Gene therapy offers hope for future AIDS cure

[Aurora Moon]

In a bold new approach ultimately aimed at trying to cure AIDS, scientists used genetic engineering in six patients to develop blood cells that are resistant to HIV, the virus that causes the disease.

It’s far too early to know whether this scientific first will prove to be a cure or even a new treatment. The research was meant to show only that, so far, it seems feasible and safe.

The concept was based on the astonishing case of an AIDS patient who seems to be cured after getting blood cells from a donor with natural immunity to HIV nearly four years ago in Berlin. Researchers are seeking a more practical way to achieve similar immunity using patients’ own blood cells.

The results announced Monday at a conference in Boston left experts cautiously excited.

“For the first time, people are beginning to think about a cure” as a real possibility, said Dr. John Zaia, head of the government panel that oversees gene therapy experiments. Even if the new approach doesn’t get rid of HIV completely, it may repair patients’ immune systems enough that they can control the virus and not need AIDS medicines — “what is called a functional cure,” he said.

Carl Dieffenbach, AIDS chief at the National Institute of Allergy and Infectious Diseases, agreed.

“We’re hopeful that this is sufficient to give the level of immune reconstitution similar to what was seen with the patient from Germany,” he said.

This is the first time researchers have deleted a human gene permanently and infused the altered cells back into patients. Other gene therapy attempts tried to add a gene or muffle the activity of one and have not worked against HIV.

The virus can damage the immune system for years before people develop symptoms and are said to have AIDS — acquired immune deficiency syndrome. The virus targets special immune system soldiers called T-cells. It usually enters these cells through a protein receptor, or “docking station,” called CCR5.

Some people (about 1 percent of whites, fewer of minorities) lack both copies of the CCR5 gene and are naturally resistant to HIV. One such person donated blood stem cells in 2007 to an American man living in Berlin who had leukemia and HIV.

The cell transplant appears to have cured both problems, but finding such donors for everyone with HIV is impossible, and transplants are medically risky.

So scientists wondered: Could a patient’s own cells be used to knock out the CCR5 gene and create resistance to HIV?

A California biotechnology company, Sangamo BioSciences Inc., makes a treatment that can cut DNA at precise locations and permanently “edit out” a gene.

Dr. Jacob Lalezari, director of Quest Clinical Research of San Francisco, led the first test of this procedure with the company and colleagues at the University of California in San Francisco and Los Angeles.

He warned that it would be “way overstated” to suggest that the results so far are a possible cure.

“It’s an overreach of the data. There are a lot of people out there with hopes and dreams around the C-word,” so caution is needed.

In the study, six men with HIV had their blood filtered to remove a small percentage of their T-cells. The gene-snipping compound was added in the lab, and about one-fourth of the cells were modified successfully. The cells were mixed with growth factors to make them multiply and then infused back into the patients.

Three men received about 2.5 billion modified cells. Three others received about 5 billion.

Three months later, five men had three times the number of modified cells expected. As much as 6 percent of their total T-cells appear to be the new type — resistant to HIV, Dr. Lalezari said.

The sixth man also had modified cells, but fewer than expected. In all six patients, the anti-HIV cells were thriving nearly a year after infusion, even in tissues that can hide HIV when it can’t be detected in blood.

“The cells are engrafting: They’re staying in the bloodstream; they’re expanding over time,” said Dr. Lalezari, who has no personal financial ties to Sangamo, the study’s sponsor.

The only side effect was two days of flulike symptoms. It will take longer to determine safety, but several AIDS experts said they were encouraged so far.

“It is a huge step” and a first for the field of genetics, said John Rossi, a researcher at City of Hope in Duarte, Calif., where he and Dr. Zaia plan another study to test Sangamo’s approach. “The idea is if you take away cells the virus can infect, you can cure the disease.”

On Wednesday, Dr. Carl June, a gene therapy expert at the University of Pennsylvania, will report partial results from a second, federally funded study of 10 people testing Sangamo’s product. He treated his first patient with it in July 2009.

Many questions remain:

• People born without the CCR5 gene are generally healthy, but will deleting it have unforeseen consequences?

• Will HIV find another way into cells? Certain types of the virus can use a second protein receptor, though this is less common and usually when AIDS is advanced. Sangamo is testing a similar approach aimed at that protein, too.

• How long will the modified cells last? Will more be needed every few years?

• Could doctors just infuse Sangamo’s product rather than removing cells and modifying them in the lab?

• What might this cost?

Sangamo spokeswoman Liz Wolffe said it’s too early in testing to guess, but it would be “a premier-priced” therapy — in the neighborhood of Dendreon Corp.’s new prostate cancer immune therapy, Provenge — $93,000.

Yet AIDS drugs can cost $25,000 a year, so this could still be cost-effective, especially if it’s a cure.

Jay Johnson, 50, who works for Action AIDS, an advocacy and service organization in Philadelphia, had the treatment there in September.

“My results are excellent,” he said. “The overall goal is to not have to take medication and then hopefully lead maybe to a cure.”

Matt Sharp, 54, of suburban San Francisco also had the treatment in September.

“I would trade anything to not have to take a handful of medications every day for the rest of my life and suffer all the consequences and side effects,” he said.

“I may not live long enough to see the cure, but I always hoped for a chance.”

http://www.washingtontimes.com/news/2011...re/?page=1

[Palladin]

This kind of r&d seems to have tons of good prospects. Good on them.

[mv]

This kind of r&d costs a lot of public money and solves a problem that can be solved on the cheap by education and culling.

[Aurora Moon]

even if it could be solved by education and culling as you seem to believe, MV, it's still good to have an ACTUAL cure.

besides, there's been people who weren't overly promiscuous who caught aids though other means... like though transplants, blood transfusions.

even though people are now more careful about checking for that kind of thing, shit like that still happens.

so were it those people's fault somehow that they got aids though blood transfusions or some such thing?

is some little kid with aids somehow deserving of your scorn? It just seems like you're buying into all the myths about people who has aids.

otherwise, you wouldn't be talking about how it can be "cured" simply by culling people and though education.

[Canuknucklehead]

Acronyms

AIDS = Adios Infected Dick Sucker

GAY - Got Aids Yet

[Aurora Moon]

it's funny because it's untrue. and just one of the many myths about AIDs.

Cumulative AIDS Cases - US and Dependent Areas

* Cumulative AIDS Cases - 988,376

* Adult and Adolescent Cases
o Males - 761,723
o Females - 181,802

o Total - 943,525

* AIDS Deaths
o Adults and Adolescents - 525,442
o Children under 13 - 4,865

o Total - 550,394

Please Note - Because totals were calculated independently of sub-populations, sub-population totals may not equal the totals.

Cumulative AIDS Cases by Age

* Under 13 - 9,112
* Ages 13 to 14 - 1,065
* Ages 15 to 19 - 5,289
* Ages 20 to 24 - 34,795
* Ages 25 to 29 - 114,141
* Ages 30 to 34 - 193,926
* Ages 35 to 39 - 208,505
* Ages 40 to 44 - 164,697
* Ages 45 to 49 - 102,732
* Ages 50 to 54 - 56,950
* Ages 55 to 59 - 30,424
* Ages 60 to 64 - 16,493
* Ages 65 and older - 14,503

Cumulative AIDS Cases By Race or Ethnicity

* White, not Hispanic - 385,537
* Black, not Hispanic - 397,548
* Hispanic - 155,179
* Asian/Pacific Islander - 7,659
* American Indian/Alaska Native - 3,238

# Hemophilia / transfusion / perinatal / not identified

* Male: 14,191
* Women: 6,535
* Total: 20,726

# Heterosexual contact

* Male: 61,914
* Women: 102,936
* Total: 164,850

AIDS Cases Among Children Younger Than 13 Years By Exposure Category

Here is the distribution of reported AIDS cases among children by exposure category.

* Hemophilia/coagulation disorder: 236

* Mother with or at risk for HIV infection: 8,629
Decreasing the Risk of HIV Transmission from Mother to Child

* Receipt of blood transfusion, blood components, or tissue: 390

* Risk not reported or identified: 45

Hmm, a lot of people...... and more than half of them hadn't much contact with gay people.

So it sounds like for some of those people they just had awful bad luck and were in circumstances that was beyond their control. do they deserve to be infected with Aids anyway?

information taken from here:
http://aids.about.com/od/toptenlists/tp/hivmyths.htm
http://aids.about.com/od/dataandstatisti...sindex.htm

[mv]

iand more than half of them hadn't much contact with gay people.

And how do we come to this interesting conclusion exactly?

Is this another "urine is solid" type of claim?

[Gunnen4u]

Yea, why are we assuming it's gay people that are the biggest group of carriers and people who spread AIDS?

Sorry, had to make it more plain. I belabor the obvious whenever I can.

[Palladin]

I don't care what causes it myself,BUT,unless my math is wrong,that chart there shows ~85% gay males.


BTW,

1) if we found a way to cure AIDS,it might inadvertently lead to other cures.

2) Most problems of humanity are caused by stupid decisions,so if we're going to use the logic we shouldn't try to find/fund cures for man made problems,we need to stop funding lots of medical research cause most of the problems are based on our conduct.

I drink and I did smoke and I eat what I please and I am a couch potato,so it is not unlikely cirrhosis,heart disease shall visit me someday soon. It will be my fault largely if so.

3) Gay political activism has achieved an inordinate amount of funding for AIDS research relative to breast cancer,lung,heart disease research etc,there can be no denying that. It's taking from much more prevalent problems of the nation,that is a fact.

[quadrat]

be careful with those statistics, "AIDS-deaths" were people who were infected with HIV and died, but the deaths may be due to any cause. still, in 30 years since its arrival, hiv has infected 0.3% of americans, and the 500,000 "aids deaths" are nothing compared to the millions who died of cancers, diabetes, etc. i don't think a cure will be found, hiv is such a beautiful business. the research can go on forever, and the drugs costs up to $26,000 per month in the usa i read a couple of days ago. Here in thailand, it's $30 per month. i looked if i can find a statistic of aids-deaths year by year because i think it has been a long time since an american who can afford the drugs has died with aids, but no success.

[ghoullio]

According to Duke U, over 200,000 children break a limb on playground equipment EVERY YEAR! That is many many times more children than suffer from HIV. http://www.dukehealth.org/health_library...oken_bones

Clearly, we must either remove recess and other breaks from school or stop children from playing altogether. Maybe science can create stronger bones in children.

[John L]

This is really encouraging: Study: Bee Venom Kills HIV..

[Ron Lambert]

MV, I read that celebrated science fiction writer Isaac Asimov died of AIDS--which he contracted from a blood transfusion when he was in the hospital. I wish he could have been cured.

Homosexuality obviously is not in harmony with God's original purpose in creating the human race. It was the normal heterosexual relation that God pronounced "good." And there are a few verses in the Bible that clearly identify homosexual behavior as sin--because it is contrary to the will of God. BUT there are many things that God puts up with, such as (at times) polygamy. And adultery does more direct harm to marriage. It is unfortuate that the gay lobby wants to make everyone recognize homosexual unions as "marriages," which directly challenges God's authority. They could be called something else, and still have legal rights established. However bad and damaging homosexuality may be to human society, it is surely not the worst sin of all. It does not even compare to murder. In fact, it is not even directly mentioned in the Ten Commandments, and 1 John 3:4 says: "Sin is the transgression of the law."

[mv]

MV, I read that celebrated science fiction writer Isaac Asimov died of AIDS--which he contracted from a blood transfusion when he was in the hospital. I wish he could have been cured.

this is accurate; however, I wish he did not get infected.---if he would have not been if not for legalization of homosexuality in 1970s.

[Ron Lambert]

What I heard was that the AIDS virus came into this country in some refugee Haitian homosexual men, who were at first confined in a temporary holding camp. The CDC wanted to quarantine them, which would have prevented the AIDS virus from spreading into the general population. But the gay lobby interepreted that as discrimination against gays, and demanded that there be no quarantine. As a result, AIDS became the primary bane and killer of homosexuals, and also spread into the general community in America and then internationally by various means (such as intravenous drug use).

[mv]

Yes, something like this (there was also a theory that many initial cases of AIDS were linked to a gay airline steward).

[mv]

Here is something tangentially related: NYC sex workers avoiding condoms ....

I suspect that this story is somehow a reflection of Doomberg's screwy ideas.... but the question is how many more cases would this generate...

[Gunnen4u]

Well, healthcare, abortion, and childbirth is going to be free or state subsidized anyhow so what is the worry?