A Cure for Multiple Sclerosis?

needleA research study published June 2016 in the prestigious journal, The Lancet, details a very risky but effective cure for patients with aggressive multiple sclerosis (1). In multiple sclerosis (MS), the immune system attacks its own insulating material called myelin which usually covers nerve cells in the brain and spinal cord. There is currently no cure, but symptoms may be managed through the use of medications that manage inflammation and inhibit the immune system to dampen further demyelinating damage. Symptoms vary among individuals but can include numbness, weakness in the limbs, loss of balance, fatigue, and blurred vision. More severe cases can involve paralysis. The medical procedure described in The Lancet relies on the harvesting of a patient’s own hematopoietic stem cells to be used as grafts after almost completely destroying the patient’s existing immune system. This autologous hematopoietic stem-cell harvest, which selects for CD34+ progenitor cells, is taken from the patient after stimulation (mobilization) to increase the numbers of circulating myeloid stem cells (2). This is done with the chemotherapy drug, cyclophosphamide, and filgrastim, a granulocyte colony-stimulating factor analog which promotes the rapid increase and differentiation of a type of white blood cell called granulocytes. A cure to MS is possible through the ablation of the current faulty immune system of MS, and the subsequent replacement with hematopoietic stem cells that can then become healthy immune blood cells.

The study began in 2000, and the paper describes Phase 2 of the study at three hospitals in Canada. Twenty-four patients participated, and one has died during the study due to liver complications. The treatment is initially quite toxic, and strong chemotherapy drugs are given to destroy the immune cells. After immune cells are rendered null through the use of busulfan, cyclophosphamide, and rabbit anti-thymocyte globulin, the patient is left vulnerable to infection. The chemotherapy is toxic to sperm or eggs, and women enter early menopause. Hair and fingernails fall off. Yet, MS patients desperately searching for a cure have chosen this option and experienced not only a cessation of symptoms, but also some recovery from previous MS-induced damage. Patients were followed up for up to thirteen years after autologous hematopoietic stem-cell transplantation, and even though they were not on any medications, they were found to be free of relapses and without any new brain lesions in MRI scans.

Interestingly, this process of “resetting” the immune system was found to be effective for MS quite accidentally. Initially, people with both leukaemia and MS were being treated for leukaemia, a cancer of the white blood immune cells. This cancer starts in the bone marrow, so autologous blood stem cells from the bone marrow are first collected and rid of any cancerous cells. The cells are then reintroduced into the body as a graft to elicit new, healthy immune cells after the existing, cancer-ridden immune system is destroyed by toxic chemotherapy. The results were found to be effective not only for leukaemia, but for MS as well. It is hoped that a similar approach can be used to cure other autoimmune diseases such as Crohn’s disease, scleroderma, and lupus. The risk associated with deleting a person’s entire immune system is great, but by adjusting the balance between toxicity and maximal therapeutic results, a safer medical intervention can be found. Larger trials can be conducted to determine the best dosage and effectiveness so that hopes can be high for people suffering from MS and other debilitating autoimmune diseases.

 

Sierra Delarosa

 

References

  1. http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(16)30169-6/abstract
  2. http://www.ncbi.nlm.nih.gov/pubmed/12592344

FOLLOW DR. KO: The Unfortunate Case of the “Drug Mule”

Screen Shot 2013-12-31 at 6.08.10 PMOn a crisp Saturday morning, Internal Medicine Residents from all over the state of New York trickled into the University of Rochester’s School of Medicine, sporting ties and dresses in lieu of their usual white coats and scrubs. In their hands they clutched a precious cargo, a cylinder that protected a poster to be used for a presentation about their research or about an unusual clinical case.

I was among the ninety-seven young (when does one cease to be called young, I wonder?) residents who had been selected to present their work at the annual New York American College of Physicians’ abstract competition. I had to rush to the airport right after work on a Friday afternoon, and then with barely six hours of sleep, (no, six hours is not enough) drag myself up and out into the cold air of Rochester, firmly gripping my poster, all the while wondering why I was giving myself all this extra work on my only free weekend of the month.

But then, as soon as I got a glimpse of other young physician’s work and ideas, my somnolence evaporated. How stimulating and motivating it was so see the talent out there! I especially remember one resident who was doing a double residency in Internal Medicine and Pediatrics. One residency is a daunting enough task, but two! And then I watched in amazement as he juggled his research poster with one hand, and his toddler with the other. It never ceases to amaze me how people can raise children and be in residency at the same time. His poster illustrated two years of laboratory work during which he exposed mice to different degrees of radiation and then extracted their stem cells to measure the relationship between the radiation exposure and the death of the cells. Fancy stuff. When I asked him how he had time for residency, family and research, he simply replied: “I don’t, really.”

My presentation was not nearly as fancy, but did make for what I figured would be a captivating story. It was about a “drug mule” I had encountered during my rotation in the Intensive Care Unit (ICU). She was a young woman barely out of her teens who was brought into our Emergency Department after suffering a seizure on the street. She subsequently went into cardiac arrest and was brought back to life after more than twenty minutes of CPR. Before her heart stopped, she was able to tell us that she had tried cocaine for the first time.

While in the ICU, our patient was kept on mechanical ventilator support for over a week because she could not breathe on her own. A number of people claiming to be her relatives showed up daily to visit, and her alleged mother raised concerns about my patient’s enlarged abdomen. We did an abdominal X-ray and saw nothing unusual. Eight days later, a nurse found pellets of a white substance in her diaper! That’s when we opted for a CT scan and found numerous other drug pellets that had not been picked up on the X-ray. It turned out that she had been paid to smuggle a load of cocaine into the U.S., probably never realizing the risk she was taking.

I found only one study comparing the sensitivity and specificity of different imaging modalities for detection of concealed drugs in the human body. A CT scan, although costly and laborious, is definitely the way to go when someone is suspected of concealing drugs in their body. This is especially so when there is imminent danger to the individual’s life, as was the case with our patient. Given that one or more of the drug pellets had already burst, a steady stream of cocaine was leaking into her system causing havoc.

When I first set out to become a doctor, research was not at all on my mind. I wanted to be a clinician, to be with patients. Forget about lab rats and data collection, forget about grant applications and writing papers. That “stuff” wasn’t for me, so I thought. But that changed during my third year of medical school, when I first got my feet wet in research on Transient Ischemic Attacks. Despite the small role I played in the project, I felt an indescribable satisfaction from giving back to the scientific community. I was, in my own very small way, contributing to the advancement of Medicine!

The judges seemed taken by my presentation about the cocaine overdose. I felt I had told a story, showed images, presented some data, and had effectively conveyed the message I wanted my audience to take home. However, I had to rush back to the airport before the announcement of the winner of the poster competition was made. I had entered this contest for fun, really just “for the heck of it”. But then a very welcome surprise came through my phone as I was to board my plane: I had won! Out of the fifty-seven contestants in my category, I came in first. Certainly worth the plane rides and the early mornings! All those years working at the McGill Office for Science and Society had seasoned me properly!

Who would have thought back then that I would be representing the state of New York at the National American College of Physicians conference? Not me.

Melody Ko

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