Castleman Disease Collaborative Network Survey

The Birth Defect Team at is participating in partnership with the Castleman Disease Collaborative Network (CDCN) and the Chan Zuckerberg Initiative.

For American Residents only, anyone can take this Survey, we need your help, please share this page link.

For American Residents only

Dear EA/TEF and OA/TOF Community,

We would appreciate your insight on a new project!

The Birth Defect Team at is participating in partnership with the Castleman Disease Collaborative Network (CDCN) and the Chan Zuckerberg Initiative. The goal is to understand the state of drug repurposing in rare diseases and create a shared, free resource that will make it easier to pursue successful drug repurposing.

If you’d like to join us in this important work, please click the link below to complete the survey as a patient, loved one of a patient, physician, or researcher.

CLICK here for Survey

  • ly/repurposingsurvey   
  • Expected duration: 15-20 minutes
  • Deadline: October 26, 2021.
  • A Spanish version is available / Hay una versión en español para pacientes

If you have any further questions, please contact project lead Ania Korsunska at or check out our website

Thank you so much for joining us in this effort to revolutionize drug repurposing!

Best Regards,
Sue Paul and Steve Wyles

Additional Background: 95% of the ~7,000 rare diseases do not have a single FDA-approved therapy. Since incentives for new drug development for rare diseases are limited, drug repurposing provides a promising way to identify effective treatments for rare diseases faster and cheaper. There are many treatments out there which could be useful for many rare diseases, but to date, there has been no systematic effort to gather such a large and diverse data set of information and experiences, and no centralized resource exists that can help guide drug repurposing. With your support, we can help create it! You can read more about the project here:

Rare disease is anything but rare. As many as 7,000 rare diseases affect 400 million people globally. The vast majority are not well understood, and less than 5 percent have approved treatments. Yet worldwide, patients are meeting these challenges head-on. The Rare As One project is committed to uniting these communities in their quest for cures.

About the Project

There are ∼7000 rare diseases affecting approximately 30,000,000 individuals in the U.S.A. and 95% of these rare diseases do not have a single Food and Drug Administration-approved therapy. Since the incentives for new drug development for rare diseases are limited, drug repurposing provides a promising way to identify effective treatments for rare diseases faster and cheaper than developing new drugs. Still, many challenges exist in the drug repurposing space, namely:

  1. lack of a consensus of the roles that various stakeholders play (patient advocacy organizations, researchers, physicians, government, pharmaceutical companies, etc.)
  2. lack of a “roadmap” of how rare disease organizations should go about pursuing drug repurposing,
  3. lack of support (data, finances, resources, guidelines, etc.) during the drug repurposing process.

The Repurposing Of All Drugs, Mapping All Paths (ROADMAP) project proposes to fill some of these knowledge gaps through document analysis, surveys and interviews grounded in a participatory design approach. Ultimately, this project aims to not only answer some fundamental questions about the experience of drug repurposing for various stakeholders, but to also design a solution to some of the challenges rare disease organizations are facing through the creation of an interactive tool.

Watch the below video to see project lead Ania Korsunska give a brief overview of the project at Find A Cure’s Drug Repurposing conference:

Project Goals

In this research, we seek to:
  1. To answer fundamental questions about the experience of various stakeholders (rare disease organizations, physicians, researchers, patients and loved ones) in drug repurposing.
  2. To understand the information and knowledge sharing practices of rare disease non profit organizations (RDNPs) within the rare disease space in regards to drug repurposing
  3. To build a network of RDNPs and their collaborators in order to better understand the rare disease space in general.
  4. To produce a “roadmap” for RDNPs to support the pursuit of drug repurposing.

We will utilize a combination of methods to pursue our research questions as follows: document analysis, surveys, network analysis, interviews, and participatory design sessions.

Want to Participate? 

For this project, we are looking to collect data from rare disease organizations, patients, loved ones, researchers, and physicians treating rare disease patients, as well as representatives of pharmaceutical companies and government organizations.

There are varying levels of participation available to interested rare disease organizations. Prior experience with drug repurposing not required.

Drug Repurposing what does it mean?

Drug repurposing involves identifying FDA-approved drugs that may be effective for diseases they’re not currently approved for. We believe that this is a much more efficient process than new drug research and development for rare diseases like Castleman disease. The CDCN is committed to discovering new uses for existing drugs to save patients’ lives and helping support other patient organizations in their drug repurposing efforts.

Fast facts about rare disease treatment:

  • Currently, there are ~2,500 FDA approved drugs that treat 25% of the ~10,000 human diseases
  • No treatments exist for over 7,000 diseases, many of which are rare and deadly
  • Developing a new drug can cost $2-3 billion and can take 13-15 years before becoming approved for widespread use
  • New drug development is also very risky. The drug may turn out to be unsafe or ineffective at hitting its target. Repurposed drugs have a known safety profile and established effect.
  • Many rare diseases are heterogeneous and/or idiopathic.
  • By definition, rare diseases do not affect a large patient population and often do not present enough of a commercial incentive for pharmaceutical companies to develop new treatments.

Given that centuries will be needed to develop a new drug for all rare diseases, drug repurposing provides a potential alternative solution. The basic idea of drug repurposing is that many diseases share similar defects and thus may benefit from the same treatments. Once a treatment has been FDA-approved and shown to be safe and effective for at least one disease, it is more likely to be safe and potentially effective in a new disease area than new, untested compounds. Research is required to identify and match potential treatments to specific conditions. This approach is often much faster and cheaper than new drug development.

Thus, redirecting more funding and research towards maximizing the uses of existing treatments, rather than focusing solely on the expensive, time-consuming and risky development of novel drugs, is potentially one of the fastest and most effective ways to get treatments to patients with rare diseases.

Drug Repurposing – CDCN

Castleman Disease Collaborative Network Survey

Castleman disease is a group of rare disorders characterized by lymph node enlargement, specific microscopic changes to the lymph nodes, and a broad range of symptoms and laboratory findings. The lymph nodes, and the cells that reside in them (lymphocytes), are an integral part of our immune system that help us fight invading organisms. For reasons unknown, these lymph nodes undergo some transformations that result in overproduction of lymphocytes and other inflammatory compounds.


The two main subtypes are Unicentric Castleman disease (UCD) and Multicentric Castleman disease (MCD). As the name suggests, the enlarged lymph nodes in UCD appear in only one region of the body. UCD tends to have milder symptoms and rarely affects organs. The multicentric types are characterized by lymph node enlargement in multiple regions of the body and the symptoms tend to be more severe than UCD.

The multicentric subtype can be further classified into two categories: HHV-8 positive Multicentric Castleman disease and idiopathic Multicentric Castleman disease. HHV-8 is a common virus that does not result in disease for the overwhelming majority of individuals. Unfortunately, a small proportion of individuals with this virus, especially those with suppressed immune systems, may develop a form of MCD. Although we know that the virus is involved, we still do not know why only a few patients with this virus develop the disease.

The other category is idiopathic Multicentric Castleman disease (iMCD). Idiopathic refers to the fact that we do not know the causative agent. We know that these individuals are HHV-8 negative and therefore have a different origin for the disease. iMCD also has important differences in symptoms, disease course, and treatment from HHV-8 positive disease. Furthermore, some iMCD patients can develop a specific subtype called TAFRO characterized by Thrombocytopenia (low platelet count), Anasarca (ascites, swelling), Fever or elevated C-reactive protein (inflammation marker), Reticulin fibrosis (evaluated in bone marrow biopsy), and Organomegaly (hepatomegaly/splenomegaly). Diagnosis of iMCD-TAFRO requires thrombocytopenia, anasarca, fever/elevated C-reactive protein, and either reticulin fibrosis or organomegaly.

CLICK THE BANNER LINK for more on this