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StarWise Therapeutics LLC & HNF Join Forces for Treatment of CMT2A

by | Mar 26, 2018 | 5 comments

starwise therapeutics

News Flash –  StarWise Therapeutics LLC and the Hereditary Neuropathy Foundation join forces to expedite the advancement of a new drug entity to the clinic for the treatment of CMT2A.

StarWise Therapeutics LLC based in Madison, WI has developed novel, safe HDAC6 inhibitors for use in the treatment of various neurological disorders.  On the top of its list is the use of its new drugs in the potential treatment of the genetic disorder known as Charcot-Marie-Tooth disease, or CMT for short.

CMT is a progressive genetic disease of which there are many variants. CMT2 is a mutation in the sequence of the mitofusin 2 gene or MFN2. MFN2 is a protein that plays an essential role in the function of mitochondria which generate energy in the cells. In MFN2 patients, the energy produced by neuronal mitochondria is insufficient to sustain axons. As a consequence, axonal degeneration gradually occurs, causing muscle weakness, atrophy and deformities in the feet, legs, arms and hands. Those with CMT often lose the ability to walk and may become dependent upon assistive devices to remain mobile. Severe, chronic pain is common, and there is no cure.  

StarWise Therapeutics and Waikato University in NZ have demonstrated that their key drug is able to restore sensory and motor function in the CMT2A mutant animals to performance levels akin to those of the wild type animals.

The Hereditary Neuropathy Foundation (HNF) is a non-profit 501(c)3 global organization whose mission is to increase awareness and accurate diagnosis of CMT, support patients and families with critical information to improve quality of life, and support research. HNF has notably developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government, and industry to support and develop treatments for CMT.

starwise therapeuticsThese two entities have now linked their expertise in order to advance the StarWise agents to the clinic. Key medicinal chemistry, biology, and clinical experts will be meeting in NYC this week to make plans for the filing of an Investigation New Drug (IND) application with the FDA for the treatment of this disorder. And this couldn’t be  more timely with the upcoming HNF-hosted FDA externally-led Patient-Focused Drug Development (PFDD) Meeting on September 28 in Washington, DC. StarWise and HNF are forging the way to accelerating therapies to the clinic for many CMT sub-types and for the first time CMT2A will be added to the that list. Stay-tuned as we will be reporting with research and clinical updates!

Learn more on this topic

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Quality of Life and CMT Research

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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.

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  1. Nancy Gebert

    I have CMT2A and live in Middleton, WI. I would be very interested in any follow-up of this research and if, or when, any patient studies might be needed.

  2. Mohamed

    It is very good. Thanks god

  3. Ritchie dugang

    Pls let us know how ee can support you..

  4. Rick Cole

    This is great news and I can’t wait to see what’s in store down the road with the agents being researched for CMT2A. I am so very thankful to HNF and partners.

  5. Karen Martine

    My 11 year old son has CMT2A and so does my 78 year old mom. I would love to see a cure soon. They suffer in pain everyday. Falls, breaks, fractures and sprained are a constant occurrence.


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