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

Double Your Donation

Corporate matching gift programs are charitable giving programs setup by corporations in which the company matches donations made by employees to eligible nonprofit organizations. For example, if a donor works for Bank of America and donates $100 to the Hereditary Neuropathy Foundation, Bank of America will double the donation by also writing a check for $100.

Taking Matters Into Your Own Hands

Do you have a rare form of CMT?

We were contacted recently from a patient with CMT2D who was looking for advice on how to make a difference. Our recommendations for all people with an ultra-rare form of CMT who want to get involved:

Now GRIN is Accessible in 141 Different Languages!

Now GRIN is Accessible in 141 Different Languages!

The Global Registry for Inherited Neuropathies (GRIN) has implemented Google Translate. Why did we choose Google? Because Google is leading the way in teaching computers how to interpret meaning, avoiding the traditional method of decoding language.

Accelerating Patient Access to Investigational Drugs in 2015

Accelerating Patient Access to Investigational Drugs in 2015

Currently the FDA is working to update the process for physicians applying for accelerated patient access to investigational drugs, while the drug or biological product is being tested in clinical trials. This will also be important for the CMT community as clinical trials for this disease are being launched.

Do You Have Charcot-Marie-Tooth Type 4 (CMT4)? – We Need Your Help

Autosomal recessive demyelinating CMT4 patients present with earlyonset and slowly progressing symptoms. These include progressive distally accentuated weakness and atrophy of muscles in the lower limbs. In addition, patients may have weakness and atrophy of hands, sensory loss and pes cavus (high-arched feet), and walking difficulties. Further information on CMT4 can be found at Orphanet.

Hot Off the Press – Potential Treatment for CMT1A

Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.

Support CMT Therapeutic Alliance

HNF has entered into a joint venture – the CMT Therapeutic Alliance – with a unique non profit organization (BioPontis Alliance for Rare Diseases) that brings professional drug discovery capabilities to translate our research results into potential treatments.

Scientific Advisory Board Meeting

Scientific Advisory Board Meeting

On the 7th of November we convened our scientific advisory board meeting at the HNF offices in NY. We have written a detailed review that has been published and captures all of the discussion and make this freely available to the scientific community.

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