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georgejjl   Monday, 03/11/19 07:23:07 PM
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UPDATED: Early data snapshots of Axovant’s gene therapies for Parkinson’s

Fol­low­ing the col­lapse of its neuro-fo­cused strat­egy, Ax­o­vant’s foray into gene ther­a­pies — with their po­ten­tial for one-shot, long-term cures — has gen­er­ated con­sid­er­able in­ter­est. On Mon­day, the biotech pro­vided an early pos­i­tive snap­shot of two of its pro­grams: in Parkin­son’s and in­fan­tile Tay-Sachs dis­ease.

Two pa­tients were given the low­est dose of Ax­o­vant’s Parkin­son’s gene ther­apy — AXO-Lenti-PD — as part of a mid-stage study called SUN­RISE-PD. Pa­tients were as­sessed three months after their dose, hav­ing been washed out of their oral lev­odopa ther­apy — the gold stan­dard treat­ment for Parkin­son’s. On a physi­cian-rated scale (UPDRS) as­sess­ing motor func­tion (the Part III score), both pa­tients ex­pe­ri­enced an im­prove­ment. The scale mea­sures scores rang­ing from 0 to 108, with lower scores in­di­cat­ing im­prove­ment. At three months, one pa­tient saw an im­prove­ment of 14 points, the other 36 points — trans­lat­ing to an av­er­age im­prove­ment of 25 points or a mean 42% from base­line, Ax­o­vant said.

Progress was also ob­served on other parts of the UPDRS scale. In ac­tiv­i­ties of daily liv­ing (UPDRS Part II), pa­tients ex­pe­ri­enced an av­er­age im­prove­ment of 22 points from base­line, while for com­pli­ca­tions of ther­apy (UPDRS Part IV), they saw a mean im­prove­ment of 7 points from base­line.

Ax­o­vant li­censed the in­ves­ti­ga­tional gene ther­apy, as well as the pre­de­ces­sor prod­uct ProSavin, from Ox­ford Bio­Med­ica last year for $30 mil­lion up­front. AXO-Lenti-PD is de­signed to de­liver three genes — ty­ro­sine hy­drox­y­lase, cy­clo­hy­dro­lase 1, and aro­matic L-amino acid de­car­boxy­lase — via a lentivi­ral vec­tor to en­code a set of crit­i­cal en­zymes re­quired for dopamine syn­the­sis to re­store steady lev­els of dopamine in the brain in one shot.

These re­sults sug­gest that the low­est dose of AXO-Lenti-PD at three months may have greater ef­fi­cacy com­pared to the high­est dose of ProSavin pre­vi­ously tested, Ax­o­vant said.

“The mech­a­nism of ac­tion of AXO-Lenti-PD…as well as our prior clin­i­cal ex­pe­ri­ence with ProSavin, led us to ex­pect that the major ben­e­fit would be in im­prov­ing the OFF state – and the re­sults so far are very en­cour­ag­ing in this re­gard,” said Roger Barker, one of the prin­ci­pal in­ves­ti­ga­tors on the SUN­RISE-PD study.

Based on feed­back from the data mon­i­tor­ing com­mit­tee, Ax­o­vant is going to test a sec­ond dose of the ther­apy, and the first ad­min­is­tra­tion of that new dose is ex­pected in the sec­ond quar­ter, the com­pany said.


Now follow this Shiela Roy as in the previous ProSavin trial. Her improvement was in step jumps over several years at least and she wrote a book or books after treatment.

“There is hope for turning this condition around”
PERSPECTIVES Author: Sheila RoyPublished: 9 April 2015 Sheila Roy (centre)
At a time when trial subjects are in short supply, people like Sheila Roy (pictured above, centre) ensure the Parkinson’s community benefits from the experimental research. One of the first people in the world to test ProSavin, she has written the following account four years into her trial – recounting the highs, the lows and ultimately the “life-changing” improvement to her quality of life

I was diagnosed with Parkinson’s in 1995 and have lived with this life affecting condition for 20 years, one third of my life. This disease has challenged my ability to function in every possible way. I lost confidence, dignity and hope. For the first 18 years I experienced mobility problems, falls, constant pain, sleep deprivation, screaming nightmares, dyskinesia, ‘freezing’, and in my case, spontaneous closure of my vocal cords and being unable to breath.

I was spiralling downwards with little hope. My day was so unpredictable. The transition from extreme involuntary movements to completely frozen took four seconds, which meant that I was unable to get into a safe position. Often the freezing would last for up to two and half hours, and took some time to return to moving around again.

At this time I spent about 60% of each day ‘off’ which meant that I spent long periods sitting and waiting until I came back ‘on’ again. Only 20% of my day was ‘on’ but this was blighted by involuntary movement making it hard to do anything. It led to a withdrawal from society and I was exhausted by the constant movement. My sleeping was erratic and I would frequently wake my husband up with screaming nightmares.

All of these symptoms changed me into a person that I no longer recognised. In 2010, when I was thinking that things could not get any worse, they did just that. One night the house caught fire and my husband became very ill while my horse and cat died in the damage. Inevitably, my PD worsened. My daily medication regime at that time – consisting of around 20 different daily doses of various tablets – was becoming less effective and my response was to increase the dose, which made the symptoms even worse.

Ideal candidate

In February 2011 Dr Roger Barker referred me to Dr Philip Buttery, a colleague of his at Addenbrooke’s Hospital, to consider my suitability for deep brain stimulation. During this consultation he told me about the ProSavin study for the first time. Dr Buttery said that they were looking for three people to take part in assessing the safety, efficacy and dose evaluation of ProSavin in people with mid-stage Parkinson’s, who were experiencing reduced benefit on levodopa medication – I was the ideal candidate. The study evaluated three dose levels in a total of 15 patients with PD. Six people received the higher dose, and I was the first of these in the UK.

“As soon as I heard about the study on ProSavin I knew it was for me”
ProSavin delivers the genes for three enzymes that are required to make dopamine. This is injected deep into the brain in order to convert cells into a dopamine factory replacing the lost source of this neurotransmitter.

As soon as I heard about the study on ProSavin I knew it was for me. I felt very confident that, if possible, I should go for it. Many people tried to persuade me otherwise, but I felt strongly that it was right for me.

It has not been an easy process. I have to watch my body for involuntary movement, and reduce my intake of oral dopamine. This has been hard to do, and gets harder the more I reduce the intake. My improvement is not a gradual incline but feels like a step up in response to dyskinetic movements and a reduction in my medication. It has taken me two years to half my dosage of Stalevo tablets from ten to five.

Life-changing improvements

I am not cured, but I now have a little factory in my brain that produces dopamine. I still have involuntary movements and ‘off/on’ times, but they are not as severe as they were. Now I know when they are coming, and can at least function when ‘off’.

The most important improvement is in my ability to think, analyse and articulate better. I couldn’t hold a good conversation a year ago, but now I can. I can also express myself in ways I couldn’t before such as writing, which I love, and laughing. My mobility is much improved too as well as my sleeping pattern. I used to only get about three hours sleep in 24 hours – now I’m getting around seven which has made a tremendous difference allowing my body to heal. I still get terrible nightmares where I scream the house down – nothing seems to make that any better.

I have an increased ‘on’ period – the time when I’m fully functional. I’ve gone from four hours to 11 hours a day – so most of my day is active and it makes such a difference. I just have more time to enjoy myself and do the things I want to do. There’s less falling asleep all the time and my mood has really picked up meaning I’m much more sociable. Overall, I’m much more confident than I was. It’s a big change.

Since starting this study I feel like I’ve improved significantly and I’m really encouraged by that. It means that there is hope for turning this condition around.

Would I do it again? Yes, I’d do it again tomorrow! Because for me it’s been a life-changing improvement.


Sheila Roy, a woman on an experimental gene therapy treatment for Parkinson’s disease, never imagined three years ago that she would be well enough to write a book.


Watch the video


Good luck and GOD bless,

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