Cell treatment of neurological diseases

In October 2005, there was an international symposium on stem cells transplants and similar forms of treatment in the Hyatt Hotel in Newport Beach, just south of Los Angeles, California. A closed group of about 50 experts reviewed the work groups that carry out these treatments in more advanced countries did. The symposium was organized by the transplant group from the City of Hope, a large hospital for cancer in southern California, with the support of several governmental and non-governmental institutions. The symposium was a mark in the U.S. strategy. Till then, the Americans were late in the use of these treatments for immune disorders, in particular multiple sclerosis, though they were the leaders in stem cells transplant in the treatment of cancer. From that moment on, they started investing heavily in immune disorders, based on basic science research with rats that helped to resolve the technical details of what to do with patients. A little after that came the Europeans and then several new studies started in North America.

The reason for the Americans to change their strategy is that in the last 5 years new techniques of stem cells transplant were developed, mainly in Italy, and the mortality in transplants fell from more than 10% to nearly zero. The doctors who work in this area call themselves the "transplanters". They are hematologists, rheumatologists and oncologists who have a sub-specialty. Transplanters from Netherlands, Canada, Italy and the USA presented reviews in this international symposium in California. In short, in Europe and North America around 400 stem cells transplants were made in patients with immune disorders since a Greek doctor, Dr Fassias, who worked in Thessaloniki, published the first multiple sclerosis cases in 1995. From all transplants, half were multiple sclerosis cases: 183 in Europe and 13 in Canada. The others were cases of systemic lupus erythematosus, rheumatoid arthritis and systemic sclerosis also called dermatomyositis.

The results of transplants in these 10 years summarize as follows. The mortality in the transplant itself was 10% in multiple sclerosis and 13% in the other 3 diseases. The survivors, approximately 1/3, do very well, being healed from the disease for many years. In a second group, over 1/3 of the patients also do well, although there remained some basic disease activity or chronic complications of the transplant and though not completely healed as the first group, they show great improvement. The remaining third, around 30% of the patients, showed no important improvement, but still with a better prognosis than the ones that did not do the transplant. This summary of results does not apply to rheumatoid arthritis only, in which the outcome of the transplant is not as good because less than 50% have a complete or nearly complete remission for a long period of time. That is, around 50% of the rheumatoid arthritis transplants have the same outcome that 70% of the multiple sclerosis, lupus or systemic sclerosis, transplants.

There were some unexpected results in 30% of the patients who got along very well. Patients improved and in some cases improved a lot. This improvement was particularly unexpected in patients with multiple sclerosis who presented a recovery of neurological function and in people with systemic sclerosis or dermatomyositis, a disease were patients develop a hardening of the skin and subcutaneous tissue that in advanced stages of the disease prevents the blinking of the eyes, the smile and other facial expressions as well as hardening of arms, hands and legs that stiffen as if they were strewed. This hardening also occurs in internal organs like the lungs and is due to a deposit of hard tissue, such as scarring, which is added under the skin, lungs and other internal organs. The name sclerosis in medicine means hardening and what in multiple sclerosis is in small lesions in the central nervous system, in systemic sclerosis is in the skin, lungs, esophagus and occasionally in other peripheral organs, but not in the central nervous system. The improvement in seemingly immutable sequels in these two diseases broke a dogma in medicine, turning upside down the knowledge experts had. These improvements led many doctors and especially lay people including the media to imagine that the treatment could lead to tissue regeneration.

The dogma broken in neurology and rheumatology, meaning the possibility sequels apparently definitive in people who are in very serious and tragic situations, in the general population raised an expectation that everything could be regenerated, including brain damage, epilepsy, old age sclerosis and even dementia. Nothing of the kind is even close to reality yet, but there is the hope now closer linked to science due to lab experiment in rats. Many �Institutes for Regeneration Medicine� and many �Institutes for Cell Therapy� are opening, in special in the US, even in very traditional universities. The fact is that the huge repercussion of these studies over the last years caused many donations, for example, the ones related to Christopher Reeve, Superman.

Two neurologists presented results in the symposium, one representing Johns Hopkins Hospital, Baltimore, USA, and the other the below undersigned representing a group that worked in Hospital Santa Cruz, Hospital Nossa Senhora das Gra�as and Unineuro in Curitiba, Brazil. Dr Douglas Kerr and the group from Curitiba presented similar experiences, though the group from Baltimore called their treatment HiCy (high dose cyclophosphamide) and the group from Curitiba called their treatment stem cell mobilization. We presented 12 cases and Dr Kerr presented 8 cases, meaning that we, in Curitiba, were the most experienced in the world in the new treatment. None of the groups had any deaths or significant sequel resulting from the treatment. Besides these 12 cases, in Curitiba we still have the first case, published in the Acta Neurologica Scandinavica, a patient that has been healed for 8 years and the paralysis in her legs has improved significantly.

There were no other groups representing Latin American in the symposium in Newport Beach, no Brazilians other than the undersigned as well as no African or Asian groups. In several bibliographic reviews, the cases treated in Brazil by the group from Ribeir�o Preto and S�o Paulo do not appear, indicating they have not been published in international indexed journals yet.

Both Americans and Europeans are now starting the so-called phase 3 in controlled studies. Studies to date are considered stage 1 or 2, meaning experimental. In phase 3, patients are randomly chosen, so that half receives a transplant and the other half receives the best treatment available for the active disease. In all protocols the best existing treatment, the one to be compared to the transplant, is the same cyclophosphamide used in Curitiba and in Baltimore, or, more rarely the metrotexate and azathioprine, all powerful immunosuppressants. For example, treatments as active as corticoids, interferons and anti-inflammatory traditionally used worldwide are not considered, not even in Brazil.

Newport Beach, October 8th 2005 - Prof. Dr. Paulo R M de Bittencourt, PhD - Translated and adapted by Marilia Bittencourt, January 2009