HOPE for patients with Duchenne Muscular Dystrophy-associated heart disease

It’s an exciting week for CIRM-funded clinical trials. Yesterday, we blogged about a young man named Kris Boesen who is responding positively to a stem cell therapy in a Phase 1/2a CIRM-funded clinical trial for spinal cord injury run by Asterias Biotherapeutics. Paralyzed from the chest down after a terrible car accident, Kris now has regained some use of his arms and hands following the stem cell transplant.

screen-shot-2016-09-08-at-9-18-46-amYesterday, Capricor Therapeutics also announced news about the progress of its CIRM-funded clinical trial that’s testing the safety and efficacy of a cardiac cell therapy called CAP-1002 for Duchenne Muscular Dystrophy-associated cardiomyopathy. Capricor has completed their Phase 1/2 trial enrollment of 25 patients. These patients are young boys (12 years of age or above) suffering from a build-up of scar tissue in their hearts due to DMD-associated cardiomyopathy. Reaching full enrollment is a key milestone for any clinical trial.

Duchenne Muscular Dystrophy (DMD) is an inherited disease that attacks muscle, causing muscle tissue to become weak and degenerate. The disease mainly appears in young boys between the ages of two and three. Patients with DMD often suffer from cardiomyopathy or weakened heart muscle caused by the thickening and hardening of the heart muscle and accumulation of scar tissue. DMD-associated cardiomyopathy is one of the leading causes of patient deaths.

President and CEO of Capricor, Dr. Linda Marban, believes there’s a potential for their CAP-1002 stem cell therapy to help DMD patients suffering from cardiomyopathy. She explained in a press release:

“In DMD, scar tissue progressively aggregates in the heart, leading to a deterioration of cardiac function for which treatment options are limited. We believe CAP-1002 is the only therapeutic candidate in development for the treatment of DMD that has been clinically shown to reduce scar tissue in the damaged heart.”

The Capricor trial was approved by the CIRM Board in March 2016 and since then Capricor has worked quickly to enroll patients in its HOPE-Duchenne trial (HOPE stands for Halt cardiomyopathy progression in Duchenne).

Dr. Marban commented on the trials recent progress:

Linda Marban, CEO of Capricor Therapeutics

Linda Marban, CEO of Capricor Therapeutics

“The rate of patient enrollment into HOPE-Duchenne far surpassed our expectations, signifying the need for therapeutic options as well as the desire of the DMD community to address the heart disease that is highly prevalent in this population. We look forward to announcing top-line six-month results from HOPE-Duchenne in the first quarter of next year, in which we will report on the safety as well as the potential efficacy of CAP-1002.”

Half of the enrolled patients will receive an infusion of the CAP-1002 cardiac cell therapy while the other half will receive regular care without the infusion. Capricor will monitor all these patients to make sure that the cell therapy is well tolerated and doesn’t cause any harm. It will also look for any positive signs that the therapy is benefiting patients using a series of tests that measure changes in scar tissue and heart function.

HOPE is high for this trial to succeed as there is currently no treatment that can successfully reduce the amount of cardiac scar tissue in patients suffering from DMD-associated cardiomyopathy. The Capricor trial is in its early stages, but check in with the Stem Cellar for an update on the safety and efficacy data from this trial in early 2017.


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Young man with spinal cord injury regains use of hands and arms after stem cell therapy

kris-boesen

Kris Boesen – Photo courtesy USC

Hope is such a fragile thing. We cling to it in bad times. It offers us a sense that we can bear whatever hardships we are facing today, and that tomorrow will be better.

Kris Boesen knows all about holding on to hope during bad times. On March 6th of this year he was left paralyzed from the neck down after a car accident. Kris and his parents were warned the damage might be permanent.

Kris says at that point, life was pretty bleak:

“I couldn’t drink, couldn’t feed myself, couldn’t text or pretty much do anything, I was basically just existing. I wasn’t living my life, I was existing.”

For Kris and his family hope came in the form of a stem cell clinical trial, run by Asterias Biotherapeutics and funded by CIRM. The Asterias team had already enrolled three patients in the trial, each of whom had 2 million cells transplanted into their necks, primarily to test for safety. In early April Kris became the first patient in the trial to get a transplant of 10 million stem cells.

Within two weeks he began to show signs of improvement, regaining movement and strength in his arms and hands:

“Now I have grip strength and do things like open a bottle of soda and feed myself. Whereas before I was relying on my parents, now after the stem cell therapy I am able to live my life.”

The therapy involves human embryonic stem cells that have been differentiated, or converted, into cells called oligodendrocyte progenitors. These are capable of becoming the kind of cells which help protect nerve cells in the central nervous system, the area damaged in spinal cord injury.

The surgery was performed by Keck Medicine of USC’s Dr. Charles Liu. In a news release about the procedure, he says improvements of the kind Kris has experienced can make a huge difference in someone’s life:

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Dr. Charles Liu, Keck School of Medicine: Photo courtesy USC

“As of 90 days post-treatment, Kris has gained significant improvement in his motor function, up to two spinal cord levels. In Kris’ case, two spinal cord levels means the difference between using your hands to brush your teeth, operate a computer or do other things you wouldn’t otherwise be able to do, so having this level of functional independence cannot be overstated.”

We blogged about this work as recently as last week, when Asterias announced that the trial had passed two important safety hurdles.  But Kris’ story is the first to suggest this treatment might actually be working.

Randy Mills, CIRM’s President & CEO, says:

 “With each patient treated in this clinical trial we learn.  We gain more experience, all of which helps us put into better context the significance of this type of event for all people afflicted with debilitating spinal cord injuries. But let us not lose sight of the individual here.  While each participant in a clinical trial is part of the group, for them success is binary.  They either improve or they do not.  Kris bravely and selflessly volunteered for this clinical trial so that others may benefit from what we learn.  So it is fitting that today we celebrate Kris’ improvements and stop to thank all those participating in clinical trials for their selfless efforts.”

For patient advocates like Roman Reed, this was a moment to celebrate. Roman has been championing stem cell research for years and through his Roman Reed Foundation helped lay the groundwork for the research that led to this clinical trial:

This is clear affirmative affirmation that we are making Medical History!  We were able to give a paralyzed quadriplegic patient back the use of his hands! With only half a clinical dosage. Now this person may hold and grasp his loved ones hands in his own hands because of the actions of our last two decades for medical research for paralysis CURE! CARPE DIEM!”

It’s not unheard of for people with the kind of injury Kris had to make a partial recovery, to regain some use of their arms and hands, so it’s impossible to know right now if the stem cell transplant was the deciding factor.

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Kris at home: photo courtesy USC

Kris’ dad, Rodney, says he doesn’t care how it happened, he’s just delighted it did:

“He’s going to have a life, even if (the progress) stops just this second, and this is what he has, he’s going to have a better life than he would have definitely had before, because there are so many things that this opens up the world for him, he’s going to be able to use his hands.”


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Stem cell stories that caught our eye: functioning liver tissue, making new bone, stem cells and mental health

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

Functioning liver tissue. Scientists are looking to stem cells as a potential alternative treatment to liver transplantation for patients with end-stage liver disease. Efforts are still in their early stages but a study published this week in Stem Cells Translational Medicine, shows how a CIRM-funded team at the Children’s Hospital Los Angeles (CHLA) successfully generated partially functional liver tissue from mouse and human stem cells.

Biodegradable scaffold (left) and human tissue-engineered liver (right) (Photo courtesy of The Saban Research Institute at Children’s Hospital Los Angeles)

Biodegradable scaffold (left) and human tissue-engineered liver (right) (Photo courtesy of The Saban Research Institute at Children’s Hospital Los Angeles)

The lab had previously developed a protocol to make intestinal organoids from mouse and human stem cells. They were able to tweak the protocol to generate what they called liver organoid units and transplanted the tissue-engineered livers into mice. The transplants developed cells and structures found in normal healthy livers, but their organization was different – something that the authors said they would address in future experiments.

Impressively, when the tissue-engineered liver was transplanted into mice with liver failure, the transplants had some liver function and the liver cells in these transplants were able to grow and regenerate like in normal livers.

In a USC press release, Dr. Kasper Wang from CHLA and the Keck school of medicine at USC commented:

“A cellular therapy for liver disease would be a game-changer for many patients, particularly children with metabolic disorders. By demonstrating the ability to generate hepatocytes comparable to those in native liver, and to show that these cells are functional and proliferative, we’ve moved one step closer to that goal.”

 

Making new bone. Next up is a story about making new bone from stem cells. A group at UC San Diego published a study this week in the journal Science Advances detailing a new way to make bone forming cells called osteoblasts from human pluripotent stem cells.

Stem cell-derived osteoblasts (bone cells). Image credit Varghese lab/UCSD.

Stem cell-derived osteoblasts (bone cells). Image credit Varghese lab/UCSD.

One way that scientists can turn pluripotent stem cells into mature cells like bone is to culture the stem cells in a growth medium supplemented with small molecules that can influence the fate of the stem cells. The group discovered that by adding a single molecule called adenosine to the growth medium, the stem cells turned into osteoblasts that developed vascularized bone tissue.

When they transplanted the stem cell-derived osteoblasts into mice with bone defects, the transplanted cells developed new bone tissue and importantly didn’t develop tumors.

 In a UC newsroom release, senior author on the study and UC San Diego Bioengineering Professor Shyni Varghese concluded:

“It’s amazing that a single molecule can direct stem cell fate. We don’t need to use a cocktail of small molecules, growth factors or other supplements to create a population of bone cells from human pluripotent stem cells like induced pluripotent stem cells.”

 

Stem cells and mental health. Brad Fikes from the San Diego Union Tribune wrote a great article on a new academic-industry partnership whose goal is to use human stem cells to find new drugs for mental disorders. The project is funded by a $15.4 million grant from the National Institute of Mental Health.

Academic scientists, including Rusty Gage from the Salk Institute and Hongjun Song from Johns Hopkins University, are collaborating with pharmaceutical company Janssen and Cellular Dynamics International to develop induced pluripotent stem cells (iPSCs) from patients with mental disorders like bipolar disorder and schizophrenia. The scientists will generate brain cells from the iPSCs and then work with the companies to test for potential drugs that could be used to treat these disorders.

In the article, Fred Gage explained that the goal of this project will be used to help patients rather than generate data points:

Rusty Gage, Salk Institute.

Rusty Gage, Salk Institute.

“Gage said the stem cell project is focused on getting results that make a difference to patients, not simply piling up research information. Being able to replicate results is critical; Gage said. Recent studies have found that many research findings of potential therapies don’t hold up in clinical testing. This is not only frustrating to patients, but failed clinical trials are expensive, and must be paid for with successful drugs.”

“The future of this will require more patients, replication between labs, and standardization of the procedures used.”

Clearing the first hurdle: spinal cord injury trial passes safety review

Jake 2

Jake Javier, participant in Asterias clinica trial

Starting a clinical trial is like taking a step into the unknown. It’s moving a potential therapy out of the lab and testing it in people. To reach this point the researchers have done a lot of work trying to ensure the therapy is safe. But that work was done in the lab, and on mice or other animals. Now it’s time to see what happens when you try it in the real world.

It can be quite nerve wracking for everyone involved: both the researchers, because years of hard work are at stake, and the patients, because they’re getting something that has never been tested in humans before; something that could, potentially, change their lives.

Today we got some good news about one clinical trial we are funding, the Asterias Biotherapeutics spinal cord injury trial. Asterias announced that its Data Monitoring Committee (DMC) has reviewed the safety data from the first two groups of patients treated and found no problems or bad side effects.

That’s an important first step in any clinical trial because it shows that, at the very least, the therapy is not going to make the patient’s condition any worse.

The big question now, is will it make their condition better? That’s something we’ll come back to at a later date when we have a better idea how the people treated in the trial are doing. But for now let’s take a deeper dive into the safety data.

Asterias – by the numbers

This current trial is a Phase 1/2a trial. The people enrolled have all experienced injuries in the C5-C7 vertebrae – that’s high up in the neck – and have essentially lost all feeling and movement below the injury site. All are treated between two weeks and one month after the injury was sustained.

The therapy involves transplants of Asterias’ AST-OPC1 cells which were made from human embryonic stem cells. The AST-OPC1 cells have been turned into oligodendrocyte progenitors, which are capable of becoming the kind of cells which help protect nerve cells in the central nervous system, the area damaged in spinal cord injury.

The first group of three patients in the Asterias trial was given 2 million cells. The second group of five patients received 10 million cells. The DMC said the safety data from those patients looked fine, that there were no signs of problems.

As Dr. Edward Wirth, the Chief Medical Officer at Asterias, said in a news release, this means the company can plan for its next phase:

“The positive safety data in the previous phase 1 study and in the ongoing phase 1/2a study gives us the confidence to now proceed to administration of 20 million cells, which based on our significant pre-clinical research is likely well within the dosing range where we would expect to see clinically meaningful improvement in these patients.”

Asterias is now looking to enroll 5-8 patients for this 20 million cell phase.

jake and family

For people like Jake Javier this news is not about numbers or data, it’s personal. Earlier this summer Jake broke his neck at a pool party, celebrating graduating from high school. It left him paralyzed from the chest down with extremely limited use of his arms and hands. On July 7th Jake was enrolled in the Asterias trial, and had ten million cells transplanted into his neck.

It could be months, even as much as one year, before we know if those cells are having any beneficial effect on Jake. But at least for now we know they don’t seem to be having any negative effects.

“First do no harm” is the cardinal rule that all budding physicians are taught. This trial seems to be meeting that benchmark. Our hope now is that it will do a lot more, and truly make a difference in the lives of people like Jake.

As Randy Mills, CIRM’s President and CEO, said in a news release:

“I recently met with Jake and heard first-hand what he and his family are going through in the aftermath of his injury. But I also saw a young man with remarkable courage and determination. It is because of Jake, and the others who volunteer to take part in clinical trials, that progress is possible. They are true heroes.”


* On a side note, Roman Reed, a great champion of stem cell research and a patient advocate extraordinaire, helped make much of this story happen. He helped Jake enroll in the Asterias trial ,and the research that led to this therapy was pioneered by Dr. Hans Keirstead who was funded by the Roman Reed Spinal Cord Injury Research Act.

 

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Seeing is believing: how some scientists – including two funded by CIRM – are working to help the blind see

retinitis pigmentosas_1

How retinitis pigmentosa destroys vision – new stem cell research may help reverse that

“A pale hue”. For most of us that is a simple description, an observation about color. For Kristin Macdonald it’s a glimpse of the future. In some ways it’s a miracle. Kristin lost her sight to retinitis pigmentosa (RP). For many years she was virtually blind. But now, thanks to a clinical trial funded by CIRM she is starting to see again.

Kristin’s story is one of several examples of restoring sight in an article entitled “Why There’s New Hope About Ending Blindness” in the latest issue of National Geographic.  The article explores different approaches to treating people who were either born without vision or lost their vision due to disease or injury.

Two of those stories feature research that CIRM has funded. One is the work that is helping Kristin. Retinitis pigmentosa is a relatively rare condition that destroys the photoreceptors at the back of the eye, the cells that actually allow us to sense light. The National Geographic piece highlights how a research team at the University of California, Irvine, led by Dr. Henry Klassen, has been working on a way to use stem cells to replace and repair the cells damaged by RP.

“Klassen has spent 30 years studying how to coax progenitor cells—former stem cells that have begun to move toward being specific cell types—into replacing or rehabilitating failed retinal cells. Having successfully used retinal progenitor cells to improve vision in mice, rats, cats, dogs, and pigs, he’s testing a similar treatment in people with advanced retinitis pigmentosa.”

We recently blogged about this work and the fact that this team just passed it’s first major milestone – – showing that in the first nine patients treated none experienced any serious side effects. A Phase 1 clinical trial like this is designed to test for safety, so it usually involves the use of relatively small numbers of cells. The fact that some of those treated, like Kristin, are showing signs of improvement in their vision is quite encouraging. We will be following this work very closely and reporting new results as soon as they are available.

The other CIRM-supported research featured in the article is led by what the writer calls “an eyeball dream team” featuring University of Southern California’s Dr. Mark Humayun, described as “a courteous, efficient, impeccably besuited man.” And it’s true, he is.

The team is developing a stem cell device to help treat age-related macular degeneration, the leading cause of vision loss in the US.

“He and his fellow principal investigator, University of California, Santa Barbara stem cell biologist Dennis Clegg, call it simply a patch. That patch’s chassis, made of the same stuff used to coat wiring for pacemakers and neural implants, is wafer thin, bottle shaped, and the size of a fat grain of rice. Onto this speck Clegg distributes 120,000 cells derived from embryonic stem cells.”

Humayun and Clegg have just started their clinical trial with this work so it is likely going to be some time before we have any results.

These are just two of the many different approaches, using several different methods, to address vision loss. The article is a fascinating read, giving you a sense of how science is transforming people’s lives. It’s also wonderfully written by David Dobbs, including observations like this:

“Neuroscientists love the eye because “it’s the only place you see the brain without drilling a hole,” as one put it to me.”

For a vision of the future, a future that could mean restoring vision to those who have lost it, it’s a terrific read.

 

A look back at the last year – but with our eyes firmly on the future

Randy

CIRM President & CEO Randy Mills doesn’t want “good”, he wants “better”

Better.

With that single word Randy Mills, our President and CEO, starts and ends his letter in our 2015 Annual Report and lays out the simple principle that guides the way we work at CIRM.

Better.

But better what?

“Better infrastructure to translate early stage ideas into groundbreaking clinical trials. Better regulatory practices to advance promising stem cell treatments more efficiently. Better treatments for patients in need.”

“Better” is also the standard everyone at CIRM holds themselves to. Getting better at what we do so we can fulfill our mission of accelerating stem cell treatments to patients with unmet medical needs.

The 2015 Annual Report highlights the achievements of the last year, detailing how we invested $135 million in 47 different projects at all levels of research. How our Board unanimously passed our new Strategic Plan, laying out an ambitious series of goals for the next five years from funding 50 new clinical trials, to creating a new regulatory process for stem cell therapies.

Snapshot of CIRM's 2015 Funding

The report offers a snapshot of where our money has gone this year, and how much we have left. It breaks down what percentage of our funding has gone to different diseases and how much we have spent on administration.

Jonathan Thomas, the Chair of our Board, takes a look back at where we started, 10 years ago, comparing what we did then (16 awards for a total of $12.5 million) to what we are doing today. His conclusion; we’re doing better.

But we still have a long way to go. And we are determined to get even better.

P.S. By the way we are changing the way we do our Annual Report. Our next one will come out on January 1, 2017. We figured it just made sense to take a look back at the last year as soon as the new year begins. It gives you a better (that word again) sense of what we did and where we  are heading. So look out for that, coming sooner than you think.

Here’s a new gene editing strategy to treat genetic blood disorders

If you’re taking a road trip across the country, you have a starting point and an ending point. How you go from point A to point B could be one of a million different routes, but the ultimate outcome is the same: reaching your final destination.

Yesterday scientists from St. Jude Children’s Research Hospital published exciting findings in the journal Nature Medicine on a new gene editing strategy that could offer a different route for treating genetic blood disorders such as sickle cell disease (SCD) and b-thalassemia.

The scientists used a gene editing tool called CRISPR. Unless you’ve been living under a rock, you’ve heard about CRISPR in the general media as the next, hot technology that could possibly help bring cures for serious diseases.

In simple terms, CRISPR acts as molecular scissors that facilitate cutting and pasting of DNA sequences at specific locations in the genome. For blood diseases like SCD and b-thalassemia, in which blood cells have abnormal hemoglobin, CRISPR gene editing offers ways to turn on and off genes that cause the clinical symptoms of these diseases.

Fetal vs. Adult hemoglobin

Before I get into the meat of this story, let’s take a moment to discuss hemoglobin. What is it? It’s a protein found in red blood cells that transports oxygen from the lungs to the rest of the body. Hemoglobin is made up of different subunits and the composition of these hemoglobin subunits change as newborns develop into adults.

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Healthy red blood cell (left), sickle cell (right).

Fetal hemoglobin (HbF) is comprised of a and g subunits while adult hemoglobin (HbA) is typically comprised of a and b subunits. Patients with SCD and b-thalassemia typically have mutations in the b globin gene. In SCD, this causes blood cells to take on an unhealthy, sickle cell shape that can clog vessels and eventually cause premature death. In b-thalassemia, the b-globin gene isn’t synthesized into protein at the proper levels and patients suffer from anemia (low red blood cell count).

One way that scientists are attempting to combat the negative side effects of mutant HbF is to tip the scales towards maintaining expression of the fetal g-globin gene. The idea spawned from individuals with hereditary persistence of fetal hemoglobin (HPFH), a condition where the hemoglobin composition fails to transition from HbF to HbA, leaving high levels of HbF in adult blood. Individuals who have HPFH and are predisposed to SCD or b-thalassemia amazingly don’t have clinical symptoms, suggesting that HbF plays either a protective or therapeutic role.

The current study is taking advantage of this knowledge in their attempt to treat blood disorders. Mitchell Weiss, senior author on the study and chair of the St. Jude Department of Hematology, explained the thought process behind their study:

“It has been known for some time that individuals with genetic mutations that persistently elevate fetal hemoglobin are resistant to the symptoms of sickle cell disease and beta-thalassemia, genetic forms of severe anemia that are common in many regions of the world. We have found a way to use CRISPR gene editing to produce similar benefits.”

CRISPRing blood stem cells for therapeutic purposes

Weiss and colleagues engineered red blood cells to have elevated levels of HbF in hopes of preventing symptoms of SCD. They used CRISPR to create a small deletion in a sequence of DNA, called a promoter, that controls expression of the hemoglobin g subunit 1 (HBG1) gene. The deletion elevates the levels of HbF in blood cells and closely mimics genetic mutations found in HPFH patients.

Weiss further explained the genome editing process in a news release:

Mitchell Weiss

Mitchell Weiss

“Our work has identified a potential DNA target for genome editing-mediated therapy and offers proof-of-principle for a possible approach to treat sickle cell and beta-thalassemia. We have been able to snip that DNA target using CRISPR, remove a short segment in a “control section” of DNA that stimulates gamma-to-beta switching, and join the ends back up to produce sustained elevation of fetal hemoglobin levels in adult red blood cells.”

The scientists genetically modified hematopoietic stem cells and blood progenitor cells from healthy individuals to make sure that their CRISPR gene editing technique was successful. After modifying the stem cells, they matured them into red blood cells in the lab and observed that the levels of HbF increased from 5% to 20%.

Encouraged by these results, they tested the therapeutic potential of their CRISPR strategy on hematopoietic stem cells from three SCD patients. While 25% of unmodified SCD blood stem cells developed red blood cells with a sickle cell shape under low-oxygen conditions (to induce stress), CRISPR edited SCD stem cells generated way fewer sickle cells (~4%) and had a higher level of HbF expression.

Many routes, one destination

The authors concluded that their genome editing technique is successful at switching hemoglobin expression from the adult form back to the fetal form. With further studies and safety testing, this strategy could be one day be developed into a treatment for patients with SCD and b-thalassemia

But the authors were also humble in their findings and admitted that there are many different genome editing strategies or routes for developing therapies for inherited blood diseases.

“Our results represent an additional approach to these existing innovative strategies and compare favorably in terms of the levels of fetal hemoglobin that are produced by our experimental system.”

My personal opinion is the more strategies thrown into the pipeline the better. As things go in science, many of these strategies won’t be successful in reaching the final destination of curing one of these diseases, but with more shots on goal, our chances of developing a treatment that works there are a lot higher.


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Dr. Deborah Deas joins CIRM Board

Deborah Deas has been appointed dean of the UCR School of Medicine

Deborah Deas, MD, MPH, UCR School of Medicine

Dr. Deborah Deas is clearly not someone who opts for the quiet life. If she were, she would have stayed home in Adams Run, the tiny town in rural South Carolina where she was born.

The website, NeighborhoodScout.com describes Adams Run (current population 1,492) as:

“One of the quietest neighborhoods in America. When you are here, you will find it to be very quiet. If quiet and peaceful are your cup of tea, you may have found a great place for you.”

Dr. Deas obviously wasn’t a tea drinker because she packed her bags and went off to college in Charleston. That was the first step on a journey that led the self-described “farmer’s daughter” to become an MD, then an MPH (Masters in Public Health), before assuming a leadership role at the Medical University of South Carolina (MUSC). More recently she headed to California’s Inland Empire where she was named the Dean and CEO for Clinical Affairs of the UC Riverside School of Medicine.

And now we are delighted to add to that list of achievements by announcing she is the newest member of the CIRM Board.

She was appointed to the Board by state Treasurer John Chiang who praised her for her:

“Passion to improve  health for underserved populations and to diversify the health care work force. She is committed to making the benefits of advanced medicine available to all Californians.”

 

In a news release our CIRM Board Chair, Jonathan Thomas, was equally fulsome in his praise and welcome to Dr. Deas.

 “We are delighted to have someone with Dr. Deas’ broad experience and expertise join us at CIRM. Her medical background and her commitment to diversity and inclusion are important qualities to bring to a Board that is striving to deliver stem cell treatments to patients, and to reflect the diversity of California.”

To say that she brings a broad array of skills and experience to the Board is something of an understatement. She is board certified in adult psychiatry, child and adolescent psychiatry and addiction psychiatry, and is widely regarded as a national leader in research into youth binge drinking, adolescent nicotine dependence, marijuana use and panic disorder, and pharmaceutical treatment of pediatric depressive disorder.

As if that wasn’t enough, she has also been named as one of the best doctors in the U.S. by U.S. News & World Report for the last eight years.

But the road to UC Riverside and CIRM hasn’t always been easy. In a first person perspective in Psychiatric News.

she said that at MUSC she was just one of two African Americans among the 500 residents in training:

“It was not uncommon for me to be mistaken by many for a social worker, a secretary, or a ward clerk despite wearing my white coat with Deborah Deas, M.D., written on it. This mistake was even made by some of my M.D. peers. I found that the best response was to ask, “And just why do you think I am a social worker?”

She says the lessons she learned from her parents and grandparents helped sustain her:

“They emphasized the importance of setting goals and keeping your eyes on the prize. Service was important, and the ways that one could serve were numerous. The notion that one should learn from others, as well as teach others, was as common as baked bread. My parents instilled in me that education is the key to a fruitful future and that it is something no one can take away from you.”

Her boss at UC Riverside, the Provost and Executive Vice Chancellor, Paul D’Anieri said Dr. Deas is a great addition to the CIRM Board:

“Deborah is a public servant at heart. Her own values and goals to help underserved patient populations align with the goals of CIRM to revolutionize medicine and bring new, innovative treatments to all patients who can benefit. I am confident that Dr. Deas’ service will have a lasting positive impact for CIRM and for the people of California.”

Dr. Deas ends her article in Psychiatric News saying:

“The farmer’s daughter has come a long way. I have stood on the shoulders of many, pushing forward with an abiding faith that there was nothing that I could not accomplish.”

She has indeed come a long way. We look forward to being a part of the next stage of her journey, and to her joining CIRM and bringing that “abiding faith” to our work.

 

 

Young Minds Shine Bright at the CIRM SPARK Conference

SPARK students take a group photo with CIRM SPARK director Karen Ring.

SPARK students take a group photo with CIRM SPARK director Karen Ring.

Yesterday was one of the most exciting and inspiring days I’ve had at CIRM since I joined the agency one year ago. We hosted the CIRM SPARK conference which brought together fifty-five high school students from across California to present their stem cell research from their summer internships.

The day was a celebration of their accomplishments. But it was also a chance for the students to hear from scientists, patient advocates, and clinicians about the big picture of stem cell research: to develop stem cell treatments and cures for patients with unmet medical needs.

Since taking on the role of the CIRM SPARK director, I’ve been blown away by the passion, dedication, and intelligence that our SPARK interns have shown during their short time in the lab. They’ve mastered techniques and concepts that I only became familiar with during my PhD and postdoctoral research. And even more impressive, they eloquently communicated their research through poster presentations and talks at the level of professional scientists.

During their internships, SPARK students were tasked with documenting their research experiences through blogs and social media. They embraced this challenge with gusto, and we held an awards ceremony to recognize the students who went above and beyond with these challenges.

I’d like to share the winning blogs with our readers. I hope you find them as inspiring and motivating as I do. These students are our future, and I look forward to the day when one of them develops a stem cell treatment that changes the lives of patients. 

Andrew Choi

Andrew Choi

Andrew Choi, Cedars-Sinai SPARK student

Am I crying or is my face uncontrollably sweating right now? I think I am doing both as I write about my unforgettable experiences over the course of the past 6 weeks and finalize my poster.

As I think back, I am very grateful for the takeaways of the research field, acquiring them through scientific journals, lab experiments with my mentor, and both formal and informal discourses. It seems impossible to describe all the episodes and occurrences during the program in this one blog post, but all I can say is that they were all unique and phenomenal in their own respective ways.

Gaining new perspectives and insights and being acquainted with many of the techniques, such as stereology, immunocytochemistry and immunohistochemistry my peers have utilized throughout their careers, proved to me the great impact this program can make on many individuals of the younger generation.

CIRM SPARK not only taught me the goings on behind the bench-to-bedside translational research process, but also morals, work ethics, and effective collaboration with my peers and mentors. My mentor, Gen, reiterated the importance of general ethics. In the process of making my own poster for the program, her words resonate even greater in me. Research, education, and other career paths are driven by proper ethics and will never continue to progress if not made the basic standard.

I am thankful for such amazing institutions: California Institute of Regenerative Medicine (CIRM) and Cedars-Sinai Medical Center for enabling me to venture out into the research career field and network. Working alongside with my fellow seven very brilliant friends, motivated me and made this journey very enjoyable. I am especially thankful my mentor, Gen, for taking the time to provide me with the best possible resources, even with her busy ongoing projects. She encouraged me to be the best that I am.

I believe, actually, I should say, I KNOW Cedars-Sinai’s CIRM SPARK program does a SUPERB and astounding job of cultivating life-long learners and setting exceptional models for the younger generation. I am hoping that many others will partake in this remarkable educational program.

I am overall very blessed to be part of a successful summer program. The end of this program does not mark the end of my passions, but sparks them to even greater heights.

Jamey Guzman

Jamey Guzman

Jamey Guzman, UC Davis SPARK student

When I found out about this opportunity, all I knew was that I had a fiery passion for learning, for that simple rush that comes when the lightbulb sputters on after an unending moment of confusion. I did not know if this passion would translate into the work setting; I sometimes wondered if passion alone would be enough to allow me to understand the advanced concepts at play here. I started at the lab nervous, tentative – was this the place for someone so unsure exactly what she wanted to be ‘when she grew up,’ a date now all too close on the horizon? Was I going to fit in at this lab, with these people who were so smart, so busy, people fighting for their careers and who had no reason to let a 16-year-old anywhere near experiments worth thousands of dollars in cost and time spent?

I could talk for hours about the experiments that I worked to master; about the rush of success upon realizing that the tasks now completed with confidence were ones that I had once thought only to belong to the lofty position of Scientist. I could fill pages and pages with the knowledge I gained, a deep and personal connection to stem cells and cell biology that I will always remember, even if the roads of Fate pull me elsewhere on my journey to a career.

The interns called the experience #CIRMSparkLab in our social media posts, and I find this hashtag so fitting to describe these last few months. While there was, of course, the lab, where we donned our coats and sleeves and gloves and went to work with pipets and flasks…There was also the Lab. #CIRMSparkLab is so much more than an internship; #CIRMSparkLab is an invitation into the worldwide community of learned people, a community that I found to be caring and vibrant, creative and funny – one which for the first time I can fully imagine myself joining “when I grow up.”

#CIRMSparkLab is having mentors who taught me cell culture with unerring patience and kindness. It is our team’s lighthearted banter across the biosafety cabinet; it is the stories shared of career paths, of goals for the present and the future. It is having mentors in the best sense of the word, trusting me, striving to teach and not just explain, giving up hours and hours of time to draw up diagrams that ensured that the concepts made so much sense to me.

#CIRMSparkLab is the sweetest ‘good-morning’ from scientists not even on your team, but who care enough about you to say hi, to ask about your projects, to share a smile. It is the spontaneity and freedom with which knowledge is dispensed: learning random tidbits about the living patterns of beta fish from our lab manager, getting an impromptu lecture about Time and the Planck Constant from our beloved professor as he passes us at lunch. It is getting into a passionate, fully evidence-backed argument about the merits of pouring milk before cereal that pitted our Stem Cell team against our Exosome team: #CIRMSparkLab is finding a community of people with whom my “nerdy” passion for learning does not leave me an oddball, but instead causes me to connect instantly and deeply with people at all ages and walks of life. And it is a community that, following the lead of our magnificent lab director, welcomed ten interns into their lab with open arms at the beginning of this summer, fully cognizant of the fact that we will break beakers, overfill pipet guns, drop gels, bubble up protein concentration assays, and all the while never stop asking, “Why? Why? Why? Is this right? Like this? WHY?”

I cannot make some sweeping statement that I now know at age 16 exactly what I want to do when I grow up. Conversely, to say I learned so much – or I am so grateful – or you have changed my life is simply not enough; words cannot do justice to those sentiments which I hope that all of you know already. But I can say this: I will never forget how I felt when I was at the lab, in the community of scientists. I will take everything I learned here with me as I explore the world of knowledge yet to be obtained, and I will hold in my heart everyone who has helped me this summer. I am truly a better person for having known all of you.

Thank you, #CIRMSparkLab. 

Adriana Millan

Adriana Millan

Adriana Millan, CalTech SPARK student

As children, we all grew up with the companionship of our favorite television shows. We enjoyed sitcoms and other animations throughout our childhood and even as adults, there’s no shame. The goofy and spontaneous skits we enjoyed a laugh over, yet we did not pay much attention to the lessons they attempted to teach us. As a child, these shows play crucial roles in our educational endeavors. We are immediately hooked and tune in for every episode. They spark curiosity, as they allow our imaginations to run wild. For me, that is exactly where my curiosity stemmed and grew for science over the years. A delusional young girl, who had no idea what the reality of science was like.

You expect to enter a lab and run a full day of experimentations. Accidentally mix the wrong chemicals and discover the cure for cancer. Okay, maybe not mix the incorrect chemicals together, I learned that in my safety training class. The reality is that working in a lab was far from what I expected — eye opening. Working alongside my mentor Sarah Frail was one of the best ways I have spent a summer. It was not my ideal summer of sleeping in until noon, but it was worthwhile.

My experience is something that is a part of me now. I talk about it every chance I get, “Mom, can you believe I passaged cells today!” It changed the way I viewed the principles of science. Science is one of the most valuable concepts on this planet, it’s responsible for everything and that’s what I have taken and construed from my mentor. She shared her passion for science with me and that completed my experience. Before when I looked at cells, I did not know exactly what I was supposed to observe. What am I looking at? What is that pink stuff you are adding to the plate?

However, now I feel accomplished. It was a bit of a roller coaster ride, with complications along the way, but I can say that I’m leaving this experience with a new passion. I am not just saying this to please the audience, but to express my gratitude. I would have never even looked into Huntington’s Disease. When I first arrived I was discombobulated. Huntington’s Disease? Now I can proudly say I have a grasp on the complexity of the disease and not embarrass my mentor my calling human cells bacteria – quite embarrassing in fact.  I’m a professional pipette handler, I work well in the hood, I can operate a microscope – not so impressive, I have made possibly hundreds of gels, I have run PCRs, and my cells love me, what else can I ask for.

If you are questioning what career path you are to take and even if it is the slightest chance it may be a course in science, I suggest volunteering in a lab. You will leave with your questioned answered. Is science for me? This is what I am leaving my experience with. Science is for me.

Other SPARK 2016 Awards

Student Speakers: Jingyi (Shelly) Deng (CHORI), Thomas Thach (Stanford)

Poster Presentations: Jerusalem Nerayo (Stanford), Jared Pollard (City of Hope), Alina Shahin (City of Hope), Shuling Zhang (UCSF)

Instagram Photos: Roxanne Ohayon (Stanford), Anna Victoria Serbin (CHORI), Diana Ly (UC Davis)

If you want to see more photos from the CIRM SPARK conference, check out our Instagram page @CIRM_Stemcells or follow the hashtag #CIRMSPARKLab on Instagram and Twitter.

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.