Vital Science

Imagine giving birth to a baby girl and noticing pieces of skin missing from her body almost immediately.

For Sharmila Nikapota, this was the case with her firstborn, Sohana. Genetic testing revealed that she was one of the over 500,000 people globally suffering from Epidermolysis Bullosa (EB), a “constantly painful and debilitating” skin condition where minimal contact can lead to blisters, wounds, tissue damage, eating difficulties, and eye injuries, among other symptoms. After seeking answers for her daughter’s prognosis and not getting any, Sharmila started Cure EB to educate people and eventually discover ways to manage this painful condition. With the UK approving a first-of-its-kind topical treatment for EB, she hopes it’s the first of many that can give patients like her daughter a better quality of life.

Join Sharmila as she discusses how Sohana’s experience with EB has shaped her personality, the origin and mission of Cure EB, the challenges of managing Sohana’s condition, and what you can do to further Cure EB’s ultimate goals.

Show Notes

Cure EB - Accelerating Research to End Painful Skin
Birch Bark Extract: A Review in Epidermis Bullosa
Charles River | Cell and Gene Therapy
Charles River | Rare Disease
Eureka Blog | Living Rare
Krystal Biotech Touts Topical Gene Therapy Uptake

Stimulant use in the United States has exploded into a nationwide crisis, as deaths and emergency room visits resulting from methamphetamine and cocaine use are rising at an alarming rate.

The overall substance abuse disorder crisis is further exacerbated by the high level of fentanyl use, which has long been referred to as an opioid epidemic, and the increasing use of more than substance at once. One fundamental limitation on the ability to respond to the crisis is that, to date, there is no FDA-approved therapeutic to treat overdose from stimulants like methamphetamine and cocaine, or polydrug. Users who want to get clean and rid themselves of methamphetamine, cocaine, and/or fentanyl often find it difficult, essentially “chaining” themselves into a cycle of use that’s mostly impossible to break.

First responders and emergency department physicians lack sufficient tools to treat overdose. But what if there was a treatment that could eliminate toxins and restore normal bodily function within minutes? That’s the aim of Clear Scientific, a small biotech in Cambridge, MA that’s currently testing a treatment (CS-1103) using small molecules to “eat” toxins and deactivate their harmful effects almost instantly. With Phase I of a first-in-human (FIH) clinical trial ongoing, this revolutionary therapeutic is offering hope to those suffering from drug misuse and addiction a “chance to get better.”

Join us as Mitch Zakin, Co-founder and Executive Vice President of Innovation, and Winston Henderson, Co-founder and General Counsel, discuss their therapeutic approach, offer an update on their progress with CS-1103, how collaboration has enhanced their development, and how they hope it will eventually impact this epidemic.

Show Notes

Clear Scientific

World Congress: Drug Modality Game Changers

Clear Scientific to Start Phase 1 Trial for Stimulant Antidote

Charles River | IND-Enabling Studies

Charles River | Small Molecule

The effects of Angelman Syndrome (AS) on young children are debilitating. From an early age, they face an uphill battle with this neurogenetic disease’s unrelenting symptoms, including seizures, cognitive impairments, and loss of motor function.

It can also be tough on caretakers – just ask Dr. Allyson Berent and Jennifer Panagoulias. They both have personal connections to this disease, which in Dr. Berent’s case led to her creating the Foundation for Angelman Syndrome Therapeutics (FAST) in 2008. Today, Panagoulias, whose niece has AS, serves as their head of Regulatory and Policy, tasked with the responsibility to find a path that will develop treatments to improve the quality of life for both patients and caregivers.

Join Jennifer as we discuss the origins of FAST, the science behind Angelman Syndrome, the crucial role outside partnerships have played in research, and what’s in the pipeline for potential therapeutics.

Show Notes

• Foundation for Angelman Syndrome Therapeutics
• ASOs Rescue Brain Rhythms, Sleep Patterns in Angelman Mouse Model
• A Big Year for Angelman Syndrome
• Antisense Therapies and Angelman's Syndrome
• Unsilencing Quincy
• The Quest to Cure Quincy
• Development of Potential Outcome Measures for AS Clinical Trials – Angelman Syndrome Foundation
  

Twins share many unique bonds, and for Jaci and Alex, fighting the same rare disease is one of them. Hear this emotional tale through the perspective of their mom, Lori, and their uncle, Mike, about how they have battled their illness, knowing the risks, and helped to develop a new treatment for this rare form of ALS.

To learn more about Jaci’s story, please visit Cowgirl Up for Jaci: Roping in a CURE for ALS. For more information about ALS, please visit Project ALS.

ACKNOWLEDGMENTS

Hosted by: Chris Garcia
Produced by: Meaghan Root, Ryan Stevenson, and Laura Robinson
Sound and Music by: Ryan Stevenson

Special thanks to: Lori Hermstad, and Mike Hadden
In memoriam: Jaci Hermstad and Alex Hermstad

Meet our hosts Gina Mullane and Chris Garcia as they introduce Vital ScienceTM, a brand new podcast that shares the personal stories of those impacted by life science. Subscribe so you don't miss us, starting October 2019.

ACKNOWLEDGMENTS

Hosted by: Gina Mullane and Chris Garcia
Produced by: Meaghan Root and Ryan Stevenson
Sound and Music by: Ryan Stevenson