Revimmune for Autoimmune Disease
31 Jul 2007
Autoimmune diseases afflict more than 8 million people in the U.S. and impact on virtually every medical specialty.
http://www.pipelinereview.com/joomla/content/view/13539/109/
TAMPA, FL, USA | July 31, 2007 | Revimmune uses an ultra-high intensity, short-course of an intravenous formulation of an approved drug (cyclophosphamide), in a new patent-pending method to "reboot" a patient's immune system, thereby eliminating the autoimmunity, whereas current therapies including oral cyclophosphamide are used chronically to try to suppress the inflammation of autoimmunity. Based on long-term follow-up showing complete remissions, there is substantial evidence that Revimmune has the potential to cure cases of severe refractory autoimmune diseases such as aplastic anemia and myasthenia gravis. Accentia's lead indication for Revimmune is multiple sclerosis (MS).

Autoimmune Diseases Afflict Millions of Patients
Autoimmune diseases afflict more than 8 million people in the U.S. and impact on virtually every medical specialty. While many autoimmune disorders (e.g., vitiligo, thyroiditis, pernicious anemia) are relatively indolent and easily managed, severe cases of systemic lupus erythematosus, rheumatoid arthritis, aplastic anemia, multiple sclerosis, etc., can cause severe morbidity and even mortality.

To date, more than 30 neurologic diseases have been recognized either to be caused primarily by autoimmune mechanisms, or to have important autoimmune components. Although many of these diseases can be treated clinically by currently available conventional immunosuppressive regimens, important problems remain: some patients are refractory to standard immunotherapy, and others respond only partially.

In nearly all cases, immunotherapy must be continued indefinitely, maintaining an impaired immune system, and often resulting in cumulative adverse side effects. Despite this, the vast majority of patients on conventional immunomodulatory treatment for MS continue to accrue disability.

Revimmune Therapy for Autoimmune Disease
Based on long-term follow-up showing complete remissions, there is substantial evidence that Revimmune has the potential to cure cases of severe refractory autoimmune diseases such as aplastic anemia and myasthenia gravis. Accentia's lead indication for Revimmune is multiple sclerosis (MS).

Developed by Dr. Richard Jones, Dr. Robert Brodsky, and colleagues at the Johns Hopkins University School of Medicine, Revimmune works by temporarily eliminating peripheral immune cells, including the immune cells causing the autoimmunity, while selectively sparing the stem cells in the bone marrow. Investigators at Hopkins discovered that stem cells uniquely have high levels of a particular protective enzyme that can be measured in advance of therapy, which makes them impervious to Revimmune, and allows the surviving stem cells to give rise to the new immune system over 2 to 3 weeks. The newly reconstituted peripheral immune system typically lacks the misdirected immunity to self-antigens, which is characteristic of autoimmune diseases.

Studies at Johns Hopkins University School of Medicine by Dr. Jones, Dr. Brodsky, and colleagues have demonstrated the potential benefits of Revimmune in a variety of autoimmune diseases.

According to information from the National Multiple Sclerosis Society, there are approximately 400,000 people in the US with Multiple Sclerosis. For the clinical course, 85% of patients are in the category of relapsing-remitting. Based upon a paper by D. Hirtz et al.(1), "How common are the 'common' neurologic disorders?," the annual incidence of Multiple Sclerosis in the U.S. was approximately 4.2 new cases per 100,000 population in 2005.
Revimmune Therapy for Multiple Sclerosis:

Revimmune treatment of 20 Multiple Sclerosis patients has resulted in the following successful outcomes in 2 published studies from C. Krishnan, D. Kerr et al.(2) and D. Gladstone et al.(3):

-- All patients have had a reduction or elimination of new and enhancing lesions on the MRI

-- No patient has had a clinical exacerbation following treatment and most patients have had a reduction in EDSS and an improvement in the MSFC following treatment

-- During follow-up, no patients increased their baseline EDSS scores by more than 1.0

-- No patient had a new lesion on brain magnetic resonance imaging; no patient showed any enhancing lesions

Systemic Lupus
Investigators have treated 40 severe systemic lupus erythematosus (SLE) patients in clinical studies with Revimmune. A significant improvement in the SLE diseases activity index was observed. There were 5 durable complete responses. Among severe, refractory cases, approximately 80% of patients treated had a complete or partial response when treated.
Myasthenia Gravis:

Using Revimmune, investigators have treated 11 patients with myasthenia gravis refractory to conventional immunosuppressive therapy. Nine of the subjects in the study markedly improved, and have returned to full activity.

Aplastic Anemia
Acquired severe aplastic anemia (SAA) is a severe, life-threatening autoimmune disease wherein a patients' immune system mistakenly attacks their own stem cells in their bone marrow. Most SAA patients will die within a year of diagnosis. Investigators have treated 75 SAA patients with Revimmune and the majority of patients have achieved a complete remission without the need of other immunosuppressive agents.

Autoimmune Hemolytic Anemia
Investigators have treated 10 patients with refractory autoimmune hemolytic anemia. After Revimmune treatment, all patients responded and became transfusion independent. There were 6 patients that achieved complete remission and 3 patients that achieved partial remission. There were no relapses at a median follow-up of 15 months and 7 of the 9 patients were able to discontinue steroids.

Experience with other autoimmune diseases
Investigators have reported favorable case experience with refractory scleroderma, acquired pemphigus, rheumatoid arthritis, and Crohn's Disease.

References

(1)"How common are the 'common' neurologic disorders?;" D. Hirtz, D. J. Thurman, K. Gwinn-Hardy, M. Mohamed, A. R. Chaudhuri, and R. Zalutsky; Neurology 2007 68: 326-337.

(2)"High-Dose Cyclophosphamide in the Treatment of Aggressive Multiple Sclerosis;" Chitra Krishnan, Daniel Drachman, Justin McArthur, David Irani, Avindra Nath, Carlos Pardo-Villamizar, David Yousem, Robert Brodsky, Peter Calabresi, Douglas A. Kerr, Baltimore, MD. AAN Abstract (P01.072); April 4, 2006.

(3)"High-Dose Cyclophosphamide for Moderate to Severe Refractory Multiple Sclerosis;" Douglas E. Gladstone, MD; Kenneth W. Zamkoff, MD; Lauren Krupp, MD; Robert Peyster, MD; Patrick Sibony, MD; Christopher Christodoulou, PhD; Emily Locher, RN; Patricia K. Coyle, MD Arch Neurol/Vol 63, Published Online August 14, 2006.

SOURCE: Accentia BioPharmaceuticals

Revimmune for Autoimmune Disease
PipelineReview.com (press release) - Barcelona,Spain
Accentia's lead indication for Revimmune is multiple sclerosis (MS). Autoimmune diseases afflict more than 8 million people in the US and impact on ...
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