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.5M research chair holds hope for Parkinson patients


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Matthew Farrer, appointed to the Dr. Donald Rix B.C. Leadership Chair in Genetic Medicine, works in his lab at the University of B.C. The Province’s Leading Edge Endowment Fund chair supports the world-class scientist’s work in identifying genes related to Parkinson’s, which is expected to lead to therapies that will arrest and prevent the disease.
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Gene therapy holds promise in the treatment of a myriad of diseases, including cancer, heart disease and diabetes, among many others. However, developing a scalable system for delivering genes to cells both efficiently and safely has been challenging.

Now a team of Northwestern University researchers has introduced the power of nanodiamonds as a novel gene delivery technology that combines key properties in one approach: enhanced delivery efficiency along with outstanding biocompatibility.

"Finding a more efficient and biocompatible method for gene delivery than is currently available is a major challenge in medicine," said Dean Ho, who led the research. "By harnessing the innate advantages of nanodiamonds we now have demonstrated their promise for gene therapy."

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A multimedia project for the 2011 Student Bio Expo by Reed McCoy, Theo Henderson, and Corbin Hughes. In recent years, Gene Therapy has emerged as a promising answer for those suffering from congenital blindness diseases associated with a malfuntioning RPE65 gene. Dr. Ben explores the field of gene therapy, RPE65′s role in the visual cycle, and the power of a working gene.

Gene Therapy for Neurological Disorders

Coedited by internationally recognized leaders in gene therapy research, this guide supplies the most recent advances, studies, and expert opinion on gene therapy for neurological disorders. Spanning conditions such as Parkinsons’s and Alzheimer’s disease, multiple sclerosis, brain tumors, stroke, epilepsy, and brain and spinal cord trauma, this reference presents the basic science and applications of recombinant gene products, immune and stem cell-mediated therapeutic strategies, and brain tumo

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(Part 1 of 2) CIRM has funded a million disease team to develop a more effective and safer bone marrow transplant to treat sickle cell disease. The team is led by Dr. Donald Kohn, director of the Human Gene Medicine Program at UCLA, a scientist with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and a professor of microbiology and pediatrics. Kohn updated the CIRM Governing Board on May 05, 2011 about his team’s progress. For more information see our sickle cell disease fact sheet: www.cirm.ca.gov To view the other video in this playlist, go to: www.youtube.com
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Stuff: Compulsive Hoarding And The Meaning of Things


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This is a mental health practitioner’s perspective on hoarding that confirms much of what I’ve observed with my clients. Some common threads: perfectionism plays a large part in hoarders getting behind with what they do with their stuff thus prompting them to become overwhelmed and then giving up on any sort of housecleaning. Hoarders have an appreciation for things beyond their obvious uses and often are very creative, but seem to lack the organizing gene.

Some interesting insights on the hoarding brain, described as having too many branches on the tree. Meaning that there are multiple paths the mind is prompted to navigate when processing which also leads to overwhelm.

I attended a day long workshop with one of the writers and the take away message from that day was that hoarders have a perception problem. They do not see that their stuff is making their home a dumpster and that they are not actually rescuing anything by picking up one more thing from the "Free" pile on the street. My experience with one client is that she has an inability to know what is important because everything is important.

Some poignant insight about causes of hoarding include absence of warmth, acceptance and support in childhood leading the child to form attachments to things and endow those things with human emotions. Thus they can’t throw an item away because there would be no one to look after it.

Little is discussed about treatment in the book. The authors have written other books to help practitioners in the field. This is largely an anecdotal book and the stories of hoarders do offer a lot of insight since you get the whole history.

I have a couple of bones to pick with the book. The authors mention the National Study Group of Chronic Disorganization which was started by one of my organizing colleagues. The authors believe the term "chronic disorganization" is a euphemism for hoarders. I think that’s unfair; a case of a hammer seeing everything as a nail. Mental health practitioners are guilty of seeing every thing in pathological terms. Everyone who has clutter is not automatically a hoarder. Hoarders have an attachment to their stuff that goes beyond the utility of the item. Plenty of people with clutter would be happy to let it go once they found the things they really needed.

The authors also don’t think that the members of the NSGCD go far enough to actually help hoarders. I think we’re changing that especially since she told us how. Cognitive therapy is the prescribed mental health treatment for hoarders. This just means that someone sits with the hoarder and asks questions that would lead them through to a rational conclusion about why they are keeping an item.

In addition I like to keep the hoarder working on sorting their stuff for so long, they are sick of it and they vow never to let it get this bad again. Then I coach them on setting limits on the incoming stuff. I also let them talk about the stuff or if they prefer, they can ask me to talk about something—anything if they want to take their minds off it. I feel this raises their self-esteem to be able to relate to me on an intellectual topic rather than wallow in the shame of their mess. Higher self-esteem often results in a higher level of energy for the task at hand. This is the Amanda theory that will either make or break me in this work.

Cognitive therapy is all about getting the client to look at their feelings while sorting through stuff. Then helping them to get past their sticking points. The theory goes that when the emotion is worked through the therapist can help diminish it, but I had a therapist tell me yesterday that this isn’t necessarily so. People just like to go on about their feelings about everything. Some therapists create a crisis around the stuff on purpose to light a fire under them. This is too messy for me. Therapists like emotional messes. I like to bypass all the guilt, blame, grief, etc., etc., in favor of just getting the job done. I have had clients tell me they appreciate this quiet efficiency. Others like the drama and I am there for that too, but I don’t go looking for it.

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Dr. Amit Nathwani's team in London cured or improved the health of six hemophilia patients with a new type of gene therapy.

Finally, some good news about gene therapy. And just in time for the holidays.

A recent study showed that giving hemophilia patients blood clotting genes can partially make up for their deficiencies and reduce bleeding. Hemophilia B is a heredity disorder caused by a defective gene for the blood clotting protein called factor IX. Researchers in Britain successfully treated six people with hemophilia by giving them a gene with a functional form of factor IX. Previous clinical trials have attempted to treat hemophilia with gene therapy, but expression of the vital factor had been short-lived. In the current study the researchers used a modified viral vector – the carrier DNA into which the factor IX DNA is inserted – that targeted the DNA specifically to the liver where factor IX is normally produced. Other studies had shown that the vector could be expressed in the livers of large animals for at least 10 years. The strategy worked, at least a year out after treatment. The participants in the study all had severe hemophilia B, meaning their bodies produced less than one percent normal factor IX levels. In a dose-dependent manner the gene raised factor IX levels in the participants between 2 and 12 percent. Not spectacular increases, but effective nonetheless. Severe hemophiliacs have to receive injections of the factor IX protein two to three times a week to avoid severe bleeding. Four of the participants were able to stop treatment injections altogether and the other two were able to extend time between injections.

Hemophilia is probably not talked about as often as many other disorders. But for people with the disease, it can be a debilitating, daily grind. Listen to Sebastian Misztal, one of the trial participants, talk about how the therapy has changed his life in the following video.

The study comes more than half a century since scientists first linked hemophilia B to a factor IX deficiency. The patient was a 10-year-old boy named Stephen Christmas. Henceforth, the disease was called “Christmas disease.” The gene therapy, however, was not without side-effects. One high-dose participant showed increased levels of transaminases, a sign of possible liver damage. Another showed a small increase in liver enzymes. The researchers treated them both with steroids.

Given that there is already a treatment for hemophilia B – factor IX concentrate injections – a fair question is: why pursue a gene therapy? One answer is cost. The cost to severe hemophiliacs who receive injections when they need them – when they’re bleeding – is about $150,000 per year. The cost for those who receive injections regularly as a prophylactic is about $300,000 per year. Over an entire lifetime the cost can exceed $20 million. The cost of the therapy is estimated at $30,000 per patient. Even if the patient has to receive multiple treatments throughout his or her lifetime it will still be the cheaper alternative by far.

One out of about every 30,000 newborn babies will have Hemophilia B. In the United States about 3,000 people have the disease. The severity of bleeding ranges widely. In the more severe cases, symptoms will be apparent early on such as when a baby is circumcised. More mild cases might not be noticed until later in life when the person experiences a trauma or undergoes surgery. It also affects men more than women as the gene for factor IX is found on the X chromosome. As with all X-linked diseases, having two X chromosomes gives women double the chance of having a normal form of the gene.

The internal bleeding of hemophilia patients can lead to dangerous swelling, joint pain, and sometimes death.

Hemophilia A, caused by a defect in clotting factor VIII, is actually much more prevalent than hemophilia B and accounts for about 80 percent of all hemophilia patients. Gene therapy scientists are starting with factor IX because it is much smaller than the factor VIII gene and more feasible to work with.

Hearing about hemophilia can conjure images of British royalty, as it is often taught in schools as the disease passed from Queen Victoria to her royal descendants all over Europe. I can still remember all those squares and circles on the science class pedigree. In 2009 scientists exhumed bones from the graves of Russian Czar Nicholas II and his family members and analyzed their DNA. The “royal disease” had been transmitted to the family from Queen Victoria. They confirmed that family members had a defective form of factor IX, or hemophilia B. Isn’t history with molecular biology fun?

But the first documentation of hemophilia goes back quite a bit further. In the Babylonian Talmud, written in the second century, Rabbi Judah commanded that “If she circumcised her first child and he died, and a second one also died, she must not circumcise her third child.”

Lucky for the third child they hadn’t yet begun to insist on an n of 3.

The current study, carried out by scientists at the University College London Cancer Institute and St. Jude Children’s Research Hospital in Memphis, Tennessee, was published in the New England Journal of Medicine. It’s a rare triumph for gene therapy, all too often delivering maladies instead of miracles. The field has proceeded much more cautiously since two French boys developed leukemia and one University of Pennsylvania teenager died after receiving gene therapy. Only six people were involved in the current study, so there’s still plenty of ground to cover before the factor IX vector can be considered a safe and effective treatment. Let’s hope the current results are not a fluke, but an indication of things to come.

[video credit: University College London via YouTube]
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