HIV clinical trials Baltimore

Nobel Laureate David Baltimore, PhD, Robert Andrews Millikan Professor of Biology, California Institute of Technology.People living with HIV will be treated with genetically engineered stem cells next month by the team of Nobel Prize-winning immunologist David Baltimore.

The goal: to create, in patients, new immune systems resistant to HIV.

“We are very hopeful we can use that as a way of effectively curing patients, and allowing them to live drug-free lives, ” Baltimore told the World Science Festival in New York on May 31.

The trial, which blends techniques both conjured in the lab and pilfered from nature, is long-awaited. Baltimore calls it a “Hail Mary Pass.” Like trials sponsored by Sangamo Biosciences, it was partly inspired by the “Berlin Patient” Timothy Brown.

A leukemia patient, Brown needed a bone marrow stem-cell transplant to replace his blood cells with those of a donor. Because he also had HIV, his doctor gave him bone marrow cells that were an immunological match, with a twist: they were from a donor who was one of the 1% of the European population resistant to HIV.

Brown’s HIV vanished. It has not returned in six years.

In a series of ongoing Sangamo trials, scientists have been disarming the gene that made Brown and the 1% resistant: the CCR5 gene coding for a receptor on CD4 T cells through which the virus enters. HIV-resistant people have two copies of the defective CCR5 gene. Sangamo has been removing patient blood cells; disarming CCR5 in their CD4 T cells (by slipping disruptive “zinc finger nucleases” (ZFNs) into their genomes via different techniques, including adenoviral vectors); returning the cells. (See related Drug Discovery and Development story here.)

Some preliminary results have been highly encouraging. But some experts say the key to long-term success may lie in altering CCR5 in stem cells, permanent cells of the bone marrow, which mature into—and give the body—a lifetime coterie of blood cells, including T cells. For that reason, many groups are now disarming CCR5 in stem cell pre-clinical work.1

LAP LAMBERT Academic Publishing The Patients Voice in HIV/AIDS Clinical Trial Participation: What motivates the willingness of HIV infected people to take part in HIV/AIDS clinical trials?
Book (LAP LAMBERT Academic Publishing)
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BMC Medical Research Methodology at the 35th Annual Conference of the ..  — BMC Pediatrics
The conference will focus on issues such as design and analysis of clinical trials, methods in biostatistics and development of clinical prediction models.

Popular Q&A

It relation to HIV clinical trials, what does ACTG stand for?

The AIDS Clinical Trials Group (ACTG) is the largest HIV clinical trials organisation in the world, playing a major role in setting standards of care.

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