Pregnancy hormone has unprecedented, powerful effect on spinal muscular atrophy

Pregnancy hormone has unprecedented, powerful effect on spinal muscular atrophy

Press Release | July 25, 2011

‘Biggest increase anyone has seen’ in the production of essential compound for deadly childhood disease

OTTAWA – July 25, 2011 – Researchers in Ottawa report new hope for the treatment of infants born with serious genetic disorder.

Over 1000 children in Canada are affected with Spinal Muscular Atrophy (SMA), a genetic disorder that causes muscle weakness and loss of motor control. In its most severe form survival of children with SMA beyond 5 years is rare. Although the disorder is caused by the loss of a specific gene, all infants and children with SMA have an untouched highly similar gene within their genetic make up. Activation of this copy gene has the potential to treat SMA, and thus has been a goal of researchers around the world. Now, researchers at the Children’s Hospital of Eastern Ontario Research Institute in Ottawa report the strongest such activation yet observed with attendant benefit on mice genetically engineered to have SMA.

PhD student Faraz Farooq working in the laboratory of University of Ottawa professor, Alex MacKenzie has discovered that the pregnancy hormone Prolactin, a Canadian discovery in itself, not only activates the copy gene but if given over time extends the lifespan of SMA mice by up to 60%. The research report is published today in the Journal of Clinical Investigation.

“Prolactin causes a dramatic regulation of copy gene SMN2 which results in high production of SMN protein, resulting in the extension in the lifespan of mice with SMA,” said Mr. Faraz Farooq. “Labs around the world have been trying to produce more protein from copy gene SMN2 but with Prolactin (an insulin like protein) we’re seeing up-regulation that’s more than tenfold. It’s the biggest increase anyone has yet seen in the SMA Field with any potential therapeutic compound. This represents a significant advance in search for a therapy for this disease.”

The laboratory testing of Prolactin on SMA not only shows an extended lifespan but also improved motor control. Prolactin has been used in clinical trials for unrelated studies, so it is expected that the path between pre-clinical validation and actual clinic trials of Prolactin with SMA patients will be reasonably short.

“News of prolactin’s role and effectiveness in SMN regulation breathes fresh hope into all of the SMA community,” said Martha Slay, president and co-founder of FightSMA. “FightSMA congratulates Dr. MacKenzie and his colleagues on this exciting breakthrough in SMA research.”

“We believe we’re moving in the direction of an effective pre-symptomatic treatment of kids with SMA,” said Dr. Alex MacKenzie, principal investigator, CHEO Research Institute. “We want to somehow stop the progress of this disorder in its tracks, and let our tiniest patients build strength. Today’s findings are not curative, but we think this is a breakthrough discovery. Hopefully by using different approaches to increase SMN protein we can develop a combination therapy for the treatment of SMA.”

###

About the CHEO Research Institute: Established in 1984, the CHEO Research Institute coordinates the research activities of the Children’s Hospital of Eastern Ontario (CHEO) and is one of the institutes associated with the University of Ottawa Teaching Hospitals. The Research Institute brings together health professionals from within CHEO to share their efforts in solving paediatric health problems. It also promotes collaborative research outside the hospital with partners from the immediate community, industry and the international scientific world.

Click HERE or on the image below to read the press release…

Advertisements

4 Responses to Pregnancy hormone has unprecedented, powerful effect on spinal muscular atrophy

  1. Many of you report to my office with a complaint of being hypothyroid only to have been told by so are you. However, they have not gone far enough to document what you know to be true. Your thyroid gland most of the time is producing adequate amounts of thyroid. However, after it leaves the gland, the brain takes over and it responds to environmental cues, sleep, stress, exercise, etc. This your primary care provider that you are normal on testing. Guess what? They are correct!! But, response changes the activity of enzymes that control the conversion of your thyroid hormone to its more active form known as T3. This has to be assessed to adequately determine your true thyroid activity. I have included a feedback chart below of this physiology

  2. office kit says:

    My coder is trying to convince me to move to .net from PHP.
    I have always disliked the idea because of the expenses. But he’s tryiong none the less.
    I’ve been using WordPress on various websites for about
    a year and am anxious about switching to another platform.
    I have heard excellent things about blogengine.net.
    Is there a way I can import all my wordpress content into it?
    Any kind of help would be really appreciated!

  3. Muhammad says:

    It’s an remarkable post in support of all the online viewers; they will tske advantage from it I
    am sure.

  4. torticolis says:

    This is my first time pay a viskt at here and i am really happoy
    to read all at one place.

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: