Q&A

For those of you who may be asking yourselves a couple of questions, here are a few Q&A’s that may help you understand the situation we are currently in.

  1. WHY IS THIS CAR-T19 TREATMENT MUCH MORE PROMISING THAN LAST YEAR?

Unlike last year, there are no plans for Matthew to receive a Bone Marrow Transplant (BMT) following the improved CAR-T treatment in Seattle. A BMT would eliminate the CAR-T cells, whereas if they are present, they can continue to kill any leukemia that may try to rematerialize.

The National Institutes of Health (NIH) has also discovered that Matthew’s DNA has a rare genetic mutation. This finding is the most important breakthrough that occurred during his stay at the NIH and probably the underlying reason why his leukemia has been non-responsive to standard therapies in the past. It also seems to be the reason why he keeps relapsing after attaining complete remissions. This mutation causes the cells to always be “activated” and constantly produce leukemia cells. With this new recent gene identification, the doctors will use a drug that should be able to halt or “deactivate” the mechanism that causes the constant replications of malignant cells. It’s a gene that has newly been discovered in leukemia patients in the USA and it is presumably not yet tested for at diagnosis in Montreal (or possibly Canada), making it exceedingly difficult to treat unique patients like Matthew. They have decided to start testing all patients at the NIH in order to give them more targeted chemotherapies initially, which is a great advancement!

The chances of Matthew staying in complete remission, with this improved CAR-T19 trial and the discovery of the new gene in his DNA, are better than ever!

  1. HOW AND WHY IS THE FRED HUTCHINSON CAR-T CELL TRIAL DIFFERENT?

The Fred Hutchinson Research Center CAR-T trial is targeting the CD19 protein, which is expressed on the entire population of Matthew’s leukemia cells. This will make it easier for the CAR-T cells to target and kill.

The advancements that have been made with immunotherapy since last year are incredible. This CAR-T treatment we are aiming for uses a genetic modification that is different than the one that was used at Memorial Sloan Kettering last year. They use a different DNA sequencing which allows the CAR-T cells to last longer in the body, without being destroyed or rejected by one’s own immune system. With this new technology, the CAR-T cells can continue to kill any remaining cancer cells over time and any re-occurring cancer cells that may appear.

Fred Hutchinson Research Center has treated over 140 patients and has had incredible high success rates. Many patients are still in remission over 3 years later, without having done any other treatments.

  1. WHY DIDN’T THE CAR-T22 IMMUNOTHERAPY TREATMENT AT THE NATIONAL INSTITUTES OF HEALTH (NIH) WORK?

To try and put this in the most layman terms as possible, Matthew’s leukemia has two different “populations”. All of his leukemia cells express the CD-19 protein, which is why he responded entirely to the CD-19 CAR-T immunotherapy he did in New York last year. However, there is also a sub-population of leukemia cells that exists that express the CD-22 protein. This protein was targeted in the trial at the NIH in Maryland (only 60-70% of his leukemia cells expressed the CD-22 protein). What doctors believe happened is that those cells expressing the CD-22 protein were properly targeted, but the remaining cell population that didn’t express the protein outsmarted the CAR-T cells and kept multiplying, keeping the leukemia active and rising.

  1. WHEN WILL THE TREATMENT AT FRED HUTCHINSON START?

– Depending on Matthew’s health status (hoping that his eligibility for the trial is not compromised in the next weeks) and on the Fred Hutchison Cancer Research Center’s ability to accommodate him, the goal is to arrive in Seattle by mid-December.

  1. ABOUT MATTHEW – REMINDER OF LAST YEAR’S CAMPAIGN

Matthew was diagnosed with ALL on August 8, 2014, at the young age of 24. In 2014, after his diagnosis, he underwent all possible treatments available in Canada (three aggressive induction chemotherapy regimens and a monoclonal antibody clinical trial treatment) all of which failed to bring his cancer into remission.

After learning that there were no treatments left for him in Canada, doctors advised Matthew that the only hope of saving his life was the CAR-T19 immunotherapy clinical trial, which was only offered in the USA. The family worked persistently with the Memorial Sloan Kettering Cancer Center (MSKCC) in New York City, NY. They provided the costs for the treatment, and demanded a deposit amount of 675,000$USD.

The trial was very promising, with a published success rate of 90% complete remissions in patients with refractory or relapsing ALL like Matthew’s disease. This was the very last option he was given, alternatively to palliative chemotherapy and care.

Matthew and his wife Katia, who he married in June 2014 only a month and a half before his diagnosis, then started a crowdfunding campaign to raise the needed funds to get him to MSKCC and receive the CAR-T19 cell immunotherapy treatment he desperately needed to survive. Due to the unbelievable support from family, friends and strangers throughout the world they were miraculously able to raise the money needed for his treatment.

On February 10, 2015, they made their way to MSKCC in New York so that Matthew could begin the procedures for the treatment. He was infused with the laboratory modified T-cells on March 18, 2015, thus marking the beginning of his treatment. After spending 10 days in the Intensive Care Unit he eventually started to recover and was back on the road to better health. Matthew was finally given the miraculous news on April 8, 2015 that he was in complete remission.

A Bone Marrow Transplant was quickly planned for May 29th, 2015. This was his greatest chance at a long term cure, even though it would eliminate all of the CAR-T cells in his body. Sadly, Matthew relapsed in December 2015. Since then it has been an inconceivable rollercoaster of temporary remissions and relapses.

Matthew refuses to give up. As long as we have options, we will keep on fighting and we will never give up!

      6. RECAP OF MATTHEW’S MEDICAL PATH

  • August 8, 2014: Diagnosis – Acute Lymphoblastic Leukemia
  • August, 2014: 1st induction chemotherapy – Hôpital Cité-de-la-Santé, Laval
  • October 2014: 2nd induction chemotherapy – Hôpital Cité-de-la-Santé, Laval
  • November 2014: 3rd induction chemotherapy – Hôpital Cité-de-la-Santé, Laval
  • December 2014: Blinatuzomab monoclonal antibody clinical trial – Hôpital Maisonneuve-Rosemont, Montreal
  • Feb to April 2015: CAR-T19 immunotherapy clinical trial – Memorial Sloan Kettering Cancer Center, New York City
  • June 2015: Bone Marrow Transplant – Hôpital Maisonneuve-Rosemont, Montreal
  • January & June 2016: Inotuzumab monoclonal antibody “bridge” treatment – Hôpital Maisonneuve-Rosemont, Montreal
  • Aug to Oct 2016: CAR-T22 immunotherapy clinical trial – National Institutes of Health, Maryland