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JUST ASK!: NCF Reports a Spanish Research Team Makes a Critical Treatment Discovery for CFIDS

From Summer 2011 Forum

The "Just Ask" column is intended to act as a means for patients to inquire aabout issues related to the NCF's research activities. This column is NOT intended to act as medical advice in any way, shape or form! The National CFIDS Foundation assumes no responsibilities for any action or treatment undertaken by readers. For medical advice, please consult your own personal healthcare providers.

Q: I assume that the NCF follows all kinds of research related to this disease. Is there anything new that you find interesting?

A: Yes, there is new research from Spain that we find quite interesting because it directly relates to therapy and is applicable to the markers found to be operative in this disease. This information comes from a patent on this disease. I am mentioning this research because of the NCF's article, in this Forum, which relates elastase generation to myelodysplasia and AML development. The research finding is that alpha-1-antitrypsin treatment appears to significantly impact CFIDS/ME. As you are probably aware, Dr. Robert Suhadolnik, at Temple University, discovered that the peripheral blood mononuclear cells (PBMCs) of patients with CFIDS/ME had hyperactive RNase L with a molecular mass of 37 kDa produced by the proteolysis of the native form of 83 kDa RNase L. The ratio of the 37 kDa to the 83 kDa form of RNase L has become a marker of disease severity. Elastase is capable of causing the proteolysis or fragmentation of RNase L. What then is the role of alpha-1-antitrypsin here? Alpha-1-antitrypsin is a glycoprotein secreted in the liver and is normally present in high concentrations in serum as well as most tissues where it acts as a serine protease inhibitor. Furthermore, alpha-1-antitrypsin is a known elastase inhibitor. These inventors first established that PBMC extracts from patients with CFIDS/ME show raised elastase activity, far higher than those PBMC extracts from healthy subjects. Next, the critical finding by these inventors is disclosed. They found that alpha-1-antitrypsin prevented the degradation of 83 kDa RNase L to generate the hyperactive form of 37 kDa RNase L. Furthermore, the inventors found that alpha-1-antitrypsin activated the expression of genes involved in the 2-5A synthetase pathway so that the administration of exogenous alpha-1-antitrypsin could re-establish normal RNase L activity and prevent its proteolysis in the PBMCs of patients with CFIDS/ME. Is alpha-1-antitrypsin available for use as a drug therapy? Therapeutic concentrates are prepared from the blood plasma of blood donors. The U.S. FDA has approved the use of three alpha 1-antitrypsin products derived from human plasma. This drug is known by several names - Prolastin, Zemaira and Aralast. These drugs are given to the patient as an intravenous infusion and have been used for augmenting alpha-1-antitrypsin levels in the blood. Initial information indicates that this therapy may cost upwards of $100,000 per year per patient. These inventors did use this therapy on CFIDS/ME patients and the therapy was successful as evaluated by a number of quantitative as well as qualitative improvements clinically speaking. The NCF plans to make this patent information available via our website.

Reference: Use of alpha-1-antitrypsin for the preparation of drugs for the treatment of chronic fatigue syndrome; Inventors: Garcia-Quintana A, Alegre Martin J; Patent # 20100331261; December 30, 2010; Assignee: Grifols S.A.- Barcelona, Spain

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