Next generation of stem cell scientists leave their mark

One of the favorite events of the year for the team here at CIRM is our annual SPARK (Summer Program to Accelerate Regenerative Medicine Knowledge) conference. This is where high school students, who spent the summer interning at world class stem cell research facilities around California, get to show what they learned. It’s always an engaging, enlightening, and even rather humbling experience.

The students, many of whom are first generation Californians, start out knowing next to nothing about stem cells and end up talking as if they were getting ready for a PhD. Most say they went to their labs nervous about what lay ahead and half expecting to do menial tasks such as rinsing out beakers. Instead they were given a lab coat, safety glasses, stem cells and a specific project to work on. They learned how to handle complicated machinery and do complex scientific experiments.

But most importantly they learned that science is fun, fascinating, frustrating sometimes, but also fulfilling. And they learned that this could be a future career for them.

We asked all the students to blog about their experiences and the results were extraordinary. All talked about their experiences in the lab, but some went beyond and tied their internship to their own lives, their past and their hopes for the future.

Judging the blogs was a tough assignment, deciding who is the best of a great bunch wasn’t easy. But in the end, we picked three students who we thought captured the essence of the SPARK program. This week we’ll run all those blogs.

We begin with our third place blog by Dayita Biswas from UC Davis.

Personal Renaissance: A Journey from Scientific Curiosity to Confirmed Passions

By Dayita Biswas

As I poured over the pages of my battered Campbell textbook, the veritable bible for any biology student, I saw unbelievable numbers like how the human body is comprised of over 30 trillion cells! Or how we have over 220 different types of cells— contrary to my mental picture of a cell as a circle. Science, and biology in particular, has no shortage of these seemingly impossible Fermi-esque statistics that make one do a double-take. 

My experience in science had always been studying from numerous textbooks in preparation for a test or competitions, but textbooks only teach so much. The countless hours I spent reading actually demotivated me and I constantly asked myself what was the point of learning about this cycle or that process — the overwhelming “so what?” question. Those intriguing numbers that piqued my interest were quickly buried under a load of other information that made science a static stream of words across a page. 

That all changed this summer when I had the incredible opportunity to work in the Nolta lab under my mentor, Whitney Cary. This internship made science so much more tangible and fun to be a part of.  It was such an amazing environment, being in the same space with people who all have the same goals and passion for science that many high school students are not able to truly experience. Everyone was so willing to explain what they were doing, and even went out of their way to help if I needed papers or had dumb questions.

This summer, my project was to create embryoid bodies and characterize induced pluripotent stem cells (iPSCs) from children who had Jordan’s Syndrome, an extremely rare neurodevelopmental disease whose research has applications in Alzheimer’s and autism.

 I had many highs and lows during this research experience. My highs were seeing that my iPSCs were happy and healthy. I enjoyed learning lab techniques like micro-pipetting, working in a biological safety hood, feeding, freezing, and passaging cells. My lows were having to bleach my beloved iPSCs days after they failed to survive, and having unsuccessful protocols. However, while my project consistently failed, these failures taught me more than my successes.

I learned that there is a large gap between being able to read about techniques and being “book smart” and actually being able to think critically about science and perform research. Science, true science, is more than words on a page or fun facts to spout at a party. Science is never a straight or easy answer, but the mystery and difficulty is part of the reason it is so interesting. Long story short: research is hard and it takes time and patience, it involves coming in on weekends to feed cells, and staying up late at night reading papers.         

The most lasting impact that this summer research experience had was that everything we learn in school and the lab are all moving us towards the goal of helping real people. This internship renewed my passion for biology and cemented my dream of working in this field. It showed me that I don’t have to wait to be a part of dynamic science and that I can be a small part of something that will change, benefit, and save lives.

This internship meant being a part of something bigger than myself, something meaningful. We must always think critically about what consequences our actions will have because what we do as scientists and researchers— and human beings will affect the lives of real people. And that is the most important lesson anyone can hope to learn.

                                                                                                   

And here’s a bonus, a video put together by the SPARK students at Cedars-Sinai Medical Center.

Join us tomorrow at noon for “Ask the Stem Cell Team about Sickle Cell Disease”, a FaceBook Live Event

As an early kick off to National Sickle Cell Awareness Month – which falls in September every year – CIRM is hosting a “Ask the Stem Cell Team” FaceBook Live event tomorrow, August 28th, from noon to 1pm (PDT).

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The live broadcast will feature two scientists and a patient advocate who are working hard to bring an end to sickle cell disease, a devastating, inherited blood disorder that largely targets the African-American community and to a lesser degree the Hispanic community.

You can join us by logging onto Facebook and going to this broadcast link: https://bit.ly/2o4aCAd

Also, make sure to “like” our FaceBook page before the event to receive a notification when we’ve gone live for this and future events. If you miss tomorrow’s broadcast, not to worry. We’ll be posting it on our Facebook video page, our website, and YouTube channel shortly afterwards.

We want to answer your most pressing questions, so please email them directly to us beforehand at info@cirm.ca.gov.

For a sneak preview here’s a short video featuring our patient advocate speaker, Adrienne Shapiro. And see below for more details about Ms. Shapiro and our two other guests.

Adrienne Shapiro [Video: Todd Dubnicoff/CIRM]

  • Dr. Donald B. KohnUCLA MIMG BSCRC Faculty 180118

    Donald Kohn, MD

    Don Kohn, M.D. is a professor in the departments of Pediatrics and Microbiology, Immunology and Molecular Genetics in UCLA’s Broad Stem Cell Research Center. Dr. Kohn has a CIRM Clinical Stage Research grant in support of his team’s Phase 1 clinical trial which is genetically modifying a patient’s own blood stem cells to produce a correct version of hemoglobin, the protein that is mutated in these patients, which causes abnormal sickle-like shaped red blood cells. These misshapen cells lead to dangerous blood clots, debilitating pain and even death. The genetically modified stem cells will be given back to the patient to create a new sickle cell-free blood supply.

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    Mark Walters, MD

    Mark Walters, M.D., is a pediatric hematologist/oncologist and is director of the Blood & Marrow Transplantation Program at UCSF Benioff Children’s Hospital Oakland. Dr. Walters has a CIRM-funded Therapeutic Translation Research grant which aims to improve Sickle Cell Disease (SCD) therapy by preparing for a clinical trial that might cure SCD after giving back sickle gene-corrected blood stem cells – using cutting-edge CRISPR gene editing technology – to a person with SCD. If successful, this would be a universal life-saving and cost-saving therapy.

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    Adrienne Shapiro

    Adrienne Shapiro is a patient advocate for SCD and the co-founder of the Axis Advocacy SCD patient education and support website. Shapiro is the fourth generation of mothers in her family to have children born with sickle cell disease.  She is vocal stem cell activist, speaking to various groups about the importance of CIRM’s investments in both early stage research and clinical trials. In January, she was awarded a Stem Cell and Regenerative Medicine Action Award at the 2018 World Stem Cell Summit.

The Five Types of Stem Cells

When I give an “Intro to Stem Cells” presentation to, say, high school students or to a local Rotary Club, I begin by explaining that there are three main types of stem cells: (1) embryonic stem cells (ESCs) (2) adult stem cells and (3) induced pluripotent stem cells (iPSCs). Well, like most things in science, it’s actually not that simple.

To delve a little deeper into the details of characterizing stem cells, I recommend checking out a video animation produced by BioInformant, a stem cell market research company. The video is introduced in a blog, “Do you know the 5 types of stem cells?” by Cade Hildreth, BioInformant’s founder and president.

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Image credit: BioInformant

Hildreth’s list categorizes stem cells by the extent of each type’s shape-shifting abilities. So while we sometimes place ESCs and iPSCs in different buckets because the methods for obtaining them are very different, in this list, they both belong to the pluripotent stem cell type. Pluri (“many”) – potent (“potential”) refers to the ability of both stem cell types to specialize into all of the cell types in the body. They can’t, though, make the cells of the placenta and other extra-embryonic cells too. Those ultimate blank-slate stem cells are called toti (“total”) – potent (“potential”).

When it comes to describing adult stem cells in my talks, I often lump blood stem cells together with muscle stem cells because they are stem cells that are present within us throughout life. But based on their ability to mature into specialized cells, these two stem cell types fall into two different categories in Hildreth’s list:  blood stem cells which can specialize into closely related cell types – the various cell types found in the blood – are considered “oligopotent” while muscle stem cells are “unipotent” because the can only mature into one type of cell, a muscle cell.

For more details on the five types of stem cells based on their potential to specialize, head over to the BioInformant blog. And scroll to the very bottom for the video animation which can also viewed on FaceBook.

Video: Behind the scenes of a life-saving gene therapy stem cell treatment

“We were so desperate. When we heard about this treatment were willing to do anything to come here.”

In the above quote from Zahraa El Kerdi, “here” refers to UCLA, a world away from her hometown in Lebanon. In September 2015, Zahree gave birth to a son, Hussein, who appeared perfectly healthy. But by six months, he was barely clinging to life due to an inherited blood disorder, ADA-SCID, also called Bubble Baby disease. The disorder left Hussein without a functioning immune system so even a common cold could prove deadly. In fact, SCID babies rarely survive past one year of age. Up until now, no treatment options existed for the disease.

But Zahraa and her husband Ali heard about a CIRM-funded clinical trial, led by Donald Kohn, M.D. at UCLA, that could modify Hussein’s blood stem cells to fix the gene problem that’s causing his disease. The El Kerdi’s 7500-mile journey to save Hussein’s life is captured in a wonderful, five-minute video produced by UCLA’s Broad Stem Cell Research Center.

With before and after scenes of Hussein’s treatment as well as animation describing how the therapy works, the short documentary is equal parts heart wrenching, uplifting and educational. Basically, what I’m trying to say is, it’s a must-see and available to view above.

World Sickle Cell Day: Managing the disease today for tomorrow’s stem cell cures

Today is World Sickle Cell Day, a day to promote awareness about sickle cell disease (SCD), an inherited, chronic blood disorder which can cause severe pain, stroke, organ failure, and other complications, including death. Sadly, it’s estimated that this year 300,000 babies around the world will be born with SCD.

To recognize World Sickle Cell Day, we’re sharing a one-minute clip from a video interview we filmed last week with Adrienne Shapiro, a tireless advocate for sickle cell patients and the development of stem cell-based cures.

Shapiro, the fifth generation of mothers in her family to have a child born with SCD, is co-founder of Axis Advocacy, a Southern California organization whose mission is to improve the lives of patients and caregivers who are dealing with this chronic illness.

In the video, Shapiro says that just the promise of stem cell-based therapies for SCD, “relieves that pain and suffering and guilt of having passed this (inherited disorder) along as well as knowing that I can really be the last mother, the last generation to fight for my child’s life.”

Speaking of stem cell therapies, CIRM is currently funding two clinical trials related to SCD. A UCLA team is testing a stem cell and gene therapy product from the patient’s own blood to correct the mutation that causes the production of abnormal, sickle-like shaped red blood cells. And City of Hope scientists are testing a novel blood stem cell transplant procedure that uses a milder, less toxic chemotherapy treatment that allows donor stem cells to engraft and create a healthy supply of non-diseased blood cells without causing an immune reaction in the patient.

While Shapiro’s Axis Advocacy and CIRM provide critical support here in California, other organizations like the American Society of Hematology and the Sickle Cell Disease Coalition have their efforts set on the developing world, particularly in sub-Saharan Africa, where an estimated 50–90 percent of infants born with SCD will die before their fifth birthday.

To do something about this heartbreaking statistic, these organization are debuting a public service announcement and short documentary – watch the video playlist below – to help improve newborn screening and early care for children in Africa living with sickle cell disease.

As Shapiro explained to us during her interview, it’s important to provide the support and education needed to manage the disease so that when the cure comes, the patients will be alive to receive it.

CCSF’s CIRM Bridges scholars: the future of stem cell research is in good hands

In need of an extra dose of inspiration? You might read a great book or listen to that podcast your friend recommended. You might even take a stroll along the beach. But I can do you one better: go to a conference poster session where young stem cell scientists describe their research.

That’s what I did last week at the City College of San Francisco’s (CCSF) Bioscience Symposium held at UC San Francisco’s Genentech Hall. It’s a day-long conference that showcases the work of CCSF Bioscience interns and gives them a chance to present the results of their research projects, network with their peers and researchers, hear panelists talk about careers in biotechnology and participate in practice job interviews.

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CCSF’s CIRM Bridges Scholars (clockwise from top left): Vanessa Lynn Herrara, Viktoriia Volobuieva, Christopher Nosworthy and Sofiana E. Hamama.

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CCSF’s CIRM Bridges Scholars (clockwise from top left): Seema Niddapu, Mark Koontz, Karolina Kaminska and Iris Avellano

Eight of the dozens of students in attendance at the Symposium are part of the CIRM-funded Bridges Stem Cell Internship program at CCSF. It’s one of 14 CIRM Bridges programs throughout the state that provides paid stem cell research internships to students at universities and colleges that don’t have major stem cell research programs. Each Bridges internship includes thorough hands-on training and education in stem cell research, and direct patient engagement and outreach activities that engage California’s diverse communities.

In the CCSF Bridges Program, directed by Dr. Carin Zimmerman, the students do a 9-month paid internship in top notch labs at UCSF, the Gladstone Institutes and Blood System Research Institute. As I walked from poster to poster and chatted with each Bridges scholar, their excitement and enthusiasm for carrying out stem cell research was plain to see. It left me with the feeling that the future of stem cell research is in good hands and, as I walked into the CIRM office the next day, I felt re-energized to tackle the Agency’s mission to accelerate stem cell treatment for patients with unmet medical needs. But don’t take my word for it, listen to the enthusiastic perspectives of Bridges scholars Mark Koontz and Iris Avellano in this short video.

The moment of truth. A video about the stem cell therapy that could help millions of people going blind.

“No matter how much one prepares, the first patient is always something very special.” That’s how Dr. Mark Humayun describes his feelings as he prepared to deliver a CIRM-funded stem cell therapy to help someone going blind from dry age-related macular degeneration (AMD).

Humayun, an ophthalmologist and stem cell researcher at USC, spent years developing this therapy and so it’s understandable that he might be a little nervous finally getting a chance to see if it works in people.

It’s quite a complicated procedure, involving turning embryonic stem cells into the kind of cells that are destroyed by AMD, placing those cells onto a specially developed synthetic scaffold and then surgically implanting the cells and scaffold onto the back of the eye.

There’s a real need for a treatment for AMD, the leading cause of vision loss in the US. Right now, there is no effective therapy for AMD and some three million Americans are facing the prospect of losing their eyesight.

The first, preliminary, results of this trial were released last week and they were encouraging. You can read about them on our blog.

Thanks to USC you can also see the team that developed and executed this promising approach. They created a video capturing the moment the team were finally taking all that hard work and delivering it where it matters, to the patient.

Watching the video it’s hard not to think you are watching a piece of history, something that has the potential to do more than just offer hope to people losing their vision, it has the potential to stop and even reverse that process.

The video is a salute to the researchers who developed the therapy, and the doctors, nurses and Operating Room team who delivered it. It’s also a salute to the person lying down, the patient who volunteered to be the first to try this. Everyone in that room is a pioneer.

Video illustrates potential path to stem cell repair for multiple sclerosis

“Can you imagine slowly losing the ability to live life as you know it? To slowly lose the ability to see, to walk, to grab an object, all the while experiencing pain, fatigue and depression?”

These sobering questions are posed at the beginning of a recent video produced by Youreka Science and Americans for Cures about multiple sclerosis (MS), a debilitating neurodegenerative disorder in which a person’s own immune system attacks cells that are critical for sending nerve signals from the brain and spinal cord to our limbs and the rest of our body.

In recognition of Multiple Sclerosis Awareness Week, today’s blog features this video. Using an easy to understand narrative and engaging hand-drawn illustrations, this whiteboard “explainer” video does a terrific job of describing the biological basis of multiple sclerosis. It also highlights promising research out of UC Irvine showing that stem cell-based therapies may one day help repair the damage caused by multiple sclerosis.

But don’t take my word for it, check out the five-minute video below:

Related Links:

Just a Mom: The Journey of a Sickle Cell Disease Patient Advocate [video]

Adrienne Shapiro will tell you that she’s just a mom.

And it’s true. She is just a mom. Just a mom who is the fourth generation of mothers in her family to have children born with sickle cell disease. Just a mom who was an early advocate of innovative stem cell and gene therapy research by UCLA scientist Dr. Don Kohn which has led to an on-going, CIRM-funded clinical trial for sickle cell disease. Just a mom who is the patient advocate representative on a Clinical Advisory Panel (CAP) that CIRM is creating to help guide this clinical trial.

She’s just a mom who has become a vocal stem cell activist, speaking to various groups about the importance of CIRM’s investments in both early stage research and clinical trials. She’s just a mom who was awarded a Stem Cell and Regenerative Medicine Action Award at last month’s World Stem Cell Summit. She’s just a mom who, in her own words, “sees a new world not just for her children but for so many other children”, through the promise of stem cell therapies.

Yep, she’s just a mom. And it’s the tireless advocacy of moms like Adrienne that will play a critical role in accelerating stem cell therapies to patients with unmet medical needs. We can use all the moms we can get.

Adrienne Shapiro speaks to the CIRM governing Board about her journey as a patient advocate

Patient’s Stage IV Cancer Held in Check by CIRM-Funded Clinical Trial [Video]

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Tom Howing

“In the last three scans, which I have every six weeks, they’re showing that there is no mestastasis (invasive cancer) anywhere in my body. [The doctors] I guess were quite blown away because they didn’t expect [the treatment] to be so quick or to be that complete.”

 

Today we’re sharing the story of Tom Howing, who took part in Forty Seven, Inc.’s CIRM-funded clinical trial that’s testing an innovative treatment for cancer.

The two-minute video below sums up Tom’s address to CIRM’s governing Board back in December. During his talk, he gave a personal perspective on his cancer diagnosis, the promising but ultimately disappointing results of standard anti-cancer treatments and the remarkable results he’s experienced from Forty Seven’s clinical trial.

Tom’s story is featured in our 2017 Annual Report (page 18), now available on our website.