200 years later, the search for a cure for Parkinson’s continues

On the surface, actor Michael J. Fox, singer Neil Diamond, civil rights activist Jesse Jackson and Scottish comedian Billy Connolly would appear to have little in common. Except for one thing. They all have Parkinson’s Disease (PD).

Their celebrity status has helped raise public awareness about the condition, but studies show that awareness doesn’t amount to an understanding of PD or the extent to which it impacts someone’s life. In fact a study in the UK found that many people still don’t think PD is a serious condition.

To try and help change that people around the world will be holding events today, April 11th, World Parkinson’s Day.

The disease was first described by James Parkinson in 1817 in “An Essay on the Shaking Palsy”. In the essay Parkinson described a pattern of trembling in the hands and fingers, slower movement and loss of balance. Our knowledge about the disease has advanced in the last 200 years and now there are treatments that can help slow down the progression of the disease. But those treatments only last for a while, and so there is a real need for new treatments.  

That’s what Jun Takahashi’s team at Kyoto University in Japan hope to provide. In a first-of-its-kind procedure they took skin cells from a healthy donor and reprogrammed them to become induced pluripotent stem cells (iPSCs), or stem cells that become any type of cell. These iPSCs were then turned into the precursors of dopamine-producing neurons, the cells destroyed by PD, and implanted into 12 brain regions known to be hotspots for dopamine production.

The procedure was carried out in October and the patient, a male in his 50s, is still healthy. If his symptoms continue to improve and he doesn’t experience any bad side effects, he will receive a second dose of dopamine-producing stem cells. Six other patients are scheduled to receive this same treatment.

Earlier tests in monkeys showed that the implanted stem cells improved Parkinson’s symptoms without causing any serious side effects.

Dompaminergic neurons derived from stem cells

Scientists at UC San Francisco are trying a different approach, using gene therapy to tackle one of the most widely recognized symptoms of PD, muscle movement.

In the study, published in the journal Annals of Neurology, the team used an inactive virus to deliver a gene to boost production of dopamine in the brain. In a Phase 1 clinical trial 15 patients, whose medication was no longer able to fully control their movement disorder, were treated with this approach. Not only were they able to reduce their medication – up to 42 percent in some cases – the medication they did take lasted longer before causing dyskinesia, an involuntary muscle movement that is a common side effect of the PD medication.

In a news article Dr. Chad Christine, the first author of the study, says this approach may also help reduce other symptoms.

“Since many patients were able to substantially reduce the amount of Parkinson’s medications, this gene therapy treatment may also help patients by reducing dose-dependent side effects, such as sleepiness and nausea.” 

At CIRM we have a long history of funding research into PD. Over the years we have invested more than $55 million to try and develop new treatments for the disease.

In June 2018, the CIRM Board awarded $5.8 million to UC San Francisco’s Krystof Bankiewicz and Cedars-Sinai’s Clive Svendsen. They are using neural progenitor cells, which have the ability to multiply and turn into other kinds of brain cells, and engineering them to express the growth factor GDNF which is known to protect the cells damaged in PD. The hope is that when transplanted into the brain of someone with PD, it will help slow down, or even halt the progression of the disease. 

The CIRM funding will hopefully help the team do the pre-clinical research needed to get the FDA’s go-ahead to test this approach in a clinical trial. 

David Higgins, CIRM Board member and Patient Advocate for Parkinson’s Disease

At the time of the award David Higgins, PhD, the CIRM Board Patient Advocate for Parkinson’s Disease, said: “One of the big frustrations for people with Parkinson’s, and their families and loved ones, is that existing therapies only address the symptoms and do little to slow down or even reverse the progress of the disease. That’s why it’s important to support any project that has the potential to address Parkinson’s at a much deeper, longer-lasting level.”

But we don’t just fund the research, we try to bring the scientific community together to help identify obstacles and overcome them. In March of 2013, in collaboration with the Center for Regenerative Medicine (CRM) of the National Institutes of Health (NIH), we held a two-day workshop on cell therapies for Parkinson’s Disease. The experts outlined the steps needed to help bring the most promising research to patients.

Around one million Americans are currently living with Parkinson’s Disease. Worldwide the number is more than ten million. Those numbers are only expected to increase as the population ages. There is clearly a huge need to develop new treatments and, hopefully one day, a cure.

Till then days like April 11th will be an opportunity to remind ourselves why this work is so important.

Targeting clinics offering bogus stem cell therapies

For some years now CIRM has been raising the alarm about the growing numbers of clinics offering unproven and unapproved stem cell therapies. But we are not alone. Now a leader of the California state Assembly is taking action, trying to ensure the clinics follow the law and don’t endanger patients.

Kevin Mullin is the Speaker pro Tem in the Assembly. He is championing a bill, AB 617, that will create a Stem Cell Clinic Regulation Advisory Group. In a news release Mullin said the motivation behind the bill is simple:

“As the Chair of the Select Committee on Biotechnology, I have heard from patients who have experienced both sides of the treatment continuum. It is clear that more must be done to ensure the proper regulation of for-profit stem cell clinics.”

Concerns about these clinics are well-founded. The clinics claim the treatments they offer – usually involving the use of the patient’s own fat or blood cells – can help address everything from arthritis to Alzheimer’s but offer little or no proof. Because the “therapies” are not approved by the FDA they are not covered by insurance, so people spend thousands, sometimes tens of thousands of dollars for something that is almost guaranteed to do little to help. In some cases, the “treatments” have had disastrous results, harming patients.

The news release from Speaker pro Tem Mullin’s office says CIRM has helped position California as a leader in stem cell research. 

“Unfortunately, not all stem cell clinics are adhering to the expected high standards of review within the industry and, as a result, patients have been subjected to unscrupulous, sometimes harmful practices. AB 617 will address those entities by creating a Stem Cell Clinic Regulation Advisory Group.”

The Advisory Group will review existing licensing and certification laws for clinics offering stem cell therapies. The Group would then make recommendations to the Legislature about ways to improve the existing rules and ensure greater protection for patients. CIRM has been working with Speaker pro Tem Mullin on AB 617 and, as our President & CEO, Maria Millan, said we will continue to do so.

“We fully support AB 617 and Speaker pro Tem Mullin’s efforts to protect California consumers from unregulated and unproven stem cell treatments.  AB 617 will help patients, their families and the medical community identify legitimate clinics that offer scientifically tested clinical trials and treatments that meet federal regulatory requirements.  The field of regenerative medicine and cell and gene therapy are coming of age and entering the realm of medical practice, so AB 617 would set up an important foundation for ensuring that the highest quality care is provided to patients seeking these treatments.”

Of Mice and Men, and Women Too; Stem cell stories you might have missed

Mice brains can teach us a lot

Last week’s news headlines were dominated by one big story, the use of a stem cell transplant to effectively cure a person of HIV. But there were other stories that, while not quite as striking, did also highlight how the field is advancing.

A new way to boost brain cells (in mice!)

It’s hard to fix something if you don’t really know what’s wrong in the first place. It would be like trying to determine why a car is not working just by looking at the hood and not looking inside at the engine. The human brain is far more complex than a car so trying to determine what’s going wrong is infinitely more challenging. But a new study could help give us a new option.

Researchers in Luxembourg and Germany have developed a new computer model for what’s happening inside the brain, identifying what cells are not operating properly, and fixing them.

Antonio del Sol, one of the lead authors of the study – published in the journal Cell – says their new model allows them to identify which stem cells are active and ready to divide, or dormant. 

“Our results constitute an important step towards the implementation of stem cell-based therapies, for instance for neurodegenerative diseases. We were able to show that, with computational models, it is possible to identify the essential features that are characteristic of a specific state of stem cells.”

The work, done in mice, identified a protein that helped keep brain stem cells inactive in older animals. By blocking this protein they were able to help “wake up” those stem cells so they could divide and proliferate and help regenerate the aging brain.

And if it works in mice it must work in people right? Well, that’s what they hope to see next.

Deeper understanding of fetal development

According to the Mayo Clinic between 10 and 20 percent of known pregnancies end in miscarriage (though they admit the real number may be even higher) and our lack of understanding of fetal development makes it hard to understand why. A new study reveals a previously unknown step in this development that could help provide some answers and, hopefully, lead to ways to prevent miscarriages.

Researchers at the Karolinska Institute in Sweden used genetic sequencing to follow the development stages of mice embryos. By sorting those different sequences into a kind of blueprint for what’s happening at every stage of development they were able to identify a previously unknown phase. It’s the time between when the embryo attaches to the uterus and when it begins to turn these embryonic stem cells into identifiable parts of the body.

Qiaolin Deng, Karolinska Institute

Lead researcher Qiaolin Deng says this finding provides vital new evidence.

“Being able to follow the differentiation process of every cell is the Holy Grail of developmental biology. Knowledge of the events and factors that govern the development of the early embryo is indispensable for understanding miscarriages and congenital disease. Around three in every 100 babies are born with fetal malformation caused by faulty cellular differentiation.”

The study is published in the journal Cell Reports.

Could a new drug discovery reduce damage from a heart attack?

Every 40 seconds someone in the US has a heart attack. For many it is fatal but even for those who survive it can lead to long-term damage to the heart that ultimately leads to heart failure. Now British researchers think they may have found a way to reduce that likelihood.

Using stem cells to create human heart muscle tissue in the lab, they identified a protein that is activated after a heart attack or when exposed to stress chemicals. They then identified a drug that can block that protein and, when tested in mice that had experienced a heart attack, they found it could reduce damage to the heart muscle by around 60 percent.

Prof Michael Schneider, the lead researcher on the study, published in Cell Stem Cell, said this could be a game changer.

“There are no existing therapies that directly address the problem of muscle cell death and this would be a revolution in the treatment of heart attacks. One reason why many heart drugs have failed in clinical trials may be that they have not been tested in human cells before the clinic. Using both human cells and animals allows us to be more confident about the molecules we take forward.”

The most popular Stem Cellar posts of 2018

The blog

You never know when you write something if people are going to read it. Sometimes you wonder if anyone is going to read it. So, it’s always fun, and educational, to look back at the end of the year and see which pieces got the most eyeballs.

It isn’t always the ones you think will draw the biggest audiences. Sometimes it is diseases that are considered “rare” (those affecting fewer than 200,000 people) that get the most attention.

Maybe it’s because those diseases have such a powerful online community which shares news, any news, about their condition of interest with everyone they know. Whatever the reason, we are always delighted to share encouraging news about research we are funding or encouraging research that someone else is funding.

That was certainly the case with the top two stories this year. Both were related to ALS or Lou Gehrig’s disease.  It’s a particularly nasty condition. People diagnosed with ALS have a life expectancy of just 2 to 5 years. So it’s probably not a big surprise that stories suggesting stem cells could expand that life span got a big reception.

Whatever the reason, we’re just happy to share hopeful news with everyone who comes to our blog.

And so, without further ado, here is the list of the most popular Stem Cellar Blog Posts for 2018.

All of us in the Communications team at CIRM consider it an honor and privilege to be able to work here and to meet many of the people behind these stories; the researchers and the patients and patient advocates. They are an extraordinary group of individuals who help remind us why we do this work and why it is important. We love our work and we hope you enjoy it too. We plan to be every bit as active and engaged in 2019.

Stories that Caught Our Eye: New ways to heal old bones; and keeping track of cells once they are inside you

broken bones

How Youth Factor Can Help Repair Old Bones

As we get older things that used to heal quickly tend to take a little longer to get better. In some cases, a lot longer. Take bones for example. A fracture in someone who is in their 70’s often doesn’t heal as quickly, or completely, as in someone much younger. For years researchers have been working on ways to change that. Now we may be one step closer to doing just that.

We know that using blood stem cells can help speed up healing for bone fractures (CIRM is funding work on that) and now researchers at Duke Health believe they have figured out how that works.

The research, published in the journal Nature Communications, identifies what the Duke team call the “youth factor” inside bone marrow stem cells. It’s a type of white blood cell called a macrophage. They say the proteins these macrophages produce help stimulate bone repair.

In a news story in Medicine News Line  Benjamin Alman, senior author on the study, says:

“While macrophages are known to play a role in repair and regeneration, prior studies do not identify secreted factors responsible for the effect. Here we show that young macrophage cells play a role in the rejuvenation process, and injection of one of the factors produced by the young cells into a fracture in old mice rejuvenates the pace of repair. This suggests a new therapeutic approach to fracture rejuvenation.”

Next step, testing this in people.

A new way to track stem cells in the body

It’s one thing to transplant stem cells into a person’s body. It’s another to know that they are going to go where you want them to and do what you want them to. University of Washington researchers have invented a device that doesn’t just track where the cells end up, but also what happens to them along the way.

The device is called “CellTagging”, and in an article in Health Medicine Network, Samantha Morris, one of the lead researchers says this could help in better understanding how to use stem cells to grow replacement tissues and organs.

“There is a lot of interest in the potential of regenerative medicine — growing tissues and organs in labs — to test new drugs, for example, or for transplants one day. But we need to understand how the reprogramming process works. We want to know if the process for converting skin cells to heart cells is the same as for liver cells or brain cells. What are the special conditions necessary to turn one cell type into any other cell type? We designed this tool to help answer these questions.”

In the study, published in the journal Nature, the researchers explain how they use a virus to insert tiny DNA “barcodes” into cells and that as the cells travel through the body they are able to track them.

Morris says this could help scientists better understand the conditions needed to more effectively program cells to do what we want them to.

“Right now, cell reprogramming is really inefficient. When you take one cell population, such as skin cells, and turn it into a different cell population — say intestinal cells — only about 1 percent of cells successfully reprogram. And because it’s such a rare event, scientists have thought it is likely to be a random process — there is some correct set of steps that a few cells randomly hit upon. We found the exact opposite. Our technology lets us see that if a cell starts down the right path to reprogramming very early in the process, all of its related sibling cells and their descendants are on the same page, doing the same thing.”

Scientists say they’re one step closer to being able to build a new you, using your own stem cells.

Organ transplant

One of the biggest obstacles to transplanting organs from one person to another is that the immune system of the person getting the new life-saving organ often tries to reject it. The immune cells see the new material as “foreign” and attacks it, sometimes destroying it.

Right now, the only way to prevent that is by using powerful immunosuppressive drugs to keep the patient’s immune system at bay and protect the new organ. It’s effective, but it also comes with some long-term health consequences.

But now researchers at Tel Aviv University in Israel say they may have found a way around that, using the patient’s own stem cells.

The team says it was able to take fatty tissue from patients and, using the iPSC procedure, turn them into other kinds of cells to help repair different kinds of tissue.

In a story in the “Times of Israel”, Prof Tal Dvir, the lead researcher, said this new approach could theoretically be used to engineer any tissue type in the body.

“We were able to create a personalized hydrogel from the materials of the biopsy, to differentiate fatty tissue cells into different cell types and to engineer cardiac, spinal cord, cortical and other tissue implants to treat different diseases. Since both the cells and the material used derive from the patient, the implant does not provoke an immune response, ensuring proper regeneration of the defected organ.”

Dvir says the research, published in the journal Advanced Materials, has only been tested in animals so far but has shown great promise, helping regenerate damaged tissues in mice and rats. Their next goal is to see if they can replicate this in people.

“Theoretically we can work in every disease or disorder that cells are involved in, where tissue is dying. We can create the tissue to fix that injury by a simple injection of materials and cells at the injury site,”

While this has long been a goal of many stem cell researchers around the world, problems translating what looks good in animals into what works in people has invariably slowed down the progress of even the most promising approach. At least so far.

Stories that caught our eye: Is a Texas law opening up access to stem cell treatments working? Another CIRM-funded company gets good news from the FDA.

TexasCapitol_shutterstock_494317324

Texas Capitol. (Shutterstock)

In 2017 Texas passed a sweeping new law, HB 810, which allowed medical clinics to provide “investigational stem cell treatments to patients with certain severe chronic diseases or terminal illnesses.” Those in favor of the law argued that patients battling life-threatening or life-changing diseases should have the right to try stem cell therapies that were involved in a clinical trial.

Now a new study, published in the journal Stem Cells and Development, looks at the impact of the law. The report says that despite some recent amendments t there are still some concerns about the law including:

  • It allows treatment only if the patient has a “severe, chronic” illness but doesn’t define what that means
  • It doesn’t have clearly defined procedures on tracking and reporting procedures so it’s hard to know how many patients might be treated and what the outcomes are
  • There is no Food and Drug Administration (FDA) oversight of the patients being treated
  • Because the treatments are unproven there are fears this will “open up the state to unsavory and predatory practices by individuals preying on vulnerable patients”

The researchers conclude:

“While HB 810 opens up access to patients, it also increases significant risks for their safety and financial cost for something that might have no positive impact on their disease. Truly understanding the impact of stem cell based interventions (SCBI) requires scientific rigor, and accurate outcome data reporting must be pursued to ensure the safety and efficacy behind such procedures. This information must be readily available so that patients can make informed decisions before electing to pursue such treatments. The creation of the SCBI registry could allow for some level of scientific rigor, provide a centralized data source, and offer the potential for better informed patient choices, and might be the best option for the state to help protect patients.”

Another CIRM-funded company gets RMAT designation

Poseida

When Congress approved the 21st Century Cures Act a few years ago one of the new programs it created was the Regenerative Medicine Advanced Therapy (RMAT) designation. This was given to therapies that are designed to treat a serious or life-threatening condition, where early clinical stage trials show the approach is safe and appears to be effective.

Getting an RMAT designation is a big deal. It means the company or researchers are able to apply for an expedited review by the FDA and could get approval for wider use.

This week Poseida Therapeutics was granted RMAT designation by the Food and drug Administration (FDA) for P-BCMA-101, its CAR-T therapy for relapsed/refractory multiple myeloma. This is currently in a Phase 1 clinical trial that CIRM is funding

In this trial Poseida’s technology takes an immunotherapy approach that uses the patient’s own engineered immune system T cells to seek and destroy cancerous myeloma cells.

In a news release Eric Ostertag, Poseida’s CEO, welcomed the news:

“Initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population. We expect to have an additional data update by the end of the year and look forward to working closely with the FDA to expedite development of P-BCMA-101.”

This means that five CIRM-funded companies have now been granted RMAT designations:

How stem cells may help children battling birth injuries

From time to time we invite patients or patient advocates to post a guest blog on the Stem Cellar. Today we are featuring Brigitta Burguess, a mother and writer from Michigan, who focuses on pregnancy, parenting, and children with disabilities. Brigitta writes for the HIE Help Center, a website that offers information and supportive resources for families of children with disabilities.

HIE-Early-Intervention

Because stem cells are the building blocks of the immune system, they possess the ability to develop into other types of cells. You can use stem cells to help repair tissues, organs, and blood vessels, and even treat a host of different diseases. This is done through stem cell harvesting and stem cell therapy. In stem cell therapy, stem cells are injected into injured tissues in the hopes of replacing damaged tissue and preserving existing tissues.

Cord Blood

Every part of the human body contains stem cells. However, many areas of the body do not contain enough stem cells to make harvesting them worthwhile. Cord blood, the leftover blood collected from a baby’s umbilical cord or a mother’s placenta after birth, is especially beneficial because:

  • It provides a rich source of stem cells that can be changed into other types of cells and help to maintain and repair tissues
  • Its stem cells are immature and have not developed the ability to attack foreign cells, which makes them perfect for transplant
  • Its stem cells differ from embryonic stem cells in that they are considered adult stem cells and do not require the destruction of an embryo to harvest
  • It can be used to treat blood disorders, immune deficiencies, and certain cancers
  • Storing cord blood can help family and community members receive gene therapy treatment for the aforementioned conditions and diseases

The Applications of Stem Cell Therapy for Kids

Today, over 2,000 total cord blood stem cell transplants are performed annually, with the total number of cord blood banks worldwide reaching over 150. The innovations in stem cell therapy have made waves over the past four decades. Today, more than 80 difference diseases are being treated with cord blood stem cells.

In 2012, many clinical trials revealed that cord blood transplants were an effective treatment for cerebral palsy. Researchers also believe that cord blood stem cells have great potential in treating the neonatal brain injuries such as hypoxic-ischemic encephalopathy (HIE). As of right now, there is no indication that stem cell therapy can cure these conditions, but there is some evidence that it can lessen the severity of symptoms.

It is important to note that there is thus far no cure for hypoxic-ischemic encephalopathy (HIE) and resulting motor, cognitive, and/or intellectual disorders. Stem cell therapy seeks to limit the damage caused by HIE and reduce the severity of disabilities caused by HIE, but it is not a cure.

Because stem cell therapy is still in clinical trials, parents should think twice before going down this untested path, as no formal guidelines about administration protocol, dosages, safety, or treatment timeline have yet been established. Clinical trials are important for ensuring that treatments are safe and effective – unregulated treatments bear significant risk.

To learn more about stem cell therapy trials for hypoxic-ischemic encephalopathy, please visit the National Institute of Health’s (NIH) Clinical Trial Recruitment Center.

 

Stem Cell Agency Invests in New Immunotherapy Approach to HIV, Plus Promising Projects Targeting Blindness and Leukemia

HIV AIDS

While we have made great progress in developing therapies that control the AIDS virus, HIV/AIDS remains a chronic condition and HIV medicines themselves can give rise to a new set of medical issues. That’s why the Board of the California Institute for Regenerative Medicine (CIRM) has awarded $3.8 million to a team from City of Hope to develop an HIV immunotherapy.

The City of Hope team, led by Xiuli Wang, is developing a chimeric antigen receptor T cell or CAR-T that will enable them to target and kill HIV Infection. These CAR-T cells are designed to respond to a vaccine to expand on demand to battle residual HIV as required.

Jeff Sheehy

CIRM Board member Jeff Sheehy

Jeff Sheehy, a CIRM Board member and patient advocate for HIV/AIDS, says there is a real need for a new approach.

“With 37 million people worldwide living with HIV, including one million Americans, a single treatment that cures is desperately needed.  An exciting feature of this approach is the way it is combined with the cytomegalovirus (CMV) vaccine. Making CAR T therapies safer and more efficient would not only help produce a new HIV treatment but would help with CAR T cancer therapies and could facilitate CAR T therapies for other diseases.”

This is a late stage pre-clinical program with a goal of developing the cell therapy and getting the data needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial.

The Board also approved three projects under its Translation Research Program, this is promising research that is building on basic scientific studies to hopefully create new therapies.

  • $5.068 million to University of California at Los Angeles’ Steven Schwartz to use a patient’s own adult cells to develop a treatment for diseases of the retina that can lead to blindness
  • $4.17 million to Karin Gaensler at the University of California at San Francisco to use a leukemia patient’s own cells to develop a vaccine that will stimulate their immune system to attack and destroy leukemia stem cells
  • Almost $4.24 million to Stanford’s Ted Leng to develop an off-the-shelf treatment for age-related macular degeneration (AMD), the leading cause of vision loss in the elderly.

The Board also approved funding for seven projects in the Discovery Quest Program. The Quest program promotes the discovery of promising new stem cell-based technologies that will be ready to move to the next level, the translational category, within two years, with an ultimate goal of improving patient care.

Application Title Institution CIRM Committed Funding
DISC2-10979 Universal Pluripotent Liver Failure Therapy (UPLiFT)

 

Children’s Hospital of Los Angeles $1,297,512

 

DISC2-11105 Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer

 

Stanford $1,415,016
DISC2-10973 Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases

 

U.C. San Diego $1,160,648
DISC2-11070 Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs

 

Scripps $1,827,576
DISC2-11183 A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs

 

University of Southern California $833,971
DISC2-11199 Modulation of the Wnt pathway to restore inner ear function

 

Stanford $1,394,870
DISC2-11109 Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome

 

Stanford $1,415,016

Finally, the Board approved the Agency’s 2019 research budget. Given CIRM’s new partnership with the National Heart, Lung, Blood Institute (NHLBI) to accelerate promising therapies that could help people with Sickle Cell Disease (SCD) the Agency is proposing to set aside $30 million in funding for this program.

barbara_lee_official_photo

Congresswoman Barbara Lee (D-CA 13th District)

“I am deeply grateful for organizations like CIRM and NHLBI that do vital work every day to help people struggling with Sickle Cell Disease,” said Congresswoman Barbara Lee (D-CA 13th District). “As a member of the House Appropriations Subcommittee on Labor, Health and Human Services, and Education, I know well the importance of this work. This innovative partnership between CIRM and NHLBI is an encouraging sign of progress, and I applaud both organizations for their tireless work to cure Sickle Cell Disease.”

Under the agreement CIRM and the NHLBI will coordinate efforts to identify and co-fund promising therapies targeting SCD.  Programs that are ready to start an IND-enabling or clinical trial project for sickle cell can apply to CIRM for funding from both agencies. CIRM will share application information with the NHLBI and CIRM’s Grants Working Group (GWG) – an independent panel of experts which reviews the scientific merits of applications – will review the applications and make recommendations. The NHLBI will then quickly decide if it wants to partner with CIRM on co-funding the project and if the CIRM governing Board approves the project for funding, the two organizations will agree on a cost-sharing partnership for the clinical trial. CIRM will then set the milestones and manage the single CIRM award and all monitoring of the project.

“This is an extraordinary opportunity to create a first-of-its-kind partnership with the NHLBI to accelerate the development of curative cell and gene treatments for patients suffering with Sickle Cell Disease” says Maria T. Millan, MD, President & CEO of CIRM. “This allows us to multiply the impact each dollar has to find relief for children and adults who battle with this life-threatening, disabling condition that results in a dramatically shortened lifespan.  We are pleased to be able to leverage CIRM’s acceleration model, expertise and infrastructure to partner with the NHLBI to find a cure for this condition that afflicts 100,000 Americans and millions around the globe.”

The budget for 2019 is:

Program type 2019
CLIN1 & 2

CLIN1& 2 Sickle Cell Disease

$93 million

$30 million

TRANSLATIONAL $20 million
DISCOVER $0
EDUCATION $600K

 

 

Hits and Myths as people celebrate Stem Cell Awareness Day

UC Davis #1

Stem Cell Awareness Day at UC Davis

Every year, the second Wednesday in October is set aside as Stem Cell Awareness Day, a time to celebrate the progress being made in the field and to remind us of the challenges that lie ahead.

While the event began here in California in 2008, with then-Governor Arnold Schwarzenegger highlighting the work of CIRM, saying: ”The discoveries being made today in our Golden State will have a great impact on many around the world for generations to come.” It has since grown to become a global event.

Here in California, for example, UC Davis and the University of Southern California (USC) both held events to mark the day.

At UC Davis Jan Nolta, PhD., the Director of the Stem Cell Program, introduced a series of speakers who highlighted the terrific work being done at the university. Peter Belafsky talked about using stem cells to repair damaged trachea and to help people who are experiencing voice or swallowing disorders. Mark Lee highlighted the progress being made in using stem cells to repair hard-to-heal broken bones. Aijun Wang focused on some really exciting work that could one day lead to a therapy for spina bifida (including some ridiculously cute video of English bulldogs who are able to walk again because of this therapy.)

USC hosted 100 local high school students for a panel presentation and discussion about careers in stem cell research. The panel featured four scientists talking about their experience, why the students should think about a career in science and how to go about planning one. USC put together a terrific video of the researchers talking about their experiences, something that can help any student around the US consider becoming part of the future of stem cell research.

Similar events were held in other institutions around California. But the celebration wasn’t limited to the Golden State. At the Texas Heart Institute in Houston, Texas, they held an event to talk to the public about the clinical trials they are supporting using stem cells to help people suffering from heart failure or other heart-related issues.

RegMedNet

Finally, the UK-based RegMedNet, a community site that unites the diverse regenerative medicine community, marked the day by exploring some of the myths and misconceptions still surrounding stem cells and stem cell research.

You can read those here.

Every group takes a different approach to celebrating Stem Cell Awareness Day, but each is united by a common desire, to help people understand the progress being made in finding new treatments and even cures for people with unmet medical needs.