Dr. Robert Califf

Advancing stem cell research in many ways

/ Kevin McCormack / 2 Comments
Speakers at the Alpha Stem Cell Clinics Network Symposium: Photo by Marco Sanchez

From Day One CIRM’s goal has been to advance stem cell research in California. We don’t do that just by funding the most promising research -though the 51 clinical trials we have funded to date clearly shows we do that rather well – but also by trying to bring the best minds in the field together to overcome problems.

Over the years we have held conferences, workshops and symposiums on everything from Parkinson’s disease, cerebral palsy and tissue engineering. Each one attracted the key players and stakeholders in the field, brainstorming ideas to get past obstacles and to explore new ways of developing therapies. It’s an attempt to get scientists, who would normally be rivals or competitors, to collaborate and partner together in finding the best way forward.

It’s not easy to do, and the results are not always obvious right away, but it is essential if we hope to live up to our mission of accelerating stem cell therapies to patients with unmet medical needs.

For example. This past week we helped organize two big events and were participants in another.

The first event we pulled together, in partnership with Cedars-Sinai Medical Center, was a workshop called “Brainstorm Neurodegeneration”. It brought together leaders in stem cell research, genomics, big data, patient advocacy and the Food and Drug Administration (FDA) to tackle some of the issues that have hampered progress in finding treatments for things like Parkinson’s, Alzheimer’s, ALS and Huntington’s disease.

We rather ambitiously subtitled the workshop “a cutting-edge meeting to disrupt the field” and while the two days of discussions didn’t resolve all the problems facing us it did produce some fascinating ideas and some tantalizing glimpses at ways to advance the field.

Alpha Stem Cell Clinics Network Symposium: Photo by Marco Sanchez

Two days later we partnered with UC San Francisco to host the Fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium. This brought together the scientists who develop therapies, the doctors and nurses who deliver them, and the patients who are in need of them. The theme was “The Past, Present & Future of Regenerative Medicine” and included both a look at the initial discoveries in gene therapy that led us to where we are now as well as a look to the future when cellular therapies, we believe, will become a routine option for patients. 

Bringing these different groups together is important for us. We feel each has a key role to play in moving these projects and out of the lab and into clinical trials and that it is only by working together that they can succeed in producing the treatments and cures patients so desperately need.

Cierra Jackson: Photo by Marco Sanchez

As always it was the patients who surprised us. One, Cierra Danielle Jackson, talked about what it was like to be cured of her sickle cell disease. I think it’s fair to say that most in the audience expected Cierra to talk about her delight at no longer having the crippling and life-threatening condition. And she did. But she also talked about how hard it was adjusting to this new reality.

Cierra said sickle cell disease had been a part of her life for all her life, it shaped her daily life and her relationships with her family and many others. So, to suddenly have that no longer be a part of her caused a kind of identity crisis. Who was she now that she was no longer someone with sickle cell disease?

She talked about how people with most diseases were normal before they got sick, and will be normal after they are cured. But for people with sickle cell, being sick is all they have known. That was their normal. And now they have to adjust to a new normal.

It was a powerful reminder to everyone that in developing new treatments we have to consider the whole person, their psychological and emotional sides as well as the physical.

CIRM’s Dr. Maria Millan (right) at a panel presentation at the Stanford Drug Discovery Symposium. Panel from left to right are: James Doroshow, NCI; Sandy Weill, former CEO Citigroup; Allan Jones, CEO Allen Institute

And so on to the third event we were part of, the Stanford Drug Discovery Symposium. This was a high level, invitation-only scientific meeting that included some heavy hitters – such as Nobel Prize winners Paul Berg and  Randy Schekman, former FDA Commissioner Robert Califf. Over the course of two days they examined the role that philanthropy plays in advancing research, the increasingly important role of immunotherapy in battling diseases like cancer and how tools such as artificial intelligence and big data are shaping the future.

CIRM’s President and CEO, Dr. Maria Millan, was one of those invited to speak and she talked about how California’s investment in stem cell research is delivering Something Better than Hope – which by a happy coincidence is the title of our 2018 Annual Report. She highlighted some of the 51 clinical trials we have funded, and the lives that have been changed and saved by this research.

The presentations at these conferences and workshops are important, but so too are the conversations that happen outside the auditorium, over lunch or at coffee. Many great collaborations have happened when scientists get a chance to share ideas, or when researchers talk to patients about their ideas for a successful clinical trial.

It’s amazing what happens when you bring people together who might otherwise never have met. The ideas they come up with can change the world.

BIO 2016: IMAGINE Curing Disease and Saving Lives Part 2

/ Karen Ring / Leave a comment

As promised, here is Part 2 of our blog coverage on the BIO International Convention currently ongoing in San Francisco. Here are a few more insights on the talks we attended and highlights of other coverage from top biotech journalists and media outlets.

Keynote with Dr. Bennet Omalu and Will Smith on “Concussion”

If you haven’t seen the movie Concussion, add it to your watch list right now. It’s certainly at the top of mine after listening to Nigerian-American doctor Bennet Omalu share his story about how he single-handedly changed the way the National Football League (NFL) and the world views concussions and brain science.

Will Smith and Dr. Bennet Omalu at #BIO2016

Will Smith and Dr. Bennet Omalu at #BIO2016

In this keynote address, Dr. Omalu sat down with actor Will Smith, who portrays Dr. Omalu in the movie, to discuss how knowledge and truth precipitates evolution. Because of his passion for seeking the truth, Omalu’s autopsy of former NFL player Mike Webster led to the first diagnosis of chronic traumatic encephalopathy (CTE). Omalu’s main message was that faith and science go hand in hand. “Faith searches for truth and science searches for truth. There is no end to truth.” He also emphasized that while the truth can be inconvenient, it’s worth pursuing because truth is empowering.

For Will Smith, portraying Dr. Omalu in Concussion, was both an honor and a duty. As a parent of a son who plays football, he was compelled to tell this story and share this knowledge with parents around the world. Smith was so motivated to take on Omalu’s character that he even watched Omalu conduct four autopsies so he could really understand both the man and the science behind CTE.

This dynamic conversation was the highlight of BIO, and you can read more details about it in this article by Eleena Korban of BIOtechNOW. 

Fireside chat with US FDA Commissioner Robert Califf

Robert Califf and Steve Usdin

Robert Califf and Steve Usdin

Robert Califf, the Commissioner of the US Food and Drug Administration, sat down with Steve Usdin, the Senior Editor with BioCentury, to discuss the most important topics facing the FDA right now. Here are some of his main points:

  • FDA will focus more on patient engagement. Califf said that patients should be involved from the beginning and not just be the recipients of the end product. He also touched on risk tolerance for patients and that it can vary based on disease. The FDA wants to engage patients, advocacy groups, and industry on this topic so that patients can make more educated decisions about their treatment options.
  • The cost of clinical trials is going up 3-4 times the consumer price index which is not sustainable. Califf suggested that we can use integrated health systems and already available data from electronic medical records and patient registries to reduce the costs of large clinical trials. He commented, “The question is, can you create a different playing field that would radically reduce the cost of clinical trials while actually getting us better data about what people really care about and solve their problems related to the use of our products. I think we are close to that point now.”
  • Califf mentioned the FDA’s role in President Obama’s Precision Medicine Initiative as a step towards radically accelerating the rate of drug development. The FDA is partnering with the NIH to create a cloud-based workspace where genetic information on disease can be stored, shared, and studied.
  • Lastly, Califf mentioned how the FDA is creating a virtual center of excellence for cancer research as part of the Cancer Moonshot Initiative. He said that the FDA needs to do a better job of collaborating across its different product centers and that drug devices and biologics will be brought together starting first in the oncology space, and then eventually rolled out to other disease areas. On the clinical side, they will focus on patient involvement and the needs of cancer patients.

More coverage on the FDA fireside chat from BIOtechNOW

 Final Thoughts

While BIO ends today, the partnerships, conversations, and innovation certainly will not. In just four short days, the vibrant and eager atmosphere of BIO has transformed this year’s theme of Imagination into one of hopeful reality. Curing disease and saving lives might not be in the immediate future, but after what I’ve seen at BIO, I’m confident that the groundwork has been laid out to accelerate us down this path.

Other #BIO2016 coverage

Call to Action by FDA at World Stem Cell Summit

/ Kevin McCormack / 2 Comments

Califf

FDA Deputy Commissioner Dr. Robert Califf talking at the World Stem Cell Summit

The World Stem Cell Summit annual conference in Atlanta kicked off today with a clarion call from Dr. Robert Califf, the Deputy Commissioner for the Food and Drug Administration. He told the audience:

“We want you to accelerate translation to produce safe and effective therapies that can be delivered reliably”

It was a message that everyone in the room, scientists and patient advocates, would love to be able to comply with. The question of course is how do you do that in a way that puts the emphasis on both speed, to get the therapies to patients who need them, and safety, so you don’t put those patients at risk.

That’s quite a challenge considering that, as panel moderator Julie Allickson of Wake Forest Institute for Regenerative Medicine said:

“the estimate now is it costs $2.4 billion and up to ten years to take something to the clinic.”

Even if that dollar amount is higher than many think it would take to bring a stem cell therapy to a clinical trial it is an indication of the challenge the field faces.

Califf, who has only been at the FDA for 8 months, says that regenerative medicine is:

“not the only field exploding with scientific knowledge and seeing a future that’s very different from what we see today so it’s exciting but also an enormous challenge for the FDA. One of the real eye openers for me is to be at the FDA and hear about drugs that have been on the market for 45 years and we’re still learning about them.”

He says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. “That’s an issue we struggle with every day.”

Califf was optimistic that the balance can be struck and progress can be made, but said that this can only truly be done if the patient is at the table as an active participant.

“Our national clinical research system is well intention but flawed. We need to have a new system that shares information right across the system and where patients are at the center. Patients should be driving the national research infrastructure. They are an essential part of change. It’s happening in Congress because they are hearing from constituents that this is what they want, a voice in the research being done that affects them.”

For the patients and patient advocates in the audience it was a welcome message. For years they have been calling for a louder voice in the research that affects them and their loved ones. Knowing they have a sympathetic ear in the FDA could be an encouraging sign that their voices are finally being heard.

We will be writing more as the conference unfolds so stay tuned!