The Bright Side of Science

Some good science news

Last week, I hosted a science storytelling event at a local brewery. During the event, people from our local community shared moments when science had impacted their real life (read more about it here). These weren’t just stories. They were valuable reminders that science is personal and brings lots of hope. I needed that reminder because the world is scary and overwhelming right now, so I wanted to share some examples of science wins from recent months.

None of this work would have been possible without scientific funding and people trained in science. It is work like this that is currently at risk, and it needs your support (more on that here).

A Shot of Hope: Twice-Yearly HIV Prevention

HIV is a virus that damages the immune system and never goes away. It can progress to acquired immune deficiency syndrome (AIDS) that can cause death. The risk of death has been significantly reduced in those who can access medications known as antiretroviral therapy (ART). However, HIV infections can still greatly impact a person's quality of life. This is why efforts have also focused on pre-exposure prophylaxis (PrEP) to help prevent HIV infection in those who are at risk of exposure.

The FDA just approved Yeztugo (lenacapavir), which is the first-ever injectable given twice a year to prevent HIV. This means, instead of patients needing to take a daily pill, people can now receive two injections a year to stay protected. In its clinical trials, the shot was almost 100% effective at preventing HIV infection.

Why is this so cool?

• This removes barriers to daily adherence to PreP while still protecting those at risk. This could also greatly reduce global HIV transmission rates if we help other countries have access (this is now at risk thanks to USA cuts to USAID and other programs).

CRISPR Saves a Babies Life

CRISPR is a tool that can help correct spelling mistakes or errors (mutations) in DNA that cause specific diseases. It is used in the lab to study the relationship between DNA and different diseases and processes, but now it is also being used in the clinic!

Recently, Children’s Hospital of Philadelphia treated a 6-month-old baby with a rare disease (called CPS1 deficiency) that occurs because he had a mistake in one gene in his DNA. It normally has about a 50% mortality rate.

The child’s medical team was able to design personalized CRISPR treatments to correct the mutation! This has greatly improved the infant's quality of life, and he was able to go home from the hospital. Since this is a new treatment, he will continue to be monitored to see how long the treatment lasts and what follow up care might be needed.

Why is this so cool?
This offers hope for treating or curing other rare, life-threatening genetic conditions.

Photo by CDC on Unsplash

mRNA Vaccine Meets Pancreatic Cancer

Pancreatic cancer is a severe and often deadly form of cancer, with a 5-year survival rate of 3% to 50%, depending on the type and spread. These numbers illustrate that new treatments and preventions are greatly needed. Enter mRNA vaccines.

In a small early stage (phase 1) clinical trial, researchers made personally tailored mRNA vaccines for 16 pancreatic cancer patients. Half of the patients generated strong immune responses, and six of these eight patients remained cancer-free for three years. Now, they are continuing to monitor these patients and have launched phase 2 trials to get more data from studies with more patients. So, while this isn’t available yet, it is an important step towards making these type of treatment options possible.

Why is this so cool?

• It shows promise of mRNA vaccines for personalized cancer treatment, giving cancer patients hope for a better tomorrow!

Stopping Cell Death

Normally, cells die through a careful process that occurs when it’s needed. But sometimes this process doesn’t work and can result in cells dying when they shouldn’t, such as neurons. Researchers in Australia recently discovered a small molecule that helps stop a protein that can cause cells to die. In lab studies, they showed that it prevented cells from dying. This is an important first step to eventually testing this in humans. If it works, it could open doors for new treatment for diseases where cells die or are damaged. For example, conditions ike Alzheimer’s, Parkinson’s and other neurodegenerative diseases.

Why is this cool?

• Helping prevent needed cells from dying could slow or even prevent disease progression!

Why This Matters

We often think of science as distant or abstract. That’s not true. Science offers hope. Every study you read about above is the beginning of someone’s new chapter, and/or the promise of a better future (if not for them, then for other future people). Whether it’s a parent watching their baby thrive, a patient being able to not worry about getting HIV, or someone gaining years they never thought they'd have—these breakthroughs matter because people matter.

You can help — for more on how go here!

Your Turn

Have you come across a science win? Do you have an example of how science has impacted your life that you’re willing for me to share? Do you have a disease, take a medication or enjoy hiking? Then I bet you DO have a science story that you haven’t considered. Let me know and we can talk! I’d love to feature more personal stories in future newsletters. You can reply to this email or comment below.

And if this issue made you smile, please forward it. The world could use more hope right now.

---

My science posts will always be free, but if you find my work valuable and can afford to please consider supporting my work by upgrading to a paid subscription! If you can’t, that is OK too! I appreciate everyone for being here regardless. <3