71 for Proposition 71

Proposition 71 is the state ballot initiative that created California’s Stem Cell Agency. This month, the Agency reached another milestone when the 71st clinical trial was initiated in the CIRM Alpha Stem Cell Clinics (ASCC) Network. The ASCC Network deploys specialized teams of doctors, nurses and laboratory technicians to conduct stem cell clinical trials at leading California Medical Centers.

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These teams work with academic and industry partners to support patient-centered for over 40 distinct diseases including:

  • Amyotrophic Lateral Sclerosis (ALS)
  • Brain Injury & Stroke
  • Cancer at Multiple Sites
  • Diabetes Type 1
  • Eye Disease / Blindness Heart Failure
  • HIV / AIDS
  • Kidney Failure
  • Severe Combined Immunodeficiency (SCID)
  • Sickle Cell Anemia
  • Spinal Cord Injury

These clinical trials have treated over 400 patients and counting. The Alpha Stem Cell Clinics are part of CIRM’s Strategic Infrastructure. The Strategic Infrastructure program which was developed to support the growth of stem cell / regenerative medicine in California. A comprehensive update of CIRM’s Infrastructure Program was provided to our Board, the ICOC.

CIRM’s infrastructure catalyzes stem cell / regenerative medicine by providing resources to all qualified researchers and organizations requiring specialized expertise. For example, the Alpha Clinics Network is supporting clinical trials from around the world.

Many of these trials are sponsored by commercial companies that have no CIRM funding. To date, the ASCC Network has over $27 million in contracts with outside sponsors. These contracts serve to leverage CIRMs investment and provide the Network’s medical centers with a diverse portfolio of clinical trials to address patients’’ unmet medical needs.

Alpha Clinics – Key Performance Metrics

  • 70+ Clinical Trials
  • 400+ Patients Treated
  • 40+ Disease Indications
  • Over $27 million in contracts with commercial sponsors

The CIRM Alpha Stem Cell Clinics and broader Infrastructure Programs are supporting stem cell research and regenerative medicine at every level, from laboratory research to product manufacturing to delivery to patients. This infrastructure has emerged to make California the world leader in regenerative medicine. It all started because California’s residents supported a ballot measure and today we have 71 clinical trials for 71.

 

 

Stem Cell Agency celebrates 50 clinical trials with patient #1

Yesterday the CIRM Board approved funding for our 50th clinical trial (you can read about that here) It was an historic moment for us and to celebrate we decided to go back in history and hear from the very first person to be treated in a CIRM-funded clinical trial. Rich Lajara was treated in the Geron clinical trial after experiencing a spinal cord injury, thus he became CIRM’s patient #1. It’s a badge he says he is honored to wear. This is the speech Rich made to our Board.

Rich Lajara

Hello and good afternoon everyone. It’s an honor to be here today as the 50th clinical trial has been officially funded by CIRM. It was feels like it was just yesterday that I was enrolled into the first funded clinical trial by CIRM and in turn became California’s’ 1st embryonic stem cell patient.

I became paralyzed from the waist down in September 2011. It was Labor Day and I was at a river with some close friends. There was this natural granite rock slide feature next to a waterfall, it was about 60 feet long; all you had to do was get a bucket of water to get the rocks wet and slide down into a natural pool. I ended up slipping and went down head first backwards but was too far over and I slid off a 15’ ledge where the waterfall was. I was pulled from the water and banged up pretty bad. Someone said “look at that deformity on his back” and tapped my leg and asked if I could feel that. I knew immediately I was paralyzed. I thought this was the end, little did I know this was just the beginning. I call it being in the wrong place at the right time.

So, after a few days in the hospital of course everyone, as well as myself, wanted a cure. We quickly learned one didn’t exist. A close family friend had been making phone calls and was able to connect with the Christopher & Dana Reeve Foundation and learned about a clinical trial with “stem cells”. One of my biggest question was how any people have done this? “Close to none”, I was told.

I was also told I most likely would have no direct benefit as this was a safety trial? So why do it at all? Obviously at that time I was willing to overlook the “most likely” part because I was willing to do anything to try and get my normal life back.

Looking back the big picture was laying the ground work for others like Kris or Jake (two people enrolled in a later version of this trial). At the time I had no clue that what I was doing would be such a big deal. The data collected from me would end up being priceless. It’s stories like Jake’s and Kris’ that make me proud and reinforce my decision to have participated in California’s first stem cell clinical trial funded by prop 71.

Like I said earlier it was just the beginning for me. A couple of years later I became a patient advocate working with Americans for Cures. I have been able to meet many people in the stem cell industry and love to see the glow in their face when they learn I was California’s first embryonic stem cell patient.

I can’t even fathom all the year’s of hard work and countless hours of research that had lead up to my long anticipated surgery, but when I see their glowing smile I know they knew what it took.

I also enjoy sharing my story and bridging the gap between myths and facts about stem cells, or talking to students and helping inspire the next generation that will be in the stem cell industry.  As a matter of fact, I have 13 year old sister, Maddie, dead set on being a neurosurgeon.

Fast forward to today. Life in a wheelchair is not exactly a roll in the park (no pun intended) but I have grown accustomed to the new normal. Aside from some neuropathic pain, life is back on track.

Not once did I feel sorry for myself, I was excited to be alive. Sure I have bad days but don’t we all.

In the last 14 years CIRM has funded 50 human clinical trials, published around 2750 new peer-reviewed scientific discoveries, and they’ve transformed California into the world leader in stem cell research. As I look around the posters on the wall, of the people whose lives have been transformed by the agency, I can’t help but be struck by just how much has been achieved in such a short period of time.

While my journey might not yet be over, Evie and 40 other children like her, (children born with SCID) will never remember what it was like to live with the horrible condition they were born with because they have been cured thanks to CIRM. There are hundreds of others whose lives have been transformed because of work the agency has funded.

CIRM has proven how much can be achieved if we invest in cutting-edge medical research.

As most of you here probably know CIRM’s funding from Proposition 71 is about to run out. If I had just one message I wanted people to leave with today it would be this. Everyone in this room knows how much CIRM has done in a little over a decade and how many lives have been changed because of its existence. We have the responsibility to make sure this work continues. We have a responsibility to make sure that the stories we’ve heard today are just the beginning.

I will do everything I can to make sure the agency gets refunded and I hope that all of you will join me in that fight. I’m excited for the world of stem cells, particularly in California, and can’t wait to see what’s on the horizon.

 

Stem Cell Agency Board Approves 50th Clinical Trial

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Rich Lajara, the first patient treated in a CIRM-funded clinical trial

May 4th, 2011 marked a landmark moment for the California Institute for Regenerative Medicine (CIRM). On that day the Stem Cell Agency’s Board voted to invest in its first ever clinical trial, which was also the first clinical trial to use cells derived from embryonic stem cells. Today the Stem Cell Agency reached another landmark, with the Board voting to approve its 50th clinical trial.

“We have come a long way in the past seven and a half years, helping advance the field from its early days to a much more mature space today, one capable of producing new treatments and even cures,” says Jonathan Thomas, JD, PhD, Chair of the CIRM Board. “But we feel that in many ways we are just getting started, and we intend funding as many additional clinical trials as we can for as long as we can.”

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The project approved today awards almost $6.2 million to Angiocrine Bioscience Inc. to see if genetically engineered cells, derived from cord blood, can help alleviate or accelerate recovery from the toxic side effects of chemotherapy for people undergoing treatment for lymphoma and other aggressive cancers of the blood or lymph system.

“This is a project that CIRM has supported from an earlier stage of research, highlighting our commitment to moving the most promising research out of the lab and into people,” says Maria T. Millan, MD, President & CEO of CIRM. “Lymphoma is the most common blood cancer and the 6th most commonly diagnosed cancer in California. Despite advances in therapy many patients still suffer severe complications from the chemotherapy, so any treatment that can reduce those complications can not only improve quality of life but also, we hope, improve long term health outcomes for patients.”

The first clinical trial CIRM funded was with Geron, targeting spinal cord injury. While Geron halted the trial for business reasons (and returned the money, with interest) the mantle was later picked up by Asterias Biotherapeutics, which has now treated 25 patients with no serious side effects and some encouraging results.

Rich Lajara was part of the Geron trial, the first patient ever treated in a CIRM-funded clinical trial. He came to the CIRM Board meeting to tell his story saying when he was injured “I knew immediately I was paralyzed. I thought this was the end, little did I know this was just the beginning. I call it being in the wrong place at the right time.”

When he learned about the Geron clinical trial he asked how many people had been treated with stem cells. “Close to none” he was told. Nonetheless he went ahead with it. He says he has never regretted that decision, knowing it helped inform the research that has since helped others.

Since that first trial the Stem Cell Agency has funded a wide range of projects targeting heart disease and stroke, cancer, diabetes, HIV/AIDS and several rare diseases. You can see the full list on the Clinical Trials Dashboard page on our website.

Rich ended by saying: “CIRM has proven how much can be achieved if we invest in cutting-edge medical research. As most of you here probably know, CIRM’s funding from Proposition 71 is about to run out. If I had just one message I wanted people to leave with today it would be this, I will do everything I can to make sure the agency gets refunded and I hope that all of you will join me in that fight. I’m excited for the world of stem cells, particularly in California and can’t wait to see what’s on the horizon.”

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The CIRM Board also took time today to honor Dr. Bert Lubin, who is stepping down after serving almost eight years on the Board.

When he joined the Board in February, 2011 Dr. Lubin said: “I hope to use my position on this committee to advocate for stem cell research that translates into benefits for children and adults, not only in California but throughout the world.”

Over the years he certainly lived up to that goal. As a CIRM Board member he has supported research for a broad range of unmet medical needs, and specifically for curative treatments for children born with a rare life-threatening conditions such as Sickle Cell Disease and Severe Combined Immunodeficiency (SCID) as well as  treatments to help people battling vision destroying diseases.

As the President & CEO of Children’s Hospital Oakland (now UCSF Benioff Children’s Hospital Oakland) Dr. Lubin was a leader in helping advance research into new treatments for sickle cell disease and addressing health disparities in diseases such as asthma, diabetes and obesity.

Senator Art Torres said he has known Dr. Lubin since the 1970’s and in all that time has been impressed by his devotion to patients, and his humility, and that all Californians should be grateful to him for his service, and his leadership.

Dr. Lubin said he was “Really grateful to be on the Board and I consider it an honor to be part of a group that benefits patients.”

He said he may be stepping down from the CIRM Board but that was all: “I am going to retire the word retirement. I think it’s a mistake to stop doing work that you find stimulating. I’m going to repurpose the rest of my life, and work to make sure the treatments we’ve helped develop are available to everyone. I am so proud to be part of this. I am stepping down, but I am devoted to doing all I can to ensure that you get the resources you need to sustain this work for the future.”

California’s Stem Cell Agency Accelerates Treatments to Patients

The following article is an Op Ed that appeared in today’s print version of the San Francisco Chronicle

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Biotechnology was born in California in the 1970s based on the discovery out of one of its universities and California is responsible for an industry that has impacted the lives of billions of people worldwide. In 2004, the voters of California approved Proposition 71, creating the California Institute for Regenerative Medicine and setting the state on the path to becoming a global leader in stem cell research. Today the therapies resulting from the institute’s work are not just changing lives, they are already saving lives.

Lives like Evie Vaccaro, who is alive today because of a treatment CIRM is funding. Vaccaro was born with SCID, also known as “bubble baby disease,” an immune disorder that often kills babies in their first two years. Vaccaro and dozens of other babies were given stem cell treatments thanks to the institute. All are showing improvement; some are now several years past treatment and considered cured.

An accident left Jake Javier from Danville paralyzed from the chest down on the eve of his high school graduation. Javier was treated in a CIRM-funded clinical trial. Today he has regained the use of his arms and hands, is driving a car and is a sophomore at Cal Poly San Luis Obispo. Five other patients treated at the same time as Javier have all experienced improvements meaning that instead of needing round-the-clock care, they can lead independent lives.

A study by the Tufts Center for the Study of Drug Development estimated it takes at least 10 years and $2.6 billion to develop one successful drug. In 14 years, and with just $3 billion, CIRM has funded 1,000 different projects, enrolled 900 patients, and supported 49 different clinical trials targeting diseases such as cancer, kidney failure and leukemia. Four of these programs have received an expedited designation by the U.S. Food and Drug Administration, meaning they could get faster approval to help more patients

We have created a network of world class medical clinics that have expertise in delivering treatments to patients. The CIRM Alpha Clinics offer treatments based on solid science, unlike the unlicensed clinics sprouting up around California that peddle unproven and potentially harmful therapies that cost patients thousands of dollars.

CIRM has:

  • Supported the creation of 12 stem-cell research facilities in California
  • Attracted hundreds of top-tier researchers to California
  • Trained a new generation of stem-cell scientists
  • Brought clinical trials to California — for example, one targeting ALS or Lou Gehrig’s disease
  • Deployed rigorous scientific standards and support so our programs have a “seal of approval” to attract $2.7 billion in additional investments from industry and other sources.

We recently have partnered with the National Institutes of Health to break down barriers and speed up the approval process to bring curative treatments to patients with Sickle Cell Disease.

Have we achieved all we wanted to? Of course not. The first decade of CIRM’s life was laying the groundwork, developing the knowledge and expertise and refining processes so that we can truly accelerate progress. As a leader in this burgeoning field of regenerative medicine, CIRM needs to continue its mission of accelerating stem-cell treatments to patients with unmet medical needs.

Dr. Maria T. Millan is President and CEO and Jonathan Thomas, JD, PhD, is the Board Chairman of the California Institute of Regenerative Medicine. 

 

 

Has Regenerative Medicine Come of Age?

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For the past few years the Signals blog site –  which offers an insiders’ perspectives on the world of regenerative medicine and stem cell research – has hosted what it calls a “Blog Carnival”. This is an event where bloggers from across the stem cell field are invited to submit a piece based on a common theme. This year’s theme is “Has Regenerative Medicine Come of Age?” Here’s my take on that question:

Many cultures have different traditions to mark when a child comes of age. A bar mitzvah is a Jewish custom marking a boy reaching his 13th birthday when he is considered accountable for his own actions. Among Latinos in the US a quinceañera is the name given to the coming-of-age celebration on a girl’s 15th birthday.

Regenerative Medicine (RM) doesn’t have anything quite so simple or obvious, and yet the signs are strong that if RM hasn’t quite come of age, it’s not far off.

For example, look at our experience at the California Institute for Regenerative Medicine (CIRM). When we were created by the voters of California in 2004 the world of stem cell research was still at a relatively immature phase. In fact, CIRM was created just six years after scientists first discovered a way to derive stem cells from human embryos and develop those cells in the laboratory. No surprise then that in the first few years of our existence we devoted a lot of funding to building world class research facilities and investing in basic research, to gain a deeper understanding of stem cells, what they could do and how we could use them to develop therapies.

Fast forward 14 years and we now have funded 49 projects in clinical trials – everything from stroke and cancer to spinal cord injury and HIV/AIDS – and our early funding also helped another 11 projects get into clinical trials. Clearly the field has advanced dramatically.

In addition the FDA last year approved the first two CAR-T therapies – Kymriah and Yescarta – another indication that progress is being made at many levels.

But there is still a lot of work to do. Many of the trials we are funding at the Stem Cell Agency are either Phase 1 or 2 trials. We have only a few Phase 3 trials on our books, a pattern reflected in the wider RM field. For some projects the results are very encouraging – Dr. Gary Steinberg’s work at Stanford treating people recovering from a stroke is tremendously promising. For others, the results are disappointing. We have cancelled some projects because it was clear they were not going to meet their goals. That is to be expected. These clinical trials are experiments that are testing, often for the first time ever in people, a whole new way of treating disease. Failure comes with the territory.

As the number of projects moving out of the lab and into clinical trials increases so too are the other signs of progress in RM. We recently held a workshop bringing together researchers and regulators from all over the world to explore the biggest problems in manufacturing, including how you go from making a small batch of stem cells for a few patients in an early phase clinical trial to mass producing them for thousands, if not millions of patients. We are also working with the National Institutes of Health and other stakeholders in discussing the idea of reimbursement, figuring out who pays for these therapies so they are available to the patients who need them.

And as the field advances so too do the issues we have to deal with. The discovery of the gene-editing tool CRISPR has opened up all sorts of possible new ways of developing treatments for deadly diseases. But it has also opened up a Pandora’s box of ethical issues that the field as a whole is working hard to respond to.

These are clear signs of a maturing field. Five years ago, we dreamed of having these kinds of conversations. Now they are a regular feature of any RM conference.

The simple fact that we can pose a question asking if RM has come of age is a sign all by itself that we are on the way.

Like little kids sitting in the back of a car, anxious to get to their destination, we are asking “Are we there yet?” And as every parent in the front seat of their car responds, “Not yet. But soon.”

Regenerative Medicine by the numbers: a snapshot of how the field is progressing

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Statistics don’t usually make for very exciting blog fodder, but they can be useful in charting progress. Case in point, the recent quarterly report from the Alliance for Regenerative Medicine (ARM), a global advocate and industry group for the field.

In the report ARM takes an in-depth look at cell therapy, gene therapy, tissue engineering and other trends in the regenerative medicine field.

Among the more notable findings are:

  • Companies in the regenerative medicine space collectively raised more than $4.1 billion in the second quarter of this year, up 164 percent over the same period in 2017.
  • Companies focused on cell therapy raised $2.2 billion, up 416 percent over the same period last year.
  • More and more companies in the space are turning to the public markets. So far this year they collectively raised $913.4 million in IPOs (initial public offerings – the very first sale of a company’s stock to the public), up from $254 million during all of last year.
  • Nearly 977 clinical trials testing such therapies are in progress across the globe; more than half of them are trying to treat cancer.

In a news release, Janet Lynch Lambert, ARM’s CEO, was understandably upbeat:

“There has been a tremendous amount of forward momentum during the first half of this year, both clinically and commercially. We’re excited for the continued growth of the regenerative medicine sector, and what it means for patients worldwide.”

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Can stem cells help people recover from a stroke? Join us for a Facebook Live event this Thursday, May 31 for the answers

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Stroke is one of the leading causes of death in the US and the leading cause of serious, long-term disability. But could stem cell therapies change that and help people who’ve had a brain attack?  Could stem cells help repair the damage caused by a stroke and restore a person’s ability to speak normally, to be able to walk without a limp or regain strength in their hands and arms?

To find out the answers to these and other questions joins us for “Ask the Expert”, a special Facebook Live event this Thursday, May 31, from noon till 1pm PDT

 The event will feature Dr. Gary Steinberg, the Chair of Neurosurgery at Stanford University. Dr. Steinberg is currently running a CIRM-funded clinical trial targeting stroke.

We will also be joined by CIRM Senior Science Officer Lila Collins, PhD who can talk about the broad range of other projects using stem cells to help people recover from a stroke.

We are also delighted to welcome Sonia Coontz, who suffered a devastating stroke several years ago and made a remarkable recovery after getting a stem cell therapy.

To join us for the event, all you have to do is go to our Facebook page on Thursday at noon (PDT) and you should see a video playing, which you can watch on mobile or desktop. Click the video to enter viewing mode.

Also, make sure to “like” our page before the event to receive a notification that we’ve gone live.

And we want to hear from you, so you will be able to post questions for the experts to answer or, you can email them directly to us at info@cirm.ca.gov

We look forward to seeing you there.

 

A Noble pursuit; finding the best science to help the most people

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Mark Noble. Photo by Todd Dubnicoff

Mark Noble, Ph.D., is a pioneer in stem cell research and the Director of the University of Rochester Stem Cell and Regenerative Medicine Institute in New York. He is also a member of CIRM’s Grants Working Group (GWG), the panel of independent scientific experts we use to review research applications for funding and decide which are the most promising.

Mark has been a part of the GWG since 2011. When asked how he came to join the GWG he joked: “I saw an ad on Craigslist and thought it sounded fun.”  But he is not joking when he says it is a labor of love.

“My view is that CIRM is one of the greatest experiments in how to develop a new branch of science and medicine. If you look at ventures, like the establishment of the National Institutes of Health, what you see is that when there is a concentrated effort to achieve an enormous goal, amazing things can happen. And if your goal is to create a new field of medicine you have to take a truly expansive view.”

Mark has been on many other review panels but says they don’t compare to CIRM’s.

“These are the most exciting review panels in which I take part. I don’t know of any comparable panels that bring together experts working across such a wide range of disciplines and diseases.   It’s particularly interesting to be involved in reviews at this stage because we get to look at the fruits of CIRM’s long investment, and at projects that are now in, or well on the way towards, clinical trials.

It’s a wonderful scientific education because you come to these meetings and someone is submitting an application on diabetes and someone else has submitted an application on repairing the damage to the heart or spinal cord injury or they have a device that will allow you to transplant cells better. There are people in the room that are able to talk knowledgeably about each of these areas and understand how the proposed project might work in terms of actual financial development, and how it might work in the corporate sphere and how it fits in to unmet medical needs.  I don’t know of any comparable review panels like this that have such a broad remit and bring together such a breadth of expertise. Every review panel you come to you are getting a scientific education on all these different areas, which is great.”

Another aspect of CIRM’s work that Mark admires is its ability to look past the financial aspects of research, to focus on the bigger goal:

“I like that CIRM recognizes the larger problem, that a therapy that is curative but costs a million dollars a patient is not going to be implemented worldwide. Well, CIRM is not here to make money. CIRM is here to find cures for unmet medical needs, which means that if someone comes in with a great application on a drug that is going to cure some awful disease and it’s not going to be worth a fortune, that is not the main concern. The main concern is that you might be able to cure this disease and yeah, we’ll put up money to help you so that you might be able to get into clinical trials, to get enough information to find out if it works. And to have the vision to go all the way from, ‘ok, you guys, we want you to enter this field, we want you to be interested in therapeutic development, we are going to help you structure the clinical trials, we are going to provide all the Alpha Stem Cell Clinics that can talk to each other to make the clinical trials happen.

The goal of CIRM is to change medicine and these are the approaches that have worked really well in doing this. The CIRM view clearly is:

‘There are 100 horses in this race and every single one that crosses the finish line is a success story.’ That’s what is necessary, because there are so many diseases and injuries for which new approaches are needed.”

Mark says working with CIRM has helped him spread the word back home in New York state:

“I have been very involved in working with the New York state legislature over the years to promote funding for stem cell biology and spinal cord injury research so having the CIRM experience has really helped me to understand what it is that another place can try and accomplish. A lot of the ideas that have been worked out at CIRM have been extremely helpful for statewide scientific enterprises in New York, where we have had people involved in different areas of the state effort talk to people at CIRM to find out what best practice is.”

Mark says he feels as if he has a front row seat to history.

“Seeing the stem cell field grow to its present stage and enhancing the opportunity to address multiple unmet medical needs, is a thrilling adventure. Working with CIRM to help create a better future is a privilege.”

 

Seeing is believing. Proof a CIRM-funded therapy is making a difference

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Thelma, participant in the CAMELLIA clinical trial

You have almost certainly never heard of Thelma, or met her, or know anything about her. She’s a lady living in England who, if it wasn’t for a CIRM-funded therapy, might not be living at all. She’s proof that what we do, is helping people.

Thelma is featured in a video about a treatment for acute myeloid leukemia, one of the most severe forms of blood cancer. Thelma took part in a clinical trial, called CAMELLIA, at Oxford Cancer Centre in Oxford, UK. The clinical trial uses a therapy that blocks a protein called CD47 that is found on the surface of cancer cells, including cancer stem cells which can evade traditional therapies. The video was shot to thank the charity Bloodwise for raising the funds to pay for the trial.

Prof. Paresh Vyas of Oxford University, who was part of the clinical trial team that treated Thelma, says patients with this condition face long odds.

“Patients with acute myeloid leukemia have the most aggressive blood cancer. We really haven’t had good treatments for this condition for the last 40 years.”

While this video was shot in England, featuring English nurses and doctors and patients, the therapy itself was developed here in California, first at Stanford University under the guidance of Irv Weissman and, more recently, at Forty Seven Inc. That company is now about to test their approach in a CIRM-funded clinical trial here in the US.

This is an example of how CIRM doesn’t just fund research, we invest in it. We help support it at every stage, from the earliest research through to clinical trials. Without our early support this work may not have made it this far.

The Forty Seven Inc. therapy uses the patient’s own immune system to help fight back against cancer stem cells. It’s looking very promising. But you don’t have to take our word for it. Take Thelma’s.

Recap of the 2018 Alliance for Regenerative Medicine Cell and Gene Therapy State of the Industry

What happened in the Cell and Gene Therapy sector in 2017, and what should we be looking out for in 2018? Over 500 executives, investors, scientists and patient advocates gathered together yesterday to find out at the Alliance for Regenerative Medicine (ARM) State of the Industry Briefing in San Francisco, California.

ARM Chairman, Robert Preti, and ARM CEO, Janet Lynch Lambert, kicked off the session by discussing how 2017 marked an inflection point for the sector. They underscored the approval of three cell/gene therapies (see slide below) by the U.S. Food and Drug Administration (FDA), a “bright and robust” future pipeline that should yield over 40 approved therapies in the next five years, and an improving regulatory environment that’s accelerating approvals of regenerative medicine therapies. This year alone, the FDA has granted 12 Regenerative Medicine Advanced Therapy (RMAT) designations through the 21st Century Cures Act (see slide below for companies/products that received RMAT in 2017).

In 2017, a total of four cell/gene therapies were approved and the US FDA awarded 12 RMAT designations. This slide is from the 2018 ARM Cell and Gene Therapy State of the Industry Briefing presentation.

Next up was a snapshot of the clinical landscape highlighting a total of 946 ongoing clinical trials at the end of 2017, and their breakdown by disease (see chart below). Oncology (cancer) is the clear winner comprising over 50% of the trials while Cardiovascular (heart) took second with 8.6% and diseases of the central nervous system (brain and spinal cord) took third with 6.5%.

Lambert also gave a brief overview of finances in 2017 and listed some impressive numbers. $7.5 Billion in capital was raised in 2017 compared to $4.2 Billion in 2016. She also mentioned major acquisitions, mergers, partnerships and public financings that paved the way for this year’s successes in cell and gene therapy.

Lambert concluded that while there was significant progress with product approvals, growing public awareness of successes in the sector, regulatory advances and financial maturity, there is a need for further commercial support and a focus on policy making, industrialization and manufacturing.

The Industry Update was followed by two panel sessions.

The first panel focused on cell-based cancer immunotherapies and featured company leaders from Juno Therapeutics, Mustang Bio, Adaptimmune, Novartis, and Fate Therapeutics.

In the cancer field, companies are aggressively pursuing the development of cell-based immunotherapies including Chimeric Antigen Receptor T (CAR-T) cells, modified T-cells and Natural Killer (NK) cells, to name a few. These therapies all involve engineering or modifying human immune cells to identify and target cancer cells that resist first-line cancer treatments like radiation or chemotherapy.

The panelists spoke of a future that involved the development of combination therapies that partner cell-based immunotherapies with other drugs and treatments to better target specific types of cancer. They also spent a significant portion of the panel discussing the issues of manufacturing and reimbursement. On manufacturing, the panel argued that a centralized cell manufacturing approach will be needed to deliver safe products to patients. On reimbursement, they addressed the difficulty of finding a balance between pricing life-saving therapies and navigating reimbursements from insurance companies.

The second panel focused on the state of gene therapy and the outlook for 2018. This panel featured company and academic leaders from CRISPR Therapeutics, Sangamo Therapeutics, BioMarin Pharmaceutical, Adverum Biotechnologies, and the Gladstone Institutes.

ARM Gene Therapy Panel: Martha Rook (MilliporeSigma), Deepak Srivastava (Gladstone Institutes), Amber Salzman (Adverum Biotechnologies), Bill Lundberg (CRISPR Therapeutics), Geoff Nichol (BioMarin Pharmaceutical), Sandy Macrae (Sangamo Therapeutics)

The panel spoke about the difference between gene editing (fixing an existing gene within a cell) and gene therapy (adding a new gene into a cell) technologies and how the delivery of these therapies into tissues and cells is the biggest challenge in the area right now.

Sandy Macrae, President and CEO of Sangamo Therapeutics, made an interesting point when he said that for gene therapy to be successful, companies need to plan two to three years in advance for a phase III trial (the final stage before a product is approved) because manufacturing gene therapies takes a long time. He said the key for success is about having medicines that are ready to launch, not just reporting good results.

Overall, ARM’s State of the Industry provided an exciting overview of the progress made in the Cell and Gene Therapy Sector in 2017 and shared outlooks for 2018 and beyond.

You can access the Live Webcast of ARM’s State of the Industry Briefing including both panel sessions on the ARM website. Be sure to check out our blog featuring our 2018 Stem Cell Conference Guide for more ARM events and other relevant stem cell research meetings in the coming year.