|[June 25, 2014]
Calimmune Approved to Treat Second Group in HIV Stem Cell Gene Modification Study
SAN DIEGO --(Business Wire)--
Calimmune, Inc. today announced that encouraging results from a first
group of participants indicates the company is ready to begin treating a
second cohort in a clinical trial involving the use of Cal-1, an
innovative gene-based stem cell therapy to help protect individuals
infected with HIV from progressing to AIDS.
Calimmune was given approval to move ahead following a review of safety
data by their Data Safety Monitoring Board (DSMB). The DSMB confirmed
that none of the participants experienced any serious adverse events or
dangerous side effects from the therapy.
"We are very excited and encouraged by this development," says Louis
Breton, Chief Executive Officer of Calimmune. "This recommendation from
the DSMB is an important step in bringing this potential one-time
therapy to the patients, and takes us closer to our ultimate goal of
Breton continued: "We are proud of our ongoing partnership with the
California Institute for Regenerative Medicine (CIRM), and are most
appreciative of their continued financial support of this critical
The Phase I/II clinical trial focuses on a protein called CCR5 that
plays a critical role in enabling HIV to infect cells. Blocking CCR5
expression may provide the cells a protective shield against HIV, which
in turn would help retain immune system functionality.
"The mission of CIRM is to efficiently accelerate the development o
stem cell treatments for patients suffering from unmet medical
conditions," said, C. Randal Mills, Ph.D., President and CEO of CIRM.
"While still early in clinical development, this announcement
demonstrates real progress towards this mission. The accomplishment of
Calimmune's team is a great example of how CIRM partnerships are working
to impact patient's lives today."
In the first phase of this study 4 HIV-positive participants were
infused with their own blood stem cells as well as mature T cells that
had been modified to carry a gene that blocks production of CCR5. The
hope is that those stem cells will then create a new blood system that
is resistant to HIV. To guard against the virus forming resistance, the
team has used a second mechanism to prevent the virus from fusing with
the patient's cells.
The participants had all previously been on anti-retroviral drugs but
had discontinued taking them because of side effects or treatment
The second group of 3-4 participants will not only get Cal-1 but will
also get a preconditioning regimen with the aim of making the therapy
The goal of the trial - which is being conducted in San Francisco and
Los Angeles - is to assess the safety of the therapy, to determine the
ease of use and feasibility of the approach for HIV/AIDS patients and to
evaluate what, if any, side effects there may be.
"With more than one million Americans living with HIV, there is clearly
an urgent need for a therapy that does more than just hold the virus at
bay," says Jonathan Thomas, Ph.D., J.D., Chair of the stem cell agency's
governing Board. "Current medications are effective, but come with a big
cost both in terms of dollars and side effects. Our goal is to find an
approach that effectively cures people with HIV/AIDS."
Calimmune is a clinical-stage HIV gene medicines company focused on
developing innovative cell-based therapies for HIV. The company's stem
cell technology was discovered in the labs of Nobel (News - Alert) Laureate, Dr. David
Baltimore (Caltech) and Dr. Irvin Chen (UCLA AIDS Institute). Calimmune
is also developing a rich product candidate pipeline to address the
needs of different types of individuals at different states of HIV
infection and with different levels of treatment experience.
About CIRM: CIRM was established in November 2004 with the
passage of Proposition 71, the California Stem Cell Research and Cures
Act. The statewide ballot measure, which provided $3 billion in funding
for stem cell research at California universities and research
institutions, was overwhelmingly approved by voters, and called for the
establishment of an entity to make grants and provide loans for stem
cell research, research facilities, and other vital research.
[ InfoTech Spotlight's Homepage ]