1) The Bottom Line of Dollars-and-Cents: The Cost of Immunoglobulin Replacement Therapy (IRT), be it intravenous (IVIg) or subcutaneous (SCIg) is exorbitant. The cost of lifelong infusions now averages between $6,500 to $9,000 per-month. Factor in the average monthly cost of infusion support, be it home ($310) or hospital-based ($2,200), and the cost mounts astronomically. Based on the average, though markedly shortened, life expectancy (in comparison to healthy individuals) of a patient with Common Variable Immunodeficiency, the lifelong cost of life-sustaining IRT can average $3.0 to $4.5 Million per-patient.
Already this year has seen a movement by both Medicare and insurance companies to redefine criteria for CVID diagnosis and access to IRT. There is no doubt we are very expensive patients.
Gene Therapy, which offers the hope of cure, rather than lifelong treatment, represents a far more cost-effective approach to both taxpayers and health maintenance organizations. Full cure, now and in the future, will cost but a mere fraction of IRT.
2) A Proven Primary Immunodeficiency Track-Record: One only need peruse the recent article in the much-revered periodical New Scientist, "'Bubble-Kid' Success Puts Gene Therapy Back On Track" to get a glimpse of the incredible strides which have been made in curing children with Severe Combined Immunodeficiency (SCID). In this version of Gene Therapy, stem cells are harvested from the patient's own marrow, provided with a working copy of a defective gene, and re-injected. The overwhelming result has been that children who almost always died before their 5th birthday are being provided with fully-functioning immune systems.
In this regard, Gene Therapy for Primary Immunodeficiency Disorders has a curative precedent, a road-map with which to begin; the groundwork has already been laid. The process works. With incredible, almost daily, inroads in the gene-sequencing of chronic and rare diseases - "supercomputers" are now on the job - CVID's somewhat wide array of mutations should be clearly spelled out in the near future.
3) The Immunoglobulin G "Well" Is In Danger of Running Dry: The usage of IRT in medicine is proliferating astronomically - almost doubling in quantity since 2008. What was once primarily a drug for Primary Immunodeficiency Disorders is now being used widely in the fields of Cardiology, Nephrology, Opthalmology, Rheumatology, Obstetrics, and more. For the first time, during mid-2014, Neurology is expected to surpass and quickly outpace Immunology in Ig usage. Demand for IVIg and SCIg, according to many experts, is in real and imminent danger of outstripping supply.
Gene Therapy for CVID would be a means of reducing demand and alleviating impending shortages. An entire group of patients, could be removed from the IRT treatment roster.
4) IRT Is Only A Partial Treatment, Not a Cure: IVIg and SCIg contain only Immunoglobulin G in therapeutically relevant amounts. Many, if not a majority of, CVID patients have additional deficiency of antibodies with crucial functions: "A," "M," and "E." These additional antibodies are irreplaceable and remain deficient despite IRT. For this reason, although IRT reduces instances of severe infections, many with CVID continue to experience severe and oft-times life-threatening illnesses. Many still die as a result of these infections.
Rather than a partial treatment, Gene Therapy represents the hope for complete immune system restoration for those with CVID. It represents the hope for medical and immunological normalcy.
5) Deadly Cancers: The rates of malignancies in CVID are astronomical, perhaps higher than in any other disease. Rates of leukemia, lymphoma, and other cancers are increased on average 20 to 50-fold in CVID in comparison to the rates of healthy counterparts. "Weakened surveillance against oncogenic viruses, premalignant or malignant cells, or both in CVID, allow for malignancies which would normally be defeated and eliminated, to take root and multiply.
The bright future of cancer treatment, the revolutionary new treatments written of almost daily, "Immunotherapy" and "Cancer Vaccines," rests in "training" the immune system to fight cancer. It is with good reason, based on its wild success, that Immunotherapy was named Science Magazine's 2013 "Breakthrough of the Year." This more natural approach will soon crush traditional oncology: chemotherapy and radiation which are toxic and deplete the immune system.
Without Gene Therapy, these "immune-training" cancer breakthroughs will pass CVID and other Primary Immunodeficiency Disorders right by. Our immune systems are completely incapable of this "higher-education." Success in reducing the untenable rates of deadly malignancies in CVID rests in the restoration of onco-surveillence which a newly-functioning immune-system can provide. Then, and only then will malignancy, which has surpassed infection as the number-one killer of those with CVID, be reduced, and the immune-system be capable of undergoing "training" to defeat cancer if and when the need arises.
6) An Enthusiastic and Highly-Motivated Patient-Base: CVID patients are vocal, enthusiastic, determined, and highly-motivated. We are an Army. Clinical-trial enrollment, participation and follow-through will not be an issue with this crowd. We will be energetic, galvanized, lined-up and fighting every step of the way.
Gene Therapy is where the future of CVID lies. It represents the hope of cure, rather than partial and prohibitively-expensive maintenance treatment. It is cost-effective and it works.
It's high-time to get the clinical-trial ball rolling for CVID Gene Therapy!
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