X-Message-Number: 27630
Date: Fri, 17 Feb 2006 09:22:14 -0800 (PST)
From: Doug Skrecky <>
Subject: breakthrough in beating progeria

[Below, in 20 weeks six of 14 untreated progeria mice died, while only one
of 13 of those treated expired. Here's hoping that the
farnesyltransferase inhibitor used is OK'ed for clinical use quickly!]


Cancer drug 'treats early ageing'

Hayley Okines, eight, from Bexhill, Surrey is affected by Progeria

A cancer drug could help children with the premature ageing disorder
progeria, a study has suggested.

University of California, Los Angeles, researchers found a
farnesyltransferase inhibitor (FTI) appeared to reduce the effects of
the disease in mice.

Progeria is a rare genetic disease, affecting one in four million

Experts in the condition said the study, in Science, offered hope the
drug could be safely given to children affected by progeria.

 This type of evidence will help us to ensure that children can safely
take this drug

Dr Leslie Gordon, Progeria Research Foundation

The condition, which is actually called Hutchinson-Gilford Progeria
Syndrome (HGPS), stems from a mutation that leads to the accumulation of
an abnormal protein on the scaffolding of the cell nucleus.

The abnormal protein causes misshapen cell nuclei, and ultimately leads
to all of the disease findings of progeria.

Those affected can experience dwarfism, baldness, wrinkles, hardened
arteries, and osteoporosis.

Most children with the condition die from heart disease before the age
of 15.

Bone health

The UCLA researchers gave a farnesyltransferase inhibitor (FTI) in mice
with progeria.

FTIs were initially developed to treat cancer, and are now given to
patients with breast and bowel cancer as well as leukaemia patients and
those with multiple myeloma - cancer of the bone marrow.

They had already shown that FTIs could prevent misshapen nuclei in
progeria cells. It works preventing the abnormal protein from reaching
the scaffolding of the cell nucleus.

By the end of the 20-week study, six of 14 untreated progeria mice had
died compared to only one of 13 FTI-treated mice.

There were only two rib fractures in the mice given the drug, compared
with 14 in untreated animals.

The majority of treated mice also showed improvements in body weight,
bone health, grip strength, and survival compared with the untreated

Dr Loren Fong, an assistant professor of medicine at the David Geffen
School of Medicine at UCLA, who worked on the study, said: "This is the
first study in an animal model to show that an FTI could be useful in
treating Progeria and related conditions.

"We believe that these studies should give some hope to Progeria
patients and their families."

However, Dr Fong said that although the study showed the drug appeared
to benefit those animals given it, it was not a cure.

But he speculated a higher dose might be more effective.

Dr Leslie Gordon, director of the Progeria Research Foundation, said:
"This study gives us pieces of information critical to our movement
toward clinical trials in children with progeria.

"This type of evidence will help us to ensure that children can safely
take this drug."

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