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.

Women who have changed, and are changing, the world

The problem with trying to write about something like Women’s History Month is where do you start? Even if you narrow it down to women in science the list is vast.

Marie Curie

I suppose you could always start with Maria Salomea Skłodowska who is better known as Marie Curie. She not only discovered radium and polonium, but she was also the first woman to win a Nobel Prize (in Physics). When she later won another Nobel (in Chemistry) she became the first person ever to win two Nobels and is still the only person ever to win in two different fields. Not a bad place to start.

Agnes Pockels

Or how about Agnes Pockels (1862–1935). Even as a child Agnes was fascinated by science but, in Germany at the time, women were not allowed to attend university. So, she depended on her younger brother to send her his physics textbooks when he was finished with them. Agnes studied at home while taking care of her elderly parents. Doing the dishes  Agnes noticed how oils and soaps could impact the surface tension of water. So, she invented a method of measuring that surface tension. She wrote a paper about her findings that was published in Nature, and went on to become a highly respected and honored pioneer in the field.

Jennifer Doudna (left) and Emmanuelle Charpentier: Photo courtesy Nature

Fast forward to today we could certainly do worse than profile the two women who won the 2020 Nobel Prize in Chemistry for their work with the gene-editing tool CRISPR-Cas9; Jennifer Doudna at the University of California, Berkeley, and Emmanuelle Charpentier at the Max Planck Unit for the Science of Pathogens in Berlin. Their pioneering work showed how you could use CRISPR  to make precise edits in genes, creating the possibility of using it to edit human genes to eliminate or cure diseases. In fact, some CIRM-funded research is already using this approach to try and cure sickle cell disease.

In awarding the Nobel to Charpentier and Doudna, Pernilla Wittung Stafshede, a biophysical chemist and member of the Nobel chemistry committee, said: “The ability to cut DNA where you want has revolutionized the life sciences. The ‘genetic scissors’ were discovered just eight years ago but have already benefited humankind greatly.”

Barbara McClintock: Photo courtesy Brittanica

Appropriately enough none of that work would have been possible without the pioneering work of another woman, Barbara McClintock. She dedicated her career to studying the genetics of corn and developed a technique that enabled her to identify individual chromosomes in different strains of corn.

At the time it was thought that genes were stable and were arranged in a linear fashion on chromosomes, like beads on a string. McClintock’s work showed that genes could be mobile, changing position and altering the work of other genes. It took a long time before the scientific world caught up with her and realized she was right. But in 1983 she was awarded the Nobel Prize in Medicine for her work.

Katherine Johnson at her desk at Langley Research Center: Photo courtesy NASA /AFP

Katherine Johnson is another brilliant mind whose recognition came later in life. But when it did, it made her a movie star. Kind of. Johnson was a mathematician, a “computer” in the parlance of the time. She did calculations by hand, enabling NASA to safely launch and recover astronauts in the early years of the space race.

Johnson and the other Black “computers” were segregated from their white colleagues until the last 1950’s, when signs dictating which restrooms and drinking fountains they could use were removed. She was so highly regarded that when John Glenn was preparing for the flight that would make him the first American to orbit the earth he asked for her to manually check the calculations a computer had made. He trusted her far more than any machine.

Johnson and her co-workers were overlooked until the 2016 movie “Hidden Figures” brought their story to life. She was also awarded the Presidential Medal of Freedom, America’s highest civilian honor, by President Obama.

There are so many extraordinary women scientists we could talk about who have made history. But we should also remind ourselves that we are surrounded by remarkable women right now, women who are making history in their own way, even if we don’t recognized it at the moment. Researchers that CIRM funds, Dr. Catriona Jamieson at UC San Diego, Dr. Jan Nolta at UC Davis, Dr. Jane Lebkowski with Regenerative Patch technologies and so many others. They’re all helping to change the world. We just don’t know it yet.

If you would like to learn about other women who have made extraordinary contributions to science you can read about them here and here and here.

A Patient Advocate’s Personal Manifesto

Janni and Obama

President Obama and Janni Lehrer-Stein

Janni Lehrer-Stein was just 26 when she was diagnosed with a degenerative eye disease and told she was going to be blind within six months. The doctor who gave her the news told her “But don’t worry, people like you are usually hit and killed by a bus long before they go completely blind.”

At the time she was recently married, had just graduated law school and landed her dream job with the government in Washington DC, litigating workplace discrimination. The news about her eyesight stopped her in her tracks.

But not for long. If you ever met Janni you would know that nothing stops her for long.

I was fortunate enough to hear Janni talk at a Foundation Fighting Blindness event in the San Francisco Bay Area last weekend. I was part of a panel discussion on new approaches to treating vision loss, including the research that CIRM is funding.

Janni didn’t talk about stem cells, instead she focused on the importance of the patient advocate voice, community, and their determination. She said one of the most important things anyone battling a life-threatening or life-changing disease or disorder needs to remember is that it’s not about disability, it’s about capability. It’s about what you can do rather than what you cannot.

Janni laid out her “manifesto” for things she says will help you keep that thought uppermost in your mind.

1) Show up. It’s that simple and that important. You have to show up. You have to get educated, you have to learn all you can about your condition so you know what you can do and what you can’t do. You have to share that information with others. You have to be there for others. Don’t just show up for yourself. Show up for others who can’t be there.

2) Share this information. Janni talked about a website called My Retina Tracker which is helping drive research into the causes of retinal diseases like retinitis pigmentosa and macular degeneration, and hopefully will lead to treatments and even cures. She says the more people work together, the more we combine our resources, the more effective we can be.

3) Support the researchers. Janni says while raising awareness is important, raising money is just as important. Without money there can be no research, and without research no treatments or cures. Janni says it doesn’t matter how you do it – a charity walk, a Go Fund me campaign, petitioning your state or federal elected representatives to urge them to fund research – everything counts, every dollar helps.

4) Remember you are part of a wider community. Janni says no one ever won a battle on their own; it takes a lot of people to fight and win the right to be treated equally. And it takes a lot of effort to stop those rights from being rolled back.

Janni hasn’t let losing her sight hold her back. In 2011, she was appointed by President Obama, and confirmed by the U.S. Senate, to the National Council on Disability where she served two terms advising the President and Congress on national disability policy.

Now she has returned home to the San Francisco Bay Area, but she is no less determined to make a difference and no less determined to fight for the rights of patients and patient advocates.

In an article on Medium she shares her feelings about being a patient advocate:

“The America that I so deeply respect is one that embraces, values and respects the contributions of us all. My America includes every one of us, regardless of our gender, race, age or disability. Our America is a place where, regardless of whether we are sighted or blind, we have the same opportunities, for which we are equally considered. Our America includes every one of us who wishes to make the world a more peaceful, responsible, and inclusive environment that is tolerant of all differences and abilities, physical or otherwise. To me, those differences make our lives richer, give our contributions more meaning, and lead to a brighter future for the next generation.”

 

National honor for helping “the blind see”

Those of us fortunate to have good health take so many things for granted, not the least of which is our ability to see. But, according to the World Health Organization, there are 39 million people worldwide who are blind, and another 246 million who are visually impaired. Any therapy, any device, that can help change that is truly worthy of celebration.

Dr.MarkHumayun2 copy

Dr. Mark Humayun: Photo courtesy USC

That’s why we are celebrating the news that Professor Mark Humayun has been awarded the National Medal of Technology and Innovation, the nation’s top technology honor, by President Obama.

Humayun, a researcher at USC’s Keck School of Medicine and a CIRM grantee, is being honored for his work in developing an artificial retina, one that enables people with a relatively rare kind of blindness to see again.

But we are also celebrating the potential of his work that we are funding that could help restore sight to millions of people suffering from the leading cause of blindness among the elderly. But we’ll get back to that in a minute.

First, let’s talk about the invention that has earned him this prestigious award. It’s called the Argus II and it can help people with retinitis pigmentosa, an inherited degenerative disease that slowly destroys a person’s vision. It affects around 100,000 Americans.

The Argus II uses a camera mounted on glasses that send signals to an electronic receiver that has been implanted inside the eye. The receiver then relays those signals through the optic nerve to the brain where they are interpreted as a visual image.

In a story posted on the USC website, USC President C. L. Max Nikias praised Humayun’s work:

“He dreamed the impossible: to help the blind see. With fearless imagination, bold leadership and biomedical expertise, he and his team made that dream come true with the world’s first artificial retina. USC is tremendously proud to be Professor Humayun’s academic home.”

At CIRM we are tremendously proud to be funding the clinical trial that Humayun and his team are running to find a stem cell therapy for age-related macular degeneration (AMD), the leading cause of vision loss in the world.  It’s estimated that by 2020 more than 6 million Americans will suffer from AMD.

Humayun’s team is using embryonic stem cells to produce the support cells, or RPE cells, needed to replace those lost in AMD. We recently produced this video that highlights this work, and other CIRM-funded work that targets vision loss.

In a statement released by the White House honoring all the winners, President Obama said:

“Science and technology are fundamental to solving some of our nation’s biggest challenges. The knowledge produced by these Americans today will carry our country’s legacy of innovation forward and continue to help countless others around the world. Their work is a testament to American ingenuity.”

Which is why we are honored to be partners with Humayun and his team in advancing this research and, hopefully, helping find a treatment for millions of people who dream of one day being able to see again.