An Incredible New Development!
Dear DADA2 Community:
I am breaking with our standard newsletter format to share an incredible development in our 10 years of DADA2 research.
2024 marks ten years since researchers in Israel and the National Institutes of Health (NIH) in Bethesda, MD, first identified the genetic basis of Deficiency of Adenosine Deaminase 2 (DADA2). For many of us, however, that breakthrough in February 2014 was simply giving a name and genetic origin to a disease we had been living with for years.
Back then, only 39 patients worldwide were known, and just one major phenotype was identified. Fast forward to today: we have identified over 630 patients and discovered three distinct phenotypes. But we also know there are likely 35,000 more who are undiagnosed!
The Next Chapter: A Potential Breakthrough in Treatment
The most significant challenge? There are still no formally approved treatments for DADA2. But that could change in the near future!
Last year, the DADA2 Foundation secured the donation of a promising compound which holds great promise as a new treatment specifically for DADA2 patients. Today, we hold the intellectual property rights and 39 patents in 31 countries for pegylated-ADA2 (PEG-ADA2). This remarkable achievement positions us as one of the few rare disease advocacy organizations to own a potential enzyme replacement therapy (ERT).
While many DADA2 patients benefit from anti-TNF therapies, these treatments are currently used “off label” and are not FDA-approved for DADA2. Moreover, patients suffering from bone marrow failure or immunodeficiency often do not respond to anti-TNFs. PEG-ADA2 could become the first approved therapy that addresses the needs of all DADA2 patients.
Why PEG-ADA2 Could Be a Game-Changer
Years ago, Dr. Dan Kastner of the NIH remarked, “DADA2 patients don’t need TNF inhibitors, they need ADA2!” Current therapies only alleviate symptoms; they don’t address the root cause. Replacing the missing ADA2 enzyme with PEG-ADA2 has the potential to drastically reduce patients’ symptoms and improve their quality of life.
What’s Next? The Path Forward for PEG-ADA2
Bringing a new drug to market is a complex, lengthy, and costly journey. Fortunately, the pharmaceutical industry has extensive experience with enzyme replacement therapies, including for ADA1 Deficiency, and ERTs have a strong track record of FDA approvals. It took four years to develop and approve PEG-ADA1 therapy, and we are optimistic we can accelerate that timeline for PEG-ADA2.
The DADA2 Foundation is actively developing a strategic plan for PEG-ADA2 and exploring partnerships. By owning the intellectual property, we can reinvest potential returns into critical research, driving progress where it’s needed most.
A Message of Gratitude and Hope
None of this would have been possible without your unwavering support. As we enter this season of giving, we are deeply grateful for your contributions that have brought us to this exciting moment. Together, we are on the brink of scientific breakthroughs that could transform the lives of DADA2 patients worldwide.
In just ten years, our global DADA2 community has achieved extraordinary milestones. With your continued support, imagine what the next decade will bring… The future holds extraordinary promise!
Chip Chambers, M.D.
Founder & President, DADA2 Foundation
P.S. As we work toward bringing PEG-ADA2 to patients, every bit of support helps. Whether sharing our mission with others, making a donation, or simply sending words of encouragement, you’re making a difference. Thank you for being an essential part of this journey!
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