starwise alliance

StarWise Therapeutics & HNF Form A Strategic Alliance to Bring a NextGen HDAC6 Drug Therapy to CMT2A (MFN2) Patients

by | Dec 29, 2018 | 0 comments

starwise

StarWise Therapeutics:  

  • Founder Prof. Alan Kozikowski of Starwise has a successful track record of drug development and licensing of his first generation of HDAC to leading pharmaceutical companies  
  • In a 2011 published study in Nature Medicine, Prof. Kozikowski and one of the leading HDAC6 research experts, Ludo Van Den Bosch, PhD VIB-KU Leuven Center for Brain & Disease Research and others demonstrated that a selective HDAC6 inhibitors can be used to treat CMT*
  • NextGen HDAC6 are novel, safe inhibitors for use in the treatment of various neurological disorders, specifically targeting CMT2A
  • Lead CMT2A researcher, Prof. Brett Langley from Waikato University in NZ has demonstrated that this key drug is able to restore sensory and motor function in the CMT2A mutant animals

Hereditary Neuropathy Foundation:

  • Funding the drug optimization to accelerate the path to human clinical trials  
  • Prof Brett C. Langley and Prof. Dianna E. Willis, Burke Neurological Institute & Brain and Mind Research Institute, Weill Cornell Medicine, both served on the HNF Scientific Advisory Board for more than a decade and have published several papers on the impact of HDAC on mitrochondrial diseases such as CMT2A
  • Expertise in clinical trial design, identifying patients for the trial and FDA regulatory filings

* Nat Med. 2011 Jul 24;17(8):968-74. doi: 10.1038/nm.2396.

HNF has been committed to funding HDAC inhibitor research since 2014. Andrew Grierson, PhD of University of Sheffield study titled: Pre-clinical testing of HDAC6 inhibitors in a zebrafish model of CMT2A, showed promise as a potential treatment. Funding was made possible through the Help Elliot Live Proud (H.E.L.P.) Fund founded by Iris and Nathaniel Adler and Grace’s Courage Crusade (GCC) founded by Marybeth and Christopher Calderone.

To support HNF’s efforts and to help us get to phase 1 clinical trials, please consider making a donation to our CMT2A funds:

DONATE TODAY!

H.E.L.P. Fund www.hnf-cure.org/h-e-l-p-for-cmt/

GCC Fund www.hnf-cure.org/graces-courage-crusade-cmt2a/

Learn more on this topic

Related Blog Posts

Quality of Life and CMT Research

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal.

Help us answer questions that your doctors and the CMT Research Community aren’t too sure about.

Did you know that you can become part of a community in therapy development and further research for all forms of CMT and inherited neuropathies? The mission of the Global Registry for Inherited neuropathies (GRIN) is to collect clinical and genetic information from patients with ALL forms of Charcot-Marie-Tooth (CMT) and other related rare and ultra rare inherited neuropathies.

Hot off the press

It seems like almost weekly there is another new publication on CMT with interesting basic biology. While an earlier “Hot off the press” highlighted the work of Cherry and co-workers at the University of Texas Southwestern Medical Center who showed show that neurons lacking a gene for rab7 result in neuropathy.

New Study on CMT Type 2B

If we are to learn more about CMT and the effectiveness of rehabilitation it is worth asking the patient and their caregiver. A recent Italian study by Padua et al recently described a survey of CMT patients and caregivers and their perspectives and perceptions of rehabilitation efficacy and needs.

Allison Moore is going to be published!

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

HNF-WVU-NIOSH Study update:

We are pleased to update our readers on the progress of the WVU-NIOSH study, “A Bi-Directional Translational Model of Exercise Training in the Treatment of Charcot-Marie-Tooth Disease”

Join the conversation

Leave a Comment

0 Comments

Submit a Comment

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

Newsletter

Join for notifications on events, campaigns, & news