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Neurologist: Well, he has CMT and yes, it gets worse over time.

Me: Silence

Neurologist: There is really nothing much you can do about it.

Me – Silently feeling FREAKED OUT!

Neurologist: Young man, at the beginning of the game, you are dealt a specific hand of cards. Some people receive a great hand, and others, well, others get cards that kind of suck. In the end, it does not matter.  Life is not successful because you hold good cards or bad cards. It’s what you do with those cards that will make or break you.

Me- Silent, trying to figure out why he was talking about a game of cards.

Neurologist: I’ll see you back here in a year.

Me: Silent, thinking that wow, we came for a diagnosis and we are leaving with an intro to philosophy.

That evening, I looked through the handouts he shared with us and I came upon the URL of the Charcot Marie-Tooth Association or CMTA. One thing led to another and I’ve been heavily involved ever since. Looking back over the past 15 years, I’m in awe of what we’ve accomplished: an abundance of CMT resources, branches, patient advocacy efforts, CMT awareness programs, and a promising research program-Strategy to Accelerate Research or STAR.

Today, I’d like to expound upon STAR.

 

 

Highlights

• In 2008, the CMTA launched STAR or Strategy to Accelerate Research which was innovative and unique at the time because we required scientists to work together and collaborate to advance CMT research. No more silos. No more individual superstars. No more “me” and a lot more “we”.
• Since many of the genes that cause many types of CMT are known, the CMTA’s work toward finding a treatment has advanced that much quicker.
• Clinical planning has started! With the Inherited Neuropathies Consortium (INC), the CMTA is developing crucial clinical infrastructures: new centers, more clinicians, patient information, and history. Get involved with CMT research: https://www.rarediseasesnetwork.org/cms/inc/

 

• We are developing ways to measure disease progression (biomarkers) in people with CMT (this is important to see if a treatment works or not). The CMTA’s STAR has developed and is using cellular and animal models of CMT to help pharmaceutical companies test their drugs quickly.

• We have partnered with pharmaceutical companies and labs to apply breakthrough genetic therapies (like gene therapy, RNAi, and CRISPR) to CMT.

Today- The Bottom Line

• The CMTA currently has 25+ active research projects with top labs around the world.
• Since 2009, we’ve spent over $8M to advance CMT research.
• We have 25 world-renowned scientists on our Scientific Advisory Board (https://www.cmtausa.org/research/star-advisory-board/).
• We have partnership agreements with about 20 pharmaceutical, biotech and service companies across three continents.
• They are using state-of-the-art technologies (CRISPR, gene therapy, gene silencing, axon, and muscle regeneration) and are working with the CMTA to use the assets we’ve developed through STAR.
• We have 26 CMTA Centers of Excellence, so the infrastructure for upcoming clinical trials is ready to roll. (https://www.cmtausa.org/resource-center/finding-help/cmta-centers-of-excellence/)
• We will continue to include more and more types of CMT in our research work.
• And we have forged relationships with many, many families with CMT. You will have a role in drug discovery. It’s a group effort.

 

 

 

 

 

What Now?

All the major elements are in place and we are giving it our all to bring treatments to people with CMT. We estimate “kneading” $10,000,000 in the upcoming years to help bring CMT drugs to market. Why?

Bringing a new drug to market costs between $400 million and $1 billion. And that’s a lot of dough!

The CMTA does not have this kind of money. And I sure don’t either. But, as I wrote above, pharmaceutical companies are reaching out to us. They want to collaborate with us because our scientific work or assets are superb.  In the end, they will carry the ball over the line for us. That’s why we are so excited that the number of partners has exploded in the past 2 years from a handful to about 20.

So, if you want to be part of these very exciting times:

-Donate to the CMTA (https://www.cmtausa.org/donate/make-a-donation/)

-Participate in a CMT Walk or Fundraiser (https://www.cmtausa.org/upcomingevents/map/)

-Fundraise for the CMTA  (https://www.cmtausa.org/fundraise-for-a-world-without-cmt/

 

 

-Register/Attend/Underwrite my family’s annual fundraiser – The Cycle (and Walk!) 4 CMT event (www.cycle4cmt.com).

–Sponsor me – Elizabeth Ouellette-  in the Cycle (and Walk!) 4 CMT fundraiser, which is taking place on August 26, 2018 in Charlotte, VT.  Every dollar invested in the Cycle (and Walk!) 4 CMT event is dedicated to advancing CMTA Research toward finding a cure. (https://cmta.akaraisin.com/pledge/Participant/Home.aspx?seid=16550&mid=9&pid=3311373)

Now, play your cards right and play them well. Get Involved. Get Sassy. Get a Move On. The future is OURS!

 

 

 

 

 

 

 

 

If you have questions, email me: elizabetho@outlook.com