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.

Regulated, reputable, and reliable – distinguishing legitimate clinical trials from predatory clinics

Here at CIRM, we get calls every day from patients asking us if there are any trials or therapies available to treat their illness or an illness affecting a loved one. Unfortunately, there are some predatory clinics that try to take advantage of this desperation by advertising unproven and unregulated treatments for a wide range of diseases such as Diabetes, Alzheimer’s, Parkinson’s, Amyotrophic Lateral Sclerosis (ALS), and Multiple Sclerosis (MS).

A recent article in the Los Angeles Times describes how one of these predatory stem cell clinics is in a class action lawsuit related to false advertising of 100% patient satisfaction. Patients were led to believe that this percentage was related to the effectiveness of the treatment, when in fact it had to do with satisfaction related to hospitality, hotel stay, and customer service. These kinds of deceptive tactics are commonplace for sham clinics and are used to convince people to pay tens of thousands of dollars for sham treatments.

But how can a patient or loved one distinguish a legitimate clinical trial or treatment from those being offered by predatory clinics? We have established the “fundamental three R’s” to help in making this distinction.

REGULATED

The United States Food and Drug Administration (FDA) has a regulated process that it uses in evaluating potential treatments from researchers seeking approval to test these in a clinical trial setting.  This includes extensive reviews by scientific peers in the community that are well informed on specific disease areas. Those that adhere to these regulations get an FDA seal of approval and are subject to extensive oversight to protect patients participating in this trial. Additionally, these regulations ensure that the potential treatments are properly evaluated for effectiveness. The 55 clinical trials that we have currently funded as well as the clinical trials being conducted in our Alpha Stem Cell Clinic Network all have this FDA seal of approval. In contrast to this, the treatments offered at predatory clinics have not gone through the rigorous standards necessary to obtain FDA approval.

REPUTABLE

We have partnered with reputable institutions to carry out the clinical trials we have funded and establish our Alpha Stem Cell Clinic Network. These are institutions that adhere to the highest scientific standards necessary to effectively evaluate potential treatments and communicate these results with extreme accuracy. These institutions have expert scientists, doctors, and nurses in the field and adhere to rigorous standards that have earned these institutions a positive reputation for carrying out their work.  The sites for the Alpha Stem Cell Clinic Network include City of Hope, UCSF, UC San Diego, UCLA, UC Davis, and UC Irvine.  In regards to the clinical trials we have directly funded, we have collaborated with other prestigious institutions such as Stanford and USC.  All these institutions have a reputation for being respected by established societies and other professionals in the field. The reputation that predatory clinics have garnered from patients, scientists, and established doctors has been a negative one. An article published in The New York Times has described the tactics used by these predatory clinics as unethical and their therapies have often been shown to be ineffective.

RELIABLE

The clinical trials we fund and those offered at our Alpha Stem Cell Clinic Network are reliable because they are trusted by patients, patient advocacy groups, and other experts in the field of regenerative medicine. A part of being reliable involves having extensive expertise and training to properly evaluate and administer treatments in a clinical trial setting. The doctors, nurses, and other experts involved in clinical trials given the go-ahead by the FDA have extensive training to carry out these trials.  These credentialed specialists are able to administer high quality clinical care to patients.  In a sharp contrast to this, an article published in Reuters showed that predatory clinics not only administer unapproved stem cell treatments to patients, but they use doctors that have not received training related to the services they provide.

Whenever you are looking at a potential clinical trial or treatment for yourself or a loved one, just remember the 3 R’s we have laid out in this blog.

Regulated, reputable, and reliable.

Breaking bad news to stem cell researchers

It’s never easy to tell someone that they are too late, that they missed the deadline. It’s particularly hard when you know that the person you are telling that to has spent years working on a project and now needs money to take it to the next level. But in science, as in life, it’s always better to tell people what they need to know rather than what they would like to hear.

And so, we have posted a notice on our website for researchers thinking about applying for funding that, except in a very few cases, they are too late, that there is no money available for new projects, whether it’s Discovery, Translational or Clinical.

Here’s that notice:

CIRM anticipates that the budget allocation of funds for new awards under the CIRM clinical program (CLIN1, CLIN2 and CLIN3) may be depleted within the next two to three months. CIRM will accept applications for the monthly deadline on June 28, 2019 but will suspend application submissions after that date until further notice. All applicants should note that the review of submitted applications may be halted at any point in the process if funds are depleted prior to completion of the 3-month review cycle. CIRM will notify applicants of such an occurrence. Therefore, submission and acceptance of an application to CIRM does not guarantee the availability of funds or completion of a review cycle.

The submission of applications for the CIRM/NHLBI Cure Sickle Cell Initiative (CLIN1 SCD, CLIN2 SCD) are unaffected and application submissions for this program will remain open.

We do, of course, have enough money set aside to continue funding all the projects our Board has already approved, but we don’t have money for new projects (except for some sickle cell disease projects).

In truth our funding has lasted a lot longer than anyone anticipated. When Proposition 71 was approved the plan was to give CIRM $300 million a year for ten years. That was back in 2004. So what happened?

Well, in the early years stem cell science was still very much in its infancy with most of the work being done at a basic or Discovery level. Those typically don’t require very large sums so we were able to fund many projects without hitting our $300m target. As the field progressed, however, more and more projects were at the clinical trial stage and those need multiple millions of dollars to be completed. So, the money went out faster.

To date we have funded 55 clinical trials and our early support has helped more than a dozen other projects get into clinical trials. This includes everything from cancer and stroke, to vision loss and diabetes. It’s a good start, but we feel there is so much more to do.

Followers of news about CIRM know there is talk about a possible ballot initiative next year that would provide another $5.5 billion in funding for us to help complete the mission we have started.

Over the years we have built a pipeline of promising projects and without continued support many of those projects face a difficult future. Funding at the federal level is under threat and without CIRM there will be a limited number of funding alternatives for them to turn to.

Telling researchers we don’t have any money to support their work is hard. Telling patients we don’t have any money to support work that could lead to new treatments for them, that’s hardest of all.

Rallying to support CIRM and stem cell research

Will CIRM be funding stem cell research after this year?

From even before we were created by the passage of Proposition 71 back in 2004, the voices of patients and patient advocates have been at the heart of CIRM’s existence. Today they are every bit as vital to the work we do, and even more essential if we are to be able to continue doing that work.  

In 2004, the patient advocate community recognized that the research we fund could help them or a loved one battling a deadly disease or disorder. And over the last 15 years that’s exactly what we have done, trying to live up to our mission of accelerating stem cell treatments to patients with unmet medical needs. And with 54 clinical trials already under our belt we have made a good start.  

But it’s just a start. We still have a lot to do. The problem is we are quickly running out of money. We expect to have enough money to fund new projects up to the end of this year. After that many great new ideas and promising projects won’t be able to apply to us for support. Some may get funding from other sources, but many won’t. We don’t want to let that happen.  

That’s why we are holding a Patient Advocate event next Tuesday, June 25th from 6-7pm in Petree Hall C., at the Los Angeles Convention Center at 1201 South Figueroa Street, LA 90015.

The event is open to everyone and it’s FREE. We have created an Eventbrite page where you can get all the details and RSVP if you are coming. And if you want to get there a little early that’s fine too, we’ll be there from 5pm onwards so you’ll have a chance to ask us any questions you might have beforehand.

It’s going to be an opportunity to learn about the real progress being made in stem cell research, thanks in no small part to CIRM’s funding. We’ll hear from the researchers who are saving lives and changing lives, and from the family of one baby alive today because of that work.

We will hear about the challenges facing CIRM and the field, but also about a possible new ballot initiative for next year that could help re-fund CIRM, giving us the opportunity to continue our work.

That’s where you, the patients and patient advocates and members of the public come in. Without you we wouldn’t be here. Without you we will disappear. Without us the field of stem cell research loses a vital source of support and funding, and potentially-life saving therapies fall by the wayside.  

We all have a huge stake in this. So we hope to see you next Tuesday, at the start of what may be the next chapter in the life of CIRM.  

Clinical trials: separating the wheat from the chaff

What do you do when the supposed solution to a problem actually turns out to be a part of the problem? That’s the situation facing people who want to direct patients to scientifically sound clinical trials. Turns out the site many were going to may be directing patients to therapies that are not only not scientifically sound, they may not even be safe.

The site in question is the www.clinicaltrials.gov website. That’s a list of all the clinical trials registered with the National Institutes of Health. In theory that should be a rock-solid list of trials that have been given the go-ahead by the Food and Drug Administration (FDA) to be tested in people. Unfortunately, the reality is very different. Many of the trials listed there have gone through the rigorous testing and approval process to earn the right to be tested in people. But some haven’t. And figuring out which is which is not easy.

The issue was highlighted by a terrific article on STAT News this week. The article’s title succinctly sums up the piece: “Stem cell clinics co-opt clinical-trials registry to market unproven therapies, critics say.”

The story highlights how clinics that are offering unproven and unapproved stem cell therapies can register their “clinical trial” on the site, even if they haven’t received FDA approval to carry out a clinical trial.

Leigh Turner, a bioethicist at the University of Minnesota and a long-time foe of these clinics, said:

“You can concoct this bogus appearance of science, call it a clinical study, recruit people to pay to participate in your study, and not only that: You can actually register on clinicaltrials.gov and have the federal government help you promote what you’re doing. That struck me as both dangerous and brilliant.”

At CIRM this is a problem we face almost every day. People call or email us asking for help finding a stem cell therapy for everything from cancer and autism to diabetes. If we are funding something or if there is one underway at one of our Alpha Stem Cell Clinics we can direct them to that particular trial. If not, the easiest thing would be to direct them to the clinicaltrials.gov site. But when you are not sure that all the programs listed are legitimate clinical trials, that’s not something we always feel comfortable doing.

As the STAT piece points out, some of the “trials” listed on the site are even being run by companies that the FDA is trying to shut down because of serious concerns about the “therapies” they are offering. One was for a Florida clinic that had blinded four people. Despite that, the clinic’s projects remain on the site where other patients can find them.

Being listed on clinicaltrials.gov gives clinics offering unproven therapies an air or legitimacy. So how can you spot a good trial from a bad one? It’s not always easy.

One red flag is if the trial is asking you to pay for the treatment. That’s considered unethical because it’s asking you to pay to be part of an experiment. Only a very few legitimate clinical trials ask patients to pay, and even then, only with permission from the FDA.

Another warning sign is anything that has a laundry list of things it can treat, everything from arthritis to Alzheimer’s. Well-designed clinical trials tend to be targeted at one condition not multiple ones.

We have put together some useful tools for patients considering taking part in a clinical trial. Here is a link to a video and infographic that tell people the questions they need to ask, and things they need to consider, before signing up for any clinical trial.

So why does the NIH continue to allow these clinics to “advertise” their programs on its website? One reason is that the NIH simply doesn’t have the bandwidth to check every listing to make sure they are legit. They have tried to make things better by including a warning, stating:

“Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Before participating in a study, talk to your health care provider and learn about the risks and potential benefits.”

The bottom line is that if you are in the market for a stem cell therapy you should approach it the way you would any potentially life-changing decision: caveat emptor, buyer beware.

CIRM-funded study helps unlock some of the genetic secrets behind macular degeneration

Retina affected by age-related macular degeneration

Age-related macular degeneration (AMD) is the leading cause of vision loss in people over 60. It affects 10 million Americans. That’s more than cataracts and glaucoma combined. The causes of AMD are not known but are believed to involve a mixture of hereditary and environmental factors. There is no treatment for it.

Now, in a CIRM-funded study, researchers at UC San Diego (UCSD) have used stem cells to help identify genetic elements that could provide some clues as to the cause, and maybe give some ideas on how to treat it.

Before we get into what the researchers did let’s take a look at what AMD does. At a basic level it attacks the retina, the thin layer of tissue that lines the back of the eye. The retina receives light, turns it into electrical signals and sends it to the brain which turns it into a visual image.

The disease destroys the macula, the part of the retina that controls our central vision. At first, sight becomes blurred or fuzzy but over time it progresses to the point where central vision is almost completely destroyed.

To try and understand why this happens the team at UCSD took skin samples from six people with AMD and, using the iPSC method, turned those cells into the kinds of cell found in the retina. Because these cells came from people who had AMD they now displayed the same characteristics as AMD-affected retinal cells. This allowed the researchers to create what is called a “disease-in-a-dish” model that allowed them to see, in real time, what is happening in AMD.

They were able to identify a genetic variant that reduces production of a protein called VEGFA, which is known to promote the growth of new blood vessels.

In a news release Kelly Frazer, director of the Institute for Genomic Medicine at UCSD and the lead author of the study, said the results were unexpected.

Kelly Frazer, PhD, UC San Diego

“We didn’t start with the VEGFA gene when we went looking for genetic causes of AMD. But we were surprised to find that with samples from just six people, this genetic variation clearly emerged as a causal factor.”

Frazer says this discovery, published in the journal Stem Cell Reports, could ultimately lead to new approaches to developing new treatments for AMD.

CIRM already funds one clinical trial-stage project targeting AMD.

The Past, the Present, and the Uncertain Future of Stem Cell Research

Ronnie, a boy who was born without a functioning immune system but who is thriving today because of CIRM funded research

When CIRM was created in 2004 the field of stem cell research was still very much in its infancy. Fast forward 15 years and it’s moving ahead at a rapid pace, probably faster than most scientists would have predicted. How fast? Find out for yourself at a free public event at UC San Diego on May 28th from 12.30 to 1.30p.

In the last 15 years CIRM has funded 53 clinical trials in everything from heart disease and stroke, to spinal cord injury, vision loss, sickle cell disease and HIV/AIDS.

UCSD was one of the first medical centers chosen to host a CIRM Alpha Stem Cell Clinic – a specialist center with the experience and expertise to deliver stem cell therapies to patients – and to date is running more than a dozen clinical trials for breast cancer, heart failure, leukemia and chronic lower back pain.

Clearly progress is being made. But the field is also facing some challenges. Funding at the federal level for stem cell research is under threat, and CIRM is entering what could be its final phase. We have enough money left to fund new projects through this year (and these are multi-year projects so they will run into 2021 or 2022) but unless there is a new round of funding we will slowly disappear. And with us, may also disappear the hopes of some of the most promising projects underway.

If CIRM goes, then projects that we have supported and nurtured through different phases of research may struggle to make it into a clinical trial because they can’t get the necessary funding.

Clearly this is a pivotal time in the field.

We will discuss all this, the past, the present and the uncertain future of stem cell research at the meeting at UC San Diego on May 28th. The doors will open at noon for registration (snacks and light refreshments will also be available) and the program runs from 12.30p to 1.30p.

The speakers are:

  • Dr. Catriona Jamieson, Director of the UC San Diego Health CIRM Alpha Stem Cell Clinic and Sanford Stem Cell Clinical Center.
  • Dr. Maria Millan, President and CEO of CIRM
  • Dr. David Higgins, CIRM Board member and Patient Advocate for Parkinson’s Disease.

And of course, we want to hear from you, so we’ll leave plenty of time for questions.

Free parking is available.

Go here for more information about the event and how you can register

Free free to share this with anyone you think might be interested in joining us and we look forward to seeing you there.

CIRM Board Approves Funding for New Clinical Trials in Solid Tumors and Pediatric Disease

Dr. Theodore Nowicki, physician in the division of pediatric hematology/oncology at UCLA. Photo courtesy of Milo Mitchell/UCLA Jonsson Comprehensive Cancer Center

The governing Board of the California Institute for Regenerative Medicine (CIRM) awarded two grants totaling $11.15 million to carry out two new clinical trials.  These latest additions bring the total number of CIRM funded clinical trials to 53. 

$6.56 Million was awarded to Rocket Pharmaceuticals, Inc. to conduct a clinical trial for treatment of infants with Leukocyte Adhesion Deficiency-I (LAD-I)

LAD-I is a rare pediatric disease caused a mutation in a specific gene that affects the body’s ability to combat infections.  As a result, infants with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations.  Those that survive infancy experience recurrent severe infections, with mortality rates for severe LAD-I at 60-75% prior to the age of two and survival very rare beyond the age of five.

Rocket Pharmaceuticals, Inc. will test a treatment that uses a patient’s own blood stem cells and inserts a functional version of the gene.  These modified stem cells are then reintroduced back into the patient that would give rise to functional immune cells, thereby enabling the body to combat infections.  

The award is in the form of a CLIN2 grant, with the goal of conducting a clinical trial to assess the safety and effectiveness of this treatment in patients with LAD-I.

This project utilizes a gene therapy approach, similar to that of three other clinical trials funded by CIRM and conducted at UCLA by Dr. Don Kohn, for X-linked Chronic Granulomatous Disease, an inherited immune deficiency “bubble baby” disease known as ADA-SCID, and Sickle Cell Disease.

An additional $4.59 million was awarded to Dr. Theodore Nowicki at UCLA to conduct a clinical trial for treatment of patients with sarcomas and other advanced solid tumors. In 2018 alone, an estimated 13,040 people were diagnosed with soft tissue sarcoma (STS) in the United States, with approximately 5,150 deaths.  Standard of care treatment for sarcomas typically consists of surgery, radiation, and chemotherapy, but patients with late stage or recurring tumor growth have few options.

Dr. Nowicki and his team will genetically modify peripheral blood stem cells (PBSCs) and peripheral blood monocular cells (PBMCs) to target these solid tumors. The gene modified stem cells, which have the ability to self-renew, provide the potential for a durable effect.

This award is also in the form of a CLIN2 grant, with the goal of conducting a clinical trial to assess the safety of this rare solid tumor treatment.

This project will add to CIRM’s portfolio in stem cell approaches for difficult to treat cancers.  A previously funded a clinical trial at UCLA uses this same approach to treat patients with multiple myeloma.  CIRM has also previously funded two clinical trials using different approaches to target other types of solid tumors, one of which was conducted at Stanford and the other at UCLA. Lastly, two additional CIRM funded trials conducted by City of Hope and Poseida Therapeutics, Inc. used modified T cells to treat brain cancer and multiple myeloma, respectively.

“CIRM has funded 23 clinical stage programs utilizing cell and gene medicine approaches” says Maria T. Millan, M.D., the President and CEO of CIRM. “The addition of these two programs, one in immunodeficiency and the other for the treatment of malignancy, broaden the scope of unmet medical need we can impact with cell and gene therapeutic approaches.”

Facebook Live: Ask the Stem Cell Team About Clinical Trials

Every day at CIRM we get emails and calls from people looking for a stem cell clinical trial to help them. Some have arthritis in the knee or hip and want to avoid surgery. Some have a child with autism and want something that will ease the symptoms. Some have cancer and conventional therapies no longer work for them. Many have run out of options. Some are running out of time.

It’s hard to tell someone who is desperate that you don’t have anything that can help them, that there are no stem cell clinical trials that would be appropriate for them. Many often push back, saying they’ve seen ads online and visited websites for companies that claim to have stem cell therapies that can help them. When I say those therapies have not been approved by the Food and Drug Administration, or even been shown to be safe let alone effective, I can hear the disappointment in their voice.

I know some will go on to try those therapies anyway, because they have nothing else. I don’t blame them. I might do the same myself.

But before making an informed decision about any therapy it is important for people to have all the facts in front of them.

That’s why we are holding a special Facebook Live “Ask the Stem Cell Team About Clinical Trials” event on Thursday, April 25th from noon till 1pm PDT.

We are bringing together three experts who will help us all understand what’s a good clinical trial, and what’s a bogus one. They will talk about:

  • Red flags that a stem cell “clinic” might be more interested in making money than making you better
  • Key things to look for to choose a bona fide stem cell clinical trial
  • What are the questions you need to ask before signing up for any clinical
  • What are good sources of information to turn to for guidance

The Stem Cell Team will talk about CIRM’s Alpha Stem Cell Clinics Network, contrasting the time and resources they devote to offering patients stem cell clinical trials that are endorsed by the FDA, with clinics that promise people their own fat or blood cells can fix everything from bad knees to multiple sclerosis.

Our experts include a doctor and a nurse from the Alpha Clinics Network with years of experience in running and managing clinical trials, plus our own Geoff Lomax who helps support the entire network.

It will be an eye opening, informative and engaging hour and we want you to be part of it.  You can either join us on the day and post questions for the panel to answer, or you can email them directly to us beforehand at info@cirm.ca.gov.

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

In the days leading up to the broadcast we’ll give you the broadcast link that will take you to the event itself.

We look forward to having you join us for this really important Facebook Live event.

If you are in the San Francisco Bay Area this week you can join us at our fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium where the topic of how to choose a clinical trial that’s right for you will be front and center.

The symposium is on Thursday, April 18th from 8.30am to 4.30pm. It’s open to the public and it’s free.

You can find details about the event, including how you can register, HERE.

Mending Stem Cells: The Past, Present & Future of Regenerative Medicine

UCSF’s Mission Bay Campus

For years we have talked about the “promise” and the “potential” of stem cells to cure patients. But more and more we are seeing firsthand how stem cells can change a patient’s life, even saving it in some cases. That’s the theme of the 4th Annual CIRM Alpha Stem Cell Clinics Network Symposium.

It’s not your usual symposium because this brings together all the key players in the field – the scientists who do the research, the nurses and doctors who deliver the therapies, and the patients who get or need those therapies. And, of course, we’ll be there; because without CIRM’s funding to support that research and therapies none of this happens.

We are going to look at some of the exciting progress being made, and what is on the horizon. But along the way we’ll also tackle many of the questions that people pose to us every day. Questions such as:

  • How can you distinguish between a good clinical trial offering legitimate treatments vs a stem cell clinic offering sham treatments?
  • What about the Right to Try, can’t I just demand I get access to stem cell therapies?
  • How do I sign up for a clinical trial, and how much will it cost me?
  • What is the experience of patients that have participated in a stem cell clinical trial?

World class researchers will also talk about the real possibility of curing diseases like sickle cell disease on a national scale, which affect around 100,000 Americans, mostly African Americans and Hispanics. They’ll discuss the use of gene editing to battle hereditary diseases like Huntington’s. And they’ll highlight how they can engineer a patient’s own immune system cells to battle deadly cancers.

So, join us for what promises to be a fascinating day. It’s the cutting edge of science. And it’s all FREE.

Here’s where you can go to find out more information and to sign up for the event.