CIRM Board Appoints Dr. Maria Millan as President and CEO

Dr. Maria Millan, President and CEO of CIRM, at the September Board meeting. (Todd Dubnicoff, CIRM)

Yesterday was a big day for CIRM. Our governing Board convened for its September ICOC meeting and appointed Dr. Maria Millan as our new President and CEO. Dr. Millan has been serving as the Interim President/CEO since July, replacing former President Dr. Randal Mills.

Dr. Millan has been at CIRM since 2012 and was instrumental in the development of CIRM’s infrastructure programs including the Alpha Stem Cell Clinics Network and the agency’s Strategic Plan, a five-year plan that lays out our agency’s goals through 2020. Previously, Dr. Millan was the Vice President of Therapeutics at CIRM, helping the agency fund 23 new clinical trials since the beginning of 2016.

The Board vote to appoint Dr. Millan as President and CEO was unanimous and enthusiastic. Chairman of the Board, Jonathan Thomas, shared the Board’s sentiments when he said,

“Dr. Millan is absolutely the right person for this position. Having seen Dr. Millan as the Interim CEO of CIRM for three months and how she has operated in that position, I am even more enthusiastic than I was before. I am grateful that we have someone of Maria’s caliber to lead our Agency.”

Dr. Millan has pursued a career devoted to helping patients. Before working at CIRM, she was an organ transplant surgeon and researcher and served as an Associate Professor of Surgery and Director of the Pediatric Organ Transplant Program at Stanford University. Dr. Millan was also the Vice President and Chief Medical Officer at StemCells, Inc.

In her permanent role as President, Dr. Millan is determined to keep CIRM on track to achieve the goals outlined in our strategic plan and to achieve its mission to accelerate treatments to patients with unmet needs. She commented in a CIRM press release,

“I joined the CIRM team because I wanted to make a difference in the lives of patients. They are the reason why CIRM exists and why we fund stem cell research. I am humbled and very honored to be CIRM’s President and look forward to further implementing our agency’s Strategic Plan in the coming years.”

The Board also voted to fund two new Alpha Stem Cell Clinics at UC Davis and UC San Francisco and five new clinical trials. Three of the clinical awards went to projects targeting cancer.

The City of Hope received $12.8 million to fund a Phase 1 trial targeting malignant gliomas (an aggressive brain cancer) using CAR-T cell therapy. Forty Seven Inc. received $5 million for a Phase 1b clinical trial treating acute myeloid leukemia. And Nohla Therapeutics received $6.9 million for a Phase 2 trial testing a hematopoietic stem cell and progenitor cell therapy to help patients suffering from neutropenia, a condition that leaves people susceptible to deadly infections, after receiving chemotherapy for acute myeloid leukemia.

The other two trials target diabetes and end stage kidney failure. ViaCyte, Inc. was awarded $20 million to fund a Phase 1/2 clinical trial to test its PEC-Direct islet cell replacement therapy for high-risk type 1 diabetes. Humacyte Inc. received $14.1 million to fund a Phase 3 trial that is comparing the performance of its acellular bioengineered vessel with the current standard of dialysis treatment for kidney disease patients.

The Board also awarded $5.2 million to Stanford Medicine for a late stage preclinical project that will use CRISPR gene editing technology to correct the sickle cell disease mutation in blood-forming stem cells to treat patients with sickle cell disease. This award was particularly well timed as September is Sickle Cell Awareness month.

The Stanford team, led by Dr. Matthew Porteus, hopes to complete the final experiments required for them to file an Investigational New Drug (IND) application with the FDA so they can be approved to start a clinical trial hopefully sometime in 2018. You can read more about Dr. Porteus’ work here and you can read our past blogs featuring Sickle Cell Awareness here and here.

With the Board’s vote yesterday, CIRM’s clinical trial count rises to 40 funded trials since its inception. 23 of these trials were funded after the launch of our Strategic Plan bringing us close to the half way point of funding 50 new clinical trials by 2020. With more “shots-on-goal” CIRM hopes to increase the chances that one of these trials will lead to an FDA-approved therapy for patients.


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CIRM Bridges Student Researcher Discovers Mentoring is a Two-Way Street

Jasmine Carter is a CIRM Bridges Scholar a Sacramento State University. She currently is interning in the lab of Dr. Kyle Fink at UC Davis and her research focuses on developing induced neurons from skin cells to model neurological disorders and develop novel therapeutics. Jasmine was a mentor to one of our UC Davis CIRM SPARK high school students this summer, and we asked her to share her thoughts on the importance of mentorship in science.

I began my scientific journey as an undergraduate student in the biomedical sciences, determined to get into medical school to become a surgeon. But I was perpetually stressed, always pushing towards the next goal and never stopping to smell the roses. Until one day, I did stop because a mentor encouraged me to figure out how I wanted to contribute to the medical field. In the midst of contemplating this important question, I was offered an undergraduate research position studying stem cells. It wasn’t long before I realized I had found my calling. Those little stem cells were incredibly fascinating to me, and I really enjoyed my time in a research lab. Being able to apply my scientific knowledge at the lab bench and challenge myself to solve biological problems was truly enjoyable to me so I applied to and was accepted into Sacramento State’s CIRM Bridges Program.

Jasmine working with stem cells in the cell culture hood.

To say I was excited to learn more about stem cell biology would be an understatement. I started volunteering in the Translational Research Lab at the Institute for Regenerative Cures at UC Davis as soon as I could. And I started to feel way outside my comfort zone as I walked into the lab because the seemingly endless rows of research benches and all the lab equipment can be a lot to take in when you first begin your research journey. When I started to actually run experiments, I worried that I may have messed the experiment up. I worried that I might SAY or DO something that would make me appear less intelligent because everyone was so knowledgeable. I struggled with figuring out whether or not I was cut out for the research environment.

I have now started my formal research internship and am constantly amazed at the mentorship I receive and collaboration I witness every day; everyone is always willing to lend a helping hand or simply be a sounding board for ideas. I have learned an immense amount of knowledge about stem cell research and its potential to improve knowledge for the scientific community and treatment options for patients. But I would not have had the opportunity to grow as an intern and learn from experts in various disciplines if it were not for the CIRM Bridges Program. The Bridges Program has allowed me to apply basic biological principles as I learn about stem cell biology and the applications of stems cells while completing a Master’s research project. Diving into the research environment has been challenging at times, but guidance from knowledgeable and encouraging mentors in the Translational Research Laboratory has helped to shape me into a more confident researcher.

Jasmine and Yasmine.

As fate would have it, just as I was becoming more and more confident in myself as a researcher, I found myself becoming a mentor to our CIRM SPARK high school intern, Yasmine. During Yasmine’s first week, I saw the exact same feelings of doubt on her face that I had experienced when I first volunteered in the laboratory. I saw how she challenged herself to absorb and understand every word and concept we said to her. I saw that familiar worried expression she’d displayed when unsure if she just messed up on an experiment or the hesitation when trying to figure out if the question she was about to ask was the “right” one. Because I had faced the same struggles, I could assure her that the internship was a learning experience and that each success and setback she encountered while working on her project would make her a better scientist.

During Yasmine’s eight-week summer internship, she observed and helped members of our team on various experiments while conducting her own research project. At the end of the first week, Yasmine commented on how diligent all the researchers in the lab were; how she hadn’t known the amount of effort and work that’s required to develop and complete a research project. Yasmine’s project focused on optimizing the protocols, or recipes, for editing genes in different types of cells for use as potential treatments for neurological disorders. Many days, you’d find Yasmine peering into the microscope and imaging cells – for her project or one of ours. Being able to visually assess the success of our experiments was exciting for her. The time we spent trying to track down just one fluorescent cell was a great opportunity for us to review the experiment and brainstorm the next set of experiments we wanted to run. I enjoyed explaining the science behind the experiments we set up, and Yasmine’s thought provoking questions sometimes led to a learning session where we figured out the answer together. Yasmine even used the knowledge she was acquiring in a graduate level Good Manufacturing Practice (GMP) course to explain her flow cytometry results to our team during a lab meeting.

Yasmine at the microscope.

It was actually during one of these lab meetings when I was practicing my poster presentation for the 2017 Annual CIRM Bridges Trainee Meeting when Yasmine said, “I finally understand your project”. She and I had frequently discussed my project, but towards the end of the internship she was integrating what she learned in lectures, whiteboard review sessions and scientific papers to the research we were doing at the lab bench. It was incredibly gratifying to see how much she had learned and how her confidence as a young scientist grew while she interned with us. The internship was an invaluable experience for Yasmine because it helped to reinforce her commitment to improving the lives of patients who suffer from brain cancer. She hopes to use the research skills that the SPARK program provided to seek out research opportunities in college.

But the learning wasn’t one-sided this summer because I was also learning from Yasmine. The CIRM SPARK students are encouraged to document their internship on social media. And with Yasmine’s encouragement, I have started to document my experiences in the Bridges program by showing what the day to day life of a graduate student looks like, what experiments are going well and how I am trouble-shooting the failed experiments. Sometimes those failed experiments can be discouraging, but taking the time to discuss it with a mentor, mentee or an individual on social media can help me to figure out how I should change the experiment. So, when self-doubt sprouted back up as I began to document my experiences in the program, I reminded myself that being pushed outside my comfort zone is a great way to learn. But one of the greatest lessons I learned from Yasmine’s summer internship is the importance of sharing in a mentor-mentee relationship. After sharing my knowledge with Yasmine, I got to watch her confidence shine when she took the reins with experiments and then shared the fruits of her labor with me.

There can be a lot of ups and downs in research. However, opportunities for mentorship and learning with such bright, enthusiastic and dedicated students has certainly validated the importance of the CIRM Bridges and SPARK programs. The mentorship and collaboration that occurs between high school interns, undergraduates, graduate students, post-docs and principal investigators to develop therapies for patients with unmet medical needs is truly amazing.

Mentorship leads to productive careers and friendships.

Jasmine Carter is also an avid science communicator. You can follow her science journey on Instagram and Twitter.

Stem Cell Stories That Caught Our Eye: Halting Brain Cancer, Parkinson’s disease and Stem Cell Awareness Day

Stopping brain cancer in its tracks.

Experiments by a team of NIH-funded scientists suggests a potential method for halting the expansion of certain brain tumors.Michelle Monje, M.D., Ph.D., Stanford University.

Scientists at Stanford Medicine discovered that you can halt aggressive brain cancers called high-grade gliomas by cutting off their supply of a signaling protein called neuroligin-3. Their research, which was funded by CIRM and the NIH, was published this week in the journal Nature. 

The Stanford team, led by senior author Michelle Monje, had previously discovered that neuroligin-3 dramatically spurred the growth of glioma cells in the brains of mice. In their new study, the team found that removing neuroligin-3 from the brains of mice that were transplanted with human glioma cells prevented the cancer cells from spreading.

Monje explained in a Stanford news release,

“We thought that when we put glioma cells into a mouse brain that was neuroligin-3 deficient, that might decrease tumor growth to some measurable extent. What we found was really startling to us: For several months, these brain tumors simply didn’t grow.”

The team is now exploring whether targeting neuroligin-3 will be an effective therapeutic treatment for gliomas. They tested two inhibitors of neuroligin-3 secretion and saw that both were effective in stunting glioma growth in mice.

Because blocking neuroligin-3 doesn’t kill glioma cells and gliomas eventually find ways to grow even in the absence of neuroligin-3, Monje is now hoping to develop a combination therapy with neuroligin-3 inhibitors that will cure patients of high-grade gliomas.

“We have a really clear path forward for therapy; we are in the process of working with the company that owns the clinically characterized compound in an effort to bring it to a clinical trial for brain tumor patients. We will have to attack these tumors from many different angles to cure them. Any measurable extension of life and improvement of quality of life is a real win for these patients.”

Parkinson’s Institute CIRM Research Featured on KTVU News.

The Bay Area Parkinson’s Institute and Clinical Center located in Sunnyvale, California, was recently featured on the local KTVU news station. The five-minute video below features patients who attend the clinic at the Parkinson’s Institute as well as scientists who are doing cutting edge research into Parkinson’s disease (PD).

Parkinson’s disease in a dish. Dopaminergic neurons made from PD induced pluripotent stem cells. (Image courtesy of Birgitt Schuele).

One of these scientists is Dr. Birgitt Schuele, who recently was awarded a discovery research grant from CIRM to study a new potential therapy for Parkinson’s using human induced pluripotent stem cells (iPSCs) derived from PD patients. Schuele explains that the goal of her team’s research is to “generate a model for Parkinson’s disease in a dish, or making a brain in a dish.”

It’s worth watching the video in its entirety to learn how this unique institute is attempting to find new ways to help the growing number of patients being diagnosed with this degenerative brain disease.

Click on photo to view video.

Mark your calendars for Stem Cell Awareness Day!

Every year on the second Wednesday of October is Stem Cell Awareness Day (SCAD). This is a day that our agency started back in 2009, with a proclamation by former California Mayor Gavin Newsom, to honor the important accomplishments made in the field of stem cell research by scientists, doctors and institutes around the world.

This year, SCAD is on October 11th. Our Agency will be celebrating this day with a special patient advocate event on Tuesday October 10th at the UC Davis MIND Institute in Sacramento California. CIRM grantees Dr. Jan Nolta, the Director of UC Davis Institute for Regenerative Cures, and Dr. Diana Farmer, Chair of the UC Davis Department of Surgery, will be talking about their CIRM-funded research developing stem cell models and potential therapies for Huntington’s disease and spina bifida (a birth defect where the spinal cord fails to fully develop). You’ll also hear an update on  CIRM’s progress from our President and CEO (Interim), Maria Millan, MD, and Chairman of the Board, Jonathan Thomas, PhD, JD. If you’re interested in attending this event, you can RSVP on our Eventbrite Page.

Be sure to check out a list of other Stem Cell Awareness Day events during the month of October on our website. You can also follow the hashtag #StemCellAwarenessDay on Twitter to join in on the celebration!

One last thing. October is an especially fun month because we also get to celebrate Pluripotency Day on October 4th. OCT4 is an important gene that maintains stem cell pluripotency – the ability of a stem cell to become any cell type in the body – in embryonic and induced pluripotent stem cells. Because not all stem cells are pluripotent (there are adult stem cells in your tissues and organs) it makes sense to celebrate these days separately. And who doesn’t love having more reasons to celebrate science?

Stem cell treatment helps puppies born with spina bifida walk again

Just when you thought puppies couldn’t get any cuter, this video appears in your twitter feed.

These adorable English bulldog puppies are named Darla and Spanky, and they were born with a birth defect called spina bifida where the bones and tissue surrounding the spinal cord fail to fuse completely. Spina bifida occurs in 1500-2000 children in the US each year and can cause serious problems such as paralysis and issues with walking, cognition, and bladder or bowel control. Dogs born with this condition usually cannot use their hind legs, and as a sad consequence, are typically put down at a young age.

Cutting edge research from UC Davis is now giving these unfortunate puppies hope. Diana Farmer, a fetal surgeon at UC Davis Health, and scientists from the university’s Veterinary Institute for Regenerative Cures have developed a combination surgery and stem cell transplant, using placenta-derived mesenchymal stromal cells (PMSCs), to treat puppies with spina bifida. Because prenatal screening for spina bifida is not done in dogs, Darla and Spanky received the treatment when they were ten weeks old.

With funding from a CIRM preclinical development award, Farmer has done similar surgeries in lambs that are still in the womb. A UC Davis news release provided historical background on Farmer’s work on spina bifida,

“Farmer pioneered the use of surgery prior to birth to improve brain development in children with spina bifida. She later showed that prenatal surgery combined with human placenta-derived mesenchymal stromal cells (PMSCs), held in place with a cellular scaffold, helped research lambs born with the disorder walk without noticeable disability.”

As you can see from the video, the surgeries were a success. Darla and Spanky are now able to live up to their full puppy potential and will live happily ever after with their adoptive family in New Mexico.

Looking forward, Farmer and her team would like to treat more dogs with spina bifida so they can improve another negative consequence of spina bifida called incontinence, or an uncontrollable bladder. The UC Davis release explained that, “while Darla and Spanky are very mobile and doing well on their feet, they still require diapers.” (Side note: this video proves that puppies can make anything look cute, even dirty diapers.)

Additionally, the team is hoping to receive regulatory approval from the US Food and Drug Administration to launch a clinical trial testing this therapy in humans. If this stem cell treatment proves to be both safe and effective in clinical trials, it could potentially prevent spina bifida from ever happening in animals and in humans.

English Bulldog undergoing spina bifida surgery at UC Davis Veterinary Medical Teaching Hospital. (Gregory Urquiaga/UC Davis)

Treatments, cures and clinical trials: an in-person update on CIRM’s progress

Patients and Patient Advocates are at the heart of everything we do at CIRM. That’s why we are holding three free public events in the next few months focused on updating you on the stem cell research we are funding, and our plans for the future.

Right now we have 33 projects that we have funded in clinical trials. Those range from heart disease and stroke, to cancer, diabetes, ALS (Lou Gehrig’s disease), two different forms of vision loss, spinal cord injury and HIV/AIDS. We have also helped cure dozens of children battling deadly immune disorders. But as far as we are concerned we are only just getting started.

Over the course of the next few years, we have a goal of adding dozens more clinical trials to that list, and creating a pipeline of promising therapies for a wide range of diseases and disorders.

That’s why we are holding these free public events – something we try and do every year. We want to let you know what we are doing, what we are funding, how that research is progressing, and to get your thoughts on how we can improve, what else we can do to help meet the needs of the Patient Advocate community. Your voice is important in helping shape everything we do.

The first event is at the Gladstone Institutes in San Francisco on Wednesday, September 6th from noon till 1pm. The doors open at 11am for registration and a light lunch.

Gladstone Institutes

Here’s a link to an Eventbrite page that has all the information about the event, including how you can RSVP to let us know you are coming.

We are fortunate to be joined by two great scientists, and speakers – as well as being CIRM grantees-  from the Gladstone Institutes, Dr. Deepak Srivastava and Dr. Steve Finkbeiner.

Dr. Srivastava is working on regenerating heart muscle after it has been damaged. This research could not only help people recover from a heart attack, but the same principles might also enable us to regenerate other organs damaged by disease. Dr. Finkbeiner is a pioneer in diseases of the brain and has done ground breaking work in both Alzheimer’s and Huntington’s disease.

We have two other free public events coming up in October. The first is at UC Davis in Sacramento on October 10th (noon till 1pm) and the second at Cedars-Sinai in Los Angeles on October 30th (noon till 1pm). We will have more details on these events in the coming weeks.

We look forward to seeing you at one of these events and please feel free to share this information with anyone you think might be interested in attending.

License to heal: UC Davis deal looks to advance stem cell treatment for bone loss and arthritis

Nancy Lane

Wei Yao and Nancy Lane of UC Davis: Photo courtesy UC Davis

There are many challenges in taking even the most promising stem cell treatment and turning it into a commercial product approved by the Food and Drug Administration (FDA). One of the biggest is expertise. The scientists who develop the therapy may be brilliant in the lab but have little experience or expertise in successfully getting their work through a clinical trial and ultimately to market.

That’s why a team at U.C. Davis has just signed a deal with a startup company to help them move a promising stem cell treatment for arthritis, osteoporosis and fractures out of the lab and into people.

The licensing agreement combines the business acumen of Regenerative Arthritis and Bone Medicine (RABOME) with the scientific chops of the UC Davis team, led by Nancy Lane and Wei Yao.

They plan to test a hybrid molecule called RAB-001 which has shown promise in helping direct mesenchymal stem cells (MSCs) – these are cells typically found in the bone marrow and fat tissue – to help stimulate bone growth and increase existing bone mass and strength. This can help heal people suffering from conditions like osteoporosis or hard to heal fractures. RAB-001 has also shown promise in reducing inflammation and so could prove helpful in treating people with inflammatory arthritis.

Overcoming problems

In a news article on the UC Davis website, Wei Yao, said RAB-001 seems to solve a problem that has long puzzled researchers:

“There are many stem cells, even in elderly people, but they do not readily migrate to bone.  Finding a molecule that attaches to stem cells and guides them to the targets we need provides a real breakthrough.”

The UC Davis team already has approval to begin a Phase 1 clinical trial to test this approach on people with osteonecrosis, a disease caused by reduced blood flow to bones. CIRM is funding this work.

The RABOME team also hopes to test RAB-001 in clinical trials for healing broken bones, osteoporosis and inflammatory arthritis.

CIRM solution

To help other researchers overcome these same regulatory hurdles in developing stem cell therapies CIRM created the Stem Cell Center with QuintilesIMS, a leading integrated information and technology-enabled healthcare service provider that has deep experience and therapeutic expertise. The Stem Cell Center will help researchers overcome the challenges of manufacturing and testing treatments to meet FDA standards, and then running a clinical trial to test that therapy in people.

Life after SPARK: CIRM high school intern gets prestigious scholarship to Stanford

As part of our CIRM scholar blog series, we’re featuring the research and career accomplishments of CIRM funded students.

Ranya Odeh

Ranya Odeh

Meet Ranya Odeh. She is a senior at Sheldon high school in Elk Grove, California, and a 2016 CIRM SPARK intern. The SPARK program provides stem cell research internships to underprivileged high school students at leading research institutes in California.

This past summer, Ranya worked in Dr. Jan Nolta’s lab at UC Davis improving methods that turn mesenchymal stem cells into bone and fat cells. During her internship, Ranya did an excellent job of documenting her journey in the lab on Instagram and received a social media prize for her efforts.

Ranya is now a senior in high school and was recently accepted into Stanford University through the prestigious QuestBridge scholarship program. She credits the CIRM SPARK internship as one of the main reasons why she was awarded this scholarship, which will pay for all four years of her college.

I reached out to Ranya after I heard about her exciting news and asked her to share her story so that other high school students could learn from her experience and be inspired by her efforts.


How did you learn about the CIRM SPARK program?

At my high school, one of our assignments is to build a website for the Teen Biotech Challenge (TBC) program at UC Davis. I was a sophomore my first year in the program, and I didn’t feel passionate about my project and website. The year after, I saw that some of my friends had done the CIRM SPARK internship after they participated in the TBC program. They posted pictures about their internship on Instagram, and it looked like a really fun and interesting thing to do. So I decided to build another website (one that I was more excited about) in my junior year on synthetic biology. Then I entered my website in the TBC and got first prize in the Nanobiotechnology field. Because I was one of the winners, I got the SPARK internship.

What did you enjoy most about your SPARK experience?

For me, it was seeing that researchers aren’t just scientists in white lab coats. The Nolta lab (where I did my SPARK internship) had a lot of personality that I wasn’t really expecting. Working with stem cells was so cool but it was also nice to see at the same time that people in the lab would joke around and pull pranks on each other. It made me feel that if I wanted to have a future in research, which I do, it wouldn’t be doing all work all the time.

What was it like to do research for the first time?

Ranya taking care of her stem cells!

Ranya taking care of her stem cells!

The SPARK internship was my first introduction to research. During my first experiment, I remember I was changing media and I thought that I was throwing my cells away by mistake. So I freaked out, but then my mentor told me that I hadn’t and everything was ok. That was still a big deal and I learned a lesson to ask more questions and pay more attention to what I was doing.

Did the SPARK program help you when you applied to college?

Yes, I definitely feel like it did. I came into the internship wanting to be a pharmacist. But my research experience working with stem cells made me want to change my career path. Now I’m looking into a bioengineering degree, which has a research aspect to it and I’m excited for that. Having the SPARK internship on my college application definitely helped me out. I also got to have a letter of recommendation from Dr. Nolta, which I think played a big part as well.

Tell us about the scholarship you received!

I got the QuestBridge scholarship, which is a college match scholarship for low income, high achieving students. I found out about this program because my career counselor gave me a brochure. It’s actually a two-part scholarship. The first part was during my junior year of high school and that one didn’t involve a college acceptance. It was an award that included essay coaching and a conference that told you about the next step of the scholarship.

The second part during my senior year was called the national college match scholarship. It’s an application on its own that is basically like a college application. I submitted it and got selected as a finalist. After I was selected, they have partner colleges that offer full scholarships. You rank your choice of colleges and apply to them separately with a common application. If any of those colleges want to match you and agree to pay for all four years of your college, then you will get matched to your top choice. There’s a possibility that more than one college would want to match you, but you will only get matched with the one that you rank the highest. That was Stanford for me, and I am very happy about that.

Why did you pick Stanford as your top choice?

It’s the closest university to where I grew up that is very prestigious. It was also one of the only colleges I’ve visited. When I was walking around on campus, I felt I could see myself there as a student and with the Stanford community. Also, it will be really nice to be close to my family.

What do you do in your free time?

I don’t have a lot of free time because I’m in Academic Decathalon and I spend most of my time doing that. When I do have free time, I like to watch Netflix, blogs on YouTube, and I try to go to the gym [laughs].

Did you enjoy posting about your SPARK internship on Instagram?

I had a lot of fun posting pictures of me in the lab on Instagram. It was also nice during the summer to see other SPARK students in different programs talk about the same things. We shared jokes about micropipettes and culturing stem cells. It was really cool to see that you’re not the only one posting nerdy science pictures. I also felt a part of a larger community outside of the SPARK program. Even people at my school were seeing and commenting on what I was doing.

UC Davis CIRM SPARK program 2016

UC Davis CIRM SPARK program 2016

I also liked that I got feedback about what I was doing in the lab from other SPARK students. When I posted pictures during my internship, I talked about working with mesenchymal stem cells. Because we all post to the same #CIRMSPARKlab hashtag, I saw students from CalTech commenting that they worked with those stem cells too. That motivated me to work harder and accomplish more in my project. Instagram also helped me with my college application process. I saw that there were other students in the same position as me that were feeling stressed out. We also gave each other feedback on college essays and having advice about what I was doing really helped me out.

Do you think it’s important for students to be on social media?

Yes, I think it’s important with boundaries of course. There are probably some people who are on social media too often, and you should have a balance. But it’s nice to see what other students are doing to prepare for college and to let loose and catch up with your friends.

What advice would you give to younger high school students about pursuing science?

I feel like students can’t expect things to be brought to them. If they are interested in science, they need to take the initiative to find something that they are going to want to do. The CIRM internship was brought to my attention. But I have friends that were interested in medicine and they found their own internships and ways to learn more about what they wanted to do. So my advice is to take initiative and not be scared of rejection, because if you’re scared of rejection you’re not going to do anything.

To hear more about Ranya’s SPARK internship experience, read her blog “Here’s what you missed this summer on the show coats.” You can also follow her on Instagram and Twitter. For more information about the CIRM SPARK internship program, please visit the CIRM website.


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Trash talking and creating a stem cell community

imilce2

Imilce Rodriguez-Fernandez likes to talk trash. No, really, she does. In her case it’s cellular trash, the kind that builds up in our cells and has to be removed to ensure the cells don’t become sick.

Imilce was one of several stem cell researchers who took part in a couple of public events over the weekend, on either side of San Francisco Bay, that served to span both a geographical and generational divide and create a common sense of community.

The first event was at the Buck Institute for Research on Aging in Marin County, near San Francisco. It was titled “Stem Cell Celebration” and that’s pretty much what it was. It featured some extraordinary young scientists from the Buck talking about the work they are doing in uncovering some of the connections between aging and chronic diseases, and coming up with solutions to stop or even reverse some of those changes.

One of those scientists was Imilce. She explained that just as it is important for people to get rid of their trash so they can have a clean, healthy home, so it is important for our cells to do the same. Cells that fail to get rid of their protein trash become sick, unhealthy and ultimately stop working.

Imilce is exploring the cellular janitorial services our bodies have developed to deal with trash, and trying to find ways to enhance them so they are more effective, particularly as we age and those janitorial services aren’t as efficient as they were in our youth.

Unlocking the secrets of premature aging

Chris Wiley, another postdoctoral researcher at the Buck, showed that some medications that are used to treat HIV may be life-saving on one level, preventing the onset of full-blown AIDS, but that those benefits come with a cost, namely premature aging. Chris said the impact of aging doesn’t just affect one cell or one part of the body, but ripples out affecting other cells and other parts of the body. By studying the impact those medications have on our bodies he’s hoping to find ways to maintain the benefits of those drugs, but get rid of the downside.

Creating a Community

ssscr

Across the Bay, the U.C. Berkeley Student Society for Stem Cell Research held it’s 4th annual conference and the theme was “Culturing a Stem Cell Community.”

The list of speakers was a Who’s Who of CIRM-funded scientists from U.C. Davis’ Jan Nolta and Paul Knoepfler, to U.C. Irvine’s Henry Klassen and U.C. Berkeley’s David Schaffer. The talks ranged from progress in fighting blindness, to how advances in stem cell gene editing are cause for celebration, and concern.

What struck me most about both meetings was the age divide. At the Buck those presenting were young scientists, millennials; the audience was considerably older, baby boomers. At UC Berkeley it was the reverse; the presenters were experienced scientists of the baby boom generation, and the audience were keen young students representing the next generation of scientists.

Bridging the divide

But regardless of the age differences there was a shared sense of involvement, a feeling that regardless of which side of the audience we are on we all have something in common, we are all part of the stem cell community.

All communities have a story, something that helps bind them together and gives them a sense of common purpose. For the stem cell community there is not one single story, there are many. But while those stories all start from a different place, they end up with a common theme; inspiration, determination and hope.

 

A Dream made me change my mind. Almost.

Dream Alliance

Dream Alliance: photo courtesy Daily Telegraph, UK

On Friday I was faced with the real possibility that a horse had made an ass out of me.

Over the years we have written many articles about the risks of unproven stem cell therapies, treatments that have not yet been shown in clinical trials to be safe and effective. Often we have highlighted the cases of high profile athletes who have undergone stem cell treatments for injuries when there is little evidence that the treatments they are getting work.

Well, on Friday I saw an athlete who bounced back from a potentially career-ending injury to enjoy an amazing career thanks to a stem cell treatment. I wondered if I was going to have to revise my thoughts on this topic. Then my wife pointed out to me that the athlete was a horse.

We had been watching the movie Dark Horse, a truly delightful true story about a group of working class people in a Welsh mining village who bred and raised a horse that went on to great success as a race horse – often beating out thoroughbreds that were worth millions of dollars.

 

At one point the horse, Dream Alliance, suffered an almost fatal injury. Everyone assumed his career was over. But thanks to a stem cell treatment he was able to return to the track and became the first horse to win a major race after undergoing stem cell surgery.

It shouldn’t be too surprising that stem cells can help heal serious injuries in horses, the researchers at UC Davis have been using them to help treat horses for years – with great success. The danger comes in then assuming that just because stem cells work for horses, they’ll work for people. And that if they can cure one kind of injury, why not another.

That thought was driven home to me on Saturday when I was giving a talk to a support group for ALS or Lou Gehrig’s disease. ALS is a nasty, rapidly progressive disease that attacks the motor nerve cells in the brain and spinal cord, destroying a person’s ability to move, eat, speak or breath.

One person asked about a clinic they had been talking to which claimed it might be able to help them. The clinic takes fat from the person with ALS, isolates the stem cells in the fat and injects it back into the person. The clinic claims it’s been very effective in treating injuries such as torn muscles, and that it also works for other problems like Parkinson’s so it might help someone with ALS.

And that’s the problem. We hear about one success story that seems to prove stem cells can do amazing things, and then we are tempted to hope that if it works for one kind of injury, it might work for another, or even for a neurodegenerative disease.

And hope doesn’t come cheap. The cost of the procedure was almost $10,000.

If you have a disease like ALS for which there is no cure, and where the life expectancy is between two to five years, you can understand why someone would be tempted to try anything, no matter how implausible. What is hard is when you have to tell them that without any proof that it works, and little scientific rational as to why it would work, that it’s hard to recommend they try using their own fat cells to treat their ALS.

At CIRM we are investing more than $56.5 million in 21 different projects targeting ALS.   We are hopeful one of them, Clive Svendsen’s research at Cedars-Sinai Medical Center,  will soon get approval from the FDA to start a clinical trial.

Much as we would like to believe in miracles, medical breakthroughs usually only come after years of hard, methodical work. It would be great if injecting your own fat-derived stem cells into your body could cure you of all manner of ailments. But there’s no evidence to suggest it will.

The movie Dark Horse shows that for one horse, for one group of people in a small Welsh mining village, stem cells helped create a happy ending. We are hoping stem cells will one day offer the same sense of hope and possibility for people battling deadly diseases like ALS. But that day is not yet here.

 

 

Brave new world or dark threatening future: a clear-eyed look at genome editing and what it means for humanity

Frankenstein

   Is this the face of the future?

“Have you ever wished that there were something different about yourself? Maybe you imagined yourself taller, thinner or stronger? Smarter? More attractive? Healthier?”

That’s the question posed by UC Davis stem cell researcher (and CIRM grantee) Paul Knoepfler at the start of his intriguing new book ‘GMO Sapiens: The Life-Changing Science of Designer Babies’.

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You can find GMO Sapiens on Amazon.com

The book is a fascinating, and highly readable, and takes a unique look at the dramatic advances in technology that allow us to edit the human genome in ways that could allow us to do more than just create “designer babies”, it could ultimately help us change the definition of what it means to be human.

Paul begins by looking at the temptation to use technologies like CRISPR (we have blogged about this here), to genetically edit or alter human embryos so that the resulting child is enhanced in some ways. It could be that the editing is used to remove a genetic mutation that could cause a deadly disease (such as the BRCA1 gene that puts women at increased risk of breast and ovarian cancer) or it could be that the technique is used to give a baby blue eyes, to make it taller, more athletic, or to simply eliminate male pattern baldness later in life.

Paul says those latter examples are not as ridiculous as they sound:

Paul Knoepfler

Paul Knoepfler

“If you think these ideas sound far-fetched, consider that Americans alone spend tens of billions of dollars each year on plastic surgery procedures and creams to try to achieve these kinds of goals. Some of the time elective cosmetic surgery is done on children. In the future, we might have “cosmetic genetic surgeons” who do “surgery” on our family’s genes for cosmetic reasons. In other countries the sensibilities and cultural expectations could lead to other kinds of genetic modifications of humans for “enhancements”.

While the technology that enables us to do this is new, the ideas behind why we would want to do this are far from new. Paul delves into those ideas including a look at the growth of the eugenics movement in the late 19th and early 20th century advocating the improvement of human genetic traits through higher reproductive rates for people considered “superior”. And there was a darker side to the movement:

“Indiana had instituted the first law for sterilization of “inferior” people in the world in 1907. Astonishingly this state law and then similar laws (the original was revoked, but a new law was passed later) stayed on the books in that state until 1974.

This led to approximately 2,500 governmentally forced sterilizations. The poor, uneducated, people of color, Native Americans, and people with disabilities were disproportionately targeted.”

Paul explores the ethical and moral implications of changing our genetic code, changes that can then be passed on to future generations. While he understands the desire to use these technologies to create positive changes, he is also very clear in his concerns that we don’t yet have enough knowledge to be able to use them in a safe manner.

“CRISPR can literally re-write the genomic book inside of us. However, it remains unknown how often it might go to the wrong page or paragraph, so to speak, or stay on the right page, but make an undesired edit there.”

Tiny errors in editing the genome, particularly at such an early stage in an embryo’s development, could have profound and unintended consequences years down the road, resulting in physical or developmental problems we can’t anticipate or predict. For example, you might remove the susceptibility to one disease only to create an even larger problem, one that is now embedded in that person’s DNA and ready to be passed on to subsequent generations.

The book includes interviews with key figures in the field – scientists, bioethicists etc. – and covers a wide range of views of what we should do. For example, the Director of the US National Institutes of Health (NIH), Francis Collins, said that designer babies “make good Hollywood — and bad science,” while the Center for Genetics and Society has advocated for a moratorium on human genetic modification in the US.

In contrast, scientists such as Harvard professor George Church and CRISPR pioneer Jennifer Doudna of UC Berkeley, say we need to carefully explore how to harness the potential for these technologies.

For Church it is a matter of choice:

“The new technology enables parents to make choices about their children just as they might with Ritalin or cleft palate surgery to ‘improve’ behavior or appearance.”

For Doudna it’s acknowledging the fact that you can’t put the genie back in the bottle:

“There’s no way to unlearn what is learned. We can’t put this technology to bed. If a person has basic knowledge of molecular biology they can do it. It’s not realistic to think we can block it…We want to put out there the information that people would need to make an informed decision, to encourage appropriate research and discourage forging ahead with clinical applications that could be dangerous or raise ethical issues.”

The power of Paul’s book is that while it does not offer any easy answers, it does raise many important questions.

It’s a wonderfully well-written book that anyone can read, even someone like me who doesn’t have a science background. He does a good job of leading the reader through the development of these technologies (from the basic idea of genetically altering plants to make them disease resistant) to the portrayal of these concepts in literature (Frankenstein and Brave New World) to movies (Gattaca – 4 stars on Rotten Tomatoes  a great film if you haven’t already seen it).

It’s clear where Paul stands on the issue; he believes there should be a moratorium on human genetic modification until we have a much deeper understanding of the science behind it, and the ethics and morality underpinning it:

“This is a very exciting time to be alive and we should be open to embracing change, but not blindly or in a rush. Armed with information and passion, we can have a major, positive impact on how this biotech revolution unfolds and impacts humanity.”

By the way, Paul also has one of the most widely read blogs about stem cells, where you can read more about his thoughts on CRISPR and other topics.