35 replies to this topic

[rcase13]

Ignore my last question. I read through the study again and it appears to only be open to people that have lost response and/or have a mutation.

 

I see the following listed:

Accepts Healthy Volunteers:  

No

 

So I guess they are not yet at the point where they are doing studies on new patients or healthy patients.

 

Sorry for my lack of patience. I need them to develop a cure yesterday. I have a 13 year old and it is obvious he will need to be taken care of his entire life... I don't recall being so stubborn at 13... my parents may disagree.

[r06ue1]

As part of the trial, they are also looking at patients taking only ABL001.  This may come after response, not sure as I don't have the details.  Also, if you are having too many issues with side effects of current medications you may qualify for the trial.  

 

No mention of this being a cure but it appears to work better than current TKI's and has less side affects.  My hope is that people who take it can eventually discontinue the use and have better results in staying off the drug than current drugs.

[Lynne D]

I have been in the ABL001 trial since April 2015. I had been on Gleevec, Tasigna, back to Gleevec then Sprycel. I developed a mutation (f317l) and I was the first patient of Dr. Mauro's first Clinical Trial at Memorial Sloan Kettering Cancer Center. I had never been in MMR before, I started ABL001 on my 10 year cancerversary, and I was immediately undetectable and am still undetectable 25 months later. I have less side effects than any other TKI. I started on 200mg twice a day. I lowered to 150 twice a day due to high blood pressure brought on by a bout of rhabdomyolsis because they started me on Crestor, abl001 and a workout in the gym. I was hospitalized for a week. Then they reformulated from capsules into pills so the dosage changed and I am currently on 160 mg twice a day. I hope this helps anyone. I also have a facebook group dedicated to ABL001. The link is below if anyone is interested. 

 

Thanks

Lynne

 

https://www.facebook...53028488228609/

[gerry]

Thanks Lynne - great to hear outcomes of this drug. :-)

[TeddyB]

Encouraging indeed.

 

If the trials go well, does anyone know when ABL001 is expected to "hit the market"?

And who will get it? Only those who do not respond to the other TKI`s or everyone?

I also have a feeling it is going to cost a lot?

[kat73]

This is so exciting!  An actual person who is on this and getting great results!  Thank you SO much, Lynne, for reporting in and congratulations on this tremendous success!  Please keep filling us in here, as not all of us (well, maybe I'm the last person on Earth?) don't do Facebook.  Best, best wishes for your continued well-being!

 

TeddyB's questions are pertinent:  Is the first opened-up cohort going to only include those who've failed the main TKIs or have a recognizable mutation?  Or will they include the rest of us?  How about turtles?  I would certainly bet it will be super expensive.  And, WHEN?????

[chriskuo]

In the US, the brand name drugs all cost about the same. A new drug will likely come in at about the same price
($120K-$140K) as all of the others on patent. Price is not a differentiator in the US. In other countries,
it can be because the governments negotiate lower drug prices individually with drug companies.

In the US, pricing with the insurance / PBM middlemen is a black box. They get a 30-40% discount on average
from the prices that are bandied about, but these discounts are kept secret as proprietary information.

[chriskuo]

I would assume that it would not be approved for first-line use, unless clinical trials are run on that
patient population. Does anybody know if that is happening or is scheduled to happen?

Since it is additional expense for the drug company, they may want to get the money flowing from second-line use
before they take the next step.

[Sandrea]

Expanded Phase 1 Study of ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Reveals Significant and Durable Responses in Patients with CML-Chronic Phase with Failure of Prior TKI Therapy
58th ASH Annual Meeting & Exposition,  San Diego, CA  December 3-6, 2016

[r06ue1]

It will still be a while before this drug hits the shelves, maybe 3 to 5 years.  

 

http://www.medicinen...articlekey=9877

 

If the drug shows curative abilities (which Novartis has speculated) it could get to market faster and be used as a first line drug (insurance companies are always looking to save money) but my guess is that the cost will double (at least), just look at the cost of some of the more recent drug therapies for other types of cancer for an example.

[TeddyB]

Whether it will be first, second or third line, its always good to have more options, and hopefully this will have less side effects than the other TKI`s

 

Will be interesting to follow ABL001 progressions towards market.

[Lynne D]

A little update from Dr. Mauro's first ABL001 Patient001  He calls me his ABL001 Poster Person. I was started on the max dose of 200 2x a day. As per my own body's ways, I always develop side effects as years go on. I've had a few seizures on Sprycel and 2 on ABL001. Not sure if it's related and I don't think I really care, I just want to know for sure. I have minor bone "humming" and folliculitis starting again. ALL MINOR considering where I came from on 6 horrible years on Gleevec, 4 toxic months on Tasigna and 4 decent years on Sprycel before mutation. All in all, I am happy with this drug. I wish it could be once a day, which is part of the trial just not my part. My Dr. is hoping to get a trial within the trial and I hope he is succesful. It is a STOP trial. I've never been undetectable ever and now it will be 3 years in April. It isn't within the guidelines to have a trial within a trial I believe, but I'd be willing to try it. I never thought I would but I'd give it a whirl.

 

They are planning a combo trial with Bosulif also and currently doing combos with Gleevec. I'm thinking they are about 2 years from FDA approval. The ABL001 straight trial, no combo, is closed unless it's a compassionate case. I've been very happy with ABL001 and I think it is going to make a huge difference. The way it works is different than the other tki's.  I am attaching the link to the slides to Dr Mauro's webinar back in September. He also used many of the slides at the New York City/New Jersey Conference at the Grand Hyatt this month. Very informative. I will continue to keep you all posted.     

 http://www.lls.org/s...gram Slides.pdf

[chriskuo]

 In Dr Mauro's presentation, I see a slide about a trial of ABL001 vs Bosulif.  Have you seen or heard information about a combo trial of the two?

[tiredblood]

I️ asked my doctor and he guesstimated about 2 years for FDA approval as well. Thanks for the update and also your participation in the trial. Please keep us posted.

[Lynne D]

 In Dr Mauro's presentation, I see a slide about a trial of ABL001 vs Bosulif.  Have you seen or heard information about a combo trial of the two?

 

Dr. Mauro isn't crazy about that combo trial. He is most likely going to take another path in his clinical trials. If anyone here is in Manhattan on Dec 16, I am having a "connections" meeting for CML patients and their caregivers. It is for another non-profit organization but it's very small and not competition for LLS. I am going to have more info on the trials going on. Hopefully, Dr. Mauro will able to stop by. If you are interested just sign up here https://www.eventbri...ets-40094189801

 

I hope that doesn't get me booted, we are all just trying to help each other. 

[Red Cross Kirk]

Bump.