Stem cell treatment restores man’s sight in right eye after 25 years

James O’Brien, recipient of a stem cell treatment that restored the vision in his right eye

At 18 years old, there are several life-changing moments that young people look forward to. For some, it involves graduating from high school, starting college, and being able to cast a vote in an election. For others, this momentous occasion symbolizes the official start of adulthood.

For James O’ Brien, this milestone was marked by a rather unfortunate event where ammonia was thrown at his face in a random attack. As a result of this incident, the surface of his right eye was burned and he was left completely blind in his right eye.

Fast forward 25 years and thanks to an experimental stem cell treatment, James is able to see out of his right eye for the first time since the attack.

“Being able to see with both eyes – it’s a small thing that means the world. Basically I went from near-blindness in that eye to being able to see everything.” said O’Brien in a news release from Daily Heralds.

Dr. Sajjad Ahmad and a team of surgeons at the Moorfields Eye Hospital in London removed healthy stem cells from O’Brien’s left eye and grew these cells in a lab for months. After an adequate number of healthy stem cells from O’Briens left eye were grown, the surgeons then cut the scar tissue in his right eye and replaced it with the healthy stem cells.

They then waited a year after the procedure for the cells to settle down before inserting a cornea – which plays a key role in vision and focuses light – from a deceased donor.

“This is going to have a huge impact. A lot of these patients are young men so it affects their work, their lives, those around them. It’s not just the vision that drops, it’s the pain.” said Dr. Ahmad in the news release previously mentioned.

The procedure used took over 20 years to develop and Dr. Ahmad hopes to continue to develop the procedure for patients that have been blinded in both eyes by chemicals or have lost their vision through degenerative conditions.

CIRM has funded three clinical trials in vision loss to date. Two of these trials are being conducted by Dr. Henry Klassen for an eye condition known as retinitis pigmentosa and have shown promising results. The third trial is being conducted by Dr. Mark Humayun for another eye condition known as age-related macular degeneration (AMD) which has also shown promising results.

See video below for a news segment of James O’Brien on BBC News:

When Google turns on you, you know you are in trouble

For years CIRM and others in the stem cell community (hello Paul Knoepfler) have been warning people about the dangers of going to clinics offering unproven and unapproved stem cell therapies. Recently the drum beat of people and organizations coming out in support of that stand has grown louder and louder. Mainstream media – TV and print – have run articles about these predatory clinics. And now, Google has joined those ranks, announcing it will restrict ads promoting these clinics.

“We regularly review and revise our advertising policies. Today, we’re announcing a new Healthcare and medicines policy to prohibit advertising for unproven or experimental medical techniques such as most stem cell therapy, cellular (non-stem) therapy, and gene therapy.”

Deepak Srivastava: Photo courtesy Gladstone Institutes

The president of the International Society for Stem Cell Research (ISSCR) Dr. Deepak Srivastava quickly issued a statement of support, saying:

“Google’s new policy banning advertising for speculative medicines is a much-needed and welcome step to curb the marketing of unscrupulous medical products such as unproven stem cell therapies. While stem cells have great potential to help us understand and treat a wide range of diseases, most stem cell interventions remain experimental and should only be offered to patients through well-regulated clinical trials. The premature marketing and commercialization of unproven stem cell products threatens public health, their confidence in biomedical research, and undermines the development of legitimate new therapies.”

Speaking of Deepak – we can use first names here because we are not only great admirers of him as a physician but also as a researcher, which is why we have funded some of his research – he has just published a wonderfully well written article criticizing these predatory clinics.

The article – in Scientific American – is titled “Don’t Believe Everything You Hear About Stem Cells” and rather than paraphrase his prose, I think it best if you read it yourself. So, here it is.

Enjoy.

Don’t Believe Everything You Hear about Stem Cells

The science is progressing rapidly,but bad actors have co-opted stem cells’ hope and promise by preying on unsuspecting patients and their families

Stem cell science is moving forward rapidly, with potential therapies to treat intractable human diseases on the horizon.Clinical trials are now underway to test the safety and effectiveness of stem cell–based treatments for blindness,spinal cord injury,heart disease,Parkinson’s disease, and more,some with early positive results.A sense of urgency drives the scientific community, and there is tremendous hope to finally cure diseases that, to date, have had no treatment.


But don’t believe everything you hear about stem cells. Advertisements and pseudo news articles promote stem cell treatments for everything from Alzheimer’s disease,autism and ALS, to cerebral palsy and other diseases.The claims simply aren’t true–they’re propagated by people wanting to make money off of a desperate and unsuspecting or unknowing public.Patients and their families can be misled by deceptive marketing from unqualified physicians who often don’t have appropriate medical credentials and offer no scientific evidence of their claims.In many cases, the cells being utilized are not even true stem cells.

Advertisements for stem cell treatments are showing up everywhere, with too-good-to-be-true claims and often a testimonial or two meant to suggest legitimacy or efficacy.Beware of the following:

    •       Claims that stem cell treatments can treat a wide range of diseases using a singular stem cell type. This is unlikely to be true.

    •       Claims that stem cells taken from one area of the body can be used to treat another, unrelated area of the body. This is also unlikely to be true.     •       Patient testimonials used to validate a particular treatment, with no scientific evidence. This is a red flag.

    •       Claims that evidence doesn’t yet exist because the clinic is running a patient-funded trial. This is a red flag; clinical trials rarely require payment for experimental treatment.

    •       Claims that the trial is listed on ClinicalTrials.gov and is therefore NIH-approved. This may not be true. The Web site is simply a listing; not all are legitimate trials.

    •       The bottom line: Does the treatment sound too good to be true? If so, it probably is. Look for concrete evidence that the treatment works and is safe.

Hundreds of clinics offer costly, unapproved and unproven stem cell interventions, and patients may suffer physical and financial harm as a result.A Multi-Pronged Approach to Deal with Bad Actors 

The International Society for Stem Cell Research (ISSCR)has long been concerned that bad actors have co-opted the hope and promise of stem cell science to prey on unsuspecting patients and their families.

We read with sadness and disappointment the many stories of people trying unproven therapies and being harmed, including going blind from injections into the eyes or suffering from a spinal tumor after an injection of stem cells.Patients left financially strapped, with no physical improvement in their condition and no way to reclaim their losses, are an underreported and underappreciated aspect of these treatments.

Since late 2017, the Food and Drug Administration has stepped up its regulatory enforcement of stem cell therapies and provided a framework for regenerative medicine products that provides guidelines for work in this space.The agency has alerted many clinics and centers that they are not in compliance and has pledged to bring additional enforcement action if needed.

A Multi-Pronged Approach to Deal with Bad Actors  The International Society for Stem Cell Research (ISSCR) has long been concerned that bad actors have co-opted the hope and promise of stem cell science to prey on unsuspecting patients and their families.

We read with sadness and disappointment the many stories of people trying unproven therapies and being harmed, including going blind from injections into the eyesor suffering from a spinal tumor after an injection of stem cells.Patients left financially strapped, with no physical improvement in their condition and no way to reclaim their losses, are an underreported and underappreciated aspect of these treatments.

Since late 2017, the Food and Drug Administration has stepped up its regulatory enforcement of stem cell therapies and provided a framework for regenerative medicine products that provides guidelines for work in this space.The agency has alerted many clinics and centers that they are not in compliance and has pledged to bring additional enforcement action if needed.

In recent weeks, a federal judge granted the FDA a permanent injunction against U.S. Stem Cell, Inc. and U.S. Stem Cell Clinic, LLC for adulterating and misbranding its cellular products and operating outside of regulatory authority.We hope this will send a strong message to other clinics misleading patients with unapproved and potentially harmful cell-based products.

The Federal Trade Commission has also helped by identifying and curtailing unsubstantiated medical claims in advertising by several clinics. Late in 2018 the FTC won a $3.3-million judgment against two California-based clinics for deceptive health claims. The Federal Trade Commission has also helped by identifying and curtailing unsubstantiated medical claims in advertising by several clinics. Late in 2018 the FTC won a $3.3-million judgment against two California-based clinics for deceptive health claims.

These and other actions are needed to stem the tide of clinics offering unproved therapies and the people who manage and operate them.

Improving Public Awareness

We’re hopeful that the FDA will help improve public awareness of these issues and curb the abuses on ClinicalTrials.gov,a government-run Web site being misused by rogue clinics looking to legitimize their treatments. They list pay-to-participate clinical trials on the site, often without developing, registering or administering a real clinical trial.

The ISSCR Web site A Closer Look at Stem Cellsincludes patient-focused information about stem cells,with information written and vetted by stem cell scientists.The site includes how and where to report adverse events and false marketing claims by stem cell clinics.I encourage you to visit and learn about what is known and unknown about stem cells and their potential for biomedicine.The views expressed are those of the author(s) and are not necessarily those of Scientific American.

Moving a great idea targeting diabetes out of the lab and into a company

Tejal Desai in her lab at UCSF: Photo courtesy Todd Dubnicoff

It’s always gratifying to see research you have helped support go from being an intriguing idea to something with promise to a product that is now the focus of a company. It’s all the more gratifying if the product in question might one day help millions of people battling diabetes.

That’s the case with a small pouch being developed by a company called Encellin. The pouch is the brainchild of Tejal Desai, Ph.D., a professor of bioengineering at UCSF and a CIRM grantee.

Encellin’s encapsulation device

“It’s a cell encapsulation device, so this material can essentially protect beta cells from the immune system while allowing them to function by secreting insulin. We are placing stem cell-derived beta cells into the pouch which is then implanted under the skin. The cells are then able to respond to changes in sugar or glucose levels in the blood by pumping out insulin.  By placing the device in a place that is accessible we can easily remove it if we have to, but also we can recharge it and put in new cells as well.”

While the pouch was developed in Dr. Desai’s lab, the idea to take it from a promising item and try to turn it into a real-world therapy came from one of Dr. Desai’s former students, Crystal Nyitray, Ph.D.

Crystal Nyitray: Photo courtesy FierceBiotech

After getting her PhD, Nyitray went to work for the pharmaceutical giant Sanofi. In an article in FierceBiotech she says that’s where she realized that the pouch she had been working on at UCSF had real potential.

“During that time, I started to realize we really had something, that everything that pharma or biotech was looking at was something we had been developing from the ground up with those specific questions in mind,”

So Dr. Nyitray went to work for QB3, the institute created by UC San Francisco to help startups develop their ideas and get funding. The experience she gained there gave her the confidence to be the co-founder and CEO of Encellin.

Dr. Desai is a scientific advisor to Encellin. She says trying to create a device that contains insulin-secreting cells is not new. Many previous attempts failed because once the device was placed in the body, the immune system responded by creating fibrosis or scarring around it which blocked the ability of the cells to get out.

But she thinks their approach has an advantage over previous attempts.

“This is not a new idea, the idea has been around for 40 or more years but getting it to work is hard. We have a convergence of getting the right cell types and combining that with our knowledge of immunology and then the material science where we can design materials at this scale to get the kind of function that we need.

Dr. Nyitray ““If we can reduce fibrosis, it really helps the cells get nutrients better, survive better and signal more effectively. It’s really critical to their success.”

Dr. Desai says the device is still in the early stages of being tested, but already it’s showing promise.

“We have done testing in animals. Where the company is taking this is now to see if we can take this to larger animals and then ultimately people.”

She says without CIRM’s support none of this would have happened.

“CIRM has been really instrumental in helping us refine the cell technology piece of it, to get really robust cells and also to support the development to push the materials, to understand the biology, to really understand what was happening with the cell material interface. We know we have a lot of challenges ahead, but we are really excited to see if this could work.”

We are excited too. We are looking forward to seeing what Encellin does in the coming years. It could change the lives of millions of people around the world.

No pressure. 

“Brains” in a dish that can create electrical impulses

Brain organoids in a petri dish: photo courtesy UCSD

For several years, researchers have been able to take stem cells and use them to make three dimensional structures called organoids. These are a kind of mini organ that scientists can then use to study what happens in the real thing. For example, creating kidney organoids to see how kidney disease develops in patients.

Scientists can do the same with brain cells, creating clumps of cells that become a kind of miniature version of parts of the brain. These organoids can’t do any of the complex things our brains do – such as thinking – but they do serve as useful physical models for us to use in trying to develop a deeper understanding of the brain.

Now Alysson Muotri and his team at UC San Diego – in a study supported by two grants from CIRM – have taken the science one step further, developing brain organoids that allow us to measure the level of electrical activity they generate, and then compare it to the electrical activity seen in the developing brain of a fetus. That last sentence might cause some people to say “What?”, but this is actually really cool science that could help us gain a deeper understanding of how brains develop and come up with new ways to treat problems in the brain caused by faulty circuitry, such as autism or schizophrenia.

The team developed new, more effective methods of growing clusters of the different kinds of cells found in the brain. They then placed them on a multi-electrode array, a kind of muffin tray that could measure electrical impulses. As they fed the cells and increased the number of cells in the trays they were able to measure changes in the electrical impulses they gave off. The cells went from producing 3,000 spikes a minute to 300,000 spikes a minute. This is the first time this level of activity has been achieved in a cell-based laboratory model. But that’s not all.

When they further analyzed the activity of the organoids, they found there were some similarities to the activity seen in the brains of premature babies. For instance, both produced short bursts of activity, followed by a period of inactivity.

Alysson Muotri

In a news release Muotri says they were surprised by the finding:

“We couldn’t believe it at first — we thought our electrodes were malfunctioning. Because the data were so striking, I think many people were kind of skeptical about it, and understandably so.”

Muotri knows that this research – published in the journal Cell Stem Cell – raises ethical issues and he is quick to say that these organoids are nothing like a baby’s brain, that they differ in several critical ways. The organoids are tiny, not just in size but also in the numbers of cells involved. They also don’t have blood vessels to keep them alive or help them grow and they don’t have any ability to think.

“They are far from being functionally equivalent to a full cortex, even in a baby. In fact, we don’t yet have a way to even measure consciousness or sentience.”

What these organoids do have is the ability to help us look at the structure and activity of the brain in ways we never could before. In the past researchers depended on mice or other animals to test new ideas or therapies for human diseases or disorders. Because our brains are so different than animal brains those approaches have had limited results. Just think about how many treatments for Alzheimer’s looked promising in animal models but failed completely in people.

These new organoids allow us to explore how new therapies might work in the human brain, and hopefully increase our ability to develop more effective treatments for conditions as varied as epilepsy and autism.

Bridges to the Future: 10 Years and Counting!

Bridges conference 2019

When Californians voted for Proposition 71 in 2004, they were investing in hope… the hope that unraveling the mysteries of stem cells could lead to new types of treatments and perhaps one day, even cures for some of the most devastating illnesses and injuries known to mankind. Making this hope a reality, however, requires much more than scientific discovery, it requires a dedicated and skilled work force that can recognize and tackle the challenges that come with such an ambitious dream.

To jump start the nascent stem cell/regenerative medicine community in California, CIRM began offering Training Grants to major research and medical institutions to attract talented PhD students and postdoctoral fellows into the field. A few years later, a second type of training program was born to attract a different, yet equally important cadre of professionals – the undergraduate, Bachelors and Master’s level scientists who are the bread and butter of any successful research endeavor.

Bridges students

Over the past 10 years, CIRM has supported 16 of these programs, which have proven to be among the most popular and successful CIRM initiatives to date. As of 2019, the Bridges programs have trained well over 1400 scientists, about half of whom are working full time in research positions at biotechnology companies or academic laboratories, and another third of whom are currently enrolled in a graduate or professional school.

Today, there are 14 active Bridges Programs around the state, each with unique attributes, but all sharing the core elements of stem cell-based coursework, hands-on-training through internships at world-class laboratories or biotechnology companies, and formal activities involving patient engagement and community outreach. Every year, the programs produce up to 140 well-rounded, highly skilled individuals that are ready to hit the ground running.

Poster presentations at the Bridges conference

Each July, the most recent cohort of Bridges trainees gather for an Annual Conference to share their research outcomes, network with their peers, and learn more about the current opportunities and challenges facing the regenerative medicine community.

This year, the 10th Annual Bridges Conference was held in San Mateo, CA and included inspiring talks from scientists performing cutting edge research and running some of the first FDA-approved stem-cell based clinical trials in the state.

Anna Simos

Perhaps the biggest highlights were hearing the real-life stories of brave individuals like Anna Simos, whose experience with life-threatening complications from diabetes inspired her life’s work of providing hope and education to those facing similar challenges.

Byron Jenkins

Equally moving was the testimonial of Byron Jenkins, a multiple myeloma patient who received an experimental new CAR-T therapy in a CIRM-supported clinical trial sponsored by Poseida Therapeutics.

Ronnie Kashyup with parents Upasana and Pawash

Last but not least, little Ronnie Kashyup, recently cured of Bubble Baby Disease through another CIRM-funded clinical trial, charmed all attendees with his larger-than-life personality while his father, Pawash Priyank, shared the story of Ronnie’s diagnosis and treatment.

In the video segments to follow:

CIRM Bridges student Sneha Santosh at San Jose State University discusses the role CIRM plays in bridging together the patient advocates with the groundbreaking research conducted by scientists.

Samori Dobson and Esther Nair, CIRM Bridges students at California State University, San Marcos, briefly discuss the positive impact that the program has had on their lives.

Below are some pictures form the 10th Annual Bridges Conference in San Mateo, CA.

For more information about CIRM Bridges Programs, see the following link and video below:

CIRM-funded internship programs

CIRM board member Lauren Miller Rogen appointed to California Alzheimer’s Task Force

Lauren Miller Rogen, Hilarity for Charity co-founder and CIRM Board Member

California has the largest aging population in the United States. The U.S. Census Bureau has estimated that one in five Californians will be 65 or older by the year 2030. Unfortunately with age comes a wide of health related issues that can arise such as Alzheimer’s.

Alzheimer’s is caused by changes in the brain that affect memory and thinking skills. The disease can progress to the point where carrying out the simplest tasks become quite a challenge. In the United States alone, 5.8 million people are living with Alzheimer’s, 630,000 of whom live in California. By 2050, the number of people with Alzheimer’s in the United States is expected to increase to almost 14 million.

To address this growing problem, California Governor Gavin Newsom announced the creation of a California Alzheimer’s Task Force comprised of scientists, politicians, and other individuals dedicated to addressing the needs of the Alzheimer’s community and the impact the disease has on California. The new task force has been tasked with releasing a report on the disease and ways to address the challenges it poses by 2020.

One of these task force members is our very own Lauren Miller Rogen, who is a dedicated member of our governing Board and the co-founder of Hilarity for Charity, a charity organization that raises awareness about and funds for research into Alzheimer’s. In addition to her advocacy work, Lauren is also a screenwriter and actress, staring alongside her husband Seth Rogen in movies such as 50/50 and Superbad.

“I’m so honored to join the Task Force to fight for the 670,000 Californians currently living with Alzheimer’s and for those who care for them,” Miller Rogen said. “This is a tremendous and diverse group who intend to create and propose real ideas to change the course of this disease.”

For Lauren, her journey towards becoming an advocate for Alzheimer’s is a very personal one. Her grandfather died of Alzheimer’s when she was just 12 years old and her grandmother died of the disease six years after that. Now, her mother is struggling with Alzheimer’s, having been diagnosed at the age of 55.

You can read more about Lauren’s story on a previous blog post.

CIRM have given awards totaling over $56 million throughout the years dedicated towards Alzheimer’s related research.

A future scientist’s journey

All this week we have been highlighting blogs from our SPARK (Summer Program to Accelerate Regenerative medicine Knowledge) students. SPARK gives high school students a chance to spend their summer working in a world class stem cell research facility here in California. In return they write about their experiences and what they learned.

The standard for blogs this year was higher than ever, so choosing a winner was particularly tough. In the end we chose Abigail Mora, who interned at UC San Francisco. We felt the obstacles she overcame in getting to this point made her story all the more remarkable and engaging.

Abigail Mora

When I was 15, my mother got sick and went to several doctors. Eventually, she found out that she was pregnant with a 3-month-old baby. A month after, my mom fell from the stairs, which were not high but still dangerous. Luckily, everything seemed to be okay with the baby. In the last week of her six-month pregnancy, she went in the clinic for a regular check-up but she ended up giving birth to my brother, who was born prematurely. She stayed in the clinic for a month and my brother also had to stay so that his lungs could develop properly.

When he came home, I was so happy. I spent a lot of time with him and was like his second mom. After an initial period of hard time, he grew into a healthy kid. Then I moved to San Francisco with my aunt, leaving my parents and siblings in Mexico so that I could become a better English speaker and learn more about science. My experience with my brother motivated me to learn more about the condition of premature babies, since there are many premature babies who are not as fortunate. I want to study neurodevelopment in premature kids, and how it may go wrong.

I was so happy when I got into the SEP High School Program, which my chemistry teacher introduced me to, and I found the research of Eric Huang’s lab at UCSF about premature babies and stem cell development in the brain super interesting. I met Lakisha and Jean, and they introduced me to the lab and helped me walk through the training process.

My internship experience was outstanding: I enjoyed doing research and how my mentor Jiapei helped me learn new things about the brain. I learned that there are many different cell types in the brain, like microglia, progenitor cells, and intermediate progenitors.

As all things in life can be challenging, I was able to persevere with my mentor’s help. For example, when I first learned how to cut mouse brains using a cryostat, I found it hard to pick up the tissue onto slides. After practicing many times, I became more familiar with the technique and my slices got better. Another time, I was doing immunostaining and all the slices fell from the slide because we didn’t bake the slides long enough. I was sad, but we learned from our mistakes and there are a lot of trials and errors in science.

I’ve also learned that in science, since we are studying the unknown, there is not a right or wrong answer. We use our best judgement to draw conclusions from what we observe, and we repeat the experiment if it’s not working.

The most challenging part of this internship was learning and understanding all the new words in neuroscience. Sometimes, I got confused with the abbreviations of these words. I hope in the future I can explain as well as my mentor Jiapei explained to me.

My parents are away from me but they support me, and they think that this internship will open doors to better opportunities and help me grow as a person.

I want to become a researcher because I want to help lowering the risk of neurodevelopmental disorders in premature babies. Many of these disorders, such as autism or schizophrenia, don’t have cures. These are some of the hardest diseases to cure because people aren’t informed about them and not enough research has been done. Hopefully, one day I can work on developing a cure for these disorders.

CIRM’s Stephen Lin, PhD, who heads the SPARK program and Abigail after her blog won first prize

Mind altering, life changing experience in stem cell lab

This week we are featuring the best blogs from our SPARK (Summer Program to Accelerate Regenerative medicine Knowledge) students. SPARK gives high school students a chance to spend their summer working in a world class stem cell research facility here in California. In return they write about their experiences and what they learned.

The blog that won second place comes from Emily Bunnapradist who spent her summer at Cedars-Sinai Medical Center in Los Angeles.

Emily Bunnapradist by the poster presentation of her work

When I was in the third grade, my mom took me to the allergy wing in the UCLA Medical Center, hoping to find answers to a number of issues that accompanied my seemingly never-ending list of food allergies: dairy, eggs, nuts, legumes, and so on. Unexpectedly, without even an appointment, clinician Dr. Braskett spent an hour out of her already busy schedule just talking us through our worries in the lobby, checking out skin problems that arose as a result of my allergies and promising to see us again as soon as she could. Because of her overwhelming kindness and generosity, my mom and I went home with relieved smiles and assurance that my health concerns were manageable.

That was the day that I decided that I wanted to pursue medicine, to make an impact on people the way that she had on my family and me. However, my conception of the field of healthcare was quite limited. For the majority of my life, I was convinced that the only way to make a true connection in a patient’s well-being was as a clinician.

This unfounded claim quickly changed when I was accepted into the CIRM SPARK program at Cedars-Sinai. In the most action-packed summer I have ever had the opportunity to experience, I was exposed to the diverse field of healthcare. Transitioning between the clinical and research aspects of science, I saw firsthand the direct effect that researchers had on patients in fields I had not even considered.

While touring the blood transfusion facility at Cedars-Sinai, a technician proudly boasted about her connection to patient care in labeling and testing blood donations to ensure they were suitable for those in need. Upon viewing the imaging core, the manager of the center informed us about the revolutionary advances his team was making in developing software to identify cancerous indicators in patients. In visiting the microbiology lab, multiple lab scientists informed us about the hundreds of tests they perform on a daily basis to detect diseases such as influenza and adenovirus, without which clinicians wouldn’t be able to perform their job to the fullest degree.

In these past weeks, I have spent hundreds of hours in the lab. From drawing on sections with hydrophobic markers to loading gels with protein samples, I have gained tremendous experience in navigating a research environment. However, although I now know the mechanics of Western blots and immunostaining like the back of my hand, the most essential takeaways for me are not learning the procedures but understanding their applications. While I am now able to pipette fluids with a steady hand and make buffer solutions without second-guessing my calculations, I am also able to appreciate the science behind each protein band and cell plate. Being able to contribute to my project and hear about my peers’ experiments has shown me the scope of influence research can have on extending knowledge and generating cures to diseases.

While I had initially considered research to be cold and isolating, I have found more warmth and connection here than I believed possible. The passion that my mentors possess for their line of work, as well as their endless knowledge on essentially any topic imaginable, has shown me the importance and integrity of what they do.

The CIRM SPARK students at Cedars-Sinai (Emily is front right): Photo courtesy Cedars-Sinai

I could not be more grateful to have the guidance of Dr. Mehrnoosh Ghiam and Dr. Adam Poe, who I have formed strong relationships with and have helped me accomplish what I have this summer. Their mentorship, along with the resources of Cedars-Sinai, have granted me the most productive and exciting summer I’ve had yet!

Time and money and advancing stem cell research

The human genome

Way back in the 1990’s scientists were hard at work decoding the human genome, trying to map and understand all the genes that make up people. At the time there was a sense of hope, a feeling that once we had decoded the genome, we’d have cures for all sorts of things by next Thursday. It didn’t quite turn out that way.

The same was true for stem cell research. In the early days there was a strong feeling that this was going to quite quickly produce new treatments and cures for diseases ranging from Parkinson’s and Alzheimer’s to heart disease and stroke. Although we have made tremendous strides we are still not where we hoped we’d be.

It’s a tough lesson to learn, but an important one: good scientific research moves at its own pace and pays little heed to our hopes or desires. It takes time, often a long time, and money, usually a lot of money, to develop new treatments for deadly diseases and disorders.

Many people, particularly those battling deadly diseases who are running out of time, are frustrated at the slow pace of stem cell research, at the years and years of work that it takes to get even the most promising therapy into a clinical trial where it can be tested in people. That’s understandable. If your life is on the line, it’s difficult to be told that you have to be patient. Time is a luxury many patients don’t have.

But that caution is necessary. The last thing we want to do is rush to test something in people that isn’t ready. And stem cells are a whole new way of treating disease, using cells that may stay in the body for years, so we really need to be sure we have done everything we can to ensure they are safe before delivering them to people.

The field of gene therapy was set back years after one young patient, Jesse Gelsinger, died as a result of an early experimental treatment. We don’t want the same to happen to stem cell research.

And yet progress is being made, albeit not as quickly as any of us would like. At the end of the first ten years of CIRM’s existence we had ten projects that we supported that were either in, or applying to be in, a clinical trial sanctioned by the US Food and Drug Administration (FDA). Five years later that number is 56.

Most of those are in Phase 1 or 2 clinical trials which means they are still trying to show they are both safe and effective enough to be made available to a wider group of people. However, some of our projects are in Phase 3, the last step before, hopefully, being given FDA approval to be made more widely available and – just as important – to be covered by insurance.

Other CIRM-funded projects have been given Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, a new program that allows projects that show they are safe and benefit patients in early stage clinical trials, to apply for priority review, meaning they could get approved faster than normal. Out of 40 RMAT designations awarded so far, six are for CIRM projects.

We are working hard to live up to our mission statement of accelerating stem cell treatments to patients with unmet medical needs. We have been fortunate in having $3 billion to spend on advancing this research in California; an amount no other US state, indeed few other countries, have been able to match. Yet even that amount is tiny compared to the impact that many of these diseases have. For example, the economic cost of treating diabetes in the US is a staggering $327 billion a year.

The simple truth is that unless we, as a nation, invest much more in scientific research, we are not going to be able to develop cures and new, more effective, treatments for a wide range of diseases.

Time and money are always going to be challenging when it comes to advancing stem cell research and bringing treatments to patients. With greater knowledge and understanding of stem cells and how best to use them we can speed up the timeline. But without money none of that can happen.

Our blog is just one of many covering the topic of “What are the hurdles impacting patient access to cell and gene therapies as part of Signal’s fourth annual blog carnival.

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.