A team of UCLA scientists, clinicians and clinical trialists led by Drs. Stan Nelson and Carrie Miceli have teamed with AVI and Dr. Craig McDonald to bring a novel combination therapy forward for use in DMD and sent a proposal to the California Institute of Regenerative Medicine (CIRM). It involves the UCLA discovery of an already FDA approved drug to boost exon skipping activity when used in combination with anti-sense oligo-nucleotides for Duchenne. Duchenne patient skin cells (fibroblasts) “re-programmed” to stem cells and ultimately to muscle cells were used to screen drugs and make the discovery; and the proposal relies further on these stem cells to inform dosing and trial design. (The grant proposal is aimed at taking a combination therapy for exon skipping to ‘IND’ which is the FDA process to approve human testing and 100% clinical trial readiness.) While focused currently on augmenting exon 51 skipping, the strategy may ultimately also apply to other exons. This therapy has the promise of 'fixing' the mutant DMD gene in all skeletal muscle cells. The team has already received the planning grant and are now are on the “short list” competing to receive a 20 million dollar grant for these studies. While the UCLA team was not recommended for funding at this point, an 'Extraordinary Petition' to the Independent Citizen’s Oversight Committee (ICOC) that lays out responses to reviewers has been prepared to address the issues required to enable the proposal to fund. Similar attempts to gain funding at this level have been successful in the past.

The final level of review is strongly influenced by advocacy presence, presentations and the team verbal statement at the ICOC (Independent Citizens Oversight Committee) on July 26th near San Francisco Airport. Heads of advocacy groups as well as California citizens affected by DMD will be allowed to speak in support of this proposal, the tremendous need for Duchenne treatments and the impact Duchenne has on California families.

From an advocacy point of view, the reason to move the proposal to funding is that:

  1. There is a large unmet medical need with no real therapies for Duchenne, which is one of the most common lethal childhood diseases,
  2. UCLA has a great team assembled and capable of carrying the new combination therapy forward towards FDA approval,
  3. There are relatively few childhood, genetic or muscle disease projects funded by CIRM (so this is a gap in their portfolio).

At the very least, we as a community have the opportunity for the ICOC to hear about Duchenne and consider the rationale for expanding their portfolio to include Duchenne treatments over the next 5 years of funding, which will bring some additional awareness to a group that holds the purse-strings for $3 billion in state funding.

At the very best, an additional $20 million will have been devoted to help DMD research and DMD will have gotten its foot in the door as part of a (hopefully) growing DMD centric focus within their portfolio.



Option 1- Attend the Meeting:

We invite you to join us as Duchenne disease advocates in support of funding the UCLA proposal and Duchenne research in general through your participation in the ICOC meeting. You can come representing individual families or more organized groups with prepared statements. Typically, individuals wishing to speak need to “sign in” the morning of the 26th prior to the beginning of the meeting, and must limit comments to no more than 3 minutes.



Option 2- Send personal email to the committee:

An easy way to express your support of this grant is by sending the committee an email. Below you will find some sample text. Please be sure to erase or edit what is in red and fill in your own information. For the first paragraph edit the line depending on your relationship to duchenne. We encourage you to ask your friends family and neighbors to help. 

Send your email to Gil Sambrano at GSambrano@cirm.ca.gov


Subject: Upcoming ICOC Meeting

Dear ICOC,

I am a resident of (City), California and I am writing to you today about the urgent need to advance therapies for skeletal muscle disease.  I understand that you are considering an Extraordinary Petition to fund a proposal from UCLA 'Combination Therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy'. As a parent (family member, or friend) of a boy/young man affected by Duchenne muscular dystrophy,  I strongly support research that will lead to new and effective treatments for the many affected by Duchenne.

I respectfully ask that you support this important research program so we can move the field forward toward a treatment.

(Feel free to add in any personal story about your son, relative, or friend of the family, please limit it to a few sentences. Feel free to attach a picture but do not attach large files, nothing over 1MB) 

Kind regards,

Your name

Your address


***Please note: This is for CALIFORNIA RESIDENTS ONLY. Please don't send emails if you are from out of state, however if you know of someone in California please send this their way. 

If anyone has question please let me know. This is a great advocacy opportunity, the committee needs to hear your voice!

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Comment by PPMD on July 23, 2012 at 8:56pm

Yes, that's right -- Thanks Leeandra! 

Comment by Leeandra Arhdeacon on July 23, 2012 at 6:58pm

emails should be sent to:    GSambrano@cirm.ca.gov

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