Battling COVID and turning back the clock on stem cell funding

Coronavirus

Battling the virus that causes COVID-19 is something that is top of everyone’s mind right now. That’s why CIRM is funding 17 different projects targeting the virus. But one of the most valuable tools in helping develop vaccines against a wide variety of diseases in the past is now coming under threat. We’ll talk about both issues in a live broadcast we’re holding on Wednesday, October 14th at noon (PDT).

That date is significant because it’s Stem Cell Awareness Day and we thought it appropriate to host a meeting looking at two of the most important issues facing the field.

The first part of the event will focus on the 17 projects that CIRM is funding that target COVID-19. This includes three clinical trials aiming to treat people who have been infected with the virus and are experiencing some of the more severe effects, such as damaged lungs.

We’ll also look at some of the earlier stage research that includes:

  • Work to help develop a vaccine
  • Using muscle stem cells to help repair damage to the diaphragm in patients who have spent an extended period on a ventilator
  • Boosting immune system cells to help fight the virus

The second part of the event will look at ways that funding for stem cell research at the federal level is once again coming into question. The federal government has already imposed new restrictions on funding for fetal tissue research, and now there are efforts in Congress to restrict funding for embryonic stem cell research.

The impacts could be significant. Fetal tissue has been used for decades to help develop some of the most important vaccines used today including rubella, chickenpox, hepatitis A, and shingles. They have also been used to make approved drugs against diseases including hemophilia, rheumatoid arthritis, and cystic fibrosis.

We’ll look at some of the reasons why we are seeing these potential restrictions on the medical research and what impact they could have on the ability to develop new treatments for the coronavirus and other deadly diseases.

You can watch the CIRM Stem Cell Awareness Day live event by going here: https://www.youtube.com/c/CIRMTV/videos at noon on Wednesday, October 14th.

Feel free to share news about this event with anyone you think might be interested.

We look forward to seeing you there.

CIRM funded study may help explain why some people with cystic fibrosis are less prone to infection

Dr. Kelly A. Frazer, UC San Diego School of Medicine

Cystic fibrosis is a disorder that mostly affects the lungs. It is caused by a mutation in a gene called cystic fibrosis transmembrane conductance regulator (CFTR). As a result of this mutation, cells that produce mucus (a slimy substance like the one in your nose) secrete a thicker-than-normal mucus that can create blockages in the lungs and digestive system. In the lungs, this thicker mucus is a perfect breeding ground for bacteria, leading to more chronic lung infections in those with cystic fibrosis.

However, some people with cystic fibrosis don’t develop lung infections as early or as often as others with the disorder. Thanks to a CIRM funded study, Dr. Kelly Frazer and her team at the UC San Diego School of Medicine have discovered why this might happen.

In healthy people, the CFTR protein is embedded in the membrane of most cells, where it forms a channel for chlorine ions. In people with cystic fibrosis, an inherited mutation in the CFTR gene means their channels don’t work as well and cells produce more mucus. The RNF5 protein inhibits CFTR, so people with cystic fibrosis who have genetic variations that decrease RNF5 expression have CFTR channels that function a little better, and thus aren’t as prone to infections as people with high RNF5 expression.

Before we get into that, we need to dive a bit deeper into cystic fibrosis and what causes this thicker-than normal mucus. In healthy people, CFTR is embedded in the membrane of most cells, where it forms a channel that allows chloride – a component of salt – to travel through. This flow ensures that cells have the right balance of salt and water. In people with cystic fibrosis, the CFTR mutation means that the channel doesn’t work as well, the flow of water is blocked resulting in more thick and sticky mucus. There are medications that can help boost CFTR, but they are very expensive and don’t work for everyone.

Dr. Frazer and her team discovered that a gene, called RNF5, also prevents CFTR from functioning well. People with cystic fibrosis who have lower levels of RNF5 have channels that function better, with less mucus build up, compared to people with higher levels of RNF5. This could potentially explain why some with cystic fibrosis get more chronic lung infections compared to others with the condition.

In a press release by UC San Diego School of Medicine, Dr. Frazer talks about how RNF5 could play a role in treating patients.

“The cystic fibrosis field is trying to figure out what are the modifiers across the genome that increase or decrease the probability that an individual patient will respond to these expensive drugs. RNF5 may be one of these modifier genes.”

In the same press release, Dr. Matteo D’Antonio, a project scientist in Dr. Frazer’s lab, talks about how these findings could result in more personalized treatments for people with cystic fibrosis.

“This study uncovered a new aspect of cystic fibrosis — one that could lead to new drug design and development, and allow clinicians to better tailor treatments.”

The full study was published in eLife.