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Monthly Archives: November 2013

Mastocytosis Treatment Update

11.15.13

Mastocytosis Treatment: Masitinib Enters Phase 3 of Clinical Study

Thank you to GlobeNewswire for allowing the sharing of the press release below!

 

AB Science: Final Results of Phase 3 Mastocytosis Study Expected by End of 2014

Final results of phase 3 mastocytosis study expected by end of 2014.

Indolent Systemic Mastocytosis is an orphan disease with no approved treatment so far.

Up to 20,000 treatable adult patients annually throughout countries paying for medication.

Long term follow up from phase 2 studies shows sustainability of response with masitinib.

PARIS, Nov. 12, 2013 (GLOBE NEWSWIRE) — AB Science SA (NYSE Euronext – FR0010557264 – AB), a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), following the press release from November 11th that announced the phase 3 study’s success in the futility analysis, further specifies important points concerning availability of phase 3 data, number of patients treatable and sustainability of response generated by masitinib observed in phase 2.

The read-out of the phase 3 study in mastosytosis is expected by end of 2014. Phase 3 study was accelerated and expanded recently outside of Europe and the USA, in Russia, India and Latin America.

Indolent Systemic Mastocytosis is an orphan disease with no approved drug currently registered. Masitinib received orphan drug status designation in mastocytosis, both from EMA and FDA.

Mastocytosis is an orphan disease characterized by an abnormal proliferation of mast cells either in bone marrow only or in several tissues. Mastocytosis comes in two main forms: indolent and aggressive. Indolent mastocytosis can be either cutaneous or systemic. The prevalence of Indolent Systemic Mastocytosis (ISM) is estimated at between 1/40,000 and 1/20,000[1] of the general population. The symptoms and handicaps are severe in about one third of the patients, hence an estimated target population for masitinib ranging from 1/120,000 to 1/60,000 of the general population.

Since the prevalence of Indolent Systemic Mastocytosis is reputed to be comparable across countries, the target population for masitinib could reach 20,000 adult patients in the world annually.

The fact that the phase 3 study in mastocytosis passed futility analysis is in line with phase 2 results.

Two phase 2 studies enrolled 46 patients suffering from systemic mastocytosis. Masitinib decreased the flush frequency by 54%, decreased the pruritus score by 45%, improved the depression status by 40% and decreased the fatigue score by 52% from baseline. The two studies generated results consistent with each other despite the fact that the first one enrolled patients without the c-kit 816 mutation and the second one with this mutation, suggesting that masitinib acts by inhibiting not only c-Kit but also Lyn, in blocking the release of the mediators by the mast cell.

In addition, long term follow up data of the two phase 2 studies showed sustainability of responses generated by masitinib. Two-thirds of the patients decided to enroll in the extension phase of the study ; 61% patients were treated for more than 1 year and 25% were still under masitinib after 5 years.

About masitinib

Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells, important cells for immunity, as well as a limited number of kinases that play key roles in various cancers. Owing to its novel mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. Through its activity of inhibiting certain kinases that are essential in some oncogenic processes, masitinib may have an effect on tumor regression, alone or in combination with chemotherapy. Through its activity on the mast cell and certain kinases essential to the activation of the inflammatory cells and fibrosing tissue remodeling, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases.

About AB Science

Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a new class of targeted molecules whose action is to modify signaling pathways within cells. Through these PKIs, the Company targets diseases with high unmet medical needs (cancer, inflammatory diseases, and central nervous system diseases), in both human and veterinary medicines.

AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA, and is pursuing ten on-going phase 3 studies in human medicine in GIST, metastatic melanoma expressing JM mutation of c-Kit, multiple myeloma, mastocytosis, severe persistent asthma, rheumatoid arthritis, Alzheimer’s disease, progressive forms of multiple sclerosis, and in Amyotrophic Lateral Sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Science website: www.ab-science.com.

This document contains prospective information. No guarantee can be given as for the realization of these forecasts, which are subject to those risks described in documents deposited by the Company to the Authority of the financial markets, including trends of the economic conjuncture, the financial markets and the markets on which AB Science is present.

[1] http://www.orpha.net  (Indolent systemic mastocytosis)

AB Science EN: http://hugin.info/155655/R/1742623/585910.pdf

 

Reposted with permission. Original article can be found:

http://www.globenewswire.com/news-release/2013/11/12/589193/10057632/en/AB-Science-Final-Results-of-Phase-3-Mastocytosis-Study-Expected-by-End-of-2014.html?parent=588800

Christal
Filed in: Life with Masto | Research & Studies

Masitinib Clinical Study for Mastocytosis Treatment

11.15.13

Clinical Study of New Drug for Mastocytosis Treatment

The following is a news release that was recently published regarding a new drug that is currently in clinical study for Mastocytosis treatment. Thank you to GlobeNewswire for graciously allowing us to repost the release here!

AB Science: Masitinib successfully passes futility analysis in pivotal clinical study in Mastocytosis

PARIS, Nov. 11, 2013 (GLOBE NEWSWIRE) — AB Science SA (NYSE Euronext – FR0010557264 – AB), a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), announces that the independent Data and Safety Monitoring Board (DSMB) created as part of the company pivotal clinical study evaluating masitinib in the treatment of mastocytosis has recommended continuation of the study, based upon completion of the futility analysis included in the study protocol.

The objective in this phase 3 study is to compare the safety and efficacy of masitinib to placebo in adult patients with Indolent Systemic Mastocytosis with severe handicap at baseline.

The futility analysis was included in the clinical study design to ensure that the study could be stopped early if it appeared that the study would be eventually unable to demonstrate the efficacy of masitinib in the target population of patients with Indolent Systemic Mastocytosis with severe handicap at baseline.

The analysis was performed with two-third of the target population to recruit. The futility analysis was performed by the independent Data and Safety Monitoring Board. It consisted in testing the possibility of masitinib to demonstrate superiority over placebo on the primary analysis set in the protocol, based on the EMA guideline on clinical trials in small populations (CHMP/EWP/83561/2005).

Alain Moussy, co-founder and CEO, stated “since the data remain blinded to AB Science, the efficacy results will not be known until the study is completed. However, we are very pleased that this pivotal clinical study has passed this important step successfully since the placebo effect was unknown as there has been no previous placebo controlled clinical study carried-out in this orphan disease”.

About masitinib

Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells, important cells for immunity, as well as a limited number of kinases that play key roles in various cancers. Owing to its novel mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. Through its activity of inhibiting certain kinases that are essential in some oncogenic processes, masitinib may have an effect on tumor regression, alone or in combination with chemotherapy. Through its activity on the mast cell and certain kinases essential to the activation of the inflammatory cells and fibrosing tissue remodeling, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases.

About AB Science

Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a new class of targeted molecules whose action is to modify signaling pathways within cells. Through these PKIs, the Company targets diseases with high unmet medical needs (cancer, inflammatory diseases, and central nervous system diseases), in both human and veterinary medicines.
AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA, and is pursuing ten on-going phase 3 studies in human medicine in GIST, metastatic melanoma expressing JM mutation of c-Kit, multiple myeloma, mastocytosis, severe persistent asthma, rheumatoid arthritis, Alzheimer’s disease, progressive forms of multiple sclerosis, and in Amyotrophic Lateral Sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Science website: www.ab-science.com.

This document contains prospective information. No guarantee can be given as for the realization of these forecasts, which are subject to those risks described in documents deposited by the Company to the Authority of the financial markets, including trends of the economic conjuncture, the financial markets and the markets on which AB Science is present.

AB Science EN: http://hugin.info/155655/R/1742219/585574.pdf

 

Reposted with permission. The original article can be found at:

http://www.globenewswire.com/news-release/2013/11/11/588800/10057365/en/AB-Science-Masitinib-successfully-passes-futility-analysis-in-pivotal-clinical-study-in-Mastocytosis.html

Christal
Filed in: Life with Masto | Research & Studies

Pity Parties and Grieving Loss

11.12.13

Pity Parties and Grieving Loss

Today it really hit me, I can’t eat anymore! I have tubes coming out of my body, food that is delivered in a bag, and almost always need to be in bed.  I’m really sad and honestly feeling a little sorry for myself.  I am craving some Kettle Brand Sea Salt Potato Chips and chocolate milk shake. I would love to go on a long walk with my hubbie, Mr. Hero, admiring all the changing fall leaves. Instead, I am lying in bed next to my very own IV pole, hooked up to my 16 hr a day nutrition that smells like bad baby formula.

It’s been one week since I had my PICC line placed and 6 days since I began TPN. Perhaps, I have been so caught up in remembering how all this works or maybe it’s because I am finally getting enough calories to think about something other than surviving but I am grieving over my situation. Today, I’ve been reminded that I was in this same spot six years. I feel bad needing so much help.  As the Mom, I am supposed to be the caregiver not the care taker. It’s safe to say I am having a pity party for myself. Urban dictionary defines a pity party as a way of experiencing grief, where you spend your time feeling sorry for yourself and whining endlessly about how bad your life is. The thing about pity parties is they are no fun! No one else really wants to attend a pity party with you and really they don’t even help you feel better. I have told my children for years there are two reasons to complain:

  1. to be negative
  2. to try to make the situation better

So I ask myself which of these is my motivation now? When you have a chronic disease you really need to be cautious about how much time you spend being negative because it can really adversely effect your healing process.

There has been more change in my life. In the last two weeks than many people will ever go through. Let’s face it, having a tube placed right at the top of your heart is scary! Unless you are so ill you require it for medical reasons and you get excited about someone feeding a catheter into you chest, there is probably something very wrong with you. I need and want to process those feelings and grieve appropriately to be healthy emotionally. So what is the difference between having a pity party and grieving? In my opinion it how long it lasts. It’s ok for me to be sad, it’s a sad situation. The problem comes when we get stuck there and linger too long. If I am still feeling sorry for myself about this next week or next month exactly how does that help the situation? It doesn’t, in fact, it will probably hold me back from the best possible recovery, I will more than likely make everyone around me miserable, and I will still be sad. I have given myself a time limit on pity parties, 24 hours max. I’m not say thing that is right for everyone, and I am not talking about the loss of a loved one. Only you can decide that for yourself how long is long enough to grieve. I am a bit of an “A” type personality and for me I need to process and move it along. Twenty-four hours allows me to get a good night sleep and see the world through rested eyes. Most of you know I am not a “crier” but even I don’t dispute sometimes tears are helpful. It’s ok for me to feel sad, snuggle up in my pajamas, and even have a good cry but then at some point I need to get over it.

It may sound cliché but I choose to be happy, joyful, hopeful and thankful. Yes, there will always be reasons to be sad, disappointed or discouraged but there really are so many more reasons to be thankful, we just need to look for them. It is human nature to focus on the less than positive things in our lives. Think about it, when we spotlight the negative things around us by talking about them all the time, worrying, being anxious, and allowing our lives to be consumed by those things we diminish the good all around us. We elevate the bad things to a place they should not be, a place of prominence, and if we are not very careful those negative things can actually become the very thing let take control of us. It actually changes our quality of life and will plunder our happiness. There has been more than one night I the last month I was pretty sure I would not be alive in the morning. When you live moments like these, you look back your life and take stock. You want more time with loved one, you want more time to carry out your life’s mission, and you want to have made a difference. Long term pity parties and feeling sorry for your self is the complete opposite of each of those things. You may not be going through a health crisis at this time but maybe you are struggling in relationships, finances, unrealized dreams or something else. I challenge you to look for the beauty in ashes

Instead of feeling bad that my children, Princess and Game Boy, don’t have a mom that can do everything for them, I think about the amazing life skills they have gained, how compassionate they are for people around them, and what amazing people they have become. I’m thankful the kids are older, can drive, and are pretty much adults at this point. I am thankful my parents, Beauty and The Businessman, have taken time off work to be with me after all I my surgeries and to help take care of me post op since I have such a hard time with anesthesia. I am thankful for a nationwide network of prayer warriors who are up round the clock lifting me up. I am joyful to have friends and family who make great personal sacrifices to call, text, email, visit and send cards. Instead of regretting that I can’t go on a date with Mr. Hero or that I have to be in bed by 4pm to start my food,  I’m thankful that almost every day he climbs in bed with me and says what should we watch tonight? Mr. Hero has, once again, been living up to his name. I have needed help walking so I don’t fall, wrapping my arm before a shower, plugging in and disconnecting my food, flushing my lines,  preparing my food all in a sterile environment to keep me healthy, and he has done so much more. I am blessed to have every single day with my friends and family and to be a part of their lives even if those days are modified because of my situation. Instead of thinking about my stinky food in a bag, I am hopeful when I think about how amazing it is that we live in an age that makes it possible to sustain life in the circulatory system instead of the G.I. system. Wow! God made our bodies with a back up system, that is pretty incredible. Instead of feeling defeated that I am 45 years old and need home health care, I am so blessed to have an amazing nurse, who I have come to adore in one short week.  Instead of focusing on the thought that I have a very rare disease with no cure, I am thankful that I have the world’s foremost authority on my disease as my specialist and he is located in this city. This is a health crisis not eternity. I will be well for eternity. At times like these, I really like to focus on my favorite Bible verse.

So we fix our eyes not on what is seen, but on what is unseen, since what is seen is temporary, but what is unseen is eternal.

2 Corinthians 4:18

Today I challenge you to focus on the abundant blessings all around you. Feel free to leave a comment about what you are joyful for.

Christal
Filed in: Life with Masto | relapse
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