December 19, 2000

ON A SEASON OF HOPE, NOW AND FOREVER

The brilliance of the holiday season, for the many grateful observers who celebrate it in diverse ways, is in the way it transforms a time potentially fraught with cold and deprivation -- the heartless depths of winter -- into a joy-filled celebration of family warmth, plenty and perhaps most importantly, hope.

It's an idea we can readily resonate to in our daily work at the Muscular Dystrophy Association, particularly those of us who have worked to advance MDA's mission going back to the Association's earliest decades.

You see, in those early days in the 1950s and 60s, hope was a commodity in short supply for families and individuals affected by neuromuscular diseases.

The causes of the spinal muscular atrophies, muscular dystrophies, metabolic disorders and other diseases affecting muscle and nerve were unknown.

And yet in subsequent decades, thanks to the unyielding determination of MDA-funded scientists, the darkness that surrounded these diseases began to clear. Gradually, the word "incurable," which had adhered so tenaciously to these diseases, began to lose its dread sense of implacability and in fact became outmoded -- thanks to the hope augured by such breakthroughs as the 1986 discovery of the genetic cause underlying Duchenne muscular dystrophy.

Subsequently, during the 80s and 90s, we witnessed literally dozens of gene discoveries for disorders in MDA's program -- each one providing a beacon of possibility in a landscape no longer bereft of hope and illumination.

"Sing Out!" by Milda Vizbar is one of many beautiful holiday-themed images that reside in the MDA National Art Collection.

Several MDA-funded research developments within the last two months have greatly added to that sense of hope and promise.

Early in November, in an announcement by MDA-funded researchers at Johns Hopkins and Harvard universities, we learned that damage to nerve cells in rodents affected by a condition similar to spinal muscular atrophy and amyotrophic lateral sclerosis can be repaired by use of neuronal stem cells. It's a development that doesn't signal a cure but proves the potentiality of stem cells to have a beneficial effect in counteracting conditions causing rampant destruction in the nervous system.

Then, shortly after Thanksgiving, an MDA grantee at the University of Pittsburgh was part of a team that announced a promising advance in Duchenne muscular dystrophy.

The team devised three minigenes to provide the crucial protein missing in those affected by Duchenne, and successfully delivered the minigenes to muscle cells in mice using an adeno-associated virus. The scientists involved hope to move this concept into clinical studies involving humans in the near future.

Hope is always with us. It "springs eternal in the human breast," as the poet Pope says. And yet, as long as we need hope, it means our goals have not yet been unfulfilled.

That's something we'll remember as we strive during 2001 to advance our scientific goals further, toward our ultimate destination of eradicating neuromuscular diseases.

To all the families we serve and all those who generously support MDA's cause, we offer our fondest wishes for a joyous holiday season.

With every best wish . . .

The Ross Report Archive