Disease

Moving Out of Stealth Mode: Biotech Journalists Offer Real-World Advice on Working with Media to Tell Your Story

“In this interactive panel discussion, well-known biotech reporters from print and online outlets will share their insights on how to successfully work with the media. Session attendees will learn critical needs of the media from what makes a story newsworthy to how to “pitch” a reporter to strategies for translating complicated science into a story for a broad audience.”

The Bioethics of Drug Development: You Decide

A discussion of the critical bioethical issues innovative manufacturers face in today’s healthcare ecosystem. Panelists will provide insights from a diverse set of perspectives, including investors, the patient advocacy community, bioethicists and federal regulators.”


Tuesday June 7th

Fireside Chat with Robert Califf, Commissioner of the US Food and Drug Administration (FDA)

Fireside Chat with Janet Napolitano, President of the University of California

Casting a Wider Net in Alzheimer’s Research: The Diversity of Today’s Approaches and Signs of Progress

Hear clinical researchers, biotech CEOs, and patient advocates explain how the field is pivoting from the failures of past approaches to make use of the latest generation of beta-amyloid research results as well as pursue alternative therapeutic angles to improve brain health.”

From Ebola to Zika: How Can We Go Faster in a Global Emergency?

This interactive panel of public health and industry leaders will discuss what has been learned through our global response to Ebola and what is and is not applicable to Zika or other pathogens of pandemic potential.”


Wednesday June 8th

Curative Therapies: Aligning Policy with Science to Ensure Patient Access

“The promise of curative treatments creates an urgent need to ensure access for patients, promote an environment conducive to developing new treatments, and manage the concentration of healthcare expenses in a sustainable manner.  A diverse set of panelists will tackle the tough questions around curative therapies and discern what changes are necessary for our health care delivery system to meet the challenges they pose.”

An Evolving Paradigm: Advancing the Science of Patient Input in the Drug Development and Regulatory Processes

This panel will explore advances in the field of assessing patient views and perspectives, and highlight how the patient voice is being incorporated into development programs and informing FDA review and approval decisions.”

A Media Perspective

“Any press is good press or so they say. You want your story known at the right time and in the right light, but how do you get industry journalist to notice you? What peaks their interest and how do they go about story discovery? What will they be looking to write about in the next 3 to 12 months? Three top journalists will discuss their approaches to keeping current and what makes a story newsworthy.”
Patient Advocacy Meetup

Over 40 patient advocacy organizations will be discussing their latest partnerships and developments in the areas of advancing disease research and drug development.


Thursday June 9th

Novel Advances in Cancer R&D: Meeting the Needs of the Patient

This panel will feature the views of patients and advocates, regulators, and companies who are working to change the way in which we diagnose and evaluate patients with cancer by better understanding the underlying biology of their disease.”


 To follow our coverage of BIO, visit our Stem Cellar Blog or follow us on Twitter at @CIRMNews.

If you want to accelerate stem cell therapies then create an Accelerating Center

Buckle up

Buckle up, we’re about to Accelerate

“You can’t teach fish to fly,” is one of the phrases that our CIRM President & CEO, Randy Mills, likes to throw out when asked why we needed to create new centers to help researchers move their most promising therapies out of the lab and into clinical trials.

His point is that many researchers are terrific at research but not so great at the form filling and other process-oriented skills needed to get approval from the Food and Drug Administration (FDA) for a clinical trial.

So instead of asking them to learn how to do all those things, why don’t we, CIRM, create a system that will do it for them? And that’s where we came up with the idea for the Accelerating Center (we’re also creating a Translating Center – that’s a topic for a future blog but if you can’t wait to find out the juicy details you can find them here.)

The Accelerating Center will be a clinical research organization that provides regulatory, operational and other support services to researchers and companies hoping to get their stem cell therapies into a clinical trial. The goal is to match the scientific skills of researchers with the regulatory and procedural skills of the Accelerating Center to move these projects through the review process as quickly as possible.

But it doesn’t end there. Once a project has been given the green light by the FDA, the Accelerating Center will help with actually setting up and running their clinical trial, and helping them with data management to ensure they get high quality data from the trial. Again these skills are essential to run a good clinical trial but things researchers may not have learned about when getting a PhD.

We just issued what we call an RFA (Request for Applications)  for people interested in partnering with us to help create the Accelerating Center. To kick-start the process we are awarding up to $15 million for five years to create the Center, which will be based in California.

To begin with, the Accelerating Center will focus on supporting CIRM-funded stem cell projects. But the goal is to eventually extend that support to other stem cell programs.

Now, to be honest, there’s an element of self-interest in all this. We have a goal under our new Strategic Plan of funding 50 new clinical trials over the next five years. Right now, getting a stem cell-related project approved is a slow and challenging process. We think the Accelerating Center is one tool to help us change that and give the most promising projects the support they need to get out of the lab and into people.

There’s a lot more we want to do to help speed up the approval process as well, including working with the FDA to create a new, streamlined regulatory process, one that is faster and easier to navigate. But that may take some time. So in the meantime, the Accelerating Center will help “fish” to do what they do best, swim, and we’ll take care of the flying for them.

 

 

 

New Stem Cell Treatment for ALS May Slow Disease Progression

Exciting news was published this week that will give patients suffering from ALS, also known as Lou Gehrig’s disease, something to cheer about. The journal JAMA Neurology reported that a new stem cell treatment was successful in slowing disease progression in a small group of ALS patients in a Phase 2 clinical trial.

This is big news for a fatal, incurable disease that is well known for its progressive, degenerating effects on nerve cells in the brain and spinal cord. We’ve written about ALS a lot in the Stem Cellar, so if you want more background on the disease, read our “Progress to a Cure for ALS” blog.

A patient’s own stem cells can help

The stem cell therapy involves extracting mesenchymal stem cells from the bone marrow of ALS patients. These stem cells are then manipulated in culture into cells that secrete a growth factor called NeuroTrophic Factor (NTF), which helps keep nerve cells in the brain and spinal cord healthy and alive. The NTF-secreting stem cells (called NurOwn cells) are then transplanted back into the same ALS patient (making this an autologous stem cell therapy) by injection into either the spinal fluid or the muscles.

logoThe NurOwn method was developed by BrainStorm Cell Therapeutics, a biotech company based in the US and Israel. Clinical trials to test the safety and efficacy of NurOwn stem cells began in 2011 at the Hadassah Medical Organization (HMO). So far, 26 patients have participated in the trials both in the US and in Israel.

According to the JAMA publication, patients were monitored 3 months before and 6 months after they received stem cell transplants and 6 months after. Twelve of the 26 patients participated in an early stage of the trial (phase 1/2) to test the safety and tolerability of the stem cell therapy. The other 14 patients participated in a later stage (phase 2a), dose-escalating study where their modified stem cells were injected into both their spinal fluid and muscles. Following the treatment, the scientists looked at the safety profile of the transplanted stem cells and for signs of clinical improvement in patients such as their ease of breathing or ability to control their muscle movement.

Stem cell treatment is effective in most ALS patients

Results from the clinical trial showed that a majority of the patients benefitted from the NurOwn stem cell therapy. HMO Principle scientist and senior author on the study, Dr. Dimitrios Karussis, explained:

Dr. Dimitrios Karussis (Image credit: Israel21c)

Dimitrios Karussis (Israel21c)

“The results are very encouraging.  Close to 90% of patients who were injected intrathecally through the spinal cord fluid were regarded as responders to the treatment either in terms of their respiratory function or their motor disability.  Almost all of the patients injected in this way showed less progression and some even improved in their respiratory functions or their motor functions.”

A PRNewswire press release covering this study called the stem cell therapy the “first-of-its-kind treatment for treating neurodegenerative diseases.”

Not a cure just yet

This stem cell therapy will need to be tested in more patients before the it can be determined truly effective in slowing progression of ALS. And Dr. Karussis was quick to note that the NurOwn stem cell therapy isn’t a cure for ALS, but rather an early-stage therapy that will provide significant benefit to patients by slowing disease progression.

“I am optimistic that within the foreseeable future, we may provide a treatment to ALS patients that can slow down or stop the progression. I believe we are in the early stages of something new and revolutionary with this harvested stem cell infusion therapy.  While this is absolutely by no means a cure, it is the first step in a long process in that direction.  I see this treatment as being potentially one of the major future tools to treat degenerative diseases of the brain and spinal cord, in general.”

Other stem cell treatments for ALS in the works

A single stem cell therapy that could treat multiple neurodegenerative diseases would be extremely valuable to patients and doctors. However, it’s not clear that the “one ring to rule them all” scenario (couldn’t help making a Lord of the Rings reference) will play out well for all diseases that affect the brain and spinal cord. Luckily, Dr. Karussis and Brainstem Cell Therapeutics are not the only ones pursuing stem cell therapies for ALS.

Clive Svendsen has been on a 15-year quest to develop an ALS therapy

Clive Svendsen

CIRM is currently funding 21 studies (a total of $56.6 million) that use stem cells to either study ALS or to develop therapies to treat the disease. We wrote about one recent study by Clive Svendsen at Cedars Sinai which is using a combination of gene therapy and brain stem cells to deliver growth factors to protect nerve cells in the brain and spinal cord of ALS patients. Currently, Svendsen and his team are in the latter stages of research and hope to apply for FDA approval to test their therapy in patients in the near future. Svendsen told CIRM, “we will begin recruiting patients the first week we have approval.”


Related Links:

What Went Down at ARM’s Regenerative Medicine State of the Industry

Every January, downtown San Francisco is taken over by a flock of investors, bankers, biotech companies, and scientists attending the annual JP Morgan Healthcare Conference. This meeting looks at the healthcare advancements over the past year and predicts the disease areas and technologies that will see the most progress and success in 2016.

According to some of the experts at the event, regenerative medicine and stem cell research are experiencing impressive, accelerated advancements, which has peaked the interest of investors, biotech, and pharmaceutical companies.

Because these are such fast paced fields, the Alliance for Regenerative Medicine (ARM) hosts the Annual Regenerative Medicine and Advanced Therapies State of the Industry Briefing during JP Morgan to discuss the recent progress and outlook for the industry in the coming year.

Screen Shot 2016-01-11 at 4.03.30 PM

What happened in 2015 and what’s next?

ARM’s  6th Annual Briefing was open to the public and drew over 300 people on Monday morning. The meeting opened with an industry update from Edward Lanphier, ARM Chairman and President/CEO of Sangamo BioSciences.  Then two panels featuring top leaders from biotech and pharmaceutical companies discussed the 2016 clinical data forecast and the promise of regenerative medicine and advanced therapies in oncology (cancer).

With an upbeat attitude, Lanphier gave an overview of clinical development progress in 2015, with 20 approved products worldwide and over 600 clinical trials both from academia and industry. More than 40% of these ongoing clinical trials are in cancer while approximately 12% are in heart disease/injury. These trials are not limited to Phase 1 either. In 2015, there were 376 in Phase 2 (compared to 200 in 2014) and 64 in Phase 3 (compared to 39 in 2014).

Edward Lanphier

Edward Lanphier

Two other areas Lanphier emphasized were CAR-T and other cell-based immunotherapies and gene therapy programs for rare diseases. He ended with 2015 statistics on clinical milestones in various disease and therapy programs, key company IPOs, the financial landscape, and predictions of major anticipated data from clinical trials in 2016.

It was a lot to take in, but this was definitely a good thing and a sign that the areas of regenerative medicine and advanced therapies are thriving. If you want more details, you can check out ARM’s State of the Industry presentation.

Major Theme: Data is King

The major theme that cropped up during the industry update and panel discussions was the importance of producing meaningful clinical data to get positive outcomes in regenerative medicine.

This was succinctly put by panelist Sven Kili, head of Gene Therapy Development at GlaxoSmithKline:

“I would say “Data is King”. A great idea is fantastic, passion is wonderful, and most companies will buy into a strong management team, but that only gets you so far. After that you need to have data, and you need to have a good plan for going forward.”

Kill added that there’s the need to work with the FDA to change the regulatory process, saying the FDA is, understandably, cautious about working with therapies that can alter a person’s genome permanently. However, he said there needs to be serious discussions with the FDA about how to speed up the process, to make it easier for the most promising projects to get approval.

Edward Lanphier also talked about the industry’s new focus on clinical data and the questions that arise when trying to advance regenerative medicine research into approved treatments and cures for patients:

“How do we communicate the value of curing blindness? How do we think about pricing that? What do we think about [drug] reimbursement?  For rare diseases, we aren’t trying to talk about acute treatments – we are talking about one-time, curative outcomes. And the value and benefit to patients in this is enormous. This is what we are trying to do, and on the cusp of, in terms of generating both approvable data and also the proof of concept data that then allows us to drive that next value inflection point in terms of financings.”

The Future Looks Good

After listening to the briefing, the future of regenerative medicine and advanced therapies certainly looks bright. As Jason Kolbert, head of Healthcare Research at the Maxim Group, said:

“This industry is now rapidly maturing and regenerative medicine and gene therapy have great things in store for the next decade.”

Usman Azam, Global Head of Cell and Gene Therapies at Novartis, had a similar outlook:

“We now are going from proof of concept to commercial availability of a disruptive innovation within seven years. If somebody had said that to me four years ago, I would have said, not possible. But that gives you a sense of how quickly this field is moving.”

Experts Panel

ARM Panel: 2016 Sector Forecast: Upcoming Clinical Data Events

CIRM-funded clinical trial for spinal cord injury reports promising results

Today, the Menlo Park-based biotech company Asterias Biotherapeutics reported positive results from the first three patients treated in its Phase 1/2a clinical study using stem cell therapy to treat patients with spinal cord injury. This trial is funded by a CIRM Strategic Partnerships Award grant of $14.3 million.

asteriasAsterias has developed a stem cell therapy called AST-OPC1 that uses oligodendrocyte progenitor cells (OPCs), a kind of cell found in the nervous system, to treat patients that have suffered from different types of spinal cord injury. Damage to the spinal cord causes a range of paralysis based on where it occurs. People with spinal cord trauma to the mid-back often retain the use of their hands and arms but can no longer walk and may lose bladder function. Patients with spinal cord injuries in their neck  can be paralyzed completely from their neck down.

astopc1OPCs are precursors to an important cell type in the central nervous system called the oligodendrocyte. These cells are responsible for forming a conductive sheet around nerve cells that allows nerves to send electrical signals and messages safely from one nerve to another. Both OPCs and oligodendrocytes provide support and protection to nerves in the spinal cord and brain, and they can also facilitate repair of damaged nerves by secreting survival and growth factors as well as promoting the formation of new blood vessels.

In this first part of the Phase 1/2a clinical trial three patients with complete cervical (neck) spinal cord injuries were given a “low dose” of two million AST-OPC1 cells to test the safety and feasibility of their stem cell treatment. The first patient was treated at the Shepard Center in Atlanta,  and at the two month post-injection assessment, the patient experienced no side effects and an improvement from a complete to an incomplete injury on the ASIA impairment injury scale. The other two patients received injections at the Rush University Medical Center in Chicago. Both procedures were reported to have gone smoothly, and the patients are still being monitored.

Asterias plans to treat a second group of patients with higher doses of AST-OPC1 cells (10-20 millions cells). Chief Medical Officer Dr. Edward Wirth explained their strategy:

 The safety data in the first cohort now paves the way for testing the higher doses of AST-OPC1 (10-20 million cells) that we believe correspond most closely to the doses that showed the greatest efficacy in animal studies.

If both the low dose and high dose groups report no serious side effects, Asterias will turn to the Food and Drug Administration (FDA) for approval to expand the patient population of this clinical trial phase from 13 patients up to 40. Asterias hopes that adding more patients “will increase the statistical confidence of the safety and efficacy readouts, reduce the risks of the AST-OPC1 program and position the product for potential accelerated regulatory approvals.”

Spinal cord injury affects more than 12,000 people every year. It remains a major unmet medical need without any FDA-approved therapies or medical devices that improve or restore patient spinal cord function. CIRM is hopeful that Asterias will continue to see positive results with the SCiStar trial and will be able to progress its AST-OPC1 program into late-stage clinical trials and eventually into an FDA-approved stem cell therapy for spinal cord injury.


Related links

Global stem cell market predicted to reach $40 billion in five years, even bigger when mixed with new technologies

The global consulting firm Frost and Sullivan held a webinar yesterday in which they noted health care systems everywhere are facing an increasing challenge of costly chronic care. They suggested health care providers have started to embrace regenerative medicine as a viable alternative.

Because of its power to change the course of disease, the consultants called regenerative medicine, and stem cell therapies in particular, a new paradigm in human health.

“Regenerative Medicine initiatives are now attracting new public and private funding,” said the firm’s Jane Andrews in a widely picked up press release, including this post at CNBC. “Although Stem Cell Therapy will continue to be the largest market segment of Regenerative Medicine, cross segment therapies that combine the use of immunology, genetic and stem cell therapy are rapidly advancing,”

CIRM funds projects in all these technologies so it is always nice to see others joining the fight. We recently posted a series of stories about our portfolio of clinical trials that combine cell therapy and gene therapy.

The report predicts the global stem cell therapy market will reach $40 billion in five years by 2020. It also suggests that just the US market will reach $180 billion by 2030.

The firm does raise a cautionary note about the inadequacy of funding for early stage clinical work with these therapies. Our President and CEO Randall Mills has also raised an alarm about this issue and called on industry to increase its support for this work.

Organized by the Asia-Pacific branch of Frost and Sullivan the webinar breaks out the markets for Japan, Korea and Singapore. The webinar itself is available on line.