Breaking News
September 26, 2018 - Researchers find molecule that halts, reverts neurodegeneration caused by Parkinson’s disease
September 26, 2018 - Novartis announces winners of 2018 eXcellence in Ophthalmology Vision Award
September 26, 2018 - New spinout company to tackle drug-resistant infections with novel antibiotics
September 26, 2018 - In depression the brain region for stress control is larger
September 26, 2018 - Smuggling RNA into cells can activate the immune system to fight cancer
September 26, 2018 - Special Focus Issue takes wide view of complementary and integrative medicine in cancer
September 26, 2018 - Researchers now confirm that genome duplication drives evolution of species
September 25, 2018 - Study provides evidence of beta lactamase producing, antimicrobial resistant E. coli in U.S. retail meat
September 25, 2018 - UCI study finds new cause of cerebral microbleeds
September 25, 2018 - Researchers propose mechanism by which ASTN2 protein defects lead to brain disorders
September 25, 2018 - Chinese and German researchers to cooperate more closely in future for better food
September 25, 2018 - Recent study helps predict probability of pregnant mothers to have child with autism
September 25, 2018 - New online, sound matching tool offers tinnitus sufferers potential treatment options
September 25, 2018 - UC Davis researchers take critical step in developing more effective Salmonella vaccine
September 25, 2018 - Antibiotics best paediatric treatment for children’s chronic wet cough
September 25, 2018 - Looking beyond opioids: Stanford pain psychologist briefs Congress
September 25, 2018 - Organs actively fighting back against autoimmune diseases, finds study
September 25, 2018 - Lancaster professor aims to understand how genes affect smoking cessation
September 25, 2018 - Human-oriented perspective needed to better understand Parkinson’s disease
September 25, 2018 - Physical activity may have beneficial effects for people with rare Alzheimer’s disease
September 25, 2018 - FDA Updates on Valsartan Recalls
September 25, 2018 - 3-D-printed tracheal splints used in groundbreaking pediatric surgery
September 25, 2018 - Who is the designated driver, or proxy, for your health decisions?
September 25, 2018 - New chemo-optogenetic method enables multi-directional activity control of cellular processes
September 25, 2018 - Study explores link between genetic predisposition to Alzheimer’s and cardiometabolic risk factors
September 25, 2018 - NeoTract presents new clinical data from studies of UroLift System for patients with BPH
September 25, 2018 - Statins Improve Long-Term Survival After AAA Repair
September 25, 2018 - Novel brain network linked to chronic pain in Parkinson’s disease
September 25, 2018 - Researchers reassess negative pressure wound therapy as its benefit and harm remain unclear
September 25, 2018 - Older adults with ‘fall plan of care’ less likely to suffer fall-related hospitalizations
September 25, 2018 - FDA lifts partial clinical hold that paused enrollment of new patients in tazemetosta clinical trials
September 25, 2018 - IME Medical Electrospinning establishes state-of-the-art manufacturing lab facilities
September 25, 2018 - Phase 1 and 2 clinical trials of entrectinib drug in ROS1-positive NSCLC show promising results
September 25, 2018 - How to Protect Your Eyesight
September 25, 2018 - Novel approach allows researchers to define how cells in the retina respond to diabetes
September 25, 2018 - Columbia University announces winners of 2018 Louisa Gross Horwitz Prize
September 25, 2018 - New model enables anyone to run powerful simulations, complex calculations easily
September 25, 2018 - Clinical trial investigators found non-compliant with requirement to report results on EU register
September 25, 2018 - Study analyzes quality of protein supplements in function of source, treatment and storage
September 25, 2018 - FDA grants Orphan Drug Designation to Myelo001 for treatment of Acute Radiation Syndrome
September 25, 2018 - U.S. Alzheimer’s Cases to Nearly Triple by 2060
September 25, 2018 - Improving cell replacement therapy for Parkinson’s disease
September 25, 2018 - Genervon reports new findings that drug candidate GM6 attenuates Alzheimer’s disease in mice model
September 25, 2018 - FDA approves new 5 mm diameter drug-eluting stent from Cook Medical
September 25, 2018 - New $17.8 million grant ensures USC at forefront of research on tobacco-related health risks
September 25, 2018 - Researchers analyze response to combination immunotherapy for patients with rare skin cancer
September 25, 2018 - Study sheds light on how brain protein may be involved neurodevelopmental disorders
September 25, 2018 - Where to draw the line on incentives
September 25, 2018 - Solid fuel use linked with increased risk of hospitalization or death from respiratory diseases
September 25, 2018 - ‘Trouble Brewing’ report highlights steps that governments can take to reduce alcohol-related harms
September 25, 2018 - Recurrence risk of VTE appears similar for patients with cancer and those with unprovoked VTE
September 25, 2018 - Global leaders must make bold commitments at first-ever UN tuberculosis summit
September 25, 2018 - Brief sleep intervention works long-term to prevent child obesity
September 25, 2018 - Vaping among kids and teens a growing concern
September 25, 2018 - Public launch of products and application solutions from Porvair Laboratory Division
September 25, 2018 - Harmful H. pylori may play a role in Parkinson’s disease
September 25, 2018 - Researchers develop way to measure different types of fear of falling in patients with Parkinson’s
September 25, 2018 - Fracture causes bone density losses throughout the body
September 25, 2018 - Researchers highlight potential therapy for treating rare, deadly blood-clotting disorder
September 25, 2018 - Hybrid theranostic complex shows high therapeutic efficacy against tumor cells
September 25, 2018 - FDA Issues Statement Reaffirming the Positive Benefit-Risk Profile of Nuplazid (pimavanserin) for Patients with Hallucinations and Delusions Associated with Parkinson’s Disease Psychosis
September 25, 2018 - Toxicological evaluation and dosimetry estimation of potential PET radiotracer
September 25, 2018 - 5 obstacles parents commonly face in child obesity treatment and how to overcome them
September 25, 2018 - Immunologist to study how Chikungunya causes devastating effects in older adults
September 25, 2018 - Rural borderland communities vulnerable to high stress impacting mental and physical health
September 25, 2018 - SNMMI announces recipients of 2018-2020 Wagner-Torizuka Fellowship
September 25, 2018 - Common painkiller not effective in controlling chronic pain after traumatic nerve injury
September 25, 2018 - New therapeutic vaccine helps immune cells fight HPV-related head and neck cancer
September 25, 2018 - Environmentally-induced gene activity influences IQ test performance
September 25, 2018 - Biogen and Eisai announce results of LTE Phase 1b study of aducanumab for treating MCI
September 25, 2018 - FDA Approves Copiktra (duvelisib) Capsules for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma and Follicular Lymphoma
September 25, 2018 - Medical device company settles US case over false claims
September 25, 2018 - Trying to get answers: One woman’s quest for a diagnosis
September 25, 2018 - Lung cancer patients treated with invasive surgery more likely to become chronic opioid users
September 25, 2018 - Oxford VR raises £3.2m to boost innovation in VR for mental health problems
September 25, 2018 - Gene therapy approach could help treat mitochondrial diseases
September 25, 2018 - Few Yogurt Products Qualify As Low-Sugar
September 25, 2018 - Eye disease can cause blindness, and it’s on the rise
September 25, 2018 - Pawnshop density linked to gun-related suicides, Stanford study finds
September 25, 2018 - Pioneering procedure for common prostate condition offered by The London Clinic
Can gene therapy be harnessed to fight the AIDS virus?

Can gene therapy be harnessed to fight the AIDS virus?

image_pdfDownload PDFimage_print
Can gene therapy be harnessed to fight the AIDS virus?
In this Jan. 26, 2018 photo, Matt Chappell, right, is checked by Dr. Christopher Schiessl during an appointment at a medical center in San Francisco. For more than a decade, the strongest AIDS drugs could not fully control Chappell’s HIV infection. Now his body does it by itself, thanks to the first gene editing experiments in people. (AP Photo/Jeff Chiu)

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell’s HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that made this possible.

Scientists removed some of his blood cells, disabled a gene to help them resist HIV, and returned these “edited” cells to him in 2014. So far, it has given the San Francisco man the next best thing to a cure.

“I’ve been off medications for three and a half years,” he said. He even was able to keep the virus in check despite cancer treatments last year that taxed his immune system.

Chappell was lucky, though. Only a few of the 100 others in those experiments were able to stay off HIV drugs for a couple years; the rest still need medicines to keep HIV suppressed.

Now researchers think they can improve the treatment and are trying again to tackle HIV by doctoring DNA. New studies to test these tweaked approaches in people are getting underway.

“Gene therapy techniques have advanced greatly,” said Dr. Otto Yang of the UCLA AIDS Institute, one place working on this. “A lot of people are thinking it’s the right time to go back.”

They include Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, which is funding some of the new studies. He doesn’t think the technique will become common because millions of people do well on existing treatments. But he says it could help those who can’t easily control the virus, and should be pursued because it holds potential for a cure.

Can gene therapy be harnessed to fight the AIDS virus?
In this Jan. 26, 2018 photo, Matt Chappell, left, talks with Dr. Christopher Schiessl during an appointment at a medical center in San Francisco. For more than a decade, the strongest AIDS drugs could not fully control Chappell’s HIV infection. Now his body does it by itself, thanks to the first gene editing experiments in people. (AP Photo/Jeff Chiu)

“They’re very bold, innovative techniques, mostly to try and cure people,” he said. “It’s worth trying because the science is there.”

ONE MAN’S CURE GAVE HOPE

Only one person is known to have been cured of HIV infection, a man who had a cell transplant a decade ago from a donor with natural immunity to the virus. The donor lacked a common gene that makes an entryway HIV uses to infect T cells, immune system soldiers in the blood.

The transplant gave the recipient that protection, but procedures like that are too risky and impractical for wide use. Scientists have been trying to find a way to create similar immunity by altering some of a patient’s own cells. They use a gene editing tool called zinc finger nucleases, which cut DNA at a precise spot to disable the HIV entryway gene.

The California company that makes the editing tool, Sangamo Therapeutics, sponsored the initial studies.

Can gene therapy be harnessed to fight the AIDS virus?
In this Jan. 26, 2018 photo, Matt Chappell, right, talks with Dr. Christopher Schiessl during an appointment at a medical center in San Francisco. For more than a decade, the strongest AIDS drugs could not fully control Chappell’s HIV infection. Now his body does it by itself, thanks to the first gene editing experiments in people. (AP Photo/Jeff Chiu)

“It worked, the T cells were edited,” said Sangamo’s president, Dr. Sandy Macrae. But it didn’t work quite well enough: The altered T cells were outnumbered by T cells that were not altered and could still be infected.

Now, Dr. John Zaia at City of Hope, a research center in Duarte, California, is trying the approach with a twist. He’s using blood stem cells—parent cells that produce many others. Once a stem cell is altered the benefit should multiply and last longer, Zaia said.

THE SILVER LINING

Though the initial gene editing experiments were disappointing, there was a silver lining. Patients in those studies had a big drop in the number of cells where HIV lurked in a dormant state—the so-called reservoir of silent disease.

At Case Western Reserve University in Cleveland, Dr. Rafick-Pierre Sekaly is trying to capitalize on that drop. His study will try the same gene editing—disabling the gene that makes the HIV entryway—while keeping patients on strong antiviral medicines for at least a year before discontinuing them.

Can gene therapy be harnessed to fight the AIDS virus?
In this Tuesday, Jan. 23, 2018 photo, James Riley, an HIV and gene therapy expert at the University of Pennsylvania, poses for a photograph in Philadelphia. Gene therapy has scored wins against some rare blood diseases and even a form of blindness. Now scientists are trying it for a much more common foe, HIV, the virus that causes AIDS. (AP Photo/Matt Rourke)

“As long as we’re not able to get rid of this reservoir, we’ll never be able to stop treatment,” he explained.

The hope is that the medicines plus the altered cells will knock down the virus and reduce the reservoir to a point where the body can control any residual disease by itself, as Chappell seems to be doing.

PROTECT AND ATTACK

University of Pennsylvania scientists are trying a two-part approach: Besides knocking out the gene for the HIV entryway, they’re adding a gene to help T cells recognize and kill HIV. This second part is called CAR-T therapy, a treatment approved last year for treating cancer.

The new study’s leader, scientist James Riley, is encouraged that some patients at Penn who were in the early studies kept HIV suppressed for nearly a year without drugs.

Can gene therapy be harnessed to fight the AIDS virus?
In this Tuesday, Jan. 23, 2018 photo, James Riley, an HIV and gene therapy expert at the University of Pennsylvania, speaks during an interview in Philadelphia. Gene therapy has scored wins against some rare blood diseases and even a form of blindness. Now scientists are trying it for a much more common foe, HIV, the virus that causes AIDS. (AP Photo/Matt Rourke)

“You’d never know they were sick” even though the virus could still be detected, Riley said. “At some point you’re going to have confidence that it’s not going to come back.”

Chappell’s doctor, Christopher Schiessl at One Medical, a health clinic in San Francisco, hopes that’s the case for Chappell. Although he’s doing well now, Chappell is showing signs that his immune system may be weakening, Schiessl said.

Chappell is optimistic, and believes gene therapy ultimately will provide a long-term solution.

“If we’re going to cure HIV,” he said, “this is how it’s going to happen.”


Explore further:
Gene therapy using CAR T-cells could provide long-term protection against HIV

Tagged with:

About author

Related Articles