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Creating the Medicine of the Future: Hadassah’s Goldyne Savad Institute of Gene Therapy

Monday, Dec 9 2013

The Hadassah Medical Organization's Goldyne Savad Institute of Gene Therapy is targeting its research to optimize the chances of fighting a wide range of cancers and neurodegenerative diseases with gene and cell therapy.

"The Institute has chosen to take the lead in this new arena of medicine," relates its director, Prof. Eithan Galun. "Our mission," he says, "is to translate the fruits of laboratory research into therapies that will directly benefit patients."

Established in 1999, The Goldyne Savad Institute houses the National Good Manufacturing Practice (GMP) Center for the production of biological drugs, as well as a clinical site where these new drugs can be tested as part of Phase I and II investigations.

The Institute currently has 13 research groups with 90 investigators, whose work helps physicians treat patients suffering from diseases as varied as heart failure, cancer, and macular degeneration. These treatments include the use of viral and non-viral gene delivery systems, as well as stem cell therapy.

For example, the Institute has just completed a Phase I clinical study using gene therapy to treat locally advanced pancreatic cancer in patients whose cancer is inoperable. The unprecedented results reveal that none of the patients' cancer progressed, coupled with a significant increase in overall survival. The Institute is now moving to a Phase II clinical trial.

In another study, stem cells are being generated on an ongoing basis at the Institute's GMP facility to treat patients battling amyotrophic lateral sclerosis (ALS).

Benefiting the field of cardiology, the Institute has developed a protein therapy for acute myocardial infarction, which has proven, in unprecedented pre-clinical findings, to reduce heart failure significantly. "We hope to translate this new concept and innovative treatment from research into clinical use," Prof. Galun reports.

Many current research projects are focused on eradicating blindness and cancer, using noninvasive procedures.

Age-related macular degeneration (AMD). This degenerative disease compromises the neuronal tissue of the retina. AMD affects approximately 30% of people over the age of 75. While current treatment protocols involve multiple administrations of intraocular injections that carry the risk of severe side effects, at the Goldyne Savad Institute, patients are being treated with infrared ultrafast femtosecond laser pulses (LGBT). Results are encouraging, Prof. Galun reports, and could "radically change the way retinal diseases are treated." This approach, he adds, delivers the treatment specifically to the site of retinal pathology, while avoiding unaffected areas--and at a low cost.

In addition, plans are underway to initiate a human embryonic stem cell (hESC) Phase I clinical trial for the treatment of AMD. Stem cells will be implanted into the retina of patients with AMD. Studies with animal models at Hadassah have already shown that stem cells can reverse the clinical condition and give animals with AMD their sight back.

Neuroblastoma. The most common extra-cranial solid cancer affecting children, it accounts for approximately 15% of cancer-related deaths in infancy. About one half of these children are found to have metastasis upon diagnosis. Despite aggressive treatment, these children with metastatic disease have a poor prognosis, with only a 20% survival rate 5 years following diagnosis. Dr. Rinat Abramovitch, one of the Institute's senior investigators, has been working with a novel drug combination in a mouse model aimed at combating this deadly cancer.

Hepatocellular carcinoma (HCC). This is one of the most prevalent and fatal neoplasms (abnormal masses) worldwide. The majority of HCC cases are preceded by chronic inflammation; however, the underlying molecular and cellular mechanisms connecting inflammation with HCC development are not well defined. The Savad Institute's work is focused on studying the function of the protein, galectin-1, in a mouse model at different stages of inflammation-associated hepatocarcigenesis. The results of this study, says Prof. Galun, should contribute toward identifying new potential therapies for liver cancer.

Hepatitis C infection (HCV). This infection is a major risk factor for those with hepatocellular carcinoma (HCC). The Institute is investigating inflammation-associated HCC in mice and has demonstrated that in the model, the Hepatitis C transgene promoted the development of this carcinoma. The Institute also aims to uncover the diagnostic and functional significance of certain DNA changes that take place at pre-cancerous stages of chronic liver inflammatory diseases and culminate in cancer.

Cervical carcinoma (CC). This cancer is associated with the human papilloma virus (HPV) in over 99% of cases. Currently, there is no available therapy to prevent CC development in HPV carriers. The Institute's investigation is focused on tumor suppression and its relation to two proteins, E6 and E67. The aim is to halt the progression from pre-cancerous neoplasm to invasive CC.

Radiation-induced xerostomia (RIX). The Institute has developed the hyper human interleukin-6 (HIL6), a protein that prevents RIX or the non-functioning of the salivary glands, following head and neck radiation for the treatment of cancer. At this time, there is no preventive therapy for this clinical condition, which can prove devastating to patients; it affects their quality of life, including dietary habits, speech, taste, and susceptibility to dental caries. The Institute's investigations, relates Prof. Galun, not only suggest that HIL6 has a therapeutic benefit, but also show how this is actually happening. The goal of further study, he adds, is to determine the mechanism by which HIL6 prevents RIX and the role of the salivary stem cells in this process.

The Institute has also developed a protein-based therapy for the prevention of hair loss following radiation, a leading cause of psychological distress. Its researchers expect it to prevent hair loss from chemotherapy as well as radiotherapy in patients undergoing cancer treatments.

Tissue regeneration. In cooperation with other investigators, Institute researchers are studying the mechanisms of tissue and organ regeneration with an eye toward developing new treatment approaches. They have been able to show that the designer protein, hyper IL-6 (HIL6) has significant regenerative effects in the liver and the kidney, as well as the salivary gland (as discussed above).

"During the last year, however," Prof. Galun comments, "we have generated, to our opinion, a breakthrough in this approach of using HIL6. We found that it has therapeutic effects in myocardial infarction. We were able to show that the administration of HIL6 could significantly reduce the infarct size, and prevent the deterioration of the heart function. This means we could reduce congestive heart failure--the most severe result of myocardial infarction. We are also now investigating the mechanism of this effect and have collected interesting preliminary results."

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