Dear Friends,
For the very first time – because of your support – Propel a Cure is sponsoring
not one, not two, but
three
cutting-edge research projects that will help us get closer to understanding
Crohn's disease.
These studies break the mold by focusing on players in Crohn's that have
previously not been thoroughly investigated or understood. The data these
labs acquire from these early-stage studies will help them verify hypotheses
that in turn will potentially attract major funding leading to more effective
therapies and eventually cures for patients!
We are very thankful to all of you and to the research teams leading these
studies for your willingness to try to break beyond the status quo and
what's been done before to give hope to those with Crohn's.
|
RESEARCH UPDATE
GHOSH LAB, UC SAN DIEGO
EFFECT OF MUTATION IDENTIFIED
|
|
This past April, Propel a Cure pledged $100,000 to the Ghosh Lab at the
University of California, San Diego for a groundbreaking, cure-focused
Crohn's disease study.
Generous supporters have already donated $79,000 toward this project,
which is now underway.
You can make a big difference by helping fund the remaining $21,000 of
this initiative at
propelacure.org/donate. Every gift, regardless of size, counts!
This lab is already giving us a better understanding of Crohn's:
|
Researchers at Ghosh Lab at University of California, San Diego have made an exciting discovery that helps
explain why some people with Crohn's disease have trouble clearing infections
in their gut.
Over the past few months, the team has studied a special immune sensor
inside cells called
NOD2
and a helper protein named GIV. When working together, these two proteins
help the immune system recognize harmful bacteria and get rid of them properly—keeping
inflammation under control. But when either of these proteins isn’t working,
the gut has trouble healing, and inflammation can get out of hand.
To understand this better, the team worked with mice and immune cells
called macrophages. Mice that didn’t have
GIV
developed a type of gut disease that looked very similar to Crohn’s disease
in humans, including swelling, tissue damage, and problems with the natural
balance of gut bacteria. Most importantly, the team focused on a common
version of NOD2 called "1007fs", which is found in many Crohn's patients. This version is broken—it’s missing a key piece—and the researchers have now found out
why
that matters: It can't connect to GIV. Without that connection, the immune
system can't do its job right, and bacteria aren't cleared properly. This
leads to more inflammation and damage in the gut.
This is the first time scientists have clearly shown how this precise
Crohn's risk mutation, which was discovered originally in 2000, disrupts
the immune system's ability to clear bacteria.
Now that the team understands this key piece of the puzzle, they are excited
about the next step:
exploring ways to fix or replace this broken connection
between NOD2 and GIV as a new way to treat Crohn's disease. Beyond mice,
this team has the unique ability to test all their findings and hypotheses
as well as potential drug-like small molecules and peptides in mini human
guts called organoids. The team plans on doing those studies next. Hence,
this foundational discovery opens up new doors for treatment, and it gives
hope that future therapies could help restore balance in the gut and reduce
inflammation—right at the source.
|
RESEARCH UPDATE
WOOLSTON LAB,
NORTHEASTERN UNIVERSITY
TOOL TO TEST MICROBE IN CROHN'S
|
|
Earlier this year, Propel a Cure, in partnership with the Mendez Family
Foundation, contributed $100,000 to a project at the Woolston Lab at Northeastern
University to determine the role of the gut microbe hydrogen sulfide (H2S)
in intestinal inflammation in inflammatory bowel disease (IBD), potentially paving the way to a new
nonimmunosuppressive therapeutic that could treat Crohn's disease. The
team first engineered bacteria in the lab to try to produce excess H2S
in the animal gut. After giving their engineered bacteria to animals, they
observed a greater than 5-fold increase in intestinal H2S.
This is a critical milestone because elevating H2S levels is experimentally
difficult due to its gaseous nature. The team now has a tool capable of
elevating intestinal H2S levels – mimicking what is seen in Crohn's patients
– that will help us understand its fundamental role.
In the next phase of this work, the Woolston Lab will be able to use their
engineered bacteria to study how high levels of H2S impact Crohn's pathology,
which is a significant step forward.
|
RESEARCH UPDATE
PROMAKHOS THERAPEUTICS
THERAPEUTIC TESTED IN MICE
|
|
Boston-area Promakhos Therapeutics is pursuing a study, funded in part
by Propel a Cure, investigating what they believe to be a deficiency in
the mucosal innate immune response in Crohn's patients and an oral, nonimmunosuppressive
therapy to correct it.
Over the past few months, the researchers transplanted stool from patients
with active Crohn's disease and reduced levels of the innate immune-activating
molecules into mice to establish a humanized mouse model of bowel inflammation.
Encouragingly, the team observed that mice transplanted with stool from
patients showed a 4-fold reduction in fecal levels of the bacterial molecules,
as compared to mice transplanted with stool from healthy individuals.
Once the Crohn's patient gut microbiome was established in the mice, the
researchers induced acute colon inflammation to evaluate the therapeutic
effect of their compound. In this model, inflammation worsens for 3-4 days
and then heals over the next 10 days or so. Starting at the peak of inflammation
(Day 4), the researchers administered their compound once per day through
the mouth to restore the natural activity of the bacterial molecules in
the mouse gut.
Although the study has not been finalized, the team is excited to share
that the administration of their therapeutic compound led to a reduction
in symptoms by 73% on Day 8 and by 92% on Day 10, while placebo-treated
mice showed a 43% and 67% reduction by Days 8 and 10, respectively.
These differences were statistically significant, suggesting that the restoration
of the natural levels of the bacterial molecules with their compound greatly
accelerated healing of acute colon inflammation in these mice.
The next steps are to further analyze the results of this study and to
explore different doses of this compound in this new Crohn's patient humanized
mouse model.
|
INTERVIEW
DR. JOANA TORRES
IBD PREDICTION & PREVENTION
|
|
In our recent virtual discussion with Dr. Joana Torres, she brings us
up to date on the latest in IBD prediction
and prevention! In this
video, she shares the remarkable headway that researchers are making in predicting
disease risk years before onset, as well as how we may eventually even
be able to stop IBD before symptoms begin. She further explains how preventive
treatments may someday lead to new therapies for those patients with established
disease.
Dr. Torres is based at Hospital Beatriz Ângelo (Loures, Portugal) and
Hospital da Luz (Lisbon) as a Consultant in Gastroenterology, where she
oversees the IBD clinic, actively combining clinical practice with research
and teaching. Her research focuses on populations at risk for IBD and newly
diagnosed IBD, with the aim of finding predictors of IBD development and
disease course. She is involved in several large projects in this sphere,
including the potential development of preventive interventions, such as
the PREDICT study, the EARLY-CD study, the GlycanTrigger project, and the
large European Union-funded INTERCEPT study.
|
Click
here
to watch the interview with Dr. Torres.
|
NEW PARTNER
INTRODUCING IBD CONNECT
|
|
We're excited to introduce our newest organizational partner
IBD Connect, a U.S.-based 501(c)(3) nonprofit organization that supports pediatric
IBD patients and their families.
IBD Connect offers in-person and virtual family support groups, a virtual
caregivers support group, a virtual young adult support support group,
a financial assistance program, a college scholarship program, an IBD "warrior
bag" program, a fleece blanket program, and an online, informational blog.
We encourage you to learn more about IBD Connect's fantastic resources
at ibdconnectinc.org and look forward to working with them collaboratively
in the future to bring attention to IBD and help improve the lives of patients
and their loved ones.
|
HOPE BUILDERS HONORED
|
|
We are deeply grateful to the following longtime donors who have elected
to become Propel a Cure Hope Builders. Hope Builders are our recurring
monthly, quarterly, and yearly donors and ensure a reliable pool of funding
year-round. Their donations are critical to sustaining the work of the
labs engaged in the cutting-edge Crohn's research that we support.
|
Raihaneh Bokharai
|
Stephen Hall
|
|
Sue Cragg
|
Gustavo Nonohay
|
|
Vince Cvijanovic
|
Anthea Pritchard
|
|
Natalie Muccioli Emery
|
Devin Rasmussen
|
|
Dina Fiatarone
|
Lakshmi Sampath
|
You can become a Hope Builder with a recurring donation of just $10 or
more and receive exclusive invitations to virtual Propel a Cure events
as well as recognition in our quarterly e-newsletter. To become a Hope
Builder, visit
propelacure.org/donate
and choose a frequency and amount; you can change your gift amount or
cancel at any time.
|
PERSONNEL CHANGES
|
|
|
|
Angela Clark
|
Raihaneh Bokharai
|
|
Propel a Cure is pleased to announce some changes to the organization's
Board of Directors, which also serves as its all-volunteer operational staff.
First, we will be forever grateful to Founder Annabelle Hall, whose drive
to find answers for her son after he was diagnosed with Crohn's disease
led to the founding of our organization in the quest to fund innovative
scientific research to uncover the causes of Crohn's disease and push for
cures.
This past April, after eight years of tireless efforts and helping Propel
a Cure become a force for change in Crohn's research, Annabelle made the
decision to retire and, in consultation with the Board, named Angela Clark
as its new President and CEO.
Angela, who worked closely with Annabelle after joining Propel a Cure
in 2019, became involved after the Crohn's diagnosis of a family member.
In the past, she enjoyed a career in the federal government as well as
working in local radio and television. She feels passionate about supporting
Crohn's research that takes us beyond the current therapy landscape, and
hopes to prioritize spreading Propel a Cure's message to a wider audience
as well as expanding the number of cure-focused studies the nonprofit is able
to sponsor.
Propel a Cure would also like to welcome its newest and first Europe-based
Board member, Raihaneh Bokharai, our Vice President of Strategic Partnerships.
Raihaneh is a strategic consultant with a diverse background spanning both
the for-profit and nonprofit sectors.
Raihaneh received her bachelor's degree in social work from Saxion University
of Applied Sciences and is currently completing a master's in management
with a specialization in strategy & innovation at the Open Universiteit.
Based in the Netherlands, Raihaneh became a patient after her IBD diagnosis.
She is a strong proponent of Propel a Cure's goal-oriented approach of
supporting innovative research and brilliant scientists working to advance
tangible progress toward a cure for Crohn's disease in the hope of bringing
relief to those affected.
|
RESEARCH OPPORTUNITY
RARE PATIENT VOICE RECRUITING FOR RESEARCH
|
Propel a Cure is a trusted referral partner of Rare Patient Voice (RVP).
RVP pays patients and caregivers of patients suffering from both rare and
non-rare illnesses and conditions for their input on new treatments, products,
and services, as well as for participation in clinical trials.
If you are in North America, Europe, Australia, or New Zealand, you may
register for current studies by visiting their website:
RarePatientVoice.com/rp/Propelacure
In addition to IBD, they are recruiting for many other conditions.
|
|
RESEARCH REVIEW
NEWS ABOUT IBD
|
Mapping the 'brain in the gut' may provide clues to gastrointestinal disorders
Researchers have identified 3 types of nerve cells connected to the intestinal
villi (tiny finger-like projections living in the small intestine that
play a crucial role in nutrient absorption), which could lead to a better
understanding of IBD and other diseases.
Article Link:
MEDICAL XPRESS
|
Leveraging Organ-on-Chip Models to Investigate Host–Microbiota Dynamics
and Targeted Therapies for Inflammatory Bowel Disease
This sweeping overview of organ-on-chip technology, including its superior
ability to replicate the human intestinal microenvironment, makes the case
for why we need this innovative model to play a larger role in IBD research.
Article Link:
WILEY ADVANCED
|
Inflammatory bowel diseases may be detectable at birth
In the first study to investigate inflammatory markers at birth and later
risk of IBD, scientists have found changes that may be indicative of very
early onset IBD that occurs prior to age 6.
Article Link:
MEDICAL XPRESS
|
Genetic link found between iron deficiency & Crohn's
UC Riverside researchers have identified a gene mutation that can disrupt
iron absorption in Crohn's disease, helping to explain some patients' persistent
anemia and why some fail to respond to oral iron therapy.
Article Link:
UC RIVERSIDE NEWS
|
THANKS TO YOU
YOUR SUPPORT IS IMPORTANT
|
|
Thank you for supporting
Propel a Cure! If you can get involved, please let us know. We would love to have you
as a volunteer! If you'd like to learn more about our organization or have
suggestions for topics you'd like to see covered in the next newsletter,
please send an email to
info@propelacure.org.
Together we can make a difference!
|
info@propelacure.org
|
PropelaCure.org
|
