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PostPosted: Thu Mar 29, 2007 10:41 pm 
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President, Gordon Research Institute

Medscape Critical Care Expert Interview

The Future of Gene Therapy: Interview With David M. Bodine, IV, PhD
Posted 07/11/2005

Editor's Note:
David M. Bodine, IV, PhD, is a Senior Investigator at the National Human Genome Research Institute and a member of the American Society of Gene Therapy Advisory Board. He spoke with Melinda Tanzola, PhD, on behalf of Medscape, about the organization's recent meeting June 1-5, 2005, in St. Louis, Missouri, and some new developments in the field of gene therapy.

Dr. Tanzola: What is the American Society of Gene Therapy (ASGT) and what is its mission?

Dr. Bodine: ASGT is an organization that was founded to bring together a lot of different people who are interested in gene transfer and ultimately gene therapy. I don't think there is one field of gene therapy. Rather, it is an arm of many different fields. Through the society, many different specialists, including biochemists, neurobiologists, oncologists, virologists, and other professionals are brought together.The mission of ASGT is to bring these diverse groups of people together to exchange information -- to help us understand some of the difficult problems and appreciate the successes. ASGT also promotes education to medical professionals and to the public.

Dr. Tanzola: What did you consider were some of the most promising developments presented at the recent ASGT meeting?

Dr. Bodine: By far, the most encouraging results I saw were from the SCID patients with adenosine deaminase (ADA) deficiency. The work is headed up by a group in Milan, Italy, although the children are from all over the world. These children have an ADA deficiency that results in a complete lack of lymphocytes. In the study, their bone marrow stem cells were harvested and treated with a retroviral vector containing the missing ADA gene. After the patients were treated to reduce their endogenous marrow, they then received a bone marrow transplant with their own marrow that had been corrected by gene therapy.[1]When these children came back for follow-up, all 6 patients were doing very well. They had a lot of cells making ADA, and they were able to make lymphocytes. In fact, their entire immune systems had been reconstituted so that, by the numbers, they were indistinguishable from healthy children. Most patients did not even require enzyme supplementation because the blood-forming cells were making enough ADA to detoxify the rest of the cells. Twenty years ago, people predicted that ADA would be the first condition cured with gene therapy, and I think they were right.

Dr. Tanzola: Using hematopoietic stem cells to deliver gene therapy could be a powerful therapeutic avenue for many diseases. However, there have been challenges in the past, such as in the XSCID trial. Could you give us some insight into this issue?

Dr. Bodine: As opposed to the SCID caused by ADA deficiency, X- linked SCID involves a signaling molecule, gamma-c. The adverse events have definitely been a shock, because things at one point were going so well. However, it is important to note that the group presented all 13 patients that they have attempted to treat. After starting with 13 children, in 1 patient, the graft didn't take, and in another, the gene transfer was very poor. Of the 11 remaining patients, 3 had adverse events. But 2 of these 3 patients responded immediately to therapy and now have a fully functional immune system. The third patient unfortunately did not respond to therapy. So whether the denominator is 11 or 13, we now have 10 children who are alive and well. I think if left untreated, or if given the conventional treatments, this number would not be 10. If you compare these results to the early days of bone marrow transplants and other treatments, including some chemotherapies, this is a success.The adverse event is caused by the therapy, so we do need to work on safety. However, I was impressed that the treatment worked as uniformly and completely as it did, in spite of the adverse events. I think we have to be careful about the way we make the vector and how much gamma-c protein is made. Having less protein may help a lot. However, the point is that a lot more good than bad came out of that study.

Dr. Tanzola: Speaking of safety, what progress is being made to understand and enhance the safety of gene therapy?

Dr. Bodine: I was very impressed at this year's meeting by the number of different approaches taken to compare the safety of the different vector systems for delivering gene therapy. Of all the studies presented, the animal model of X-SCID leukemia was most directly applicable. Investigators used X-SCID mice that didn't make gamma-c protein. They then also gave it the "first hit" for cancer by adding a cell cycle regulatory protein. That way, they started to see higher frequencies of tumors. Then they asked whether you would see even higher frequencies of cancer after you give back gamma-c in a gene therapy bone marrow transplant.[2]What they found was that leukemia occurred at one frequency in mice without the cell cycle protein, at a higher frequency with the X-SCID mice, and at an even higher frequency when gamma-c was replaced. What this established for me was that the adverse events were not only related to the therapy but also to the disease and to the biology of the disease.

Dr. Tanzola: Were there any particularly relevant primate studies looking into the X-SCID leukemia issue?

Dr. Bodine: There was one study in which primates were given gene therapy vector carrying only a marker to track the cells with vector. Of approximately 100 animals treated, one developed leukemia. While one animal may seem insignificant, keep in mind this is from a pool of animals treated the same way, or more aggressively, than humans, and the frequency was only 1 in 100. The animal did have a cancer similar to a rare human leukemia, and investigators were able to clone out insertion sites that might have been the first hit.Usually 2 hits are needed, and in this case, the second hit was not clear. What we need to do now is determine the mechanism, even in this one animal. It would be nice to get cell lines from the animal or see if knocking genes back down will reverse it. Overall, I was impressed with the denominator -- obviously these researchers were looking hard for the event and only found it in 1 of 100 animals.[3]

Dr. Tanzola: There were several interesting studies presented at the meeting that used RNA components for gene therapy. In one study investigating muscular dystrophy, researchers were able to promote the "skipping" of the mutated part of the gene. How does this technology work?

Dr. Bodine: Muscular dystrophy is a huge gene -- there is no way we could put this gene into a cell. Some patients have severe muscular dystrophy, either because they have a complete deletion of the gene, or because they have a stop codon truncating the gene. In this second set of patients, if we could just suppress the stop codon, the cell might then make normal protein.This gene therapy technique takes advantage of the fact that DNA has multiple coding sequences interspersed with noncoding introns that are spliced out during RNA processing. The gene therapy vector delivers a specially engineered gene, U7, which is transcribed into an RNA molecule that matches perfectly with the sequences flanking the muscular dystrophy DNA segment that contains a stop codon. This process then loops out the bad sequence that contains the stop codon and continues on to make a perfect protein.This work looks terrific in animals -- the mice that have been treated are walking around a lot more than the untreated ones. The question, though, is that humans have thousands of times more muscle cells compared with the number of muscles in a mouse. Just think of how big our large muscles are compared with a mouse. However, if this therapy could be injected locally into the diaphragm and the hands of patients with muscular dystrophy, this could make a huge difference for them.[4]

Dr. Tanzola: A second RNA technique of note is RNA interference, which was used as a potential treatment for Huntington's disease. What do you think are the implications of this work, and how do you see this technology fitting in to the future of gene therapy?

Dr. Bodine: This is a new technology that a lot of people in the society are getting excited about. RNA interference is a natural way that cells regulate levels of RNAs, and we are just harnessing it. It involves generating a double-stranded RNA. You can easily do this by making a virus vector or a DNA construct that makes a double- stranded RNA. Enzymes in the cell then cut the RNA into small pieces and release these molecules that are 21 bases long. If those short RNA molecules find a matching RNA, they can bind to it. Sometimes they target it for destruction and sometimes they just interfere with protein production. Either way, in presence of these small inhibitory RNAs, protein synthesis goes down.With Huntington's disease, you're translating an RNA that has multiple copies of glutamate. Once you get past 20 copies of this repeat, you begin to have Huntington's disease. The goal of this treatment, therefore, is to slow down the making of this polyglutamate protein. Investigators put this construct encoding the glutamate repeat into the brains of Huntington's disease mice. They showed that they could knock down the Huntington protein with the polyglutamate, and they saw a nice effect on extending the life of the animal.The interesting thing about Huntington's disease is that it is a dominant gene. It is more tricky than the ADA deficiency where you only need to add the gene back in to correct the condition. In this case, the dominant gene product is not good for you, so you have to find a way to take it away. This is a novel use of RNA interference to take away the bad Huntington protein.[5-7]

Dr. Tanzola: Finally, what advances in gene therapy would you predict are on the horizon?

Dr. Bodine: What I think is going to happen in gene therapy is that these new tools, such as genomics, RNA interference, chromosomal insulators, and others that are just starting out as techniques, will end up giving us a whole new tool box for treating these diseases. We need to keep people coming to these meetings and exchanging their ideas. The proof-of-principle for lysosomal storage diseases and Huntington's disease is there. The question is how we can crank it up to the next level.The last 10 years have been the first tries with gene therapy in the clinic, and, just like the first tries with any new therapy, we have learned a lot, but not that many people got better. Now we're going back and really studying these things and learning what the problems were. In the second stage, which may come at around 2010, we will see a lot of clinical trials, and I bet we'll do a lot better.I was very upbeat about the attitude of the field. They've taken on the safety issue, and more complex problems of scaling up the therapies, as a real mission. Instead of rushing to be the first to the clinic, investigators are really focusing on doing the best job of developing and understanding these therapies.

Dr. Tanzola: Thank you very much for sharing your thoughts with us.

Dr. Bodine: You are quite welcome.

1. Santoni de Sio F, Naldini L. Lentiviral gene transfer into HSC is enhanced by early-acting cytokines without impairing stem cell properties and involves cellular responses distinct from cell cycle control. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.
2. Uchiyama T, Kumaki S, Onodera M, et al. Application of a suicide gene to X-SCID gene therapy. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.
3. Brenner S, Ryser M, Choi U, et al. Polyclonal insertion sites in two rhesus macaques after non-myeloablative transplantation with MFGS-gp91phox transduced autologous CD34+PBPC. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.
4. Goyenvalle A, Vulin A, Kaplan JC, Leturcq F, Danos O, Garcia L. Highly efficient exon-skipping and sustained correction of muscular dystrophy using an adeno-associated viral vector. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.
5. Raoul C, Aebischer P. Lentiviral-mediated silencing of SOD1 through RNA interference delays disease onset and progression in a mouse model of ALS. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.
6. Harper SQ, Staber PD, He X, et al. AAV-delivered RNAi improves cellular and motor phenotypes in a mouse model for Huntingtons disease. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.
7. Rodriguez E, Denovan-Wright E, Lewin AS, Mandel RJ. Demonstration of in vivo off-targeting using rAAV-delivered ribozymes and shRNA molecules in a mouse model of Huntingtons disease. Program and abstracts of the American Society of Gene Therapy 2005 Annual Meeting; June 1-5, 2005; St. Louis, Missouri.

David M. Bodine, IV, PhD, Senior Investigator, National Human Genome
Research Institute, Bethesda, Maryland
Melinda Tanzola, PhD, medical writer, Atlanta, Georgia

PostPosted: Sun May 17, 2015 5:16 pm 

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The Gut Health Kit is a program to cleanse, balance, and support your digestive system by combining four of our top products and a healthy diet.

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