Stem cell and gene therapy research gets a good report card from industry leader

arm

Panel discussion at ARM State of the industry briefing: left to Right Robert Preti, Chair ARM; Jeff Walsh, bluebird bio; Manfred Rudiger, Kiadis Pharma; Barbara Sasu, Pfizer;  Thomas Farrell, Bellicum Pharmaceuticals. Photo courtesy ARM.

The state of the regenerative medicine field is strong and getting stronger. That was the bottom line verdict at the 2017 Cell and Gene Therapies State of the Industry briefing in San Francisco.

The briefing, an annual update on the field presented by the Alliance for Regenerative Medicine (ARM), gave a “by the numbers” look at the field and apart from one negative spot everything is moving in the right direction.

Robert Preti, Chair of ARM’s Board, said worldwide there are more than 750 regenerative companies working in the stem cell and gene therapy space. And those companies are increasingly moving the research out of the lab and into clinical trials in people.

For example, at the end of 2016 there were 802 clinical trials underway. That is a 21 percent growth over 2015. Those breakdown as follows:

Phase 1 – 271 (compared to 192 in 2015)

Phase 2 – 465 (compared to 376 in 2015)

Phase 3 – 66 (compared to 63 in 2015)

The bulk of these clinical trials, 45 percent, are focused on cancer. The second largest target, 11 percent, is on heart disease. The number of trials for neurological disorders and rare diseases are also growing in number.

Preti says the industry is at an important inflection point right now and that this growth is presenting new problems:

“The pipeline of products is robust and the technologies supporting that pipeline is even more robust. The technologies that are fueling the growth in clinical activity have accelerated so fast that we on the manufacturing side are playing catchup. We are at a point where we have to get serious about large scale commercial production.”

Preti also talked about “harmonization” of the regulatory process and the need to have a system that makes it easier for products approved for clinical trials in one country, to get approval for clinical trials in other countries.

Michael Werner, the executive director of ARM, said the organization has played a key role in helping promote the field and cited the recently passed 21st Century Cures Act as “a major win and a powerful statement of ARM’s leadership in this sector.”

But there was one area where the news wasn’t all positive, the ability of companies to raise capital. In 2015 companies raised $11 billion for research. In 2016 it was less than half of that, $5.3 billion.

With that somber note in mind it was appropriate that the panel discussion that followed the briefing was focused on the near-term and long-term challenges facing the field if it was to be commercially successful.

One of the big challenges was the issue of regulatory approval, and here the panel seemed to be more optimistic than in previous years.

Manfred Rüdiger of Kiadis Pharma said he was pleasantly surprised at how easy it was to work with different regulatory agencies in the US, Canada and Europe.

“We used them as a kind of free consultancy service, listening to their advice and making the changes they suggested so that we were able to use the same manufacturing process in Europe and Canada and the US.”

Jeff Walsh of bluebird bio, said the key to having a good working relationship with regulatory agencies like the Food and Drug Administration (FDA) is simple:

“Trust and transparency between you and the regulatory agencies is essential, it’s a critical factor in advancing your work. The agencies respond well when you have that trust. One thing we can’t be is afraid to ask. The agencies will tell you where their line is, but don’t be afraid to ask or to push the boundaries. This is new for everyone, companies and regulators, so if you are pushing it helps create the environment that allows you to work together to develop safe therapies that benefit patients.”

Another big issue was scalability in manufacturing; that it’s one thing to produce enough of a product to carry out a clinical trial but completely different if you are hoping to use that same product to treat millions of people spread out all over the US or the world.

And of course cost is always something that is front and center in people’s minds. How do you develop therapies that are not just safe and effective, but also affordable? How do companies ensure they will get reimbursed by health insurers for the treatments? No one had any simple answer to what are clearly very complex problems. But all recognized the need to start thinking about these now, long before the treatments themselves are even ready.

Walsh ended by saying:

“This is not just about what can you charge but what should you charge. We have a responsibility to engage with the agencies and ultimately the payers that make these decisions, in the same way we engage with regulatory agencies; with a sense of openness, trust and transparency. Too often companies wait too long, too late before turning to the payers and trying to decide what is appropriate to charge.”

 

 

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

img_1077

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”.

 

 

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

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.

Why is a cell therapy that restores sight to the blind against the law?

FDA

A lot of people are frustrated with the US Food and Drug Administration (FDA) and its woefully slow process for approving stem cell therapies. That’s one of the reasons why we started the CIRM Stem Cell Champions campaign, to gather as many like-minded supporters of stem cell research as possible and help to change the way the FDA works, to create a more efficient approval process.

You can read more about that campaign and watch a short video on what being a Stem Cell Champion involves (hint: not very much).

Now Randy Mills, our President and CEO, has teamed up with former US Senator Bill Frist to explain precisely why the FDA needs to change the way it regulates stem cells, and to offer a simple way to create the system that will best serve the needs of patients.

This Op Ed appeared on Fox News’ online Opinion section on Friday, May 20th.


Cell therapy reversed blindness for 47,000 patients in 2015. So why is it against the law?

By C. Randal Mills Ph.D., Sen. Bill Frist M.D.

As medical miracles go, restoring sight to the blind is right up there. A mother seeing her baby for the first time, or a child being able to count the stars is a beautiful gift, and its value cannot be overstated. Last year 47,000 Americans received that gift and had their blindness reversed through the transplantation of cells from a corneal donor’s final selfless act.

It is safe, it is effective, and because it is curative, it is a relatively cost effective procedure. It is medicine at its most beautiful. And according to FDA regulations, the distribution of this cell therapy is in violation of federal law.

That’s right. The regulation says that no matter how competent the surgeon, the FDA must first approve cells from donated corneas as if they were a drug—a process that takes over a decade and can costs billions of dollars — all for a practice that has been successfully restoring sight for more than 50 years. And this is only one example.

The good news: the FDA doesn’t always adhere to its regulations and has not in this case.

The bad news: inconsistent enforcement creates uncertainty, deterring innovation for other unmet medical needs such as arthritis, back pain, and diabetic ulcers.

How did a country known for pioneering medical breakthroughs get here?

Appropriate regulation of living cells that treat disease is inherently complex. Some therapies, like corneal cell transplants, are well-understood. Others are far more sophisticated and can involve forcing cells to change from one type to another, cutting out defective genes, and growing cells in culture to expand their numbers into the billions. Although this may sound like science fiction, it’s the type of very real science that will revolutionize the practice of medicine. And it is a challenging spectrum to regulate.

Unfortunately, what we have today amounts to a regulatory light switch for cell therapy; one that is either OFF or ON. For some cell therapies there is essentially no pre-market regulation. But at some point of added complexity, often arbitrarily decided by the FDA, the switch flips to ON and the cell becomes a drug in the minds of the Agency. And the consequences could not be more profound.

A product can be introduced through the OFF pathway in days with no FDA review and at very little cost. The ON pathway on the other hand, takes 10-20 years and can cost over a billion dollars. For cell therapy, there is no in between.

It is not possible to regulate the continuum of cell therapies fairly and effectively by using this binary approach. The system is broken and is impeding the hunt for safe and effective treatments for suffering patients.

Why? Because sensible people don’t invest significant capital gambling that the FDA will give them a pass out of its rules. They evaluate the time and cost of development assuming they will be forced down the ON pathway. They also assume that this arbitrary approach to regulation will (and often does) work against them by allowing a competitor to enter the market through the OFF pathway, placing them at a prohibitive disadvantage. The results speak for themselves. After 15 years under this paradigm we have had only a few cell therapies approved, all commercial disasters.

This is because the ON-OFF approach fails to adequately account for the difference in cell therapy complexity. To better understand, imagine this methodology applied to the regulation of automobiles. The government might permit low tech cars, say the Model T, to be sold without pre-market regulation. But if a manufacturer wanted to improve the vehicle by adding air conditioning, a radio or other such feature, the car would be subject to massive pre-market regulation. And not just on the new feature. Instead, the addition of the new feature would trigger a bumper-to-bumper evaluation of the entire car, increasing its development cost from basically nothing to that of a Lamborghini. The result would be streets full of hot, radio-less go-karts, except for a few ultra-high-end sports cars whose manufacturers are now defunct because they were never able to recoup the disproportionate costs of satisfying the regulatory system. This is what we see with cell therapies today: progress that is sluggish at best.

How can we move forward?

Ironically, the FDA identified a solution to the problem. In order to account for the broad spectrum inherent to cell therapy, in the late 90’s the FDA proposed a progressive, risk-based approach. The higher the risk, the greater the regulation. This guards against under regulation that might put patients at risk and prevents overregulation that can disincentivize the development of new or improved products.

In the FDA’s own words, the regulation they proposed would abide by a few basic principles:

  • “Under this tiered, risk-based approach, we propose to exert only the type of government regulation necessary to protect the public health.”
  • “The regulation of different types of human cells… will be commensurate with the public health risks…”
  • “These planned improvements will increase the safety of human cells… while encouraging the development of new products.”

It was a remarkably common sense approach that would have balanced safety with the need for innovation over an exceptionally broad range of technological complexity and risk.

It would have.

Unfortunately, the regulatory framework that was promised was never delivered, and it is time to resuscitate it. The burden placed on the development of cell therapies must accurately reflect the risks; must be balanced against the very real consequences of doing nothing (patients continuing to suffer); and must be consistently and fairly applied. In short, the FDA had it right and we need to give them the tools to deliver the regulatory paradigm they originally envisioned.

If we fix this highly fixable problem, we can create a system that will drive new innovations and better outcomes. Europe and Japan have already acted and are seeing the benefits. People with great ideas are coming off the bench, and game changing therapies are entering practice. While challenging the status quo does not sit well with some, particularly those who stand to prosper from the built-in barriers to entry the current structure provides, in the United States we have a responsibility to do better for patients and fix this broken system.

Randal Mills, Ph.D., is the President and CEO of the California Institute for Regenerative Medicine

William “Bill” H. Frist, M.D. is a nationally-acclaimed heart and lung transplant surgeon, former U.S. Senate Majority Leader, and chairman of the Executive Board of the health service private equity firm Cressey & Company.

How do you know what patients want if you never even ask them?

Picture1

Our mission at CIRM is to accelerate stem treatments to patients with unmet medical needs. But what if those needs are not just unmet, they’re also unknown? What happens when those developing treatments never even bother to ask those they are trying to help if this is what they really need, or want?

The question came up during a panel discussion at a meeting of the CIRM Alpha Stem Cell Clinics Network in San Diego earlier this month. David Higgins, a CIRM Board member and a Patient Advocate for Parkinson’s disease, highlighted the problem saying that if you ask most people what they think is the biggest problem for Parkinson’s sufferers, they would probably say the movement disorders such as tremors and muscle rigidity. But David said that if you ask people who have Parkinson’s what their biggest problems are, then movement disorder probably wouldn’t even come in the top five concerns that they really have.

David listed insomnia, severe fatigue, anxiety, and depression as far more pressing and important:

“Researchers study what they know and they look to solve the things they think they can solve, and it is sometimes very different than the things that patients would like them to solve to ease their concerns.”

That sparked a fascinating discussion about the gap between what researchers and scientists sometimes think they should be doing, the kinds of treatments they should be trying to develop, and what the people who have those conditions really want.

David Parry, who is with GlaxoSmithKline and worked in drug development and discovery for most of his career, said:

“If I told you how many times I sat in meetings with my medical discovery group and talked about what our targets should be then we’d be here all night. We focus on what we know, what we think we can fix and what will work, when maybe we need to be more mindful of what could really make a difference in the life of patients.”

Alpha clinic panelAlpha Stem Cell Clinics Network panel discussion: Left David Higgins, David Parry, Catriona Jamieson, John Zaia, John Adams

Clearly there is a gap between what we think we can fix and what we should try and fix, and the best way to close that gap is to have a conversation.

Patients and Patient Advocates need to speak up and tell researchers what their main concerns and problems are, to help the scientists understand that while they would dearly love something that saves their life, they would also appreciate something that helps improve the quality of their lives.

Researchers too need to take a step back and not just get caught up in the search for an answer to a scientific or medical puzzle, without first asking “is this a puzzle that people want solved?”

At CIRM we work hard to make sure the voices of the patients and Patient Advocates are heard at every level of the work we do; from deciding what to fund to how to design a clinical trial involving our funding. But clearly it’s important that those voices be heard at a much earlier stage, to help shape the direction the research takes long before it comes to us for funding.

Breaking down barriers

For too long there has been a communications barrier between researchers and patients. This is not something that was deliberately constructed, it is something that simply evolved over time. Now it’s time to break down that barrier, and make sure both groups are talking to each other.

When it comes to developing treatments for deadly diseases and disorders, patients and researchers should think of themselves as partners. Researchers put their minds to work developing these treatments. Patients put their bodies on the line testing them.

Without the research there is no hope. Without the patients there is no proof. So, let’s start talking to each other.

If you have any thoughts or suggestions on how we can get this conversation started we would love to hear from you.

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

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.

reimbursement_shutterstock_326604206

“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.

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.

Moving Beyond Current CIRM Funding

Delivering on CIRM’s mission of “accelerating stem cell treatments to patients with unmet medical needs” requires the participation of multiple stakeholders to span the research, development, and commercialization phases of bringing a new product to market. In this post, I am pleased to highlight two recent examples of CIRM-funded projects moving beyond their period of CIRM funding by establishing partnerships with industry and investors to further develop the underlying CIRM-funded technology.

In 2000, Dr. Jill Helms, an academic investigator at Stanford University, received a $6.5 million grant from CIRM under an Early Translational award. The title of Dr. Helms’ project was Enhancing Healing via Wnt-Protein Mediated Activation of Endogenous Stem Cells,” and the goal of the award was to develop a novel, protein-based therapeutic platform to accelerate and enhance tissue regeneration through activation of adult stem cells. The five-year award achieved many critical milestones along the way, including the initiation of two preclinical studies aimed at demonstrating the effectiveness of a protein called L-WNT3A to improve the success of spinal fusion surgery and to treat a degradative bone disease called osteonecrosis, both of which represent unmet medical needs.

Helms_bonegraft

Through CIRM funding, Dr. Jill Helms’ team was able to demonstrate that treatment with a protein called L-Wnt3a regenerates and promotes bone formation in animals models (Figs D,F: untreated; Figs E,G: Wnt3a treated). (image credit: Leucht et al. J Bone Joint Surg Am. 2013;95:1278-88)

Dr. Helms’ work attracted considerable interest from the investor community during the lifespan of her grant, and during the final year of her award Dr. Helms’ WNT3A technology platform was successfully spun out of Stanford into a newly created company called Ankasa Regenerative Therapeutics. Ankasa was established with the financial support of Avalon Ventures – a La Jolla based life sciences venture capital firm, Correlation Ventures – an analytics driven venture capital firm, and Heraeus Medical – a diversified global medical device company based in Germany with over $1 billion of annual revenue. Ankasa has raised an initial $8.5 million in the first round of the total $17 million Series A financing to continue the development of the previously CIRM-funded technology.

Moving Radially Branched Deployment_Neurosurgery_Lim

Dr. Daniel Lim’s CIRM-funded BranchPoint Device allows neurosurgeons to deliver cell based therapies to multiple areas of the brain with just one needle penetration.  (image credit: Silvestrini et al. Stereotact Funct Neurosurg 2013;91:92–103)

The second recent example comes from a CIRM Tools & Technology grant to Dr. Daniel Lim, a neurosurgeon at UCSF. Dr. Lim was awarded a $1.8 million grant to develop a more efficient device for transplanting stem cells into the brain, titled Development and Preclinical Testing of New Devices for Cell Transplantation to the Brain.” Dr. Lim successfully developed a platform technology that enables Radially Branched Deployment (RBD) of cells to multiple target locations at variable radial distances and depths along the initial brain penetration tract with real-time interventional magnetic resonance image (iMRI) guidance. This technology is a huge leap forward over the conventional and crude syringe and needle device that are typically used to inject living cells into the brain.

Dr. Lim’s work attracted the attention of Accurexa, a publicly traded medical device company that licensed the CIRM-funded technology from UCSF. Under the guidance of Accurexa, a 510(k) application was submitted to the FDA for the newly coined “BranchPoint Device.” In June of this year, Accurexa successfully raised $2.5 million in equity financing to continue the development and for commercialization of the BranchPoint Device.

Overall, there remains a lack of industry pull for early stage stem cell technologies, however, both Drs. Helms and Lim’s stories represent successful examples of CIRM providing public dollars for early stage research with the resulting potentially life-saving applications attracting interest from investors and companies. These new investors will further fund and develop the technologies well beyond current CIRM funding and, assuming they are successful, deliver them to patients with unmet medical needs.