It’s All About the Patients

Whenever you are designing something new you always have to keep in mind who the end user is. You can make something that works perfectly fine for you, but if it doesn’t work for the people who are going to work with it day in and day out, you have been wasting your time. And their time too.

At California Institute for Regenerative Medicine (CIRM), our end users are the patients. Everything we do is about them. Starting with our mission statement: to accelerate stem cell treatments to patients with unmet medical needs. Everything we do, every decision we make, has to keep the needs of the patient in mind.

So, when we were planning our recent 2020 Grantee Meeting (with our great friends and co-hosts UC Irvine and UC San Diego) one of the things we wanted to make sure didn’t get lost in the mix was the face and the voice of the patients. Often big conferences like this are heavy on science with presentations from some of the leading researchers in the field. And we obviously wanted to make sure we had that element at the Grantee meeting. But we also wanted to make sure that the patient experience was front and center.

And we did just that. But more on that in a minute. First, let’s talk about why the voice of the patient is important.

Some years ago, Dr. David Higgins, a CIRM Board member and patient advocate for Parkinson’s Disease (PD), said that when researchers are talking about finding treatments for PD they often focus on the dyskinesia, the trembling and shaking and muscle problems. However, he said if you actually asked people with PD you’d find they were more concerned with other aspects of the disease, the insomnia, anxiety and depression among other things. The key is you have to ask.

So, we asked some of our patient advocates if they would be willing to be part of the Grantee Meeting. All of them, without hesitation, said yes. They included Frances Saldana, a mother who lost three of her children to Huntington’s disease; Kristin MacDonald, who lost her sight to a rare disorder but regained some vision thanks to a stem cell therapy and is hoping the same therapy will help restore some more; Pawash Priyank, whose son Ronnie was born with a fatal immune disorder but who, thanks to a stem cell/gene therapy treatment, is now healthy and leading a normal life.

Because of the pandemic everything was virtual, but it was no less compelling for that. We interviewed each of the patients or patient advocates beforehand and those videos kicked off each session. Hearing, and seeing, the patients and patient advocates tell their stories set the scene for what followed. It meant that the research the scientists talked about took on added significance. We now had faces and names to highlight the importance of the work the scientists were doing. We had human stories. And that gave a sense of urgency to the work the researchers were doing.

But that wasn’t all. After all the video presentations each session ended with a “live” panel discussion. And again, the patients and patient advocates were a key part of that. Because when scientists talk about taking their work into a clinical trial they need to know if the way they are setting up the trial is going to work for the patients they’re hoping to recruit. You can have the best scientists, the most promising therapy, but if you don’t design a clinical trial in a way that makes it easy for patients to be part of you won’t be able to recruit or retain the people you need to test the therapy.

Patient voices count. Patient stories count.

But more than anything, hearing and seeing the people we are trying to help reminds us why we do this work. It’s so easy to get caught up in the day to day business of our jobs, struggling to get an experiment to work, racing to get a grant application in before the deadline. Sometimes we get so caught up in the minutiae of work we lose sight of why we are doing it. Or who we are doing it for.

At CIRM we have a saying; come to work every day as if lives depend on you, because lives depend on you. Listening to the voices of patients, seeing their faces, hearing their stories, reminds us not to waste a moment. Because lives depend on all of us.

Kevin McCormack,
Senior Director Public Communications & Patient Advocate Outreach, CIRM

Browse more programs in Patient Perspective – CIRM Grantee Meeting 2020.

Wildfires and Smoke

The 2020 California wildfires are among the worst in history and the wildfire season is just starting. Wildfires have been a feature of the mountain west for eons but the fires of the last few years have been catastrophic in loss of property, life and health. With increased fires at the wildfire urban interface the effects of the fires and the smoke they cause are impacting front-line firefighters and Californians all over the state, even when the fire activity is hundreds of miles away.

The duration of the wildfire season is longer, now stretching from summer into early winter, and catastrophic wildfires are increasing in size and frequency due to climate change. The fires are also having an impact on air quality with clear evidence of an association between wildfire smoke and respiratory health. Smoke also likely causes other negative health outcomes such as adverse birth outcomes.

Dr. John Balmes, UCSF Professor of Medicine, Division of Occupational and Environmental Medicine, and Division of Pulmonary and Critical Care Medicine, ZSFG/UCSF, explains the impacts of smoke on health and discusses studies underway to improve our knowledge as we continue to deal with devastating wildfires that are projected to get worse.

Watch Where There’s Wildfire, There’s Smoke.

Climate Change Making Allergies Worse

People with allergies know that daily weather determines symptoms and that symptoms vary by season. Dr. Katherine Gundling, an allergy and immunology specialist at UCSF, looks at how the warming of our planet might affect allergic respiratory disease. What is emerging from data collected at pollen counting stations around the world is that the length of pollen season is increasing, starting earlier and ending later, especially in higher latitude and higher elevations. As temperatures increase pollen concentrations rise. And increasing temperature may also cause pollen to be more potent.

There are similar indicators that climate change is increasing mold growth. Of particular concern are indoor molds that propagate in wet environments. As sea levels rise and flooding and humidity increase, so too does mold exposure which can cause severe asthma reactions, especially in children who are more vulnerable.

The good news is that we know what to do. Climate change solutions are also solutions to improving health disparities and allergic respiratory disease.

Watch Impacts of Our Changing Climate on Allergic Respiratory Disease.

Privacy, Practicality, and Potential: The Use of Technology for Healthy Aging

That wearable fitness device on your wrist is measuring so much more than your exercise levels. Digital tools offer unprecedented opportunities in health research and healthcare but it can come at the cost of privacy. Six days of step counts are enough to identify you among a million other people – and the type of inferences that can be made from other everyday behaviors is growing rapidly.

Camille Nebeker, EdD, MS is Associate Professor of Behavioral Medicine in the Department of Family Medicine & Public Health at the UC San Diego School of Medicine. She discusses the ethical considerations of informed consent and potential harms and benefits of these technologies. She also shares ideas on how we can work together to create systems that define and encourage safe digital health research and practice.

Watch — The Digital Revolution: Ethical Implications for Research on Healthy Aging

Medicine of the Future

UCSF has a long history of pioneering biomedical research and a bold vision for advancing science and seeking new ways to improve health care delivery nationwide. But, what does that actually mean in the near future and beyond?

This new series, part of the popular Mini Medical School for the Public, takes you inside the work of UCSF scientists to learn what the next decade may bring to the world of medicine. Hailing from a wide spectrum of disciplines, each explores a different topic that has the potential to impact the future of healthcare.

UCSF was the only medical school to be ranked in the top five in the nation in both research and primary care by US News and World Report, ranking fifth in biomedical research and third in primary care education. UCSF was also the only medical school ranked in the top five in all eight of the specialty areas covered by the survey in 2019.

Browse more programs in Next: UCSF Scientists Outline What’s To Come .