Researchers Debate The Utility Of Gene Therapy In Curing HIV (IAS 2011)
Is gene therapy too expensive and impractical to form the basis for a cure for HIV, or is it a promising technique with a proven track record that has the specificity required for a cure?
This question was debated by researchers at the 6th International AIDS Society Conference on HIV Pathogenesis, Treatment, and Prevention (IAS 2011) in Rome last month.
Gene therapy is an experimental approach that is currently in early stages of clinical testing. Gene therapy involves modifying a person’s DNA (the genetic information in cells) so that it becomes, for example, resistant to HIV infection (see related AIDS Beacon news).
In most cases of gene therapy, cells are taken from the patient’s body, genetically modified in the laboratory, and then injected back into the patient.
Dr. Sharon Lewin, director of the Infectious Disease Unit at Alfred Hospital in Melbourne, Australia, argued in a session titled “Controversies in HIV Cure Research” that gene therapy is not the strategy most likely to lead to a cure for HIV due to its impracticality.
“Gene therapy is scientifically flawed, high risk, and will never get to the clinic,” said Dr. Lewin in her presentation.
Dr. Lewin argued that cure strategies based on drugs that activate latent HIV are far more promising. Latent HIV is virus that lies dormant in cells during antiretroviral therapy but activates again once treatment is stopped to renew the infection.
A number of clinical trials are currently underway to test whether certain drugs can reactivate latent HIV so that it can be eliminated from the body’s cells. According to Dr. Lewin, these treatments have an advantage in that they are available now for testing, are less expensive and more general than gene therapy (which involves intensive individualized treatment for each person), and usually have side effects that are mild and reversible. This is not necessarily the case with gene therapy, which attempts to create permanent changes in a person’s genes.
In addition, she pointed out that there are at least 22 different potential drugs for targeting latent HIV that are in Phase 1, 2, or 3 clinical trials, several of which are already approved by the U.S. Food and Drug Administration for other uses.
Although she acknowledged that drugs, in general, are not as targeted as gene therapy, she also noted that researchers are working on ways to make the drugs target latent HIV more specifically, rather than affecting all the cells in the body.
Overall, Dr. Lewin criticized the practicality of gene therapy as a cure for HIV, particularly in developing countries. “We need a cure that is scalable, deliverable, and cheap,” she concluded.
Dr. Keith Jerome, from the Fred Hutchinson Cancer Research Center in Seattle, disagreed with Dr. Lewin and argued that gene therapy is indeed the correct approach for curing HIV.
“The principle has been proven in the only HIV cure to date,” said Dr. Jerome, in reference to “The Berlin Patient,” a man who received a bone marrow transplant from a carefully selected donor with a mutated form of the CCR5 gene.
HIV requires the CCR5 protein, which is on the surface of white blood cells, in order to attach to and infect the cell. People naturally born with an alternate form of the CCR5 gene are almost entirely immune to HIV. Since his transplant, no sign of HIV has been detected in The Berlin Patient. Currently, the most advanced gene therapy strategies are attempts to replicate this success in other people with HIV.
Dr. Jerome argued that HIV is a genetic disease and therefore needs genetic treatment strategies to cure it. HIV works by incorporating itself into a cell’s DNA and then using the cell’s normal processes to replicate. Current treatments cannot remove the HIV once it has been incorporated, and Dr. Jerome argued that drugs that target DNA are not specific enough and are likely to have significant effects on other cell processes.
Instead, he said, treatments need to target and change specific genes, which means gene therapy is necessary.
Dr. Jerome did admit that more work needs to be done before gene therapy is a viable option for treatment. Researchers need to work more on developing therapies that are specific, have higher success rates in modifying genes, and do not require toxic preparative treatments before therapy, he said.
However, he noted that scientists have been making significant breakthroughs in the field and that researchers now have the necessary tools to make gene therapy work.
“Gene therapy is the path to a cure,” Dr. Jerome concluded.
For more information, please see Dr. Lewin’s and Dr. Jerome’s presentations (pptx) at the IAS 2011 conference website.
Related Articles:
- Sangamo BioSciences Initiates Gene Therapy Trials Toward A Cure For HIV
- The Possible Future Of HIV Treatment – Part 2: Research Toward A Cure (CROI 2011)
- Advances and Barriers To A Cure For HIV: Part 3 – Gene Therapy And Therapeutic Vaccines
- Results From HIV Gene Therapy Trial Show Long-Term Positive Effects (CROI 2012)
- An Interview With AmfAR’s Dr. Jeffrey Laurence: Part 2 – Working Toward A Cure
Why is it a question of ‘either/or’? I think all possible treatments should be thoroughly examined!
I don’t understand why they are arguing about how to end an epidemic, the only known person to date was cured using stem cell, ok thats not widely practical but gene therapy is so if it works then they should be making every means possible to get this cure out to the massis, one mans cure should be shared with the world!
gene therapy must b seen as a cure till the actual cure is found out.its should b used to cure so that most of the hiv persons may save there lifes.is this gene therapy used in India.
if the gene theraphy is cure for hiv is any proof for cured peron
we congratulate our industrious researchers,but then arguement will not bring cure.just do your best for the world.God bless.
i know with understanding and time , the final cure will come out.but argument will not solve the prob. in fact u are doing a great job by try to find pure cure of HIV. but it must be fast, GOD BLESS.thxx alot Dr
with this argument people are losing hope for feature please focusing on a cure and leave treatmant out this
question?
Where are the statistics that HAART medication protected people infected with HIV,who did not have AIDS from getting AIDS?
It make me so angry for the pass 9 months that i found out from CNN breaking news has a good new that Dr has found a cure this guy and i am so happy this guy is cure and move on his life, and now for the pass 9 months i have heard nothing talking everything has kept quiet and no one talk about HIV/AIDS cured and now i am so tired to read that there is no cured for HIV/AIDS and all those peoples were talking blah and blah on and on about there is no cured and too risky and i am so proud Dr Gene has found and he should try for other peoples to see if it work for them but they did only one guy tim ray brown 4 years ago he has no HIV virus in his body so why they are wasting time not to cured other to give a try, i think they are playing behined my back and i want a cured just like other peoples need cured too it our life and give a try to see if it work for other and i am getting tired they talk too much and i think they are jealous of DR Gene and i am not i am happy for Dr Gene god bless you and keep up a good work to help other peoples!
Gene therapy and Medicinal cure both have a point.
In my opinion, I believe that Gene Therapy is somehow expensive, and a well, risky. But currently, the Gene modification was the only process that cured an HIV individual, whether this is an accident or not, a result is a result. If scientists make Gene Therapy a sure way for the cure, with reduced risk, even if it needs personalized treatments per person, this will greatly help. And even if Gene therapy is expensive, I think that the health sector, will make it cheaper to afford this. We should forget profits in this sickness. Let’s remember that people are struggling and are in turmoil because of aids, and I believe that there is goodness in all people, that they will help in the process.
Medicinal cure, in my opinion is easier to spread, and cheaper. I think that activating latent HIV-infected cells while on ARV will aid in the eradication of HIV.
Whether Gene Therapy or Medicinal Cure, I think they should exert more effort in these both rather than PrEP. Taking Meds daily, will increase risky and promiscuous unsafe sex thinking that they wont be infected since they are on PrEP. A real vaccine booster would be more practical, whether there’s a vaccine or not, it is more important to find the cure.
A vaccine, is made to avoid more infections. But let’s admit it, not everyone will have that vaccine. and the number of HIV+ increases everyday. A cure is much needed now to treat HIV+ people who are struggling to live, lived a long time, newly infected, and even children who have no idea of HIV.
I have just been recently diagnosed with HIV, for a few months now, but I am still hoping that in a few years time, a cure will be made. This is one reason that keeps me from living, a reason to wait and be cured.
It’s so disappointing that these people are putting a “block” already and an obstacle on whether or Gene therapy is the path to the cure. Rather than focus on the research itself before they give up and find this negative before they even start.
This are scientists, Just take ALL the option including Gene therapy and drugs that target the reservoirs make them all possible .
They are arguing, before the cure is out. Let’s do not lose time in this.
FOCUS on the possibilities, rather than the obstacles.
I found out that i have HIV last year and it’s been a real tough thing to live with, I would give anything to be free of this illness I don’t know the future but I have a good feeling that a cure is coming. I went to my doctor the other day and she said at least I didn’t get this 15 years ago and she said that something was about to happen, she said all this after I asked her had she heard about the new gene therapy experiment that was going on right now, I believe everything happens for a reason I don’t know why I have this but I do know God does things for a reason so we all have to just pray that this miricale will come to us all and we will get a second chance to live life happy and healthy again. God Bless everybody with Hiv that we all will be free of this disease one day soon.
I had just diagnosed with HIV yesterday. It was the darkest day in my life and i am still young, 28. I havent achieved my dreams in my life and i havent fulfilled my duty as a son to bring happiness to my parents. I do hope a cure will be practically introduced to the world within the next 5 years. A lot of researchers mentioend that a cure is not going to be in the near future but we must work hard to make it happen.
Wish God bless everyone of us and i prophetsize in the name of Jesus, a cure will be materialized wihtin the next 5 years. I pray in the mighty name of Jesus. Amen.
Let’s continue to pray, NON STOP. God is listening to our prayer.
@Winston I know how you feel. I have just been diagnosed in a foreign country for employment purpose last June. And now it seems I cant go back to that country EVER. I was just 21 during that time(but now I’m 22) I have a lot of good plans to do, I have given up a lot of my career. I was scared that I have infected somebody else, I’m still praying that I dont.
I am hoping for a cure soon. Yeah, maybe 5 years. Hopefully vaccines going on their Phase III trials will be succesful. Let’s all pray for that.
Hopefully SEEK’s HIV-v vaccine will be indeed prove successul for their Phase III trial next year. Or Bionor’s HIV Vacc-x4 and Vacc-c5 will all go well. As well as the Gene Therapy, hopefully all these will be successful and be available to the public.
I think that PrEPs are, Yes, quite good to prevent HIV transmission(But condoms are the penultimate barrier to infection) But in reality, not everyone can afford PrEPs, and as long as we don’t focus to find the cure, more and more people will suffer and die.
Maybe ARVs can extend lives, but to what extent? Prolong lives with discrimination? Prohibited from entering or even working in other country? Thus, I think it will just (I know you will negate this) prolong the suffering of PLHIV.
Let’s all pray that all people with or without HIV, rich or poor, will have the passion to cure one of the world’s biggest crisis, HIV.
(And let’s hope NO ONE will interfere or block the route to HIV Cure)
*Businessmen, Pharmaceuticals, I hope you won’t get greedy to stop making HIV cure a possibility. You have earned so much already!
Hi Johann, yes indeed. One of my dreams is to work at other country as well to experience different things. Vaccine is not enough, vaccine is just for those who are negative currently but it will not cure the positive. We need a cure indeed even a functional cure in the near future let say within next 2 to 3 years will be good enough.
We pray to God, God please help us. You are the only one we can rely on now. We need your help to give us wisdom how to come out with a cure for this disease. Please dont leave us alone God. We pray in the mighty name of Jesus. Amen.
Hi Johann, my email is kinkboy_pj@hotmail.my. We can be fren?
Dear all,
Let’s pray hard, a cure will be introduced to the public within next 5 years.
We pray in the mighty name of Jesus, Amen.
I discovered on the 5th of this month that I am HIV+ and I am only 23 years old.. I was supposed to celebrate my 24th anniversary in 2 weeks. And here I am sad, scared and disoriented … all my dreams are shattered… I’ve dissapointed my mother, my father and all my friends… I gave up all hope but I didn’t give up believing in God, that He will give us the wisdom to find a cure for hiv.
Let us all pray to God , so that the humankind will soon be able to discover a cure for this terrible disease…
Diagnosed just over two years ago, it was tough going for the first year, and now I feel on top of the world again. It’s not the end of the world. Gotta be strong and positive in life. Get your sleep and don’t forget medicine, otherwise, trouble. Just abstain from alcohol and that will give you a leading edge on life. I was never a drinker to begin with and the Doc said that will stand for me. Had a cd4 of 45. Yes, 45. Now holding a steady 350 and just under 30pc. Undetected ever since.
Hi Nigel,
The time when you were diagnosed, your CD4 count was only 45?
Hi Winston,
Yes, that is correct, 45. The docs are quite surprised with the massive improvement, they plan on giving me 6 months of meds as opposed to 4 months on my next visit, in October, which means I only have to get checked out twice a year. Currently on Atripla meds. I take one tablet just before bedtime. Only drawback is if I eat too late in the evng, I will experience rather unpleasant “dreams” (nightmares)> Now, I have fine tuned that to stop eating at least four hours before I take the tablet.
Rgds and stay strong, just remember, NO alcohol and regular sleep. Trust me…
hi Nigel,
thank you very much on your advice and courage. i had been diagnosed few days ago via a family doctor. i will get my report from him and consult a specialist in hospital.
Let’s dont stop our prayer, continue to pray until God allows the cure is available.
God bless every one. Amen
Hi Winston
If it happened over 25 yrs ago, I would not be here today. Big improvements and people are now LIVING. However, one must ADHERE to the tablets regime. Otherwise, it spells DISASTER. My memory was never great, having said that I have NOT missed one tablet yet. That’s the secret, otherwise, it jeopardizes your future medicines. Try not get stressed out and try and be open. I try not bottle it up, one day I arrived at work and basically told them “I’m living with a bitch called hiv”. They have a better understanding now and I’m coping much, much better. However, I did NOT tell any family member – yet. EIRE…
Hi Nigel.
Do you mind to send me an email so that we can chat more? kinkboy_pj@hotmail.my
Hi W
Living in rural Republic of ÉIRE.
Have you got a g-mail add?
Tried your email but unable to send.
Good evng from ÉIRE…
do u have msn? add me by the same address too. nop, i dont have a gmail account.
hi Nigel, i created one, sheckan@gmail.com
Hey everyone I am in the window period 12 weeks, Im afraid I have been infected with HIV. I had a rapid ELISA test at 25 days and it came back a definate negative. I have read alot of literature on the advancing steps in HIV cure/ treatment / vaccine. Im living here in Canada, i feel like my life has been completely destroyed I spend hours crying a day. I can not function I cant concentrate. Ive spent over 150 hours researching HIV medical scrpits, trails, and articles. The berlin patient story is one of hope. Mind you the doctors have known for a while of the immunity to infection that the “delta32″ holds. A “chemo-stem transplant” cure as a fuctional cure for one man, but other complications would make it unlikely to work for others. Not to mention that the largest population of suffers in the less devolped of countries could not handle this type of procedure, not to mention the price tag of $100,000 US each. Id like to see some more research put into treating a patients own blood with a zinc based treatment to remove the CCR5 protien thus attempting to turn the HIV+ blood into a delta32 type scenario. Recently some discoveries have been made that the outter membrane of the HIV cell is constructed of a cholestrol. After disrupting the cholestrol the HIV cell no longer can destroy the T cells, allowing for the immune system to deal with in a natural way. After comparing other illnesses with HIV and this particular cholestrol envolpe. Ive compilled literature to show that a certain other “child like alziemers” illness consists of an abnormal amount of cholestrol being stored in brain tissue causing serious complication. The starch based enzyme reactant beta-cyclodextrin can be used to damage the outter membrane of the HIV cell. HIV has been cured in a petri-dish many times, a cure is out there, the problem is finding one that can be used in every setting. Rich or poor, advanced hospital or simple medical set up in africa, and to be affordable to be made avaliable to everyone on this planet. Beta-cyclodextrin is a non complex mutli sugar molecule, its used in farming, pharm. and believe it or not it is even found in “febreeze” the fragrant spray. I pray that a group of doctors will use this information that is so easily avaliable to them to help all who are dealing with this chronic illness.
God bless and grant us all health.
i found it disgusting what “dr. Lewin” said during the reading of this article and was astonished that alot of the “facts” attributed to her were wrong. THERE ARE CURRENTLY NOOO DRUGS IN PHASE 2 OR 3 TRIALS TO ELIMINATE LATENT VIRUS. all tests with these drugs are in lab dishes or in animals. THE ONLY HIV TEST IN HUMAN TRIALS AND PHASE 2 TRIALS IS SANGAMOS RESEARCH—WHICH IS GENE THERAPY…. how can she be so ignorant to argue 1) the wrong facts. 2)that gene therapy wont work when ONE MAN OUT OF 60 MILLION PEOPLE WAS CURED USING GENE THERAPY. 3) even mention such a disgusting issue as cost. if we find a cure in gene therapy then this is a disgusting, horrid world we live in where we say, no we won’t use it because of money issues. i found this entire article to be untrue, distasteful, and abhorrent.
Jay, I think the issue is that the people who have the money are not necessarily the people with HIV, so they could not possibly know what it’s like to live with it. If there are two treatment and one of them is more expensive than the other (gene therapy according to this article, I admit I do not have much of a background in this topic), then there is no reason why it should be pursued if the other way is equally promising but not as expensive. And I understand that you thin that the issue of cost is disgusting, but unfortunately there’s not much you can do unless you have a fortune that you’re willing to donate to gene therapy research as a big “screw you” to all those who shot it down
I think that gene therapy is a lofty goal and should be pursued while the drugs are being improved and giving HIV-ers a precious life line. Dr. Louis Mansky’s lab published a paper regarding the lethal mutagenesis (eradication) of HIV using azacytidine (Vidaza), a known cancer drug. If we’re so gun-ho on curing HIV, why isn’t there a clinical trial for this drug?
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