Stories that caught our eye: $20.5 million in new CIRM discovery awards, sickle cell disease cell bank, iPSC insights

CIRM Board launches a new voyage of Discovery (Kevin McCormack).
Basic or early stage research is the Rodney Dangerfield of science; it rarely gets the respect it deserves. Yesterday, the CIRM governing Board showed that it not only respects this research, but also values its role in laying the foundation for everything that follows.

The CIRM Board approved 11 projects, investing more than $20.5 million in our Discovery Quest, early stage research program. Those include programs using gene editing techniques to develop a cure for a rare but fatal childhood disease, finding a new approach to slowing down the progress of Parkinson’s disease, and developing a treatment for the Zika virus.

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Electron micrograph of Zika virus (red circles). Image: CDC/Cynthia Goldsmith

The goal of the Discovery Quest program is to identify and explore promising new stem cell therapies or technologies to improve patient care.

In a news release Randy Mills, CIRM’s President & CEO, said we hope this program will create a pipeline of projects that will ultimately lead to clinical trials:

“At CIRM we never underestimate the importance of early stage scientific research; it is the birth place of groundbreaking discoveries. We hope these Quest awards will not only help these incredibly creative researchers deepen our understanding of several different diseases, but also lead to new approaches on how best to use stem cells to develop treatments.”

Creating the world’s largest stem cell bank for sickle cell disease (Karen Ring).
People typically visit the bank to deposit or take out cash, but with advancements in scientific research, people could soon be visiting banks to receive life-saving stem cell treatments. One of these banks is already in the works. Scientists at the Center for Regenerative Medicine (CReM) at Boston Medical Center are attempting to generate the world’s largest stem cell bank focused specifically on sickle cell disease (SCD), a rare genetic blood disorder that causes red blood cells to take on an abnormal shape and can cause intense pain and severe organ damage in patients.

To set up their bank, the team is collecting blood samples from SCD patients with diverse ethnic backgrounds and making induced pluripotent stem cells (iPSCs) from these samples. These patient stem cell lines will be used to unravel new clues into why this disease occurs and to develop new potential treatments for SCD. More details about this new SCD iPSC bank can be found in the latest edition of the journal Stem Cell Reports.

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Gustavo Mostoslavsky, M.D., PH.D., Martin Steinberg, M.D., George Murphy PH.D.
Photo: Boston Medical Center

In a news release, CReM co-founder and Professor, Gustavo Mostoslavsky, touched on the future importance of their new stem cell bank:

“In addition to the library, we’ve designed and are using gene editing tools to correct the sickle hemoglobin mutation using the stem cell lines. When coupled with corrected sickle cell disease specific iPSCs, these tools could one day provide a functional cure for the disorder.”

For researchers interested in using these new stem cell lines, CReM is making them available to researchers around the world as part of the NIH’s NextGen Consortium study.

DNA deep dive reveals ways to increase iPSC efficiency (Todd Dubnicoff)
Though the induced pluripotent stem (iPS) cell technique was first described ten years ago, many researchers continue to poke, prod and tinker with the method which reprograms an adult cell, often from skin, into an embryonic stem cell-like state which can specialize into any cell type in the body. Though this breakthrough in stem cell research is helping scientists better understand human disease and develop patient-specific therapies, the technique is hampered by its low efficiency and consistency.

This week, a CIRM-funded study from UCLA reports new insights into the molecular changes that occur during reprogramming that may help pave the way toward better iPS cell methods. The study, published in Cell, examined the changes in DNA during the reprogramming process.

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Senior authors Kathrin Plath and Jason Ernst and first authors Petko Fiziev and Constantinos Chronis.
Photo: UCLA

In a skin cell, the genes necessary for embryonic stem cell-like, or pluripotent, characteristics are all turned off. One way this shut down in gene activity occurs is through tight coiling of the DNA where the pluripotent genes are located. This physically blocks proteins called transcriptions factors from binding the DNA and activating those pluripotent genes within skin cells. On the other hand, regions of DNA carrying skin-related genes are loosely coiled, so that transcription factors can access the DNA and turn on those genes.

The iPS cell technique works by artificially adding four pluripotent transcriptions factors into skin cells which leads to changes in DNA coiling such that skin-specific genes are turned off and pluripotent genes are turned on. The UCLA team carefully mapped the areas where the transcription factors are binding to DNA during the reprogramming process. They found that the shut down of the skin genes and activation of the pluripotent genes occurs at the same time. The team also found that three of the four iPS cell factors must physically interact with each other to locate and activate the areas of DNA that are responsible for reprogramming.

Using the findings from those experiments, the team was able to identify a fifth transcription factor that helps shut down the skin-specific gene more effectively and, in turn, saw a hundred-fold increase in reprogramming efficiency. These results promise to help the researchers fine-tune the iPS cell technique and make its clinical use more practical.

Stem Cell Profiles in Courage: Brenden Whittaker

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Brenden Whittaker: Photo Colin McGuire

It’s not often you meet someone who says one of their favorite things in the world is mowing the lawn. But then, there aren’t many people in the world like Brenden Whittaker. In fact, as of this writing, he may be unique.

Brenden was born with severe chronic granulomatous disease (x-CGD), a rare genetic disorder that left him with an impaired immune system that was vulnerable to repeated bacterial and fungal infections. Over 22 years Brenden was in and out of the hospital hundreds of times, he almost died a couple of times, and lost parts of his lungs and liver.

Then he became the first person to take part in a clinical trial to treat x-CGD. UCLA researcher Don Kohn had developed a technique that removed Brenden’s blood stem cells, genetically re-engineered them to correct the mutation that caused the disease, and then returned those stem cells to Brenden. Over time they created a new blood system, and restored Brenden’s immune system.

He was cured.

We profiled Brenden for our 2016 Annual Report. Here’s an extended version of the interview we did with him, talking about his life before and after he was cured.

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Brenden with a CIRM Game Ball – signed by everyone at CIRM

Brenden’s story:

I still think about it, my disease, every few days or so and it’s weird because in the past I was sick so often; before this year, I was sick consistently for about 5 years and going to doctor’s appointments 2 or 3 times a week and being in the hospital. So, it’s weird having a cough and not having to be rushed to the ER, not having to call someone every time the smallest thing pops up, and not having to worry about what it means.

It’s been good but it’s been weird to not have to do that.  It’s a nice problem to have.

What are you doing now that you didn’t do before?

Cutting the grass is something I couldn’t do before, that I’ve taken up now. Most people look at me as if I’m crazy when I say it, but I love cutting grass, and I wasn’t able to do it for 22 years of my life.

People will complain about having to pick up after their dog goes to the bathroom and now I can follow my dog outside and can pick up after her. It really is just the little things that people don’t think of. I find enjoyment in the small things, things I couldn’t do before but now I can and not have to worry about them.

The future

I was in the boy scouts growing up so I love camping, building fires, just being outdoors. I hiked on the Appalachian Trail. Now I’ll be able to do more of that.

I have a part time job at a golf course and I’m actually getting ready to go back to school full time in January. I want to get into pre-med, go to medical school and become a doctor. All the experience I’ve had has just made me more interested in being a doctor, I just want to be in a position where I can help people going through similar things, and going through all this just made me more interested in it.

Before the last few months I couldn’t schedule my work more than a week in advance because I didn’t know if I was going to be in the hospital or what was going on. Now my boss jokes that I’m giving him plans for the next month or two. It’s amazing how far ahead you can plan when you aren’t worried about being sick or having to go to the hospital.

I’d love to do some traveling. Right now most of my traveling consists of going to and from Boston (for medical check-ups), but I would love to go to Europe, go through France and Italy. That would be a real cool trip. I don’t need to see everything in the world but just going to other countries, seeing cities like London, Paris and Rome, seeing how people live in other cultures, that would be great.

Advice for others

I do think about the fact that when I was born one in a million kids were diagnosed with this disease and there weren’t any treatments. Many people only lived a few years. But to be diagnosed now you can have a normal life. That’s something all on its own. It’s almost impossible for me to fathom it’s happening, after all the years and doctor’s appointments and illnesses.

So, for people going through anything like this, I’d say just don’t give up. There are new advances being made every day and you have to keep fighting and keep getting through it, and some day it will all work out.


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Avalanches of exciting new stem cell research at the Keystone Symposia near Lake Tahoe

From January 8th to 13th, nearly 300 scientists and trainees from around the world ascended the mountains near Lake Tahoe to attend the joint Keystone Symposia on Neurogenesis and Stem Cells at the Resort at Squaw Creek. With record-high snowfall in the area (almost five feet!), attendees had to stay inside to stay warm and dry, and even when we lost power on the third day on the mountain there was no shortage of great science to keep us entertained.

Boy did it snow at the Keystone Conference in Tahoe!

Boy did it snow at the Keystone Conference in Tahoe!

One of the great sessions at the meeting was a workshop chaired by CIRM’s Senior Science Officer, Dr. Kent Fitzgerald, called, “Bridging and Understanding of Basic Science to Enable/Predict Clinical Outcome.” This workshop featured updates from the scientists in charge of three labs currently conducting clinical trials funded and supported by CIRM.

Regenerating injured connections in the spinal cord with neural stem cells

Mark Tuszynski, UCSD

Mark Tuszynski, UCSD

The first was a stunning talk by Dr. Mark from UCSD who is investigating how neural stem cells can help outcomes for those with spinal cord injury. The spinal cord contains nerves that connect your brain to the rest of your body so you can sense and move around in your environment, but in cases of severe injury, these connections are cut and the signal is lost. The most severe of these injuries is a complete transection, which is when all connections have been cut at a given spot, meaning no signal can pass through, just like how no cars could get through if a section of the Golden Gate Bridge was missing. His lab works in animal models of complete spinal cord transections since it is the most challenging to repair.

As Dr. Tuszynski put it, “the adult central nervous system does not spontaneously regenerate [after injury], which is surprising given that it does have its own set of stem cells present throughout.” Their approach to tackle this problem is to put in new stem cells with special growth factors and supportive components to let this process occur.

Just as most patients wouldn’t be able to come in for treatment right away after injury, they don’t start their tests until two weeks after the injury. After that, they inject neural stem cells from either the mouse, rat, or human spinal cord at the injury site and then wait a bit to see if any new connections form. Their group has shown very dramatic increases in both the number of new connections that regenerate from the injury site and extend much further than previous efforts have shown. These connections conduct electrochemical messages as normal neurons do, and over a year later they see no functional decline or tumors forming, which is often a concern when transplanting stem cells that normally like to divide a lot.

While very exciting, he cautions, “this research shows a major opportunity in neural repair that deserves proper study and the best clinical chance to succeed”. He says it requires thorough testing in multiple animal models before going into humans to avoid a case where “a clinical trial fails, not because the biology is wrong, but because the methods need tweaking.”

Everyone needs support – even dying cells

The second great talk was by Dr. Clive Svendsen of Cedars-Sinai Regenerative Medicine Institute on how stem cells might help provide healthy support cells to rescue dying neurons in the brains of patients with neurodegenerative diseases like Amyotrophic Lateral Sclerosis (ALS) and Parkinson’s. Some ALS cases are hereditary and would be candidates for a treatment using gene editing techniques. However, around 90 percent of ALS cases are “sporadic” meaning there is no known genetic cause. Dr. Svendsen explained how in these cases, a stem cell-based approach to at least fix the cellular cause of the disease, would be the best option.

While neurons often capture all the attention in the brain, since they are the cells that actually send messages that underlie our thoughts and behaviors, the Svendsen lab spends a great deal of time thinking about another type of cell that they think will be a powerhouse in the clinic: astrocytes. Astrocytes are often labeled as the support cells of the brain as they are crucial for maintaining a balance of chemicals to keep neurons healthy and functioning. So Dr. Svendsen reasoned that perhaps astrocytes might unlock a new route to treating neurodegenerative diseases where neurons are unhealthy and losing function.

ALS is a devastating disease that starts with early muscle twitches and leads to complete paralysis and death usually within four years, due to the rapid degeneration of motor neurons that are important for movement all over the body. Svendsen’s team found that by getting astrocytes to secrete a special growth factor, called “GDNF”, they could improve the survival of the neurons that normally die in their model of ALS by five to six times.

After testing this out in several animal models, the first FDA-approved trial to test whether astrocytes from fetal tissue can slow spinal motor neuron loss will begin next month! They will be injecting the precursor cells that can make these GDNF-releasing astrocytes into one leg of ALS patients. That way they can compare leg function and track whether the cells and GDNF are enough to slow the disease progression.

Dr. Svendsen shared with us how long it takes to create and test a treatment that is committed to safety and success for its patients. He says,

Clive Svendsen has been on a 15-year quest to develop an ALS therapy

Clive Svendsen 

“We filed in March 2016, submitted the improvements Oct 2016, and we’re starting our first patient in Feb 2017. [One document is over] 4500 pages… to go to the clinic is a lot of work. Without CIRM’s funding and support we wouldn’t have been able to do this. This isn’t easy. But it is doable!”

 

Improving outcomes in long-term stroke patients in unknown ways

Gary Steinberg

Gary Steinberg

The last speaker for the workshop, Dr. Gary Steinberg, a neurosurgeon at Stanford who is looking to change the lives of patients with severe limitations after having a stroke. The deficits seen after a stroke are thought to be caused by the death of neurons around the area where the stroke occurred, such that whatever functions they were involved with is now impaired. Outcomes can vary for stroke patients depending on how long it takes for them to get to the emergency department, and some people think that there might be a sweet spot for when to start rehabilitative treatments — too late and you might never see dramatic recovery.

But Dr. Steinberg has some evidence that might make those people change their mind. He thinks, “these circuits are not irreversibly damaged. We thought they were but they aren’t… we just need to continue figuring out how to resurrect them.”

He showed stunning videos from his Phase 1/2a clinical trial of several patients who had suffered from a stroke years before walking into his clinic. He tested patients before treatment and showed us videos of their difficulty to perform very basic movements like touching their nose or raising their legs. After carefully injecting into the brain some stem cells taken from donors and then modified to boost their ability to repair damage, he saw a dramatic recovery in some patients as quickly as one day later. A patient who couldn’t lift her leg was holding it up for five whole seconds. She could also touch her arm to her nose, whereas before all she could do was wiggle her thumb. One year later she is even walking, albeit slowly.

He shared another case of a 39 year-old patient who suffered a stroke didn’t want to get married because she felt she’d be embarrassed walking down the aisle, not to mention she couldn’t move her arm. After Dr. Steinberg’s trial, she was able to raise her arm above her head and walk more smoothly, and now, four years later, she is married and recently gave birth to a boy.

But while these studies are incredibly promising, especially for any stroke victims, Dr. Steinberg himself still is not sure exactly how this stem cell treatment works, and the dramatic improvements are not always consistent. He will be continuing his clinical trial to try to better understand what is going on in the injured and recovering brain so he can deliver better care to more patients in the future.

The road to safe and effective therapies using stem cells is long but promising

These were just three of many excellent presentations at the conference, and while these talks involved moving science into human patients for clinical trials, the work described truly stands on the shoulders of all the other research shared at conferences, both present and past. In fact, the reason why scientists gather at conferences is to give one another feedback and to learn from each other to better their own work.

Some of the other exciting talks that are surely laying down the framework for future clinical trials involved research on modeling mini-brains in a dish (so-called cerebral organoids). Researchers like Jürgen Knoblich at the Institute of Molecular Biotechnology in Austria talked about the new ways we can engineer these mini-brains to be more consistent and representative of the real brain. We also heard from really fundamental biology studies trying to understand how one type of cell becomes one vs. another type using the model organism C. elegans (a microscopic, transparent worm) by Dr. Oliver Hobert of Columbia University. Dr. Austin Smith, from the University of Cambridge in the UK, shared the latest about the biology of pluripotent cells that can make any cell type, and Stanford’s Dr. Marius Wernig, one of the meeting’s organizers, told us more of what he’s learned about the road to reprogramming an ordinary skin cell directly into a neuron.

Stay up to date with the latest research on stem cells by continuing to follow this blog and if you’re reading this because you’re considering a stem cell treatment, make sure you find out what’s possible and learn about what to ask by checking out closerlookatstemcells.org.


Samantha Yammine

Samantha Yammine

Samantha Yammine is a science communicator and a PhD candidate in Dr. Derek van der Kooy’s lab at the University of Toronto. You can learn more about Sam and her research on her website.

Has the promise of stem cells been overstated?

One of the most famous stem cell scientists in the world said on Monday that the promise of stem cell treatments has in some ways been overstated.

In an interview with the New York Times, Dr. Shinya Yamanaka, one of the recipients of the 2012 Nobel Prize in Medicine for his discovery of induced pluripotent stem cells (iPS cells), said, “we can help just a small portion of patients by stem cell therapy.”

Shinya Yamanaka. (Image source: Ko Sasaki, New York Times)

Shinya Yamanaka. (Image source: Ko Sasaki, New York Times)

He explained that there are only 10 target diseases that he believes will benefit directly from stem cell therapies including, “Parkinson’s, retinal and corneal diseases, heart and liver failure, diabetes, spinal cord injury, joint disorders and some blood disorders. But maybe that’s all. The number of human diseases is enormous.”

This is a big statement coming from a key opinion leader in the field of stem cell research, and it’s likely to spur a larger conversation on the future of stem cell treatments.

Yamanaka also touched on another major point in his interview – progress takes time.

In the ten years since his discovery of iPS cells, he and other scientists have learned the hard way that the development of stem cell treatments can be time consuming. While autologous iPS cell treatments (making stem cell lines from a patient and transplanting them back into that patient) have entered clinical trials to treat patients with macular degeneration, a disease that causes blindness, the trials have been put on hold until the safety of the stem cell lines being used are confirmed.

At the World Alliance Forum in November, Yamanaka revealed that generating a single patient iPS cell line can cost up to one million dollars which isn’t feasible for the 1000’s of patients who need them. He admitted that the fate of personalized stem cell medicine, which once seemed so promising, now seems unrealistic because it’s time consuming and costly.

But with any obstacle, there is always a path around it. Under Yamanaka’s guidance, Japan is generating donor iPS cell lines that can be used to treat a large portion of the Japanese population. Yamanaka said that 100 lines would cover 100 million people in Japan and that 200 lines would be enough to cover the US population. iPS cell banks are being generated around the world, meaning that one day the millions of people suffering from the target diseases Yamanaka mentioned could be treated or even cured. Would this not fulfill a promise that was made about the potential of stem cell treatments?

Which brings me to my point, I don’t believe the promise of stem cells has been overstated. I think that it has yet to be realized, and it will take more research and more time to get there. As a community, we need to be understanding, patient, and supportive.

In my opinion (as a scientist aside from my role at CIRM), I believe that Yamanaka’s interview failed to reveal his optimism about the future of stem cell treatments. What I took from Yamanaka’s comments is that stem cell treatments can help a small number of patients with specific diseases right now. That’s not to say that stem cell research won’t produce promising treatments for other diseases in the future.

Retinal diseases and blood disorders are easier to target with stem cell treatments because only one type of cell needs to be replaced. It makes sense to tackle those diseases first and make sure that these stem cell treatments are effective and safe in patients before we focus on more complicated diseases where multiple cell types or organs are involved.

Part of the reason why scientists are unsure whether stem cell treatments can treat complex diseases is because we still don’t know the details of what causes these diseases. After we know more about what’s going wrong, including all the cell types and molecules involved, research might reveal new ways that stem cells could be used to help treat those diseases. Or on the other hand, stem cells could be used to model those diseases to help discover new drug treatments.

I’ve heard Yamanaka talk many times and recently I heard him speak at the World Alliance Forum in November, where he said that the two biggest hurdles we are facing for stem cell treatments to be successful is time and cost. After we overcome these hurdles, his outlook was optimistic that stem cell treatments could improve people’s lives. But he stressed that these advances will take time.

He shared a similar sentiment at the very end of the NY Times interview by referencing his father’s story and the decades it took to cure hepatitis C,

“You know, my father had a small factory. He injured his leg in the factory when I was in junior high. He had a transfusion, and he got hepatitis C. He passed away in 1989. Twenty-five years later, just two years ago, scientists developed a very effective cure. We now have a tablet. Three months and the virus is gone — it’s amazing. But it took 25 years. iPS cells are only 10 years old. The research takes time. That’s what everybody needs to understand.”

Yamanaka says more time is needed for stem cell treatments to become effective cures, but CIRM has already witnessed success. In our December Board meeting, we heard from two patients who were cured of genetic blood diseases by stem cell treatments that CIRM funded. One of them was diagnosed with severe combined immunodeficiency (SCID) and the other had chronic granulomatous disease (CGD). Both had their blood stem cells genetically engineered to removed disease-causing mutations and then transplanted back into their body to create a healthy immune system and cure them of their disease.

Hearing how grateful these patients and their families were to receive life-saving stem cell treatments and how this research brings new hope to other patients suffering from the same diseases, in my mind, fulfills the promise of stem cell research and makes funding stem cell treatments worth it.

I believe we will hear more and more of these success stories in the next decade and CIRM will most certainly play an important role in this future. There are others in the field who share a similar optimism for the future of stem cell treatments. Hank Greely, the Director for Law and the Biosciences at Stanford University, said in an interview with the Sacramento Bee about the future of CIRM,

Hank Greely, Stanford University

Hank Greely, Stanford University

“The next few years should determine just how good California’s investment has been. It is encouraging to see CIRM supporting so many clinical trials; it will be much more exciting when – and I do expect ‘when’ and not ‘if’ – one of those trials leads to an approved treatment.”

 


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Stem Cells Profile in Courage: Pat Furlong, Patient Advocate

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Pat Furlong: Photo by Colin McGuire – http://www.colinmcguire.com

One of the true joys for me in helping put together this year’s Annual Report was getting to know the patients and patient advocates that we profiled in the report. These are some extraordinary individuals and the short profiles we posted only touch the surface of just how extraordinary.

So, over the next few weeks we are going to feature four of these people at greater length, allowing them, in their own words, to talk about what makes them tic, and how they keep going in the face of what is often heartbreak and tragedy.

We begin with Pat Furlong, a Patient Advocate and the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (DMD).

DMD is the most common fatal, genetic childhood disorder, which affects approximately 1 out of every 3,500 boys each year worldwide. It’s a progressive muscle disorder that leads to loss of muscle function, meaning you lose your ability to walk, to use your arms, and ultimately to breathe. And because the heart is a muscle, that is often seriously affected. There is no cure, and treatment options are limited. At the time her sons were diagnosed life expectancy was in the teens.

Pat’s story:

“When my sons, Chris and Pat were diagnosed with DMD, at the ages of 4 and 6, there was nothing available for them. Doctors cared about them but they didn’t have the tools they needed, or the National Institutes of Health the money it needed to do research.

Doctors were faced with diagnosing a disease and saying “there’s nothing we can do”. And then parents like me, coming to them hearing there was nothing they could do, no hope, no help. When your son is diagnosed with something like this you are told go home and love them.

When I asked questions, I was often ignored or dismissed by some doctors.

When my sons were diagnosed with DMD I would drop them off at school and go walking and that would help me deal with the anger.

For me staying in this is to be able to say to Chris and Pat in the universe, when you were here I tried my very best and when you were gone I continued to try my best so that others would have advantages that you didn’t receive.

I haven’t stood back and said I can’t go on.

The family is all scarred, we all suffered this loss. It’s much more apparent when we are together, there are empty chairs, emptiness. If we go to a family gathering we wish Chris and Pat were here, could be married. Now there’s my husband and our two daughters. We have a granddaughter, who is wonderful, but still we are incomplete and we will live with that forever.

I am trained as a nurse and I find DMD equal parts fascinating disease, heartbreaking and painful. I try to emphasize the fascinating so I can keep going. There are frustrations; lack of money, the slow process of regulatory approval, but I have an incredible team of very smart people and we are passionate about change so that helps keep us going.

Your only interest can’t be DMD, it can’t be. For me it’s certainly a priority, but it’s not my only interest. I love to go to an art museum and see how creative people work. I love Cirque du Soleil because they do things with their muscles I can’t imagine. Going outside and seeing these things makes the world better.

I am interested in the expression of art, to see how people dress, to see how people are creative, I love creativity, I think the human spirit is pretty amazing and the creativity around it. I think we are all pretty amazing but sometimes we don’t say it enough.

I recently saw a woman on the subway with a pair of tennis shoes that said “you are beautiful” and people around her were looking at her shoes and smiling, just because of those shoes. We forget to interact, and that was such a simple way of doing that.

bucket-feet

 

I relax by doing yoga, 90-minute hot yoga, as often as I can. I’ve also done a number of half marathons, but I’m more a walker than a runner. I find getting outside or hot yoga makes me concentrate on what I’m doing so that I can’t think of anything else. I can put it down and think about nothing and whisper prayers to my sons and say am I doing the right thing, is there something I should be doing differently? It’s my time to think about them and meditate about what they think would be important.

You need to give your mind time to cope, so it’s putting your phone down and your computer away. It’s getting rid of those interruptions. To put the phone, the computer down and get in a hot room and do yoga, or run around outside, to look at a tree and think about the changing season, the universe, the sun. It’s an incredible break for the brain to be able to rest.

I think the disease has made us kinder people and more thoughtful. When Chris died, we found a notebook he kept. In it was written “the meaning of life is a life of meaning”. I think that’s where we have all landed, what we all strive for, a life of meaning.

 

 

 

What’s Your 2017 Stem Cell Resolution?

January marks the beginning of a new year and is typically a time when people make resolutions to better themselves. This year at CIRM, we’re shaking things up and making stem cell resolutions.

What’s your #StemCellResolution?

Our goal is to raise awareness about the importance of funding stem cell research and accelerating the development of safe and effective stem cell treatments for patients. We want to promote this goal not only within the scientific and patient communities but also within the general public.

That’s why we are challenging you (yes you the reader) to come up with your own stem cell resolution for 2017 and share it with us on social media during the month of January.

It’s easy and fun to participate. All you need to do is think of a resolution about stem cell research. If you’re a scientist, it could be making a resolution to apply for funding for your newest stem cell project. Don’t know anything about stem cells? How about making a resolution to learn about stem cell research for a specific disease? The options are endless!

After you decide on your resolution, you can post a selfie, video, or stem cell resolution graphic that we’ve designed (available on our website  https://www.cirm.ca.gov/stemcellresolution) on Instagram, Facebook, or Twitter. Make sure to write your resolution in your post, include the hashtag #stemcellresolution, and tag CIRM’s social media accounts.

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Have more than one resolution? No problem! Feel free to post as many stem cell resolutions during January as you want. We also encourage you to share this campaign with your friends and challenge them to participate.

Check out our video for more details on how to participate:

There be prizes!

At the end of January, we will pick the most inspiring stem cell resolutions and blog about them on the Stem Cellar. We’ll also send the people who wrote those resolutions CIRM Stem Cell Champions t-shirts.

So, what are you waiting for? We want to hear from you!

Stem cell and gene therapy research gets a good report card from industry leader

arm

Panel discussion at ARM State of the industry briefing: left to Right Robert Preti, Chair ARM; Jeff Walsh, bluebird bio; Manfred Rudiger, Kiadis Pharma; Barbara Sasu, Pfizer;  Thomas Farrell, Bellicum Pharmaceuticals. Photo courtesy ARM.

The state of the regenerative medicine field is strong and getting stronger. That was the bottom line verdict at the 2017 Cell and Gene Therapies State of the Industry briefing in San Francisco.

The briefing, an annual update on the field presented by the Alliance for Regenerative Medicine (ARM), gave a “by the numbers” look at the field and apart from one negative spot everything is moving in the right direction.

Robert Preti, Chair of ARM’s Board, said worldwide there are more than 750 regenerative companies working in the stem cell and gene therapy space. And those companies are increasingly moving the research out of the lab and into clinical trials in people.

For example, at the end of 2016 there were 802 clinical trials underway. That is a 21 percent growth over 2015. Those breakdown as follows:

Phase 1 – 271 (compared to 192 in 2015)

Phase 2 – 465 (compared to 376 in 2015)

Phase 3 – 66 (compared to 63 in 2015)

The bulk of these clinical trials, 45 percent, are focused on cancer. The second largest target, 11 percent, is on heart disease. The number of trials for neurological disorders and rare diseases are also growing in number.

Preti says the industry is at an important inflection point right now and that this growth is presenting new problems:

“The pipeline of products is robust and the technologies supporting that pipeline is even more robust. The technologies that are fueling the growth in clinical activity have accelerated so fast that we on the manufacturing side are playing catchup. We are at a point where we have to get serious about large scale commercial production.”

Preti also talked about “harmonization” of the regulatory process and the need to have a system that makes it easier for products approved for clinical trials in one country, to get approval for clinical trials in other countries.

Michael Werner, the executive director of ARM, said the organization has played a key role in helping promote the field and cited the recently passed 21st Century Cures Act as “a major win and a powerful statement of ARM’s leadership in this sector.”

But there was one area where the news wasn’t all positive, the ability of companies to raise capital. In 2015 companies raised $11 billion for research. In 2016 it was less than half of that, $5.3 billion.

With that somber note in mind it was appropriate that the panel discussion that followed the briefing was focused on the near-term and long-term challenges facing the field if it was to be commercially successful.

One of the big challenges was the issue of regulatory approval, and here the panel seemed to be more optimistic than in previous years.

Manfred Rüdiger of Kiadis Pharma said he was pleasantly surprised at how easy it was to work with different regulatory agencies in the US, Canada and Europe.

“We used them as a kind of free consultancy service, listening to their advice and making the changes they suggested so that we were able to use the same manufacturing process in Europe and Canada and the US.”

Jeff Walsh of bluebird bio, said the key to having a good working relationship with regulatory agencies like the Food and Drug Administration (FDA) is simple:

“Trust and transparency between you and the regulatory agencies is essential, it’s a critical factor in advancing your work. The agencies respond well when you have that trust. One thing we can’t be is afraid to ask. The agencies will tell you where their line is, but don’t be afraid to ask or to push the boundaries. This is new for everyone, companies and regulators, so if you are pushing it helps create the environment that allows you to work together to develop safe therapies that benefit patients.”

Another big issue was scalability in manufacturing; that it’s one thing to produce enough of a product to carry out a clinical trial but completely different if you are hoping to use that same product to treat millions of people spread out all over the US or the world.

And of course cost is always something that is front and center in people’s minds. How do you develop therapies that are not just safe and effective, but also affordable? How do companies ensure they will get reimbursed by health insurers for the treatments? No one had any simple answer to what are clearly very complex problems. But all recognized the need to start thinking about these now, long before the treatments themselves are even ready.

Walsh ended by saying:

“This is not just about what can you charge but what should you charge. We have a responsibility to engage with the agencies and ultimately the payers that make these decisions, in the same way we engage with regulatory agencies; with a sense of openness, trust and transparency. Too often companies wait too long, too late before turning to the payers and trying to decide what is appropriate to charge.”

 

 

CIRM Board member Jeff Sheehy appointed to the San Francisco Board of Supervisors

As a former journalist I love breaking news, it gets the adrenaline flowing. Usually when news is breaking it’s bad news. Today, however, I was fortunate to be present for breaking news that was, more than anything, a celebration.

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Jeff Sheehy, CIRM Board member (standing at podium) was appointed today as San Francisco’s District 8 Supervisor by Mayor Ed Lee (right of Sheehy), replacing Scott Weiner (3rd from left) who held the position before his election to the State Senate

San Francisco Mayor Ed Lee today appointed CIRM Board member, and Patient Advocate for HIV/AIDS, Jeff Sheehy, as the new Supervisor for District 8. In announcing his decision the Mayor said:

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SF Mayor Ed Lee

“This was a very important decision. I was looking for someone who is passionate, a lover of our City and our people, someone who is solution oriented. I found that person in Jeff Sheehy. He has passion and commitment. He has an intellect that is very deep and a spirit that is steeped in advocacy.”

 

Those of us at CIRM know that passion and advocacy very well. As CIRM Board Chair, Jonathan Thomas, and Vice Chair, Art Torres, said in a joint statement:

“We are delighted that Mayor Lee has chosen Jeff Sheehy to be the new Supervisor. Having worked with Jeff for many years we know that he brings intelligence, dedication and compassion to everything he does. While Jeff is the HIV/AIDS Patient Advocate member on our Board, he has always been a true champion for anyone suffering from an inadequately treated disease, making sure that their voices are heard and reflected in every decision we make. We are confident he will bring those same qualities, and that same passion to the Board of Supervisors. We are also delighted that while he takes on this new role he will still continue to be a member of the CIRM Board and help us fulfill our mission of accelerating stem cell treatments to patients with unmet medical needs.”

As the first HIV-positive person to serve on the Board Jeff said he knows there are going to be tough challenges ahead, for the LGBTQ community and the City, but he said he has one very clear goal:

“This is about the kids, they are our future. If we don’t do well for our kids, we won’t do well for our City.”

He said he is both honored and humbled to be appointed to what he calls “a very challenging job.” But anyone who knows Jeff knows that he never backs away from a challenge.

Scott Weiner, who represented District 8 before being elected to the State Senate, called Jeff “an extraordinary leader, an extraordinary thinker. Some who is tenacious and committed to serving our community.”

Congratulations to Jeff, his husband Billy and their daughter Michelle. That’s a pretty cool way to start 2017.

Cured by Stem Cells

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To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual Report for 2016, now online, is our way of checking that we are still on the right path.

I think, without wishing to boast, that it’s safe to say not only are we on target, but we might even be a little bit ahead of schedule.

The Annual Report is chock full of facts and figures but at the heart of it are the stories of the people who are the focus of all that we do, the patients. We profile six patients and one patient advocate, each of whom has an extraordinary story to tell, and each of whom exemplifies the importance of the work we support.

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Brenden Whittaker: Cured

Two stand out for one simple reason, they were both cured of life-threatening conditions. Now, cured is not a word we use lightly. The stem cell field has been rife with hyperbole over the years so we are always very cautious in the way we talk about the impact of treatments. But in these two cases there is no need to hold back: Evangelina Padilla Vaccaro and Brenden Whittaker have been cured.

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Evangelina: Cured

 

In the coming weeks we’ll feature our conversations with all those profiled in the Annual Report, giving you a better idea of the impact the stem cell treatments have had on their lives and the lives of their family. But today we just wanted to give a broad overview of the Annual Report.

The Strategic Plan was very specific in the goals it laid out for us. As an agency we had six big goals, but each Team within the agency, and each individual within those teams had their own goals. They were our own mini-maps if you like, to help us keep track of where we were individually, knowing that every time an individual met a goal they helped the Team get closer to meeting its goals.

As you read through the report you’ll see we did a pretty good job of meeting our targets. In fact, we missed only one and we’re hoping to make up for that early in 2017.

But good as 2016 was, we know that to truly fulfill our mission of accelerating treatments to patients with unmet medical needs we are going to have do equally well, if not even better, in 2017.

That work starts today.

 

Stem cell heroes: patients who had life-saving, life-changing treatments inspire CIRM Board

 

It’s not an easy thing to bring an entire Board of Directors to tears, but four extraordinary people and their families managed to do just that at the last CIRM Board meeting of 2016.

The four are patients who have undergone life-saving or life-changing stem cell therapies that were funded by our agency. The patients and their families shared their stories with the Board as part of CIRM President & CEO Randy Mill’s preview of our Annual Report, a look back at our achievements over the last year.

The four included:

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Jake Javier, whose life changed in a heartbeat the day before he graduated high school, when he dove into a swimming pool and suffered a spinal cord injury that left him paralyzed from the chest down. A stem cell transplant is giving him hope he may regain the use of his arms and hands.

 

 

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Karl Trede who had just recovered from one life-threatening disease when he was diagnosed with lung cancer, and became the first person ever treated with a new anti-tumor therapy that helped hold the disease at bay.

 

brenden_stories_of_hopeBrenden Whittaker, born with a rare immune disorder that left his body unable to fight off bacterial or fungal infections. Repeated infections cost Brenden part of his lung and liver and almost killed him. A stem cell treatment that gave him a healthy immune system cured him.

 

 

evangelinaEvangelina Padilla Vaccaro was born with severe combined immunodeficiency (SCID), also known as “bubbly baby” disease, which left her unable to fight off infections. Her future looked grim until she got a stem cell transplant that gave her a new blood system and a healthy immune system. Today, she is cured.

 

 

Normally CIRM Board meetings are filled with important, albeit often dry, matters such as approving new intellectual property regulations or a new research concept plan. But it’s one thing to vote to approve a clinical trial, and a very different thing to see the people whose lives you have helped change by funding that trial.

You cannot help but be deeply moved when you hear a mother share her biggest fear that her daughter would never live long enough to go to kindergarten and is now delighted to see her lead a normal life; or hear a young man who wondered if he would make it to his 24th birthday now planning to go to college to be a doctor

When you know you played a role in making these dreams happen, it’s impossible not to be inspired, and doubly determined to do everything possible to ensure many others like them have a similar chance at life.

You can read more about these four patients in our new Stories of Hope: The CIRM Stem Cell Four feature on the CIRM website. Additionally, here is a video of those four extraordinary people and their families telling their stories:

We will have more extraordinary stories to share with you when we publish our Annual Report on January 1st. 2016 was a big year for CIRM. We are determined to make 2017 even bigger.