October 24, 1998

Cytolog Research Progress

Pediatric Guillain Barré Syndrome:

Giullain Barré Syndrome is an autoimmune disease possibly triggered by a viral infection, which affects both axon and myelin components of the peripheral nerves, causing an acute state of painful paralysis. It has several different degrees of severity. Congenital therapy includes intravenous immunoglobulins during the first 3-5 days of disease. Severe cases, specifically those not treated with immunoglobulins, tend to have prolonged recovery phases, taking as long as 12-18 months to achieve a functional state. Some patients do have permanent nerve damage. (In Guatemala, immunoglobulins are very expensive for a lot of people, and in many cases the most affected patients are the ones who can't afford to pay for them)

We received financial help from John Mann, who was interested on a child who had GB Sx some time ago, but when the hospital authorized us to use Cytolog, this patient had already been discharged. By this time we had been authorized to work on a GB protocol with Dr. Teyda Villate, a MD/physical therapy attendant at the Children's Infectious Disease and Rehabilitation Hospital, a public institution in Guatemala City. Patients enter the hospital right after the Syndrome is settled (between the 8th and 10th day of disease, depending on the severity), and thus this excludes us from observing the effects of Cytolog on the truly acute phases of the disease process. (In order to do this, we would have to pay a species of "fee for right of research" to Roosevelt hospital, and pay all participant physicians as well). Our working hypothesis is that Cytolog reduces nerve recovery time after severe injury. We're set to include six patients in the study, and follow them over 16 weeks.

The study was started on August 3, 1998 with two patients. The first patient was Mauro, a 5 year old boy who had severe nerve damage on both hands, with no evidence of improvement after a long time of physical therapy and rehabilitation procedures. He was given 6 weeks of Cytolog and was discharged from the hospital. We have not been able to document any benefit, although it seemed to us that he did show a clinical improvement before he left. We're still pending follow-up on this case.

The second case is Cristina, an 11 year old girl who had a severe case of GB approximately 1 year ago. The family couldn't pay for the immunoglobulins. She has severe bilateral peroneal nerve damage, with bilateral "dropped foot", that makes it difficult for her to walk. The baseline EMG at study entrance was only slightly different than the one done after six weeks of therapy, but it meant a rather significant change in prognosis, that is , the possibility of nerve recovery was evident on the second exam and not the first one. Cristina is due to go for her third EMG next week. We saw some evidence of clinical improvement last week. She has gained lateral toe movement (which she had totally lost), and the dropped food seem to be less dramatic. We're really enthusiastic and full of expectations.

The third case is Patricia, a 6 year old girl also with severe disease, who entered the study about 4 weeks later. She did receive immunoglobulins. When she arrived at the hospital, she could only move her eyes, and was suffering both because she was separated from her family and was in severe pain. EMG showed severe nerve damage. After she was started on Cytolog, the pain disappeared and her mood improved immediately. Close to her eighth week of therapy, she is now able to turn herself in bed, and soon will be seated on a wheelchair. This seems to be a rather rapid course for a severe case. We're planning to document this not only with her EMG reports but also comparing this outcome with those obtained on similar cases.

Best Regards

Alejandro & Fabiola Nitsch, MD