PPMD; Our Mission

  

The Research

Hope for the Future

Foundation for a Future supports and funds leading researchers, as well as organizations and institutions engaged in cutting-edge translational research (research aimed at moving science from the lab into human clinical trials) to find a treatment or cure for Duchenne muscular dystrophy.  We partner with national Duchenne organizations to identify and fund promising research that can help save this generation of boys living with Duchenne.

Research is advancing in several promising treatments, including:

Exon Skipping

Exon skipping offers the possibility of slowing down the fast progressing Duchenne into the much milder Becker muscular dystrophy. Human clinical trials for exon skipping drugs are currently underway in Europe and the US. 

To learn more about exon skipping, watch this video from Australian Broadcasting Corporation.   http://www.abc.net.au/catalyst/stories/2879914.htm

Gene Therapy

Several scientists are developing techniques for transferring a modified dystrophin gene into muscle cells.  It is hoped that introduction of this shortened dystrophin gene would allow for the production of a truncated, but functional protein that would reduce the severity of the disease and improve quality of life.  This potential treatment is very promising, however, human clinical trials are not expected to begin for several years.

Utrophin Upregulation

There are several drugs under development to upregulate utrophin, a protein structurally and functionally similar to dystrophin. Researchers have identified several compounds that upregulate utrophin in the mdx mouse. Human clinical trials could begin in the next few years, however, additional research is necessary.

Myostatin Inhibition

Myostatin is a growth factor that regulates the size of muscles from early development through adulthood and prevents muscles from growing too large.  Decreasing levels of myostatin in patients may stimulate muscle regeneration, increase muscle size and improve strength.  Drugs that inhibits myostatin are currently under development. 

IGF-1

Insulin-like growth factor (IGF-1) plays an important role in the regeneration of damaged muscles by activating muscle cell proliferation and fusion.  Increasing levels of IGF-1 may help repair damaged muscles and preserve muscle function.  PTC Therapeutics, through Project Catalyst, is developing compounds to increase levels of IGF-1. 

FDA Approved Drugs

Reseachers are evaluating whether certain drugs that are already FDA approved for other conditions might also help to alleviate some of the symptoms of Duchenne.  The development and approval process for a new drug can take over ten years and is extremely expensive, so determining whether certain currently approved drugs are effective against some of the symptoms of Duchenne offers the potential of providing new treatments in the near term.  Clinical trials for Duchenne patients are necessary to determine dosing and efficacy for Duchenne, particularly since the drugs will be given to children. Eli Lilly is currently conducting a Phase 3 clinical trial to determine if tadalafil (Cialis) can slow the decline in walking ability of boys with Duchenne.  Scientists are also looking at other compounds that target the same pathway as Cialis. 

While these and other treatments under development each have the potential to slow the progression of Duchenne, a combination of several treatments will most likely be required to significanly slow or stop its progression.  

 

An overview of Duchenne research can be found at:  http://www.treat-nmd.eu/dmd/research-overview/introduction/