Researchers, advocates see better ALS therapies on the horizon

Editor's note: This is part of our continuing series of stories about Bruce Kramer, the former dean of the College of Education, Leadership and Counseling at the University of St. Thomas, as he copes with life after being diagnosed with ALS, also known as Lou Gehrig's Disease. You can read all the stories in the series by clicking here.

This weekend marks the anniversary of one of pro sports' most poignant moments: Baseball great Lou Gehrig, standing before microphones near home plate at Yankee Stadium on July 4, 1939 as a standing-room only crowd honored him as one of the most famous players of his time.

The moment happened shortly after he was diagnosed with a mysterious neurological condition: ALS. The anniversary offers an opportunity to take stock of where research and treatments for the disease may be headed.

"Today, I consider myself the luckiest man on the face of the Earth," Gehrig told the crowd tat day. "I may have had a tough break, but I have an awful lot to live for."

Gehrig died two years later of the disease which now bears his name. He was 37 years old.

In the 75 years after Gehrig talked about his "bad break," organizations like the ALS Association and facilities like Minnesota's Mayo Clinic are still looking for effective treatments and a cure for the disease that ultimately killed the baseball player. In those 75 years, there's been only one drug developed that buys ALS patients, at best, two to three months of survival.

At a recent downtown Minneapolis luncheon, a small group heard from one man who's been living with ALS since being diagnosed in December of 2010: Bruce Kramer.

"At the beginning of 2010, the thought that I might have ALS was a million light years away.Yet by December, ALS was all I could think of," he said. "It slept with me, it ate with me, it colonized my thinking. My waking moments, my sleeping dreams. It became my constant companion--a dark presence between me and my loved ones, my friends and my colleagues."

Kramer has been in two drug trials since being diagnosed. Each has ended, failing to reach the goals set by the drug's makers.

Jane Gilbert, the head of the national ALS Association, said, "that's not money thrown down the drain, because it is money that puts them one step further toward looking at other iterations of that drug that may be helpful."

"What we're finding in the research that's ongoing is that many of the neuromuscular diseases, there's a relationship. And so all of a sudden, all of these diseases that would have been considered 'orphan' diseases or certainly not diseases of huge population bases, there's a lot more interest in them because they are seeing that they're related," Gilbert said of diseases like Parkinson's and muscular dystrophy.

"There is a very definite feeling that they're all connected," she said. Eventually, "there will be a cocktail of drugs, or a cocktail of treatments that will in fact treat ALS."

Related ALS Association links
• ALS Navigating Life with ALS
• Care Services Roadmap

Gilbert believes stem cell trials offer the most exciting possibilities right now, "which of course we're very involved in and have supported financially, are very, very exciting. Again, are they an absolute cure-all for, you know, five years down the line? Probably not, but there's going to be a lot of information gathered from these trials and from watching them and studying them."

There are two stem cell therapy trials going on at Mayo Clinic. Both involve using stem cells grown from a participant's stomach fat. One is looking at the safety of injecting ALS patients with varying doses of their stem cells. The other trial uses a patient's stem cells, modified with growth factors, and reintroduced into the patient's spinal fluid. Researchers hope those tweaked stem cells will protect cells that control movement from further damage and death from ALS. Dr. Anthony Windebank, with the Center for Regenerative Medicine at Mayo, calls the fledgling therapy a "radically new kind of treatment."

Windebank, like Gilbert, believes a breakthrough is close for ALS.

"I honestly think that is correct," he said. "It's the closest one we'll find a treatment for. I'm an optimist, that's why I enjoy being in this field, but I'm cautious. I've been doing clinical trials for ALS for almost 30 years and I've seen many things that were hopeful turn out not to work in the end but I think the research is moving so fast now that there's a cure on the horizon."

Still, the money spent on ALS research is a fraction of that spent on other diseases. The National Institute of Health spent about $40 million on ALS last year, compared to about $5 billion spent by the National Cancer Institute, for example. Advocates say it's hard to get research money for a disease that affects less than 30,000 people at any given time.

ALS has also been a confounding puzzle to solve using traditional animal research models.

"Unfortunately, we've treated ALS successfully in mice many times and when it comes to humans it just hasn't worked," Windebank said. "And one of the paradigm shifts we made about five years ago was saying this disease is so awful that we need to go directly to the patients. If we can demonstrate that something is safe in preclinical studies, then why not treat patients rather than mice and see if that will help us move along more quickly?"

Kramer recognizes that medical advances will come too late for him. His voice is much softer than the last time we talked. The effort to converse exhausts him, which made his appearance at the local ALS chapter luncheon even more impressive. But he thanked the group for its work.

"No one should be alone as they face ALS," he said. "The healing that I have found in the care of this chapter has helped me to live a full life even though ALS continues to take away my physical capacities. There's not a day that goes by that I don't thank this association for the support they have given me. I hope you will find it in your hearts to support them, to continue and grow and find a treatment for ALS."