Lack of diversity impacts research into Alzheimer’s and dementia

THIS BLOT IS ALSO AVAILABLE AS AN AUDIOCAST ON SPOTIFY

A National Institutes of Allergy and Infectious Diseases clinical trial admissions coordinator collects information from a volunteer to create a medical record. Credit: NIAID

Alzheimer’s research has been in the news a lot lately, and not for the right reasons. The controversial decision by the Food and Drug Administration (FDA) to approve the drug Aduhelm left many people wondering how, when, or even if it should be used on people battling Alzheimer’s disease. Now a new study is raising questions about many of the clinical trials used to test medications like Aduhelm.

The research, published in the journal Jama Neurology, looked at 302 studies on dementia published in 2018 and 2019. Most of these studies were carried out in North America or Europe, and almost 90 percent of those studied were white.

In an accompanying editorial in the journal, Dr. Cerise Elliott, PhD, of the National Institute on Aging (NIA) in Bethesda, Maryland, and co-authors wrote that this limited the value of the studies: “This, combined with the fact that only 22% of the studies they analyzed even reported on race and ethnicity, and of those, a median 89% of participants were white, reflects the fact that recruitment for research participation is challenging; however, it is unacceptable that studies continue to fail to report participant demographics and that publishers allow such omissions.”

That bias is made all the more glaring by the fact that recent data from the Centers for Disease Control and Prevention shows that among people 65 and older, the Black community has the highest prevalence of Alzheimer’s disease and related dementias (13.8%), followed by Latinx (12.2%), non-Hispanic white (10.3%), American Indian and Alaskan Native (9.1%), and Asian and Pacific Islander (8.4%) populations.

The researchers admitted that the limited sample size – more than 40 percent of the studies they looked at included fewer than 50 patients – could have impacted their findings. Even so this clearly suggests there is a huge divide between the people at greatest risk of developing Alzheimer’s, or some other form of dementia, and the people being studied.

In the editorial, Elliott and his colleagues wrote that without a more diverse and balanced patient population this kind of research: “will continue to underrepresent people most affected by the disease and perpetuate systems that exclude important valuable knowledge about the disease.”


There are more details on this in Medpage Today.

An editorial in the New England Journal of Medicine highlights how this kind of bias is all too common in medical research.

“For years, the Journal has published studies that simply do not include enough participants from the racial and ethnic groups that are disproportionately affected by the illnesses being studied to support any conclusions about their treatment. In the United States, for example, Black Americans have high rates of hypertension and chronic kidney disease, Hispanic Americans have the highest prevalence of nonalcoholic fatty liver disease, Native Americans are disproportionately likely to have metabolic syndrome, and Asian Americans are at particular risk for hepatitis B infection and subsequent cirrhosis, but these groups are frequently underrepresented in clinical trials and cohort studies.”

“For too long, we have tolerated conditions that actively exclude groups from critical resources in health care delivery, research, and education. This exclusion has tragic consequences and undermines confidence in the institutions and the people who are conducting biomedical research. And clinicians cannot know how to optimally prevent and treat disease in members of communities that have not been studied.”

The encouraging news is that, finally, people are recognizing the problem and trying to come up with ways to correct it. The not so encouraging is that it took a pandemic to get us to pay attention.

At CIRM we are committed to being part of the solution. We are now requiring everyone who applies to us for funding to have a written plan on Diversity, Equity and Inclusion, laying out how their work will reflect the diversity of California. We know this will be challenging for all of us. But the alternative, doing nothing, is no longer acceptable.

A conversation with Bob Klein about the past, present and future of CIRM

Bob Klein

Anyone who knows anything about CIRM knows about Bob Klein. He’s the main author and driving force behind both Proposition 71 and Proposition 14, the voter-approved ballot initiatives that first created and then refunded CIRM. It’s safe to say that without Bob there’d be no CIRM.

Recently we had the great good fortune to sit down with Bob to chat about the challenges of getting a proposition on the ballot in a time of pandemic and electoral pandemonium, what he thinks CIRM’s biggest achievements are (so far) and what his future plans are.

You can hear that conversation in the latest episode of our podcast, “Talking ’bout (re) Generation”.

Enjoy.

We’ve got cash, here’s how you can get some

When the voters of California approved Proposition 14 last November (thanks folks) they gave us $5.5 billion to continue the work we started way back in 2014. It’s a great honor, and a great responsibility.

It’s also a great opportunity to look at what we do and how we do it and try to come up with even better ways of funding groundbreaking research and helping create a new generation of researchers.

In addition to improving on what we already do, Prop 14 introduced some new elements, some new goals for us to add to the mix, and we are in the process of fleshing out how we can best do that.

Because of all these changes we decided it would be a good idea to hold a “Town Hall” meeting and let everyone know what these changes are and how they may impact applications for funding.

The Town Hall, on Tuesday June 29, was a great success with almost 200 participants. But we know that not everyone who wanted to attend could, so here’s the video of the event, and below that are the questions that were posed by people during the meeting, and the answers to those questions.

Having seen the video we would be eternally grateful if you could respond to a short online survey, to help us get a better idea of your research and education needs and to be better able to serve you and identify potential areas of opportunity for CIRM. Here’s a link to that survey: https://www.surveymonkey.com/r/VQMYPDL

We know that there may be issues or questions that are not answered here, so feel free to send those to us at info@cirm.ca.gov and we will make sure you get an answer.

Are there any DISC funding opportunities specific to early-stage investigators?

DISC funding opportunities are open to all investigators.  There aren’t any that are specific to junior investigators.

Are DISC funding opportunities available for early-mid career researchers based out of USA such as Australia?

Sorry, you have to be in California for us to fund your work.

Does tumor immunology/ cancer immunotherapy fall within the scope of the CIRM discovery grants?

Yes, they do.  Here is a link to various CIRM DISC Awards that fall within the cancer category.  https://www.cirm.ca.gov/grants?disease_focus%5B%5D=1427&program_type%5B%5D=1230

Will Disc1 (Inception awards) and/or seed funding mechanisms become available again?

CIRM is anticipating launching a program to meet this need toward the end of this year.

For DISC award is possible to contact a grant advisor for advice before applying?

Please email discovery@cirm.ca.gov to discuss Discovery stage applications before applying

Is co-funding requirement a MUST for clinical trials?

Co-funding requirements vary.  Please refer to the following link for more information: https://www.cirm.ca.gov/sites/default/files/files/about_cirm/CLIN2_Mini_Brochure2.pdf

Hi, when will reviews for DISC 2: CIRM Quest – Discovery Stage Research Projects (deadline March 2021) be available? Thanks!

Review summaries for the March 2021 Discovery submitted applications will be available by mid-August, with final board funding decisions at the August 24th Application Review Subcommittee Meeting

Has CIRM project made it to Phase III or product launch with FDA approval? What is CIRM strategy for start-up biotech companies?

CIRM has funded several late-stage Phase III/potentially pivotal clinical trials. You can view them here: https://www.cirm.ca.gov/our-impact/funding-clinical-trials

CIRM funding supports non-profit academic grantees as well as companies of all sizes.

I am studying stem cells using mouse. Is my research eligible for the CIRM grants?

Yes it is.

Your programs more specifically into stem cell research would be willing to take patients that are not from California?

Yes, we have treated patients who are not in California. Some have come to California for treatment and others have been treated in other states in the US by companies that are based here in California.

Can you elaborate how the preview of the proposals works? Who reviews them and what are the criteria for full review?

The same GWG panel both previews and conducts the full review. The panel first looks through all the applications to identify what each reviewer believes represents the most likely to be impactful and meet the goals of the CIRM Discovery program. Those that are selected by any reviewer moves forward to the next full review step.

If you meet your milestones-How likely is it that a DISC recipient gets a TRAN award?

The milestones are geared toward preparation of the TRAN stage.  However, this is a different application and review that is not guaranteed to result in funding.

Regarding Manufacturing Public Private partnerships – What specific activities is CIRM thinking about enabling these partnerships? For example, are out of state for profit commercial entities able to conduct manufacturing at CA based manufacturing centers even though the clinical program may be primarily based out of CA? If so, what percent of the total program budget must be expended in CA? How will CIRM enable GMP manufacturing centers interact with commercial entities?

We are in the early stages of developing this concept with continued input from various stakeholders. The preliminary vision is to build a network of academic GMP manufacturing centers and industry partners to support the manufacturing needs of CIRM-funded projects in California.

We are in the process of widely distributing a summary of the manufacturing workshop. Here’s a link to it:

If a center is interested in being a sharing lab or competency hub with CIRM, how would they go about it?

CIRM will be soliciting applications for Shared Labs/Competency hubs in potential future RFAs. The survey asks several questions asking for feedback on these concepts so it would really help us if you could complete the survey.

Would preclinical development of stem cell secretome-derived protein therapies for rare neuromuscular diseases and ultimately, age-related muscle wasting be eligible for CIRM TRAN1 funding? The goal is to complete IND-enabling studies for a protein-based therapy that enhances tissue regeneration to treat a rare degenerative disease. the screening to identify the stem-cell secreted proteins to develop as therapeutics is done by in vitro screening with aged/diseased primary human progenitor cells to identify candidates that enhance their differentiation . In vivo the protein therapeutic signals to several cell types , including precursor cells to improve tissue homeostasis.

I would suggest reaching out to our Translation team to discuss the details as it will depend on several factors. You can email the team at translational@cirm.ca.gov

Here are the slides used in the presentations.

Hitting our Goals: Accelerating to the finish line

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #6 was Accelerate.

Ever wonder how long it takes for a drug or therapy to go from basic research to approval by the US Food and Drug Administration (FDA)? Around 12 years on average is the answer. That’s a long time. And it can take even longer for stem cell therapies to go that same distance.

There are a lot of reasons why it takes so long (safety being a hugely important element) but when we were sitting down in 2015 to put together our Strategic Plan we wanted to find a way to speed up that process, to go faster, without in any way reducing the focus on safety.

So, we set a goal of reducing the time it takes from identifying a stem cell therapy candidate to getting an Investigational New Drug (IND) approval from the FDA, which means it can be tested in a clinical trial. At the time it was taking us around eight years, so we decided to go big and try to reduce that time in half, to four years.

Then the question was how were we going to do that? Well, before we set the goal we did a tour of the major biomedical research institutions in California – you know, University of California Los Angeles (UCLA) UC San Francisco, Stanford etc. – and asked the researchers what would help them most. Almost without exception said “a clearing house”, a way to pair early stage investigators with later stage partners who possess the appropriate expertise and interest to advance the project to the next stage of development, e.g., helping a successful basic science investigator find a qualified partner for the project’s translational research phase.

So we set out to do that. But we didn’t stop there. We also created what we called Clinical Advisory Panels or CAPs. These consisted of a CIRM Science Officer with expertise on a particular area of research, an expert on the kind of research being done, and a Patient Representative. The idea was that CAPs would help guide and advise the research team, helping them overcome specific obstacles and get ready for a clinical trial. The Patient Representative could help the researchers understand what the needs of the patient community was, so that a trial could take those into account and be more likely to succeed. For us it wasn’t enough just to fund promising research, we were determined to do all we could to support the team behind the project to advance their work.

How did we do. Pretty good I would have to say. For our Translational stage projects, the average amount of time it took for them to move to the CLIN1 stage, the last stage before a clinical trial, was 4.18 years. For our CLIN1 programs, 73 percent of those achieved their IND within 2 years, meaning they were then ready to actually start an FDA-sanctioned clinical trial.

Of course moving fast doesn’t guarantee that the therapy will ultimately prove effective. But for an agency whose mission is “to accelerate stem cell therapies to patients with unmet medical needs”, going slow is not an option.

Hitting our goals: Making good progress

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #5 was Advance.

A dictionary definition of progression is “The act of moving forward or proceeding in a course.” That’s precisely what we set out to do when we set one of the goals in our 2015 Strategic Plan. We wanted to do all that we could to make sure the work we were funding could advance to the next stage. The goal we set was:

Advance: Increase projects advancing to the next stage of development by 50%.

The first question we faced was what did we mean by progression and how were we going to measure it? The answer basically boiled down to this: when a CIRM award completes one stage of research and gets CIRM funding to move on to the next stage or to develop a second generation of the same device or therapy.

In the pre-2016 days we’d had some success, on average getting around nine progression events every year. But if we were going to increase that by 50 percent we knew we had to step up our game and offer some incentives so that the team behind a successful project had a reason, other than just scientific curiosity, to try and move their research to the next level.

So, we created a series of linkages between the different stages of research, so the product of each successful investment was the prerequisite for the next stage of development for the research or technology.

We changed the way we funded projects, going from offering awards on an irregular basis to having them happen according to a pre-defined schedule with each program type offered multiple times a year. This meant potential applicants knew when the next opportunity to apply would come, enabling them to prepare and file at the time that was best for them and not just because we said so. We also timed these schedules so that programs could progress from one stage to the next without interruption.

But that’s not all. We recognized that some people may be great scientists at one level but didn’t have the experience or expertise to carry their project forward. So, we created both an Accelerating Center and Translating Center to help them do that. The Translating Center helped projects do the work necessary to get ready to apply to the US Food and Drug Administration (FDA) for permission to start a clinical trial. The Accelerating Center helped the team prepare that application for the trial and then plan how that trial would be carried out.

Creating these two centers had an additional benefit; it meant the work that did progress did so faster and was of a higher quality than it might otherwise have been.

Putting all those new building blocks in place meant a lot of work for the CIRM team, on top of their normal duties. But, as always, the team rose to the challenge. By the end of December 2020, a total of 74 projects had advanced or progressed to the next level, an increase of 100 percent on our pre-2016 days.

When we were laying out the goals we said that “The full implementation of these programs will create the chassis of a machine that provides a continuous, predictable, and timely pathway for the discovery and development of promising stem cell treatments.” Thanks to the voter approved Proposition 14 we now have the fund to help those treatments realize that promise.

Hitting our Goals: Playing Matchmaker

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #3 was Partner.

In the musical “Fiddler on the Roof” two of the daughters sing about their hopes of finding a husband, through the services of a matchmaker:

Matchmaker, Matchmaker,
Make me a match,
Find me a find,
Catch me a catch

While CIRM isn’t in the business of finding husbands for young ladies, we have set up ourselves as matchmakers of a very different kind. Over the course of the last five years or more we have actively tried to find deep pocketed partners for some of the researchers we are funding. You could say we are changing the last line in that verse to “Catch me some cash.” And we do.

Our goal is to help these researchers have access to the kind of money they’re going to need to move their work into clinical trials and through the Food and Drug Administration (FDA) approval process, so they are available to people who need them. To do that we created what we call our Industry Alliance Program (IAP).

The goal of the IAP is simple, to be proactive in creating partnerships between industry and our grantees, helping develop direct opportunities for industry to partner with CIRM in accelerating the most promising stem cell, gene and regenerative medicine therapy programs to commercialization.

It takes a lot of money to move a promising idea out of the lab and into the arms, or other body parts, of patients; one recent estimate put that at around $1 billion. CIRM can help with providing the funding to get projects off the ground and into clinical trials, but as you get to larger clinical trials it gets a lot more expensive. The IAP brings in well-heeled investors to help cover those expense.

Back in 2015, when we were developing our Strategic Plan, we made these partnerships one of our Big 6 goals. And, as with everything we did in that plan, we set an ambitious target of “partnering 50% of unpartnered clinical projects with commercial partners.”

So, how did we go about trying to reach that goal? Our Business Development Team (Drs Shyam Patel and Sohel Talib) worked with large companies to help identify their strategic focus and then provided them with non-confidential information about projects we fund that might interest them. If they saw something they felt had promise we introduced them to the researchers behind that project. In essence, we played matchmaker.

But it wasn’t just about making introductions. We stayed involved as the two groups got to know each other, offering both scientific and legal advice, to help them overcome any reservations or obstacles they might encounter.

So how did we do? Pretty good I would have to say. By the end of 2020 we had partnered 63% of unpartnered clinical projects, 72 events altogether, generating almost $13 billion in additional investments in these projects. That money can help move these projects through the approvals process and ultimately, we hope, into the clinic.

But we’re not done. Not by a long shot. Now that we have achieved that goal we have our eyes set on even bigger things. We are now working on creating a new Strategic Plan that is considering bringing industry in to partner with projects at earlier stages or creating public-private partnerships to ensure there is enough manufacturing capacity for all the new therapies in the pipeline.

We have a lot of work to do. But thanks to the passage of Proposition 14 we now have the time and money we need to do that work. We’ve got a lot more matchmaking to do.

Hitting our Goals: Let’s start at the beginning shall we

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #3 was Discover.

When journalists write about science a lot of the attention is often focused on clinical trials. It’s not too surprising, that’s the stage where you see if treatments really work in people and not just in the lab. But long before you get to the clinical trial stage there’s a huge amount of work that has to be done. The starting point for that work is in the Discovery stage, if it works there it moves to the Translational stage, and only after that, assuming it’s still looking promising, does it start thinking about moving into the clinic.

The Discovery, or basic, stage of research is where ideas are tested to see if they have any promise and have the potential to lead to the development of a therapy or device that could ultimately help patients. In many ways the goal of Discovery research is to gain a better understanding of how, in our case, stem cells work, and how to harness that power to treat particular diseases or disorders.

Without a rigorous Discovery research program you can’t begin to create a pipeline of promising projects that you can advance towards patients. And of course having a strong Discovery program is not much use if you don’t have somewhere for those projects to advance to, namely Translational and ultimately clinical.

So, when we were laying out our Strategic Plan goals back in 2015 we wanted to create a pipeline for all three programs, moving the most promising ones forward. So we set an ambitious goal.

Introduce 50 new therapeutic or device candidates into development.

Now this doesn’t mean just fund 50 projects hoping to develop a new therapy or device. A lot of studies that are funded, particularly at the earliest stages, have a good idea that just doesn’t pan out. In fact one quite common definition of early research – in this case from Translational Medicine Communications – is “the earliest stage of research, conducted for the advancement of knowledge, often without any concern for its practical applications.

That’s not what we wanted. We aren’t in this to do research just for its own sake. We fund research because we want it to lead somewhere, we want it to have a practical application. We want to fund projects that actually ended up with something much more promising, a candidate that might actually work and was ready to move into the next level of research to test it further.

And we almost, almost made it to the 50-candidate goal. We got to 46 and almost certainly would have made it to 50 if we hadn’t run out of money. Even so, that’s pretty impressive. There are now 46 projects ready to move on, or are already moving on, to the next level of research.

Of course, there’s no guarantee that these will ultimately end up as an FDA-approved therapy or device. But if you don’t set goals, you’ll never score. And now, thanks to the passage of Proposition 14, we have a chance to support those projects as they move forward.

Hitting our Goals: Scoring a half century

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #2 was Expand.

Scientist preparing a sample vial for automated analysis in the lab.

When CIRM first started there was an internal report that said if we managed to help get one project into a clinical trial before we ran out of money we would be doing well. At the time that seemed quite reasonable. The field was still very much in its infancy and most of the projects we were funding, particularly in the early days, were Discovery or basic research projects.

But as the field advanced we got a little bolder. By 2010 we were funding not just our first clinical trial, but the first clinical trial in the world using embryonic stem cells. This was the Geron trial targeting spinal cord injury. Sadly the excitement didn’t last very long. After treating just five patients Geron pulled the plug on the trial, deciding that targeting cancer was a better bet.

Happily, Geron returned all the money we had loaned them, plus interest, so we were able to use that to fund more research. Soon enough we had a number of other promising candidates heading towards a meeting with the US Food and Drug Administration (FDA) to try and get permission to start a clinical trial.

By 2014, ten years after we began, we actually had ten projects either running or getting ready to start a clinical trial. We thought that was really good. But at CIRM, really good is never good enough.

For our Strategic Plan in 2015 we decided to shoot for the moon and aim to get another 50 clinical trials over the next five years. At the time it seemed, to be honest, a bit bonkers. How on earth were we going to do that. But then our Therapeutics team went a hunting!

In the past we had the luxury of mostly just waiting for people with promising projects to approach us for funding. With an ambitious goal of getting 50 more clinical trials, we couldn’t afford to wait. The Therapeutics team scouted around for promising projects, inside and outside California, inside and outside the US, and pitched them on the benefits of applying for funding. Slowly the numbers started to rise.

By the end of 2016 we had 12 new trials. In 2017 we were really cruising along, adding 16 more trials. 2018 there was another 14 and that was also the year we passed the 50 clinical trials total since CIRM was created. We celebrated at a Board meeting with a balloon and a cake (we’re a state agency, our budget doesn’t extend to confetti). Initially the inscription on the cake read ‘Congratulations: 50 Clinical Trails’. Happily, we were able to fix it before anyone noticed. But even with the spelling error, it would still have tasted just fine.

Patient advocate Rich Lajara with the Big Balloon celebration for funding 50 clinical trials

By the time we got to mid-2020 we were stuck on 47 and with time, and money, running out it looked like we might miss the goal. But then our team put in one last effort and with weeks to spare we funded four more clinical trials for a total of 51 (68 since we started in 2004).

So, the moral is dream big but work hard. Now let’s see what we can dream up for our next Strategic Plan.

Hitting our goals: regulatory reform

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. We are going to start with Regulatory Reform.

The political landscape in 2015 was dramatically different than it is today. Compared to more conventional drugs and therapies stem cells were considered a new, and very different, approach to treating diseases and disorders. At the time the US Food and Drug Administration (FDA) was taking a very cautious approach to approving any stem cell therapies for a clinical trial.

A survey of CIRM stakeholders found that 70% said the FDA was “the biggest impediment for the development of stem cell treatments.” One therapy, touted by the FDA as a success story, had such a high clinical development hurdle placed on it that by the time it was finally approved, five years later, its market potential had significantly eroded and the product failed commercially. As one stakeholder said: “Is perfect becoming the enemy of better?”

So, we set ourselves a goal of establishing a new regulatory paradigm, working with Congress, academia, industry, and patients, to bring about real change at the FDA and to find ways to win faster approval for promising stem cell therapies, without in any way endangering patients.

It seemed rather ambitious at the time, but achieving that goal happened much faster than any of us anticipated. With a sustained campaign by CIRM and other industry leaders, working with the patient advocacy groups, the FDA, Congress, and President Obama, the 21st Century Cures Act was signed into law on December 13, 2016.

President Obama signs the 21st Century Cures Act.
Photo courtesy of NBC News

The law did something quite radical; it made the perspectives of patients an integral part of the FDA’s decision-making and approval process in the development of drugs, biological products and devices. And it sped up the review process by:

In a way the FDA took its foot off the brake but didn’t hit the accelerator, so the process moved faster, but in a safe, manageable way.

Fast forward to today and eight projects that CIRM funds have been granted RMAT designation. We have become allies with the FDA in helping advance the field. We have created a unique partnership with the National Heart, Lung and Blood Institute (NHLBI) to support the Cure Sickle Cell initiative and accelerate the development of cell and gene therapies for sickle cell disease.

The landscape has changed since we set a goal of regulatory reform. We still have work to do. But now we are all working together to achieve the change we all believe is both needed and possible.

A word from our Chair, several in fact

In 2005, the New Oxford American Dictionary named “podcast” its word of the year. At the time a podcast was something many had heard of but not that many actually tuned in to. My how times have changed. Now there are some two million podcasts to chose from, at least according to the New York Times, and who am I to question them.

Yesterday, in the same New York Times, TV writer Margaret Lyons, wrote about how the pandemic helped turn her from TV to podcasts: “Much in the way I grew to prefer an old-fashioned phone call to a video chat, podcasts, not television, became my go-to medium in quarantine. With their shorter lead times and intimate production values, they felt more immediate and more relevant than ever before.”

I mention this because an old colleague of ours at CIRM, Neil Littman, has just launched his own podcast and the first guest on it was Jonathan Thomas, Chair of the CIRM Board. Their conversation ranged from CIRM’s past to the future of the regenerative field as a whole, with a few interesting diversions along the way. It’s fun listening. And as Margaret Lyons said it might be more immediate and more relevant than ever before.