The Research Institute for Children’s Health was launched in 2015 to apply a pipeline approach to therapeutic development for rare, genetic diseases while taking advantage of the processes already in place for research on cystic fibrosis. Our goal as an institute is to convert scientific discoveries into therapies, or even cures, for these devastating diseases.
Despite lingering challenges of the pandemic, our researchers continue to make tremendous progress. Progress that simply could not have been realized without support from generous and engaged individuals like you.
Since the early stages of the Institute, fundraising has been used to support bold and promising new ideas, investments into instrumentation, and cultivating the next generation of young scientists. Together, these investments have accelerated our research and led to additional grant funding from a variety of sources that places our return on investments into the institute at nearly 10-to-1. In fact, this year alone our faculty have secured more than $6 million in grants that were made possible by previous support and philanthropic investment. From a scientific and medical perspective, these investments have accelerated our research immensely, shortening the time it takes to test and implement an idea by months, and in some cases even years.
Some highlights of the past year include:
Funding for Projects
| CWRU CF Summer internship program (Feb 2022-Jan 2024) | $200,000 |
| Altered Circadian Rhythm Regulation in Cystic Fibrosis (Apr 2022-Mar 2026) | $2,504,828 |
| Editing disease-causing CFTR alleles caused by segmental duplications (May 2022-Apr 2024) | $336,000 |
| Addressing barriers to delivery and editing for CF (Jan 2023-Dec 2025) | $3,158,363 |
| Therapeutic pipeline for genetic diseases, (Apr 2022-Mar 2024) | $230,000 |
| Hartwell postdoctoral fellowship for Glycogen Storage Disease 1A | $100,000 |
| Total | $6,529,191 |
Presentations
The Institute’s co-director, Rebecca Darrah, was highlighted as a plenary speaker at the North American Cystic Fibrosis Conference. She addressed 4,000 in-person attendees and 2,000 online about advances in treating cystic fibrosis and the contemporary needs of genetic counseling. To view Dr. Darrah’s presentation, click below.
Fundraising
On August 27th, Giving Kids Hope was held at Columbia Woodlands in Dover, Ohio and raised a record high $175,000+ for the institute’s biomedical research efforts and engagement of young scientists. A key component of the event’s success was emcee David McCreary, who captivated and entertained guests. Featured speaker Ryan Ress highlighted the important role research has played in his experience with cystic fibrosis.
Promoting Young Scientists
This year William Hannah, MD joined the ranks of the Research Institute. As a geneticist specializing in metabolic disorders, Dr. Hannah is developing therapeutic approaches for disorders such as glycogen storage diseases that affect the body's ability to use stored energy.
Our summer internship program provided research experiences for 11 undergraduates and 2 medical students. Six of these students attended an international research conference in November, all of whom felt it was a very positive, impactful experience.
Summer student program information and application forms can be found on our student programs page.
Upcoming Events
On February 28, 2023 The Research Institute for Children’s Health will host Rare Disease Day CLE, an annual meeting of the National Organization for Rare Diseases (NORD), to present local work on rare diseases. The meeting will run from 9:00 am - 1:00 pm and lunch will be included. If you are interested in learning more about this event or would like to attend, please contact Mitch Drumm, PhD at mitchell.drumm@case.edu.
Although the date is yet to be determined, Giving Kids Hope will be back in 2023!
You can follow us and our events on our front page