MS treatment that shows promise may not get clinical trials

From ABC 4 News in Utah:

SALT LAKE CITY -- At the end of November a young mother kissed her children goodbye, and boarded a flight with her husband. It was the beginning of a medical journey that she believes has ended with miraculous results. Michelle Colledge was treated for severe Multiple Sclerosis at Johns Hopkins, in Baltimore Maryland. She is just one of 44 patients to receive the experimental High Dose Cyclophosphamide, and may be the last.

Michelle’s husband says his wife’s MS was so severe that several times he was told to prepare for her to die. Current medications available to treat Michelle had failed. Unwilling to give up hope, they researched until they found the work being done at Johns Hopkins. It was that discovery that Michelle Colledge says saved her life, and gave her life. “It feels miraculous to me. I feel like Lazarus rising from the dead. Like was dead, and a prisoner inside my own body and now I am living again.”

In order for Michelle to live, her immune system had to die. At Johns Hopkins, she says she received the High Dose Cyclophosphamide, an older and stronger form of chemotherapy. Thirteen liters, over a course of four days, killed all of her white blood cells. She says,” I like to think of them as assassins. So, they had these instructions that were not correct, that said the brain and the spine was the enemy. So, we killed all of those cells.” Her new immune system took about three weeks to grow. Adam Kaplin M.D, PhD, at Johns Hopkins explains, “It’s a resetting of the whole immune system and these people really genuinely have a new immune system.” That immune system has no memory of MS and no longer attacks the brain and spinal chord.

Michelle says she noticed a difference within days of receiving her last chemo treatment. “The sixth day I could tell. There was clarity to my mind that hadn’t been there in about ten years. It was brilliantly clear, almost sensory overload. It’s like living in a black and white world and then all of the sudden there are all of these colors and noise.” By December 22nd, her white blood cell count was up, and her immune system strong enough to go home to her family in Utah.

As weeks pass, there have been other life changing improvements. Michelle says sensation has returned. “Losing the ability to feel things is just so devastating. About two weeks after I got back, my husband rubbed my back, just to say goodnight, and I was like hold on, I can feel that!” Michelle says she can now feel hot and cold and no longer has to ask her youngest daughter to check her bathwater with a finger before getting in. She is knitting to help her hands rehabilitate, has taught her little girl to ride a bike, and takes pleasure in the everyday mundane tasks of life. “I’m driving, filling up the gas tank, making dinner. Things that everybody takes for granted, I can do now.”

Michelle’s reversal of symptoms is not uncommon with the High Dose Cyclophosphamide treatment, ninety percent of those who have had it have no evidence of the disease, and forty percent have show improvement in function. Dr. Kaplin says “If you can turn off the inflammation, really stop the immune system from its constant picking at the brain and spinal chord, then there is a healing process.”

But it’s a process that may no be available to others. Large scale clinical trials cost millions and right now, study of the treatment is unfunded. Pharmaceutical companies are not interested in funding it because the patents on the medications being used has already lapsed and there is no money to be made. The National MS Society says it has not been approached to fund the trials needed for FDA approval. Johns Hopkins says it did submit a grant request several years ago but was denied. It hopes to put forth another request in the coming year. A spokesperson says because of the economy donations are down, and it’s uncertain there would be the money needed to fund the Johns Hopkins study.