Showing posts with label aids. Show all posts
Showing posts with label aids. Show all posts

Tuesday, March 03, 2015

$28 Million California Search for HIV/AIDS Stem Cell Cure Advancing to Clinical Trial

The California stem cell agency today announced that one of its multimillion dollar efforts to come up with a cure for HIV/AIDS is moving into the first stage of clinical trials involving human beings.

The effort includes the City of Hope in Duarte, Ca., the University of Southern California and Sangamo BioSciences, Inc., of Richmond, Ca.

John Zaia
City of Hope photo 
The $3 billion agency is pumping $5.6 million into the clinical trial. Overall, the agency has awarded the lead scientist in the project, John Zaia of the City of Hope, $28.2 million for work involving HIV/AIDS. 

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), said the FDA had approved the initial trial to determine the safety of the treatment.

CIRM’s press release said that the plan is to “take blood stem cells from HIV infected individuals, then treat them with zinc finger nucleases (ZFNs), a kind of molecular scissors, to disrupt the CCR5 gene in those cells. The hope is that this will make those stem cells, and their progeny, resistant to HIV. The modified cells will then be reintroduced into the patient with the hope that they will create a new, AIDS-resistant immune system.”

The effort is intended to replicate what occurred with the “Berlin Patient,” a man who has apparently been cured of AIDS as the result of a mutation.

Jeff Sheehy, a longtime CIRM board member and HIV/AIDS advocate, said in the press release,
“This trial is enrolling HIV patients whose immune cells have not returned to normal levels even after success in suppressing the virus with antiretroviral therapy, and even if it doesn’t lead to a cure it could still result in a therapy that offers clinical benefit to patients at risk for opportunistic infections.”

Zaia said,
‘While we have a number of drugs that are effective in holding HIV at bay, we have nothing that cures it. In addition, for many patients, these medications come with significant long-term problems so there is a real need for a therapy that can help eradicate the virus from a patient completely. That is where our work is focused.”  
The agency is also funding a clinical trial involving an alternative approach to HIV/AIDS. That effort includes UCLA and an Arizona company, Calimmune, Inc., co-founded by a former member of the CIRM governing board, David Baltimore. Calimmune also has an address near UCLA in Los Angeles.

CIRM said,
“Calimmune, an HIV gene medicines company focused on developing cell-based therapies for HIV, began its human clinical trial in July 2013 and has already shown that the first group of patients treated did well enough for the company to start treating a second group more intensively.” 
Jonathan Thomas, chairman of the CIRM board, said,
“This kind of work is too important to just try one method at a time and sit back and wait to see if it is effective.”

Tuesday, July 09, 2013

HIV Clinical Trial Hailed by California Stem Cell Agency

The California stem cell agency today scored what it called an “important milestone” with the announcement of the start of a clinical trial involving a therapy to help protect persons infected with HIV from the effects of the virus.

The trial is partially funded from a $20 million award from the stem cell agency, which is known as CIRM, to researchers at UCLA and Calimmune, a Tucson, Az., company. Calimmune's share of the award was $8.2 million.

Alan Trounson, president of the $3 billion state agency, said in a statement,
CIRM funding of this Phase l/ll trial is an important milestone for us. One of our goals is to support research that moves the most promising science out of the lab and into clinical trials in people. To be able to do that with a disease as devastating as HIV/AIDS highlights the importance of our funding and the potential impact it could have on the health of people around the world.”

The trial was announced by Calimmune this morning. The company said,

The first patient has begun treatment in a Phase I/II clinical trial designed to determine whether a pioneering genetic medicine approach can help to protect individuals infected with HIV from the effects of the virus. The study, “Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection,” utilizes a gene medicine called Cal-1, developed in the lab of Nobel Laureate Dr. David Baltimore and by Calimmune.”

Baltimore served on the CIRM board from 2004 until June 6, 2007. He resigned from the board about 18 months before the application process began for the grant round that ultimately funded Calimmune, a company he helped to found. He is currently chairman of the Calimmune board. 

Asked for comment, Jeff Sheehy, a member of the CIRM governing board and communications director for AIDS research at UC San Francisco, said,
"This trial will hopefully offer several important insights into the safety and feasibility of genetically modifying blood forming stem cells in an HIV patient as a potential therapy.  We are very early in this research, and with this Phase I trial's goal of establishing safety and the risks involved, I applaud the courage and altruism demonstrated by the patients who are willing to participate in this study."

The Calimmune press release said the principal investigators on the clinical trial are Ron Mitsayasu of UCLA and Jacob P. Lalezari of Quest Clinical Research of San Francisco. Quest is currently soliciting patients for the clinical trial as well as UCLA. (Persons interested in participating in the trial can find email contacts at this website. Twelve are needed.)

The principal investigators on the CIRM award are Irvin Chen of UCLA and Geoff Symonds of Calimmune.  

Here are links to the CIRM press release on the subject and the agency's blog item.

(An earlier version of this story did not include the fact that Calimmune's share of the CIRM award was $8.2 million or the links to the agency press release and blog.)

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