Lola -Zarnestra - New drug for myelodysplasia

[StephN]

Good morning, Lola -
Well, you have had a night to sleep on your news. I am sure your attitude is even better today!
I did a quick search and came up with some very recent news on Zarnestra. The jargon is more like our normal English, so should be easier to read!
At least this is something to watch and be aware of as there may be a way for you to benefit at some point.


"NDA Submitted for Zarnestra


Jan 24,2005


Today, Johnson & Johnson announced the completion of a NDA submission to the FDA for Zarnestra (tipifarnib), an oral farnesyltransferase inhibitor, for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients 65 years of age and older.

The NDA submission for this indication was based on data from a Phase II study that used measurements of activity and disease responsiveness as endpoints instead of survival. An international Phase III trial, initiated in October 2004, is also being conducted to illustrate the clinical benefit of Zarnestra.

In addition to receiving Fast Track and Orphan Designation, Zarnestra has been entered into the Pilot 1 Program. This program is designed to expedite the rolling NDA concept by allowing the submission of a limited number of "Reviewable Units" of a NDA in advance of the complete application. The FDA is committed to initiating the review of these sections within six months of notification of Pilot 1 status. Products that have been designated Fast Track status and have demonstrated significant therapeutic potential in clinical trials compared to available therapies for the disease or condition are eligible for the Pilot 1 Program. The ultimate goal is to get the drugs through the FDA process and onto the market more quickly.

Zarnestra is also in lower phases of development for multiple other cancer types. "

Another link to a report on the MD Anderson trial from Dec. 2004:
http://www.thedoctorslounge.net/hemalounge...rnestra_aml.htm (http://www.thedoctorslounge.net/hemalounge/conferences/ash-2004/zarnestra_aml.htm)

There is also a good report I found from a multi-center international trial, so this all looks good for this drug's approval.

[lolam]

You are really a good researcher! I didn't find a thing on any treatment. From what I read it is so rare that they hadn't even bothered to have a plan. I am just a little young it seems. I guess most people are so old they leave them alone!
This new drug sounds promising. It is a bit daunting to have to be up on two different cancers at once. I hope my body helps me out here just a little! My onc. is a total scientist. He loves a good challenge ! I hope he has kept himself in the know about these things. If not, I surely will help him out all I can.
Thanks so much for the info. I am encouraged.
Hugs, Lola

[*_jeff_*]

Lola,

I just wanted to jump in to say that I've just done a quick check on cancer.gov for trials using tipifarnib and its being tested ALL over the place in lots of different types of cancers. I hope it will be helpful to you if and when you need it.

In the meantime, I hope you continue to feel well and have many, many joyful days.

Best,
jeff

[lolam]

Thanks so much for the information. It looks like there are a couple meds to try. My onc. did not mention any treatments. Don't know if he is waiting for the chromosome report or if he is in the dark about these new treatments.

As I have read more now, My situation is a little more grave than I had known. It doesn't look like many patients survive for very long, the onset of full blown leukemia is short, the survival is not long, some treatments are killers in themselves. It is all rather discouraging.

Things always seem better to me in the morning, so I must say good- night. I really should get more sleep anywise......having preleukemia and all :) ......

Thanks again for being interested in my case when it doesn't even affect you. I don't feel so alone.....

Hugs, Lola

[StephN]

Hi Lola -
Looks like you have some information to share with your care team on possible treatment. If these docs don't go to conferences regularly and read up a lot they don't have all the latest - why we have to keep on top of our own cases! My med onc goes to all the national conferences (I bumped into him at San Antonio and brought him over to the Her2support booth to meet everyone!), which is a reason I chose him.

The only person I know who had the same disease, but caused by natural onset from aging, was my father-in-law. He was about 78 when diagnosed with the pre-leukemia and he was 82 or 83 when he passed away. He was living in France and treated there until the end, which was around the end of 2000. He did well up until his last 3 or 4 months. I had visited him for 5 days that summer just before my diagnosis and he was walking all over town, driving and going to church.

[lolam]

I am anxiously awaiting my chromosome report. I f it is irregular, it is a whole new ball game, more difficult. If it is still normal, then I have more options. I will be taking the new drug info to my onc. either way. I am concerned about BC mets as I don't know if they could use chemo on me with my other condition. Obviously, a bridge I have not come to so I try not to make it a practice to cross them!! It does take discipline of mind. We talk so positively about our struggle against BC mets and I think we all agree that if we fight we will live until the cures come rolling in. But I just don't seem as optimistic about MDS. I guess I just don't know enough about it yet. I was just getting used to all the stuff we have to keep straight about BC Mets and Her2/neu. I guess I still need to have my annual brain MRI which is coming up. I don't know what I am fighting first now. Seems BC has been relegated to the back burner, moving MDS forward. I am the type of person that could never keep alot of balls in the air!! A real one track mind. No 3 ring circuses for me. I always admired friends who could do that. I guess I will have to try new tricks in my old age!!
Thanks for your encouragement. We all need that from time to time.
Hugs, Lola

[al from canada]

Lola, Some more chemo reports on , Al

Study shows long-term benefits for new CML patients receiving
imatinib therapy
2005 JAN 3 - (NewsRx.com) -- CHU in Poitiers, France, announced results of a study
showing that newly diagnosed patients with a certain form of leukemia who are treated early
with imatinib are more likely to achieve complete cytogenetic responses (elimination of
leukemic cells) and have improved long-term outcomes.
New data from the largest study of chronic myeloid leukemia (CML) patients (1,106
patients) ever conducted, International Randomized IFN vs. ST1571 (IRIS), were presented at
the annual meeting of the American Society of Hematology (ASH).
In the study, newly diagnosed patients with chronic-phase Philadelphia chromosomepositive
(Ph+) CML who achieved cytogenetic responses early had improved rates of
progression-free survival compared to those who did not achieve early responses. Responses to
imatinib, a new type of medication that prevents and stops the growth of cancer cells, were
shown to be durable at 42 months.
The landmark analysis showed that at 42 months, 84% of patients taking imatinib
remained progression free, and only 6% progressed to the more advanced disease stages
(accelerated phase and blast crisis). Overall survival (based on CML-related deaths) in patients
treated with imatinib was 97% at 42 months. Patients taking imatinib as first treatment who
achieved a complete cytogenetic response (CcyR) within 12 months of therapy had a 93%
progression free survival rate at 42 months, compared with 74% in those without CcyR.

AND,

Peptide vaccine can produce remission in myeloid leukemia patients
2005 JAN 3 - (NewsRx.com) -- Researchers from The University of Texas M. D.
Anderson Cancer Center are reporting the first study to show that vaccination with a peptide that
is abnormally expressed on myeloid leukemia cells can produce a complete molecular remission
in some patients.
The experimental vaccine produced an immune response in 60% of patients tested -
or 20 of 33 evaluable patients, according to their study, which was presented at the annual
meeting of the American Society of Hematology.
Of those 20 patients who mounted an immune response against their cancer, 14 had
an overall survival of 4 years, and none of the 13 patients who did not have such a response lived
that long, they say. Three patients are in "molecular remission" in which there is no evidence of
the disease remaining.
"These are very promising results in a group of patients who were quite sick, and who
normally would live for just months," says the study's lead investigator, Jeffrey Molldrem, an
associate professor in the Department of Blood & Marrow Transplantation.
"Not only can we say that some of these patients had an immune response, but they
also went into remission, and that has never been demonstrated to occur after peptide vaccination
before. And for patients who showed an immune response but did not go into remission,
progression of their cancer was slowed down compared to patients who did not have an immune
response to the vaccine," he says. "Of course, we have much more research to do, especially in
testing greater numbers of patients within each disease group."
Nearly all of these patients had either active or relapsed acute myelogenous leukemia
(AML) or refractory chronic myelogenous leukemia (CML) - forms of leukemia in which there
is a dangerous accumulation of immature cells in the bone marrow - or they had high-risk
myelodysplastic syndrome (MDS), a precancerous bone marrow disorder.
The vaccine is made from the PR1 peptide, a small part of a protein found on the
inside of leukemia cells. Delivery of the vaccine induces immune cells that recognize the PR1
peptide, which are recruited to the bone marrow that contains leukemia," says Molldrem. "The
PR1 peptide is naturally present in normal bone marrow cells and since the target protein is
overexpressed on leukemia cells, it directs immune T-cells to kill the leukemia and leave normal
cells alone."
Like many vaccines used to induce immunity against infections, this one appears to
create an immune system "memory" for the PR1 peptide, conferring long-lasting protection.
"Patients only get the vaccine three times, yet we have been able to measure an immune response
in patients four years after treatment," says Molldrem.
This article was prepared by Cancer Vaccine Week editors from staff and other
reports. Copyright 2005, Cancer Vaccine Week via NewsRx.com.