James And Lindsey Team CMT Kids

Collaboration is the Key to HNF Success

by | Dec 4, 2014 | 0 comments

James And Lindsey Team CMT KidsWe at HNF are so proud of the accomplishments of those scientists we fund and are asking you to please continue to support our efforts. Our Therapeutic Research In Accelerated Discovery (TRIAD) program is a proven collaborative model in the drug discovery process. Through partnerships with leaders in academia, government and industry, we will be part of the first Phase 3 trial from Pharnext, an advanced clinical stage biopharmaceutical company for the most common form of CMT, CMT1A.  We are also committed to our continued strategy for developing treatments for CMT2A. For example, our recent funding to evaluate compounds in the zebrafish model is a giant step toward drug discovery. We are also embarking on other forms   of hereditary neuropathies to ultimately accomplish the same goals of getting treatments to patients as soon as possible. Our strategy is to put donors’ dollars to work funding the scientists who are doing translational research which will move their ideas to treatments that can ultimately be tested in the clinic. This is not an overnight process, which some organizations would like you to believe; we have to be prepared to invest for the long-haul. It usually takes well over a decade to go from a discovery in the laboratory to something that can go to the FDA for their approval. There are no guarantees. If a scientist finds a promising compound in a screen, the odds are stacked against that molecule ever becoming a drug that a patient takes, and it takes millions of dollars to bring a drug to market. Although HNF is a relatively small organization, we do know this disease better than most drug companies, allowing us to make astute investments. With the help of our Scientific Advisory Board and industry advisors, we look to fund projects that are important and may have been missed by others. You will notice we have a diversified portfolio of projects in different stages in the same way that many of you likely diversify your investments.  In 2014 alone we have announced major collaborations with Pharnext and the Biopontis Alliance for Rare Diseases. These partnerships will help develop a pipeline to bring early and late stage treatments to clinical trials for CMT1A.  We have also funded Dr. Andy Grierson working on the CMT2A zebrafish model and Dr. Lucia Notterpek working on microRNA for CMT1A. As  always we continue to push forward, and following our Scientific Advisory Board meeting in mid-November, we will have new goals to pursue as well. We will report on these in the next issue.  As the holiday season approaches it is usually a time to reflect on the past year and to position HNF for the years ahead. Our goal to fund key projects to bring treatments to the patient is a marathon, not a sprint, and we need your continued support to see us to that finish line!

Learn more on this topic

Related Blog Posts

Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!

The first disease community to receive a therapeutic gene to the spinal cord for an ultra rare inherited neuropathy is Giant Axonal Neuropathy (GAN). Congratulations to Hannah’s Hope Fund (HHF), a 501(c)3 public charity, which has driven this collaborative research in less than six years. Six million dollars has been raised to date to fund pre-clinical and clinical research on this rare disease.

A Rare Disease Patient That Does It All!

A movie script could not play out like this. There are very few occasions when a rare disease patient becomes the topic of a story that is truly uplifting. Often the stories are tragic or disheartening. That was not the case, however, with CMT patient Kim Goodsell.

Hot Off the Press

One of the ways that some CMT patients first become aware of their disease is when they are given a drug treatment for another disease. This is termed chemotherapy-induced neurotoxicity. Drugs such as paclitaxel and the vinca alkaloids that are widely used in cancer treatment cause severe peripheral neuropathy and in some patients this exacerbates CMT, revealing it perhaps for the first time.

Kara Q&A: How to “support” a friend or family member that’s affected with Charcot-MarieTooth.

My name is Courtney Hollett, Fundraising Coordinator at the Hereditary Neuropathy Foundation. This time of year I count my blessing daily and wanted to share with you a Q&A session I had with a new supporter of HNF. Kara, like myself has many family members affected with CMT and I reached out to her to share her thoughts and advice about how to “support” a friend or family member that’s affected with Charcot-MarieTooth.

Sneak Peak of Fall Newsletter: CMT Research Study Survey

My name is Elizabeth Francisco and I am a graduate student from the Genetic Counseling program at the University of North Carolina Greensboro. I am inviting you to participate in a research study. The goal of my study is to learn more about the experiences of people with Charcot-Marie-Tooth (CMT) with genetic counseling and genetic testing. Adults with CMT and parents or legal guardians of someone of any age who has a diagnosis of CMT are eligible to participate

Join the conversation

Leave a Comment


Submit a Comment

Your email address will not be published. Required fields are marked *


Join for notifications on events, campaigns, & news