CIRM's
Governing Board recently approved $15 million for four projects in
translational awards program, which promises to offer support to invent
novel stem cells transplants that can be applied to patients during
clinical trials. Wondering what "translational" might mean in studies,
involving stem cells? "Translational" implies to researches that take
the basic results of a clinical study and then advance them in a way
that they become ready to be tested in patients in various clinical
trials.
The fund for translating stem cell studies into effective therapies is divided into four researches. They are:
· Alzheimer's disease
· Sickle cell anemia
· Cartilage damage
· Osteonecrosis
Treating Alzheimer's disease:
Gladstone Institutes in San Francisco received $5.9 million from the
fund to find out a way to treat Alzheimer's disease. Alzheimer's disease
has no long-term treatment or no known cure and it affects five million
Americans.
In the process, the researchers are looking at
generating brain cells by applying pluripotent stem cells in an induced
form. Pluripotent stem cells or iPSCs are the adult cells that can be
reprogrammed to give them the ability to modify into any other cell
type.
Treating patients with sickle cell anemia:
The second project involves patients suffering from sickle cell anemia.
A fund of $4.5 million is approved to the researchers at Children's
Hospital Oakland Research Institute (CHORI) for the trial.
Sickle
cell anemia is a genetic blood disorder. It involves severe pain and can
even lead to organ damage and stroke. The disease has affected over
100,000 people in the US. Currently, the most effective and long-term
treatment for this disease involves bone marrow transplant. However, the
procedure requires a suitably matched donor, which may take months.
Even though a donor is found, the surgery may still leave a life risk.
Researchers
found out that the patient's own blood stem cells can eliminate the
complications and at the same time address the condition. To implement
the technique, a new gene-editing tool called CRISPR-Cas9 is used by the
CHORI team. This technique helps to develop a way to edit the defective
gene causing sickle cell and transform it into a healthy blood supply
for the affected people.
Treating medically untreated cartilage damage:
The third portion of the fund went to the University of Southern
California (USC). The researchers of USC have been granted with $2.5
million, so that they can develop a full-proof technique to treat
cartilage damage with stem cell transplant.
Cartilage damage might
not be life-threatening, but it affects the lifestyle of millions of
people to a great extent. And if remain untreated for a long time, it
might result in severe conditions such as chronic pain, degeneration of
joints and even arthritis.
Treating Osteonecrosis:
The fourth part of the fund, $2.09 million, went to Ankasa Regenerative
Therapeutics for a project, involving osteonecrosis. Regenerative
Therapeutics researchers hope to discover a solution to the painful
disease, which is caused due to inadequate blood flow to the bones. And
as the result, the bones begin to rot and then die.
We hope that translational awards programs with stem cell ideas result into effective therapies. Stay tuned for more updates!
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