- Some exciting news on the research work that we have been following with Eric Minikel and Sonia Vallabh:
Prion disease: potential treatment discovered by researchers and Researchers find new potential treatment for prion diseases
- Studies Show Pomegranate Supplement Slows Neurodegenerative Diseases (news article link regarding Publication)
- Delay of gCJD aggravation in sick TgMHu2ME199K mice by combining NPC transplantation and Nano-PSO administration (pdf of Publication)
Information provided by Professor Ruth Gabizon from Israel and studies into Pomegranate Seed Oil (PSO) marketed as Granagard.
LAY SUMMARY OF THE PUBLICATION:
Granagard is a nano formulation of PSO (pomegranate seed oil). PSO comprises 80-90% of Punicic Acid (omega 5) which is the strongest natural lipid antioxidant. In vivo, Punicic acid metabolizes into a specific form of conjugated linoleic acid (CLA), which is known to be a calpain inhibitor, suggested as a treatment target for several neurodegenerative conditions. While the active components, both PSO and CLA, do not enter the brain following administration of PSO and actually also CLA, following administration of the Granagard formulation, CLA is found in the brain of rodents, concomitantly with its neuroprotective effect that cannot be found when natural PSO was administrated. Most of our prion related experiments were done in a genetic model of E200K CJD. These mice are born heathy, then start to show neurological disabilities at 5-6 month of age and from there deteriorate until a terminal state at about 12 months of age. They mimic the situation of healthy carriers since they present spontaneous disease caused by a mutant PrP. Continuous Granagard administration from birth or 3 months of age delays the presentation of terminal disease in these mice by almost 6 months. The mechanism of activity of Granagard is most probably related to the maintenance of normal mitochondrial activity even under the stress caused by abnormal protein aggregates, thereby allowing neuronal survival under disease. This is applicable to a number of neurodegenerative disease diseases as well as normal aging. Several human studies are in process (Alzheimer’s disease (AD) ,Parkinson’s disease (PD). Preliminary results in a double-blind study shows that administration of Granagard as compared to placebo improves memory and cognition in MS patients under diverse treatments. As for CJD, we are mostly interested in delay/prevention in asymptomatic carriers of pathological PrP mutations. It is indeed very difficult to establish a proper clinical study to establish if carriers taking Granagard will get sick later or never as compared to those taking placebo. Nobody wants to be in the placebo group for years and most people don’t want to get tested for the mutation. Therefore, we are looking at this in a different manner. Since Granagard is a safe food supplement beneficial to the general public, we follow a large group of genetic families in Israel in which all/most the siblings in affected families are taking it. Then we look, with the help of the medical community in Israel, at the new CJD patients. We believe 30-50% of CJD affected families are taking Granagard regularly.
We have learned so far that
1: No symptomatic CJD patient has taken Granagard prior to diagnosis.
2: The general number of genetic CJD patients, which was around 20 per year for several years until 2017, has gone down to less than 10 in 2018/2019 and may be as low in 2020.
Obviously, this is all observational and more time is needed to establish if we had affected disease onset, but it looks very encouraging.
- First all-human mouse model of inherited prion disease by Public Library of Science from medicalexpress.com
- Brain targeting of 9c,11t- Conjugated Linoleic Acid, a natural calpain inhibitor, preserves memory and reduces A? and P25 accumulation in 5XFAD mice Orli Binyamin, Keren Nitzan, Kati Frid, Yael Ungar, Hanna Rosenmann & Ruth Gabizon.–lay summary. —report on clinical trial with MS patients.
- Challenging the Huntington Disease Paradigm: Evaluation of Psychosocial Issues in Persons at-risk for Genetic Prion Disease. Conducted by Case Western Reserve University
- Experimental treatment slows prion disease, extends life of mice. Very exciting research!!! As many of you know we have been communicating regularly with Eric Minikel and Sonia Vallabh and had an update via video at our annual family conference last November. Although Eric and Sonia will not be able to attend our conference again this year for personal reason they will do a video update and we hope to have a representative from Ionis, the pharmaceutical company involved with AOS to also update us. It is hoped that clinical trials may start within 5 years. Our friends at the NIH Rocky Mountain in Hamilton USA are also doing some wonderful research work. Many of you met Byron Caughey a few years ago when he presented on his work with RT-QuIC at an annual conference. I am sure you also remember Dr Cathryn Haigh and Dr Simote Foliaki, who both worked at the Collins Lab in Melbourne before relocating to NIH Rocky Mountain Lab. CJDSGN donations made possible by the generosity of so many of our families assisted both these wonderful researchers while they worked in Australia.
- From National Institute of Allergies and Infectious Diseases– Eyes of CJD Patients Show Evidence of Prions
- From Neurobiology of Disease– Mitochondrial dysfunction in preclinical genetic prion disease: A target for preventive treatment?
- “The path to an ASO drug for prion disease”. Video Presentation by Eric Minikel and Sonia Vallabh (Broad Institute, Boston, USA) . Originally shown at the 2018 11th Annual National CJD Conference, Melbourne, Australia.
- From La Trobe University- ‘Living’ brain slices reveal how nerve cells die
- Prion protein found in skin of Creutzfeldt-Jakob disease patients: NIH/Case Western Reserve University
- Research article regarding Jewish New Year fruit from Neurobiology of Disease Volume 108, Issue null, Pages 140-147:
Continues administration of Nano-PSO significantly increased survival of genetic CJD mice, Orli Binyamin, Guy Keller, Kati Frid, Liraz Larush, Shlomo Magdassi, Ruth Gabizon
- Article from The Times of Israel –Jewish New Year fruit may hold seeds of hope for brain disease sufferers
- Tedx Talk- Dr. Valerie Sim, how her curiosity brought her to discover a new way to bring science to a deeper level.
- A new article on Doxycline which we understand was found to be ineffective in prolonging life with CJD patients after several trails. Deana Simpson and I as co-chairs of the CJD International Support Alliance (CJDISA) will ask Professor Inga Zerr, who is a member of the Friends and Advisors Group of the CJDISA for an update. The full article can be found here: Doxycycline: bringing hope for early sporadic Creutzfeldt-Jakob disease patients
- Paper published by Eric Minikel and Sonia Vallabh: Quantifying prion disease penetrance using large population control cohorts.
Lay Versions: CureFFI.org: Does This Mean I’ll Definitely Get The Disease? CureFFI.org: Q&A on genetic prion disease risk
2015 & prior
- Dr Brian Appleby from Cleveland USA has provided the first summary which discusses the paper mentioned in the article How Do Prions Kill Brain Cells?
- Professor Will has provided the follow comments for which we are very grateful. We hope this helps reduce any misinterpretation surrounding iatrogenic and sporadic CJD while still maintaining an open mind to all research. New research: sporadic type of Creutzfeldt-Jakob disease (mad cow) results from surgery